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22–416 2017

EXAMINING LEGISLATION TO IMPROVE PUBLIC 

HEALTH 

HEARING 

BEFORE THE

SUBCOMMITTEE ON HEALTH 

OF THE

COMMITTEE ON ENERGY AND 

COMMERCE 

HOUSE OF REPRESENTATIVES 

ONE HUNDRED FOURTEENTH CONGRESS 

SECOND SESSION 

SEPTEMBER 8, 2016 

Serial No. 114–165 

( 

Printed for the use of the Committee on Energy and Commerce 

energycommerce.house.gov 

(II) 

COMMITTEE ON ENERGY AND COMMERCE 

FRED UPTON, Michigan 

Chairman 

JOE BARTON, Texas 

Chairman Emeritus 

JOHN SHIMKUS, Illinois 

JOSEPH R. PITTS, Pennsylvania 

GREG WALDEN, Oregon 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 

MARSHA BLACKBURN, Tennessee 

Vice Chairman 

STEVE SCALISE, Louisiana 

ROBERT E. LATTA, Ohio 

CATHY MCMORRIS RODGERS, Washington 

GREGG HARPER, Mississippi 

LEONARD LANCE, New Jersey 

BRETT GUTHRIE, Kentucky 

PETE OLSON, Texas 

DAVID B. MCKINLEY, West Virginia 

MIKE POMPEO, Kansas 

ADAM KINZINGER, Illinois 

H. MORGAN GRIFFITH, Virginia 

GUS M. BILIRAKIS, Florida 

BILL JOHNSON, Ohio 

BILLY LONG, Missouri 

RENEE L. ELLMERS, North Carolina 

LARRY BUCSHON, Indiana 

BILL FLORES, Texas 

SUSAN W. BROOKS, Indiana 

MARKWAYNE MULLIN, Oklahoma 

RICHARD HUDSON, North Carolina 

CHRIS COLLINS, New York 

KEVIN CRAMER, North Dakota 

FRANK PALLONE, JR., New Jersey 

Ranking Member 

BOBBY L. RUSH, Illinois 

ANNA G. ESHOO, California 

ELIOT L. ENGEL, New York 

GENE GREEN, Texas 

DIANA DEGETTE, Colorado 

LOIS CAPPS, California 

MICHAEL F. DOYLE, Pennsylvania 

JANICE D. SCHAKOWSKY, Illinois 

G.K. BUTTERFIELD, North Carolina 

DORIS O. MATSUI, California 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

JERRY MCNERNEY, California 

PETER WELCH, Vermont 

BEN RAY LUJA ́ N, New Mexico 

PAUL TONKO, New York 

JOHN A. YARMUTH, Kentucky 

YVETTE D. CLARKE, New York 

DAVID LOEBSACK, Iowa 

KURT SCHRADER, Oregon 

JOSEPH P. KENNEDY, III, Massachusetts 

TONY CA ́ RDENAS, California 

SUBCOMMITTEE ON HEALTH

JOSEPH R. PITTS, Pennsylvania 

Chairman 

BRETT GUTHRIE, Kentucky 

Vice Chairman 

JOHN SHIMKUS, Illinois 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 

MARSHA BLACKBURN, Tennessee 

CATHY MCMORRIS RODGERS, Washington 

LEONARD LANCE, New Jersey 

H. MORGAN GRIFFITH, Virginia 

GUS M. BILIRAKIS, Florida 

BILLY LONG, Missouri 

RENEE L. ELLMERS, North Carolina 

LARRY BUCSHON, Indiana 

SUSAN W. BROOKS, Indiana 

CHRIS COLLINS, New York 

JOE BARTON, Texas 

FRED UPTON, Michigan (ex officio) 

GENE GREEN, Texas 

Ranking Member 

ELIOT L. ENGEL, New York 

LOIS CAPPS, California 

JANICE D. SCHAKOWSKY, Illinois 

G.K. BUTTERFIELD, North Carolina 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

DORIS O. MATSUI, California 

BEN RAY LUJA ́ N, New Mexico 

KURT SCHRADER, Oregon 

JOSEPH P. KENNEDY, III, Massachusetts 

TONY CA ́ RDENAS, California 

FRANK PALLONE, JR., New Jersey (ex 

officio) 

(III) 

C O N T E N T S 

Page 

Hon. Joseph R. Pitts, a Representative in Congress from the Commonwealth 

of Pennsylvania, opening statement ................................................................... 1 

Prepared statement .......................................................................................... 2 

Hon. Gene Green, a Representative in Congress from the State of Texas, 

opening statement ................................................................................................ 4 

Hon. Marsha Blackburn, a Representative in Congress from the State of 

Tennessee, opening statement ............................................................................ 5 

Hon. G.K. Butterfield, a Representative in Congress from the State of North 

Carolina, opening statement ............................................................................... 6 

WITNESSES

Jonathan Leffert, M.D., FACP, FACE, ECNU, President-Elect, American Association of Clinical Endocrinologists ................................................................. 8 

Prepared statement .......................................................................................... 10 

Arthur Dean, Chairman and CEO, Community Anti-Drug Coalitions of America .......................................................................................................................... 16 

Prepared statement .......................................................................................... 18 

Sonja L. Banks, President and COO, Sickle Cell Disease Association of America, Inc. .................................................................................................................. 29 

Prepared statement .......................................................................................... 31 

R. Sean Morrison, M.D., Professor and Vice Chair, Brookdale Department 

of Geriatrics and Palliative Medicine, Icahn School of Medicine at Mount 

Sinai, and Director, National Palliative Care Research Center ....................... 41 

Prepared statement .......................................................................................... 43 

Brad Marino, M.D., MPP, MSCE, Chair Pediatric Congenital Heart Association ........................................................................................................................ 60

Prepared statement .......................................................................................... 62 

SUBMITTED MATERIAL

Documents for the record submitted by Mr. Pitts ................................................ 95 

Article entitled, ‘‘Current Management of Sickle Cell Anemia,’’ Cold Spring 

Harbor Perspectives in Medicine, 2013, submitted by Mr. Burgess ................ 124 

Documents for the record submitted by Mr. Bilirakis .......................................... 141 

Documents for the record submitted by Mr. Engel ............................................... 155 

(1) 

EXAMINING LEGISLATION TO IMPROVE 

PUBLIC HEALTH 

THURSDAY, SEPTEMBER 8, 2016 

HOUSE OF REPRESENTATIVES, SUBCOMMITTEE ON HEALTH, COMMITTEE ON ENERGY AND COMMERCE, Washington, DC. 

The subcommittee met, pursuant to call, at 10:00 a.m., in room 

2322, Rayburn House Office Building, Hon. Joseph R. Pitts (chairman of the subcommittee) presiding. 

Present: Representatives Pitts, Guthrie, Barton, Burgess, Blackburn, Lance, Bilirakis, Long, Ellmers, Bucshon, Brooks, Collins, 

Green, Engel, Schakowsky, Butterfield, Schrader, Kennedy, 

Cardenas, and Pallone (ex officio). 

Also Present: Representative Roybal-Allard. 

Staff Present: Paul Edattel, Chief Counsel, Health; Adrianna 

Simonelli, Professional Staff Member, Health; Heidi Stirrup, 

Health Policy Coordinator; Waverly Gordon, Minority Professional 

Staff Member; Tiffany Guarascio, Minority Deputy Staff Director 

and Chief Health Advisor; and Samantha Satchell, Minority Policy 

Analyst. 

OPENING STATEMENT OF HON. JOSEPH R. PITTS, A REPRESENTATIVE IN CONGRESS FROM THE COMMONWEALTH 

OF PENNSYLVANIA 

Mr. PITTS. The subcommittee will come to order. The chair will 

recognize himself for an opening statement. Today’s hearing will 

examine several different legislative proposals that will address 

various aspects of the Public Health Service Act. 

H.R. 1192, the National Diabetes Clinical Care Commission Act, 

amends the Public Health Service Act to foster more effective implementation and coordination of clinical care for people with 

prediabetes and the chronic diseases and conditions that result 

from diabetes. 

Today, our witnesses will also be discussing potential changes to 

legislation that will make it less disease-specific, so the focus can 

be broader, to include related autoimmune and metabolic syndromes. According to the Centers for Disease Control and Prevention, CDC, almost 29 million Americans have diabetes, and an estimated 86 million American adults have prediabetes. Diabetes is 

the seventh leading cause of death in the United States. It is the 

leading cause of kidney failure. The total national cost associated 

with diabetes in 2012, according to the CDC, exceeded $245 billion. 

One in three Medicare dollars is currently spent upon people with 

2 

diabetes. There are 35 Federal departments, agencies, and offices 

involved with implementation of Federal diabetes activities. And 

this legislation will establish a commission to evaluate, recommend 

solutions for better coordination of patient care and ways to control 

costs across all of these offices. 

And I thank my colleague, Representative Pete Olson, for sponsoring this important legislation, which will be welcome news for 

the over 100 million people afflicted with diabetes or prediabetes. 

H.R. 1717, the Sober Truth on Preventing Underage Drinking 

Reauthorization Act, or the STOP Act, sponsored by Representative 

Roybal-Allard of California, provides for programs and activities to 

prevent underage drinking. 

H.R. 1807, the Sickle Cell Disease Research, Surveillance, Prevention and Treatment Act of 2015, sponsored by Representative 

Danny Davis of Illinois and Dr. Michael Burgess of Texas, would 

reauthorize the Sickle Cell Disease Demonstration Program. Sickle 

cell disease has no cure. It leads to premature death. This legislation will hopefully move us one step closer to improving the quality 

of care and symptom management for those afflicted. 

H.R. 3119, the Palliative Care and Hospice Education Training 

Act, sponsored by Representative Engel of New York, increases the 

number of permanent faculty in palliative care at accredited 

allopathic and osteopathic medical schools, nursing schools, social 

work schools, to promote education and research in palliative care 

and hospice and to support the development of faculty careers in 

academic palliative medicine. 

H.R. 3952, the Congenital Heart Futures Reauthorization Act of 

2015, sponsored by Representative Bilirakis of Florida, coordinates 

Federal congenital heart disease research efforts and improves 

public education and awareness of congenital heart disease. 

Today, we will hear from one panel of experts and stakeholders 

as to their ideas and recommendations on these various bills. 

We welcome all of you, and I now yield to Dr. Burgess. 

[The prepared statement of Mr. Pitts follows:] 

PREPARED STATEMENT OF HON. JOSEPH R. PITTS

The Subcommittee will come to order. 

The Chairman will recognize himself for an opening statement. 

Today’s hearing will examine several different legislative proposals that will address various aspects of the Public Health Service Act. 

H.R. 1192, the ‘‘National Diabetes Clinical Care Commission Act’’ amends the 

Public Health Service Act to foster more effective implementation and coordination 

of clinical care for people with pre-diabetes, diabetes, and the chronic diseases and 

conditions that result from diabetes. Today, our witness will also be discussing potential changes to the legislation that will make it less disease specific so the focus 

can be broader to include related autoimmune and metabolic syndromes. 

According to the Centers for Disease Control and Prevention (CDC), almost 

29,000,000 Americans have diabetes and an estimated 86,000,000 American adults 

have pre-diabetes. Diabetes is the seventh leading cause of death in the United 

States and is the leading cause of kidney failure. 

Total national costs associated with diabetes in 2012 according to the CDC exceeded $245 billion. One in three Medicare dollars is currently spent on people with 

diabetes. 

There are 35 Federal departments, agencies, and offices involved with implementation of Federal diabetes activities. This legislation will establish a commission to 

evaluate and recommend solutions for better coordination of patient care and ways 

to control costs across all of these offices. 

3 

I thank my colleague, Rep. Pete Olson, for sponsoring this important legislation 

which will be welcome news for the over 100,000,000 people afflicted with diabetes 

or pre-diabetes. 

H.R. 1717, the Sober Truth on Preventing Underage Drinking Reauthorization 

Act or the STOP Act, sponsored by Rep. Roybal-Allard (CA) provides for programs 

and activities to prevent underage drinking. 

H.R. 1807, the Sickle Cell Disease Research, Surveillance, Prevention, and Treatment Act of 2015, sponsored by Rep. Danny Davis (IL) and Rep. Michael Burgess 

(TX) reauthorizes a sickle cell disease demonstration program. Sickle cell disease 

has no cure and leads to premature death. This legislation will hopefully move us 

one step closer to improving the quality of care and symptom management for those 

afflicted. 

H.R. 3119, the Palliative Care and Hospice Education and Training Act, sponsored by Rep. Engel (NY) increases the number of permanent faculty in palliative 

care at accredited allopathic and osteopathic medical schools, nursing schools, social 

work schools, to promote education and research in palliative care and hospice, and 

to support the development of faculty careers in academic palliative medicine. 

H.R. 3952, the Congenital Heart Futures Reauthorization Act of 2015, sponsored 

by Rep. Bilirakis (FL) coordinates federal congenital heart disease research efforts 

and improves public education and awareness of congenital heart disease. 

Today we will hear from one panel of experts and stakeholders as to their ideas 

and recommendations on these various bills. 

I will now yield to Dr. Burgess. 

Mr. BURGESS. Thank you, Mr. Chairman. 

And it is with a great deal of pleasure that I recognize from my 

neck of the woods, Dr. Leffert being here today. He is the president-elect of the American Association of Clinical Endocrinologists, 

and in another life, I used to refer patients to Dr. Leffert from my 

practice in Louisville, Texas, down to the big city specialist. 

So, Jonathan, it is great to see you again. It is great to have you 

here as part of this committee. 

And Ms. Banks, who is here on behalf of the Sickle Cell Disease 

Foundation, who will be talking about the disease that Danny 

Davis and I, Representative Davis and I, introduced a bill. We are 

going to be looking at legislation that seeks to identify and improve 

the overall public health of our country, and one of those bills is 

1807, the Sickle Cell Disease Research, Surveillance, Prevention 

and Treatment Act of 2015. It was back in the middle 1970s when 

I was a resident at Parkland Hospital, our obstetric service there, 

Dr. Jack Pritchard, Dr. Eric Cunningham, sickle cell disease in 

pregnancy was a special project that they put a lot of effort into. 

And as a consequence, we ended up seeing a lot of patients referred 

from around the country to the program there at Parkland Hospital 

in the middle 1970s. It had been some time since I thought about 

it again, and then, with Representative Davis at one of your meetings here on the Hill, it really struck how there really hadn’t been 

the advancements in this area that I thought there would have 

been by this time. So that is one of the things that this committee 

has been very active in the Cures for the 21st Century. We want 

those things that are supposed to be there by now, and I would include this as one of those things that we want to be there by now. 

So thank you, Mr. Chairman. I will back and await the discussion. 

Mr. PITTS. The chair thanks the gentlemen and now yields to the 

ranking member of the subcommittee, Mr. Green of Texas, 5 minutes for an opening statement. 

4 

OPENING STATEMENT OF HON. GENE GREEN, A 

REPRESENTATIVE IN CONGRESS FROM THE STATE OF TEXAS 

Mr. GREEN. Thank you, Mr. Chairman. 

I want to welcome our panel today, and thank you for taking 

your time to come before us. We are examining five pieces of legislation that will improve public health and build on this committee’s 

record of advancing and enacting very bipartisan bills. I want to 

thank the chairman for calling the hearing and our witnesses for 

being here with us this morning. 

H.R. 1192, the National Diabetes Clinical Care Commission Act, 

was introduced and championed by my colleague on our committee 

but also my neighbor in Houston, Pete Olson, Congressman Olson, 

and Dave Loebsack, who is also a member of our committee. We 

would be considering a manager’s amendment on H.R. 1192, which 

would establish a national clinical care commission to evaluate and 

offer recommendations to improve care, leverage resources, and coordinate efforts around complex metabolic, autoimmune, and insulin-related diseases. Through innovation and collaboration and 

maximizing return on investment, this important legislation provides the opportunity to address the enormous economic and 

human impact caused by diabetes and other disorders, and I am 

proud to be a cosponsor of this legislation. 

H.R. 1717, the Sober Truth on Preventing Underage Drinking 

and Reauthorization Act, or the STOP Act, was introduced by Representative Lucille Roybal-Allard. She has been a tireless champion 

for this issue. In fact, she has talked to me—I think we came to 

Congress in 1993—and she has talked to me all of the time since 

then about trying to deal with drunk driving. I want to recognize 

her, as she is here, and thank her for her leadership. 

The STOP Act will build on successful efforts to reduce underage 

drinking by reauthorizing a number of important public health programs and add an additional component of screening and intervention. 

H.R. 1807, the Sickle Cell Disease Research, Surveillance, Prevention and Treatment Act, will enhance our ability to understand 

and survey and treat sickle cell disease. Sickle cell disease is a 

group of inherited red blood disorders that affect approximately 

100,000 Americans. Unfortunately, it is difficult to diagnose, as 

symptoms can be severe, and the treatment requires comprehensive and complex care. 

H.R. 1807, introduced by Representative Danny Davis and Mike 

Burgess, also on our committee, will promote research and prevention and treatment and emphasizes collaboration of communitybased entities focusing on sickle cell disease. 

H.R. 3119, Palliative Care and Hospice Education and Training 

Act, is an important bill to improve palliative care. Representative 

Eliot Engel, also a member of our committee, introduced this legislation, recognizing that palliative care enhances the quality of life 

for individuals with serious and life-threatening disease by treating 

the symptoms, the side effects, and emotional pain experienced by 

patients. H.R. 3119 would improve training for health professionals, enhance research in palliative and hospice care, and support projects to fund the training of physicians and nonphysician 

healthcare professionals entering the field of palliative care. 

5 

Finally, we are considering H.R. 3952, the Congenital Heart Futures Reauthorization Act. Each year, approximately 4,000 babies 

are born with congenital heart defects, making it the most common 

type of birth defect in the United States. It is estimated that 1 million children and 1.4 million adults live with congenital heart disorders. They require specialized care and face a lifelong risk of disability and premature death. The cause is unknown, but several genetic and environmental factors have been linked in the diseases. 

H.R. 3952, introduced by Representative Gus Bilirakis, also from 

our committee, and Adam Schiff and Eleanor Holmes Norton, 

builds on existing efforts by requiring the Centers for Disease Control and Prevention to enhance and expand its research, surveillance, and education outreach to providers and the public about 

congenital heart diseases. Under this legislation, the CDC would 

report to Congress on a cohort study to improve the knowledge of 

epidemiology of the disease across lifespans and implement an 

awareness campaign. I am proud to support each of these important bills and thank our sponsors and our committee for their commitment to improving public health and look forward to hearing 

from our witnesses and learning more about each of these bipartisan pieces of legislation. 

Mr. Chairman, unless someone else wants my last 30 or 40 seconds, I will yield back. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize the vice chair of the full committee, the 

gentlelady from Tennessee, Ms. Marcia Blackburn, for 5 minutes 

for opening statement. 

OPENING STATEMENT OF HON. MARSHA BLACKBURN, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF TENNESSEE 

Mrs. BLACKBURN. Thank you, Mr. Chairman. 

I want to thank our witnesses for being here. 

And I am so pleased that we are taking some time to go through 

these bills. They all have a significance to public health. The diabetes bill, I had the opportunity before coming to Congress to serve 

on the Diabetes Association Board in my state, and I appreciate 

Representative Olson’s good work on looking at this issue. 

Of course, the underage drinking bill, when I was in the state 

senate, this is something at the state level where we put a tremendous amount of effort and energy, and Tennessee, my home State, 

continues to do so. I know that Reps. Davis and Burgess have 

worked hard on the sickle cell disease. It does need more attention. 

It needs more research. It needs more focus, so we are pleased to 

see that. 

We all have heard about palliative care, the importance of that, 

as we have been at home and in our districts and talked to families 

and to healthcare providers and beginning to think this through 

and look for new models. And certainly, in Nashville, we have a 

tremendous amount of research and new focus that has come to 

bear on the delivery of palliative care. So I am pleased that we are 

moving forward there. You know, I have to say that H.R. 3952, 

going back to my days as being on the Board of Friends of Children’s Hospital in Nashville and dealing with congenital heart disVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00009 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
6 

ease, and as a mom and a grandmom now, having friends and 

knowing of families, individuals from church, that have a baby that 

is born with CHD, and just seeing firsthand, living through the anguish and the desperate reach for resources that can help with 

this, that can extend the life of that child. I am so pleased that we 

are moving this forward. I do hope that we will see NIH and other 

research entities focus on how we deal with this so that these precious children will live long past that 18th birthday and will be 

able to move toward enjoying a full and productive life. 

Mr. Chairman, I will yield my time to whomever would like it. 

Mr. PITTS. Anyone on the majority seeking time? 

If not, the gentlelady yields back. 

The chair now recognizes Judge Butterfield, North Carolina, 5 

minutes for opening statement in place of Mr. Pallone. 

OPENING STATEMENT OF HON. G.K. BUTTERFIELD, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF NORTH 

CAROLINA 

Mr. BUTTERFIELD. Thank you. I too want to thank you, Chairman Pitts, for holding this important hearing on reauthorizing important programs to combat the sickle cell disease and on improving diabetes awareness and care. Let me join my colleagues in 

thanking the five witnesses for coming forward today to testify and 

to give us the benefit of your expertise. I know you just didn’t wake 

up this morning and come to this room. You have been preparing 

for this day, and we thank you for your work. 

Sickle cell disease and diabetes disproportionately affect African 

American citizens, including many in my congressional district in 

North Carolina. In fact, more than 30 years ago, I lost a first cousin to the disease. The two diseases, if not properly managed, can 

land people in the hospital multiple times. In fact, a 2010 study 

published in the journal of the American Medical Association shows 

that people with SCD are hospitalized nearly three times per year. 

Many people who have SCD are unaware of it before tests can confirm the illness. Even those who know they have SCD find themselves back in the hospital with problems with pain or other 

morbidities. SCD is a serious disease which can dramatically reduce life expectancy. A study in the New England Journal of Medicine found that the median age for men with SCD is only 42 years 

old. For women, it is 48 years. The disease is caused by a small 

genetic abnormality that deforms blood cells and causes them to 

block blood flow. SCD can lead to the development of other conditions, ranging from heart disease and stroke to kidney or liver 

problems. 

It is estimated, Mr. Chairman, that 100,000 Americans have 

SCD. Many more have sickle cell trait, although they never experience symptoms, may not even know that they have the trait. Children can inherent SCD if both of their parents have SCD or sickle 

cell trait and certain genes are passed on to them. Many of those 

who have SCD are African Americans. More than 1 out of every 

400 African Americans have SCD. That is 1 out of 400, and 1 of 

every 13 has the sickle cell trait. 

And so I applaud my friend and colleague Congressman Danny 

Davis and other colleagues that have done likewise—from ChiVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00010 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
7 

cago—for his longtime advocacy for SCD health programs and his 

reintroduction of H.R. 1807, the Sickle Cell Disease Research, Surveillance, Prevention and Treatment Act of 2015. This legislation 

is a priority for many members of the Congressional Black Caucus, 

and I am proud to support it. And this important bill would reauthorize the sickle cell disease Treatment Demonstration Program, 

improve research and surveillance of the disease, and support a 

grant program for states to develop and implement prevention and 

treatment strategies. 

This bill, Mr. Chairman, is a clean reauthorization and would not 

increase government spending. It is a meaningful first step to help 

prevent and treat SCD, and I urge my colleagues to support this 

important bill. We can, we must do more to support those with this 

disease. SCD does not currently have a cure. No treatments have 

been approved since 1998. For that reason, I have long advocated 

to include SCD in the Pediatric Priority Review Voucher Program, 

the PRV, run by the Food and Drug Administration. I am encouraged that there is currently a viable treatment in clinical trials at 

the FDA, but we must do all we can to spur innovation in rare pediatric disease spaces. More than 100,000 Americans are counting 

on us to support sickle cell disease prevention and treatment programs and need our help to find a cure. I urge my colleagues to 

support 1807, and I yield back. Thank you. 

Mr. PITTS. The chair thanks the gentleman. 

That concludes the opening statements. As usual, the written 

opening statements of all members will be made a part of the 

record. 

And we welcome, as was noted previously, Congresswoman Roybal-Allard to sit with the committee today. 

Without objection, so ordered. 

And I ask unanimous consent to submit the following for the 

record: a letter from 43 organizations representing physicians, allied health professionals, patients, community health organizers, 

and industry; as well as statements from the Academy of Nutrition 

and Dietetics, Novo Nordisk, and Diabetes Advocacy Alliance, all 

regarding H.R. 1192; and both a statement from the American Society of Hematology regarding H.R. 1807 along with their State of 

Sickle Cell Disease 2016 Report. 

Without objection, so ordered. 

[The information appears at the conclusion of the hearing.] 

Mr. PITTS. I will now introduce the panelists in the order of 

which they will speak, and as usual, your written statements will 

be made a part of the record, and you will each be recognized for 

5 minutes for a summary. 

So, first, Dr. Jonathan Leffert, Acting Director and Presidentelect of the American Association of Clinical Endocrinologists; secondly, General Arthur Dean, Chairman and CEO, Community 

Anti-Drug Coalitions of America; and Sonja L. Banks, President 

and COO of Sickle Cell Disease Association of America, Inc.; then 

Dr. Sean Morrison, Professor and Vice Chair, Brookdale Department of Geriatrics and Palliative Medicine, Icahn School of Medicine at Mount Sinai, and Director of the National Palliative Care 

Research Center; and, finally, Dr. Brad Marino, Chair, Pediatric 

Congenital Heart Association. 

8 

Thank you for coming today. 

And, Dr. Leffert, you are recognized 5 minutes for your summary 

at this time. 

STATEMENTS OF JONATHAN LEFFERT, M.D., FACP, FACE, 

ECNU, PRESIDENT-ELECT, AMERICAN ASSOCIATION OF 

CLINICAL ENDOCRINOLOGISTS; ARTHUR DEAN, CHAIRMAN 

AND CEO, COMMUNITY ANTI-DRUG COALITIONS OF AMERICA; SONJA L. BANKS, PRESIDENT AND COO, SICKLE CELL 

DISEASE ASSOCIATION OF AMERICA, INC.; R. SEAN MORRISON, M.D., PROFESSOR AND VICE CHAIR, BROOKDALE DEPARTMENT OF GERIATRICS AND PALLIATIVE MEDICINE, 

ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI, AND DIRECTOR, NATIONAL PALLIATIVE CARE RESEARCH CENTER; AND 

BRAD MARINO, M.D., MPP, MSCE, CHAIR PEDIATRIC CONGENITAL HEART ASSOCIATION 

STATEMENT OF JONATHAN LEFFERT 

Dr. LEFFERT. Thank you, Mr. Chairman, and, Dr. Burgess, for 

your kind words. My name is Jonathan Leffert, and I am a clinical 

endocrinologist from Dallas, Texas, and the current president-elect 

of the American Association of Clinical Endocrinologists. On behalf 

of our 7,000 members, I would like to thank you for this opportunity to testify about H.R. 1192, the National Diabetes Clinical 

Care Commission Act. The subcommittee should be commended for 

addressing diabetes and recommending to expand the scope of H.R. 

1192 to include other metabolic and autoimmune diseases and diseases resulting from insulin deficiency and insulin resistance. We 

appreciate the opportunity to work with the bill’s sponsors, Representative Pete Olson and Representative Dave Loebsack, in this 

subcommittee on consensus language to amend H.R. 1192. 

I will focus my comments today on diabetes, which represents a 

significant part of my medical practice as a clinical endocrinologist 

and is the most prevalent of the diseases that will be addressed by 

an amended H.R. 1192. 

According to the Centers for Disease Control and Prevention, the 

number of Americans diagnosed with diabetes over the course of 

the last 35 years has increased more than fivefold, from 5.5 million 

Americans in 1980 to 29.1 million in 2014. The CDC estimates that 

there are 86 million Americans with prediabetes, a condition 

known to progress to diabetes without appropriate intervention. Diabetes is also the catalyst for many other diseases. Diabetes is the 

leading cause of new cases of blindness among adults. Diabetes is 

the leading cause of kidney failure. Diabetes causes increased 

death rates from cardiovascular disease and higher rates of hospitalization from heart attack and stroke. Diabetes is the seventh 

leading cause of death in the United States. The total cost of diabetes to the Nation in 2012 exceeded $322 billion. Sixty-two percent 

of this cost is borne by the U.S. Government through programs like 

Medicare and Medicaid. By 2025, the total cost of diabetes is projected to reach $514 billion, a level comparable to the entire Medicare budget. Our Nation cannot afford for the current diabetes 

prevalence and cost trends to continue. Congress should not let another session go by without addressing this critical health crisis. 

9 

H.R. 1192 provides a cost-effective approach to begin to address 

diabetes and the many other diseases and diagnoses encompassed 

by this legislation. The commission established in H.R. 1192 will 

provide a venue where the expertise of specialists, primary care 

physicians, allied healthcare professionals, and patient advocates 

will help our Federal Government partner to evaluate current programs so they are meeting the goal of improving the quality of patient care. 

The commission will also facilitate improved coordination and 

communication among Federal agencies. Consider the example of 

the FDA approved continuous glucose monitors, referred to as 

CGMs. These devices are indispensable to patients with type 1 diabetes by allowing them to constantly monitor blood glucose levels. 

Patients with this device no longer fear losing consciousness from 

low blood sugar or enduring complications from constantly high 

blood sugar levels. Nearly all private insurance plans cover CGMs. 

However, once a patient turns 65 and enrolls in the Medicare program, coverage for this lifesaving device is no longer available. 

Remedies to fix these issues often require an act of Congress, 

which places Congress in a position to do the job of the regulatory 

agencies that failed to work together. 

Having the commission available to work through issues such as 

this will help all parties to find and implement meaningful solutions. The expertise on the commission would also be utilized to 

prioritize the clinician training and deployment of new revolutionary technologies, such as the artificial pancreas, to ensure patient access to these medical innovations is not comprised. 

Agencies can and must work together in a coordinated national 

response driven by research experts, specialists, healthcare professionals, and people living with diabetes. The commission established under H.R. 1192 will help achieve this important objective. 

On behalf of the American Association of Clinical 

Endocrinologists, I would like to thank the members of the committee for the opportunity to testify today on H.R. 1192, and I urge 

you to act now and move this bill forward, ensuring its passage by 

the U.S. House of Representatives as soon as possible. 

In addition to the 220 Members of Congress who have cosponsored H.R. 1192, including many who are members of this committee, I would like to thank the 45 organizations representing the 

patients, physicians, allied health professionals, community organizations, and industry, and the Diabetes Advocacy Alliance, who 

have helped to advance this legislation. 

[The prepared statement of Dr. Leffert follows:] 

10 


22416.001

~merican ~ssociation of cGnica{ ~ndocrinofoaists 

~1.~.1~!\~~-~t!~ :~-c~_th.~"· _;:~~!~- :.:.o~J_ .. Ja_~:k~~:!~:-~~-~-~~t~ -~~~~3_: !~1~:. r!S:~i-~~?~7~ · ~":_t~:~-~ 3E-3·S~E-·_ :-:.~~~~~~~~:_~_!_ 

TESTIMONY 

of 

Jonathan D. Leffert, MD, FACP, FACE, ECNU 

on Behalf of 

American Association of Clinical Endocrinologists 

before the 

U.S. House of Representatives Committee on Energy & Commerce 

Subcommittee on Health 

September 8, 2016 

Thank you Mr. Chairman. My name is Jonathan Leffert, and I am a clinical 

endocrinologist from Dallas, Texas and the current President-Elect of the American Association 

of Clinical Endocrinologists (AACE). On behalf of the 7,000 members of the AACE, I would 

like to thank you for this opportunity to testify about H.R. 1192, the National Diabetes Clinical 

Care Commission Act, which is sponsored by Representatives Pete Olson (R-TX) and Dave 

Loebsack (D-IA), and the necessity that this bipartisan legislation be enacted as soon as possible. 

The Subcommittee should be commended for addressing this devastating gateway disease and 

for broadening the scope of this bill by offering an amendment, should it be considered by this 

Subcommittee later this year, to include other metabolic and autoimmune disease, diseases 

resulting from insulin deficiency and insulin resistance and their complications. We appreciated 

the opportunity to work with our sponsors and this Subcommittee on the language for this 

amendment. 
11 


22416.002

However, I am going to focus my comments today on diabetes, which represents a 

significant part of my medical practice as a clinical endocrinologist and is one of the most 

prevalent of the diseases that will be addressed by an amended H.R. 1192. 

According to the Centers for Disease Control and Prevention (CDC), the number of 

Americans with diagnosed diabetes over the course of the last 35 years has increased more than 

five-fold, from 5.5 million Americans in 1980 to 29.1 million in 2014. The CDC estimates there 

are 86 million Americans with pre-diabetes, a condition known to progress to diabetes without 

appropriate intervention. 

Diabetes is also the catalyst for many of the other diseases in these disease clusters that 

will be addressed by this legislation as amended, such as cardiovascular disease and chronic 

kidney disease. 

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) defines 

diabetes as "a complex group of diseases with a variety of causes. People with diabetes have 

high blood glucose, also called high blood sugar or hyperglycemia. Over time, high blood 

glucose damages nerves and blood vessels, leading to complications such as heart disease, 

stroke, kidney disease, blindness, dental disease and amputations. 

The role that diabetes plays in the onset of many other diseases and debilitating 

conditions cannot be understated. 

• Diabetes is the leading cause of new cases of blindness among adults 

• Diabetes is the leading cause of kidney failure 

• 60% of all non-traumatic lower limb amputations in the U.S. occur in individuals 

with diabetes 

• 60-70% of individuals with diabetes have neuropathies or nerve disorders 

2 
12 


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• Individuals with diagnosed diabetes have increased death rates from 

cardiovascular disease and higher rates of hospitalization for heart attack and 

stroke 

Although many of the disease complications of diabetes are preventable, millions of 

Americans suffer the devastating consequences of disease progression, experiencing reduced 

quality of life, productivity and even death. Diabetes is the 7'h leading cause of death in the 

United States. 

As stewards of taxpayer funds and the federal budget, you arc probably aware of the 

economic impact of the diabetes epidemic. The total cost of diabetes to the nation in 2012 

exceeded $322 billion and 62% of this cost is borne by the U.S. government through programs 

like Medicare and Medicaid. By 2025, the total cost of diabetes is projected to reach $514 

billion a level comparable to the entire Medicare budget. Currently, $1 out of every $3 

Medicare dollars is spent on people with diabetes. 

It is not surprising that global diabetes trends arc mirroring the trajectory of the disease in 

the United States. An estimated 422 million adults were living with diabetes in 2014, compared 

to 108 million in 1980. So concerning is this trend that the World Health Organization's Global 

Report on Diabetes released earlier this year called on governments around the globe to take 

more aggressive steps in fighting the rising number of diabetes cases worldwide. 

I am sure you will agree with me that our nation cannot afford for the current diabetes 

prevalence and cost trends to continue. Maintaining the status quo with respect to this disease is 

not an option. Our country needs to better leverage the resources spent on medical research and 

patient care and use them more effectively to address diseases and disorders of the metabolic 

system, the autoimmune system and those due to insulin resistance. If not addressed, diabetes 

3 
13 


22416.004

and its disease complications have the very real potential of bankrupting the federal health 

programs that provide care to over 125 million Americans. 

While there is no "cure" at present for diabetes and its devastating complications and no 

single solution for addressing the disease, we have many tools that can improve the quality of 

lite for people with diabetes and its complications. Our healthcare system can and should do 

better for patients. Congress should not let another session go by without addressing this critical 

health crisis. 

By passing H.R. 1192, Congress will advance legislation that provides a cost-effective 

approach to begin to address diabetes and the many other disease diagnoses encompassed in this 

legislation. Through innovation, collaboration, and application of advances in care called for in 

this bill, the federal government can better leverage taxpayer dollars invested in diabetes research 

to reduce the staggering impact of diabetes and its disease complications on health care 

spending. At the same time, care will be improved for the tens of millions of Americans living 

with diabetes. 

The Commission established under this legislation will represent a partnership between 

the private sector experts who work with patients with diabetes on a daily basis and Federal 

agency representatives who are active in clinical diabetes care. The Commission will provide a 

venue where the expertise of specialists, primary care physicians, allied healthcare professionals 

and patient advocates will help our federal government partners to evaluate current programs to 

ensure they are meeting the goal of improving the quality of patient care delivered to people with 

diseases and disorders of the metabolic system, the autoimmune system and those due to insulin 

resistance. For example, improved coordination among agencies would make a difference in the 

lives of Medicare patients with Type l diabetes mellitus and their use of the continuous glucose 

4 
14 


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monitoring device (CGMs). CGMs detect and display real-time glucose levels in five minute 

intervals. This data is transmitted wirelessly, allowing individuals and those who care for them 

to constantly monitor glucose levels. This technology enables better blood glucose control and 

helps users live their life without the fear of losing consciousness from low blood sugar or 

enduring complications from constantly high blood sugar levels. These FDA-approved devices 

are indispensable for the patient with type I diabetes. Nearly all private insurance plans cover 

CGMs; however, once a patient turns 65 and enrolls in the Medicare program, coverage for the 

life-saving device is no longer available. A key tool in the patient's care plan "tool box" is 

suddenly removed, which places the patient's health and safety in jeopardy. Such short-sighted 

policies reflect poor communication and coordination of federal diabetes activities and a broken 

system that does not work for patients. Remedies to fix these issues often require an act of 

Congress, which places patients in an abyss and Congress in a position to do the job of the 

regulatory agencies that failed to work together. 

The CGM example suggests that the health care system must be better-prepared for the 

deployment of new technologies so they are accessible to patients as they are approved by the 

FDA. The coming years promise new device and medication innovations, such as the artificial 

pancreas, which will be pivotal for patients with diabetes. Having an effective, well-organized 

federal response to activate these innovations will help millions of Americans with this 

devastating disease. This will require the government to change the status quo of agencies 

working in silos to a coordinated national response, driven by research experts, specialists, health 

care professionals and people living with diabetes. 

5 
15 


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The Commission will not only help improve patient care through better communication 

and coordination among federal agencies, but it will also help facilitate the dissemination of 

innovative clinical practices and resources resulting from federal research to the physician 

practice at the local level, so patients have access to high-quality, optimal care. 

The Commission established under H.R. 1192 can play an important role in this effort as 

it provides the solution to the dual problem of cost and access in the federal government's 

response to patients with diabetes. We can attain this goal without allocating any new funds, as 

the cost of this Commission is to be paid out of the existing HHS budget. The bill language 

explicitly states that the cost of convening the Commission shall come from existing HHS funds. 

In addition to the 220 Members of Congress who have supported H.R. 1192, including 

many who are members of this Committee, I would like to thank the 45 organizations 

representing the patients, physicians, allied health professionals, community organizations and 

industry, including the Juvenile Diabetes Research Fund (JDRF) and the American Diabetes 

Association (ADA), and the Diabetes Advocacy Alliance (DAA) who have helped to advance 

this legislation. 

On behalf of AACE, I would like to thank the Members of this Committee for the 

opportunity to testify today on H.R. 1192 and I urge you to act now and move this bill forward, 

ensuring its passage by the U.S. House of Representatives as soon as possible. Please do not let 

the opportunity pass to do something to reverse the trajectory of these expensive and debilitating 

diseases and to improve the health and quality of life for patients with diabetes and its associated 

diseases and complications. 

6 
16 

Mr. PITTS. The chair thanks the gentleman for his summary and 

now recognizes General Dean 5 minutes for his summary. 

STATEMENT OF ARTHUR DEAN 

Mr. DEAN. Chairman Pitts, Ranking Member Green, and esteemed members of the subcommittee on Health, it is my pleasure 

to testify in support of H.R. 1717, the Sober Truth on Preventing 

Underage Drinking Act, so-called the STOP Act. 

I would also like to thank the bill’s sponsor, Congresswoman Lucille Roybal-Allard, for her steadfast leadership on this issue, and 

I thank you for the support and leadership you provided to us. 

I am General Arthur Dean, currently serving as the Chairman 

and CEO of Community Anti-Drug Coalitions of America, a membership based, not-for-profit organization, commonly called CADCA. 

CADCA is a national nonprofit organization, and our mission is to 

build and strengthen the capacity of local citizens, and we put 

them into what we call community coalitions designed for them to 

have the capacity and the skills to build safe, healthy, and drugfree communities in the U.S. and around the world. We work with 

more than 5,000 communities in the U.S., and it is our role to be 

here. And they are very, very concerned about the prevention and 

the reduction and combatting underage drinking. Back in 2003, the 

National Research Council and the Institute of Medicine published 

a report titled ‘‘Reducing Underage Drinking: A Collective Response.’’ This report cited serious underage drinking and recommended critical components for a national strategy to reduce alcohol consumption by minors. The original STOP Act builds upon 

these recommendations, and in 2016, the bill passed the House of 

Congress with a unanimous bipartisan support. Authorization for 

the law expired in 2010, as you know, which makes it urgent that 

Congress pass a reauthorization as soon as possible. 

H.R. 1717, the STOP Act reauthorization bill, would maintain 

and enhance the original provisions of the STOP Act, and quickly 

I would describe some of those things that we are concerned about. 

It reauthorizes the highly successful community-based coalition enhancement grants. We take trained coalitions and give them a 

small grant to work this issue. Provides grants for current and 

former drug-free community grantees and partners and allows 

them to partner with higher education to prevent underage drinking on college campuses. It reauthorizes the Interagency Coordinating Committee to Prevent Underage Drinking—we call that 

ICCPUD—which coordinates the efforts of 16 Federal agencies to 

combat this problem. It reauthorizes a highly visible national 

adult-oriented media campaign to raise the awareness of this issue 

and provide education. It reauthorizes epidemiology studies on excessive drinking and analyzes how young people drink and how 

they obtain alcohol in the relationship associated with that. 

And, lastly, 1717 creates a new grant program for pediatric 

healthcare providers. We think it is important that those that are 

treating our youth understand and focus on best practices around 

screening, brief intervention, and referral as appropriate. 

In the 10 years that have passed since STOP Act was created, 

it is clear that law’s coordinated provisions have effectively been reducing underage drinking. As we look at the most recent MoniVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00020 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
17 

toring the Future study, it shows that lifetime alcohol use by those 

in the 8th grade, 10th grade, and 12th grade is currently at the 

lowest level since each of these grades were included in the study. 

While this is welcome news—it is important news—underage 

drinking continues to be a very serious problem that is faced in 

this country: 17.2 percent, or nearly one in six, high school seniors 

still binge drink, which is unfortunate. Between 2006 and 2010, approximately 4,300 young people under the age of 21 died from excessive drinking, which is critical. And the total annual economic 

costs of underage drinking are estimated at $24.6 billion. 

So H.R. 1717 builds upon the effective data-driven, Drug-Free 

Communities Program as the most cost-effective way to prevent 

and reduce underage drinking. The community-based coalition enhancement grants included in the STOP Act are just one vital component of a comprehensive approach to improve public health and 

address underage drinking. 

I respectfully urge the committee to support swift passage of 

H.R. 1717, the Sober Truth on Preventing Underage Drinking Act. 

I will be submitting a detailed statement for the record, which includes information for your consideration. 

And we ask that, as in the past, this bill be unanimously supported and passed quickly so that it can become law during this 

session of Congress. I thank you so very much for your attention 

to this issue, and we understand that underage drinking, although 

data says we have made progress, there is much progress that still 

needs to be made, and many, many young people will be better 

served if this passes, takes place quickly. Thank you very much. 

[The prepared statement of Mr. Dean follows:] 

18 


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~CADC~ 

Written Statement of 

General Arthur T. Dean 

Chairman and CEO, Community Anti-Drug Coalitions of America (CADCA) 

for 

House Energy and Commerce Committee Hearing 

in support of H.R. 1717, the Sober Truth on Preventing Underage Drinking (STOP) Act 

September 8, 20 16 
19 


22416.008

Chairman Upton, Ranking Member Pallone, and esteemed Members of the Committee, it 

is my pleasure to testify today in support of H. R. 1717, the Sober Truth on Preventing Underage 

Drinking Act, also called the STOP Act. I am General Arthur Dean, Chairman and CEO of 

Community Anti-Drug Coalitions of America (CADCA). 

CADCA is a national non-profit whose mission is to build and strengthen the capacity of 

community coalitions to create safe, healthy, and drug-free communities globally. Working with 

the more than 5,000 member coalitions around the United States, CADCA has for a very long 

time been at the forefront of efforts to prevent, reduce, and combat underage drinking. 

Back in 2003, the National Research Council and Institute of Medicine published a report 

titled, "Reducing Underage Drinking: A Collective Responsibility." This report placed the 

seriousness of underage drinking into context for the American public, and recommended a 

number of critical components as part of a national strategy to reduce alcohol consumption by 

minors. The original STOP Act built upon these recommendations, and included all of them in the 

final bill that passed both Houses of Congress in an overwhelmingly bipartisan basis in 2006. 

CADCA, as well as other organizations as part of the National Alliance to Prevent 

Underage Drinking, were key supporters of getting the STOP Act passed at that time. 

Authorization for the law expired in 20 I 0, making it urgent that Congress pass a reauthorization 

as soon as possible, and the 21 national organizations that are members ofthe National Alliance 

to Prevent Underage Drinking have come out in strong support of this bill (see attachment 1 ). 
20 


22416.009

H.R. 1717, the STOP Act reauthorization bill, proposes to maintain and enhance the areas 

of policy development contained in the original STOP Act and adds an additional area, screening 

and brief intervention. It does all of this while staying within the existing overall authorized level 

of$18 million. Below arc detailed descriptions of each ofthe components ofH.R. 1717 (see 

attachment 2 for a detailed breakdown of comparisons of original authorization vs. reauthorization 

in H.R. 1717). 

COMMUNITY -BASED PROGRAMS TO PREVENT UNDERAGE DRINKING 

Community-Based Coalition Enhancement Grants ($6 million for each of FY 2016-2020) 

These are grants of up to $50,000 for up to four years for current and former Drug-Free 

Communities (DFC) grantees to enhance underage drinking prevention efforts. DFC grantees are 

well-placed to handle such enhancement grants because they are data-driven, they understand 

their community epidemiology, and are capable of implementing multi-sector interventions at 

preventing and reducing alcohol use. H.R. 1717 builds upon the effective, data-driven 

infrastructure of the Drug-Free Communities program as the most cost-effective 1 way to invest 

minimal federal dollars to prevent and reduce the number of youth who drink alcohol at the 

community level. As a condition of their grant, DFC grantees are required to engage in a 

tremendous amount of strategic planning, and therefore have identified the best practices and 

1 Swisher, .J.D., Scherer and Yin, I<_ The Journal of Primary Prevention. "Cost-Benetit Estimates in Prevention 

Research." 25:2, October 2004 
21 


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comprehensive, community-wide strategies that could be implemented with a minimal amount of 

resources. DFC grantees, and therefore STOP Act grantees, primarily focus on implementing 

strategies to ensure the entire community is targeted with comprehensive, multi-component 

strategies across all community sectors to change norms and reshape community environments to 

achieve population-level decreases in underage drinking rates. 

This community-based approach has been very successful (see attachment 3). The most 

recent independent national evaluation of the DFC program showed that, among all DFC grantees 

ever funded from first report to latest report, 30-day use of alcohol declined by 24.4%2

• with past 

30-day use of alcohol by high school students declining by 15.5%. 3 Individual STOP Act 

grantees, as well, have shown remarkable results: North Coastal Prevention Coalition, from 

the Tri-City area of San Diego County in California, markedly decreased past 30-day alcohol use 

and binge drinking 11th grade students between 2008 and 2011 (see attachment 4). The Coalition 

for Healthy and Safe Families, another STOP Act grantee, from Newton, New Jersey, showed a 

decrease in past 30-day use of alcohol among high school seniors from 63% in 2010 to 51.9% in 

2012 (see attachment 5). 

Some example strategies that grantees have engaged in include: 

Conducting community-wide education campaigns 

• Conducting vendor and server training 

2 Available here: 

h ttps://ww w. whitchouse.gov/sites/default/files/DFC20 I 41 ntcrim%20ReportExecutiveSummaryFinal.pdf 

3 Ibid. 
22 


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Conducting compliance checks 

• Mobilizing the community to restrict youth access to alcohol 

Strengthening and enforcing underage sales/services laws 

There is tremendous interest in and demand for these grants by the substance abuse 

prevention field, as can be seen from the chart below: 

Number of Coalition Enhancement Grants Awarded 

Number of Applicants for Number of New Grants 

Fiscal Year Appropriated Level New Grants Awarded 

2008 $4 million 419 79 

2009 $5 million 427 23 

2010 $5 million 0 0 

2011 $5 million 0 0 

2012 $5 million 364 81 

2013 $5 million 247 17 

2014 $5 million 0 0 

2015 $5 million 0 0 

2016 $5 million 0 0 

GRANTS FOR PARTNERSHIPS BETWEEN COMMUNITY COALITIONS AND 

INSTITUTIONS OF HIGHER EDUCATION ($2.5 million for each ofFY 2016-2020) 
23 


22416.012

This provision provides grants to current and former DFC grantees to partner with institutions of 

higher education to prevent and reduce the rate of underage alcohol consumption, including binge 

drinking on college campuses. Grants may be awarded up to $100,000 per year for up to four 

years. Coalitions will work hand in hand with institutions of higher education to reduce the 

consumption and abuse of alcohol through comprehensive, community-wide and evidence-based 

strategies that change environments and norms. Specific focus is on reducing incidents of alcohol 

use and abuse for students under 21 and to include the identification of incidents of violations, 

physical assaults, sexual assaults, reports of intimidation, disruption of school functions, 

disruption of students' studies, mental health referrals, illnesses, alcohol-related visits to 

emergency departments and deaths. Note that this modifies the existing authorization by 

changing eligible applicants from a State, institution of higher education, or non-profit entity to 

DFC coalitions, and cuts the funding level from $5 million to $2.5 million (see attachment 2 for 

more information). 

INTERAGENCY COORDINATING COMMITTEE TO PREVENT UNDERAGE 

DRINKING (ICCPUD); ANNUAL. REPORT ON UNDERAGE DRINKING 

PREVENTION AND ENFORCEMENT ACTIVITIES ($1 million for each ofFY 2016-

2020) 

H.R. 1717 reauthorizes the Interagency Coordinating Committee to Prevent Underage Drinking 

(ICCPUD), which coordinates the efforts and expertise of sixteen federal agencies to combat 
24 


22416.013

underage drinking. It was formed in 2004 to assist in the development of" A Comprehensive 

Plan for Preventing and Reducing Underage Drinking", which Congress called for in 2004. The 

STOP Act, enacted in 2006, formally established ICCPUD and called for enhancing its 

prevention efforts. Its purpose is, in addition to coordinating federal efforts, to provide ongoing, 

high-level leadership and guidance on policy and program developments across the federal 

government with respect to underage drinking; and also writes an Annual Report to Congress 

with a comprehensive assessment of federal efforts and the status of underage drinking 

prevention in the country, including state-specific information on reducing underage drinking 

(see attachment 6 and 7). 

NATIONAL ADULT-ORIENTED MEDIA CAMPAIGN TO PREVENT UNDERAGE 

DRINKING ($1 million for each FY 2016-2017 and such sums as necessary for FY 2018-

2020) 

The highly-visible National Adult-Oriented Media Campaign is meant to raise awareness about 

the issue of underage drinking. Carried out through the Substance Abuse and Mental Health 

Services Administration (SAMHSA), the online presence of the media campaign can be found 

on www.samhsa.gov/under-drinking. The current campaign, titled, "Talk. They Hear You", aims 

to increase parents' awareness of the prevalence and risk of underage drinking; equip parents 

with skills and understanding to prevent underage drinking; and to increase parents' taking 

action to prevent underage drinking. (see attachment 8) 
25 


22416.014

EPIDEMIOLOGICAL STUDIES ON EXCESSIVE AND UNDERAGE DRINKING ($4.5 

million for each of FY 2016-2020) 

Reauthorizes epidemiological studies on excessive drinking and underage alcohol use to provide 

better data in support of evaluation of community and other efforts to reduce and prevent 

underage drinking. The work, done at the CDC, is improving the collection and usefulness of 

data in support of effective and comprehensive community-based approaches to underage 

drinking by funding dedicated alcohol epidemiologists in at least five states. Drawing on its 

Youth Risk Behavior Surveillance System, it is analyzing and reporting more precise 

information than is currently available on how young people drink, where they obtain alcohol, 

and the relationship between youth alcohol use and a wide range of risk behaviors including 

suicide, drinking-driving, physical fighting, and risky sexual behavior. CDC uniquely tracks the 

cost of underage drinking at the federal and state levels, and monitors youth exposure to alcohol 

advertising to show how alcohol industry voluntary standards may be more effectively applied to 

reduce youth exposure. It also tracks at the national and state level the deaths and years of life 

among persons under age 21 because of excessive alcohol use. (see attachment 9). 

REDUCING UNDERAGE DRINKING THROUGH SCREENING AND BRIEF 

INTERVENTION ($3 million for each of FY 2016-2020) 
26 


22416.015

In a change from the original authorization, H.R. 1717 provides a new grant program for pediatric 

health care providers on best practices for screening children and adolescents for alcohol usc, 

offering brief interventions to discourage drinking, and referring those in need to treatment. By 

training appropriate health care providers in the Screening, Brief Intervention, and Referral to 

Treatment (SBIRT) model and disseminating best practices, SBIRT widens the net to help youth 

who have started misusing substances and has been shown to be effective at reducing 

alcohol consumption. A 2009 study on SBIRT found that individuals who were screened positive 

for hazardous drug and alcohol use noticed a 39% reduction in heavy alcohol consumption six 

months later.4 There is a great need for SBIRT for those youth who have initiated substance use 

in order to stop the trajectory towards addiction as early as possible. This new funding will 

provide critical support to the remaining provisions of the STOP Act. 

In the I 0 years since the initial STOP Act passed, it has become clear that the law's 

coordinated provisions have worked effectively to reduce underage drinking, as the most recent 

Monitoring the Future study showed that lifetime alcohol use by 81

\ I 0'\ and 121h graders is 

currently at the lowest levels since each grade was included in the study. 5 While this is very 

welcome news, underage drinking still remains a serious public health issue facing the country. 

Here are some facts on underage drinking that will show you just how serious this crisis is: 

4 Madras BK, Compton WM, Avula D. Stegbauer T, Stein JB, Clark HW. Screening, brief interventions, referral 

to treatment tor illicit drug and alcohol use at multiple healthcare sites: Comparison at intake and 6 months. 

Drug and Alcohol Dependence. 2009;99(1-3):280-295. 

5 2015 Monitoring the Future Survey. 
27 


22416.016

6 Ibid. 

7 Ibid. 

Despite the major declines in youth underage drinking rates, alcohol remains by far the 

preferred substance of choice for young people, with 35.3% of high school seniors 

drinking in the past month, compared to 11.4% for cigarettes and 23.6% for illicit drugs.6 

17 .2%, or nearly l in 6, high school seniors still binge drink on at least one occasion in 

the past two weeks. 7 

The total annual economic costs of underage drinking are estimated at $24.6 billion, with 

$3.7 billion in health care expenditures and other impacts on society, such as loss of 

productivity, car crashes, and more, at $20.9 billion. 8 

Between 2006 and 2010, approximately 4,300 young people under the age o f21 died due 

to excessive drinking. 9 

Youth who use alcohol may remember 1 0% less of what they have learned than those 

who don't drink. 10 According to recent research, 16% to 18% of teen drinkers have 

missed school or work because of alcohol use. 11 

Among college students, underage drinking was involved in 97,000 incidents of alcohol 

related sexual assault or date rape and 690,000 students were assaulted by students who 

were drinking. 12 

8 Sacks,J.J.,J. Rreber, E c Bouchery, K Gon?.ales, F. J. Chaloupka and R. D. Brewer (2013). "State costs of 

excessive alcohol consumption, 2006." American Journal of Preventive Medicine 45(4): 4 74-485. 

9 Centers for Disease Control and Prevention. Alcohol Related Disease Impact (ARDI) application, 2013. Available at 

http://apps.nccd.cdc.gov/DACH ARDI/Default.aspx. 

I 0 Brown, SA, Taper!, S.F., Granholm. c,et al. (2000). Neurocognitive functioning of adolescents: Effects of 

protracted alcohol use. Alcoholism: Clinical and experimental research, (24)(2). 

II "Teenagers and alcohol misuse in the United States: By any definition. it's a big problem." Addiction, {91)(1 0). 

1489-1503. 

12 National Institutes of Health. National Institute on Alcohol Abuse and Alcoholism. 2013) College Drinking. 

Available at http: U www. n iaaa. nih. gov I a I coho 1-health/special-popu lations-co-occurring -disorders/ co liege-drinking. 
28 


22416.017

The stakes are high to maintain national efforts at reducing underage drinking. While the 

rate and number of young people drinking alcohol has come down in the past several years, it is 

still the main substance of choice for youths. Underage drinking has devastating consequences 

not only for the children who suffer the health, safety, and mortality consequences, but for their 

families, communities, and our country as a whole. I respectfully urge you to support passage of 

H.R. 1717, the Sober Truth on Preventing Underage Drinking Act. 

Thank you for the opportunity to present CADCA's views on this important piece of 

legislation today. 
29 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize Ms. Banks 5 minutes for your summary. 

STATEMENT OF SONJA L. BANKS 

Ms. BANKS. Good morning, Chairman Pitts and Ranking Member 

Green—— 

Mr. PITTS. Could you turn on the microphone now? The light 

should be on. 

Ms. BANKS. Good morning. Chairman Pitts, Ranking Member 

Green, this distinguished committee, thank you for holding this 

hearing and allowing me the opportunity to testify in support of 

H.R. 1807, the Sickle Cell Disease Research, Surveillance, Prevention and Treatment Act. I also would like to thank Dr. Burgess, the 

lead Republican cosponsor of this legislation, and Representative 

Butterfield, for their leadership on this important legislation. We 

would also be remiss if we did not recognize Representative Danny 

Davis for not only leading this important legislation but remaining 

one of the sickle cell community’s key champions. 

I am here representing the Sickle Cell Disease Association of 

America, the Nation’s only organization working full-time on a national level to resolve issues surrounding sickle cell disease and 

sickle cell trait. Since 1971, SCDA has been on the forefront for improving the quality of life, health, and services for individuals and 

families impacted by sickle cell disease, promoting policies and research and fighting for a universal cure. 

Now allow me to take you on a journey. I want you to think 

about the worst pain that you have ever experienced in your life, 

a broken bone, a stomach virus, a flu, or maybe for you women in 

here who have children, labor pains. Now I want you to take that 

pain, and I want you to magnify it by two. Now magnify it by five. 

Now magnify it by 10. Now imagine the pain hitting you at any 

time, anywhere, with no control, no treatment, and no way to manage it. Now imagine it rearing its ugly head monthly, weekly, and 

even daily. Though not the journey we all long for, it is one that 

is a reality. These unpredictable pain episodes are the hallmark of 

sickle cell disease and the reality for those who are afflicted with 

it. They can start as early as 6 months of age and span throughout 

the lifetime, impacting school, work, and ordinary daily living. 

Sickle cell disease is an inherited blood disorder affecting approximately 100,000 Americans. This disease causes the destruction and deformation of red blood cells, producing extreme complications that could include stroke in children and adults, lung 

problems, chronic damage to organs, including kidneys, liver and 

spleen, and, yes, severe painful episodes, and even death. One in 

every 400 African American newborns have sickle cell disease, as 

does 1 in every 1,200 newborns in Hispanic descent. 

Despite its first noted discovery well over 100 years ago, progress 

has been relatively slow, and the sickle cell community still faces 

numerous challenges. For instance, the average life expectancy of 

a person with sickle cell disease is relative young, age 40 to 45. 

Presently, there is only one medication that has been FDA approved to treat this disease. There is an overwhelming shortage of 

physicians that treat or specialize in sickle cell disease, which 

makes it very difficult for patients to have a primary care physiVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00033 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
30 

cian or medical home. A vast majority of our patients make the 

emergency room their medical home. There is no comprehensive 

model here to help reduce the major healthcare complexities that 

SCD patients encounter. 

It is because of these challenges and more that H.R. 1807 is so 

crucial for the sickle cell community. Reauthorization is needed to 

assure program stability, establish more effective care coordination, 

set in motion a model of care, and allow for a broader reach into 

areas of the country where people with sickle cell disease are not 

adequately served. This legislation will allow states to receive Federal funding for patient counseling, education initiatives, and community outreach programs, set the groundwork for 25 sickle cell 

treatment centers across the country to treat our patients, support 

the continuance of a national coordinating and evaluation center, 

allow the Centers for Disease Control to establish and continue its 

surveillance program. 

Through this initiative, we are hopeful that data collected would 

help us to understand and improve current estimates about the incidence and prevalence of sickle cell disease. Distinguished leaders, 

I humbly stand—and I know I am sitting—before you as an advocate. No, I do not have sickle cell disease. I am not personally affected by it. I do not have anyone in my family with it, but I am 

an advocate. I, like you, believe that every American deserves equitable quality of life. Individuals with sickle cell disease deserve better treatment. They deserve better access to care, and more importantly, they deserve a better quality of life. So will you stand with 

me and support this legislation? It can and it will change many 

lives for the better. Thank you. 

[The prepared statement of Ms. Banks follows:] 

31 


22416.018

'"Break The Sickle Cycle"$ 

Testimony of Sonja L. Banks 

President, Sickle Cell Disease Association of America 

"Examining Legislation to Improve Public Health" 

Energy & Commerce Subcommittee on Health 

September 8, 2016 
32 


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Summary 

Historical Background 

I. Current State of Federal Law: Congress enacted and President Nixon signed into law 

the Sickle Cell Anemia Control Act in 1972. At the time, President Nixon pledged that his 

Administration would "reverse the record of neglect on the dreaded disease." Under that first 

law, Congress authorized $10 million to expand sickle cell programs in fiscal year 1972, with the 

goal of increasing funding in FY 1973 and expanding SCD-related activities at the Veterans 

Administration. 

Over 30 years later, Congress and President George W. Bush enacted the Sickle Cell 

Treatment Act of2003 (SCTA) as an amendment to the American Jobs Creation Act of2004 

(P.L. 108-357). Original cosponsors Senator Charles Schumer (D-NY), Senator Richard Burr 

(R-NC) and Representative Danny Davis (D-IL) were instrumental in introducing the SCTA 

within the Jobs Creation Act. 

II. Problem: Authorization for the SCTA expired on September 30, 2009. As a result, 

funding for SCTA programs, while not entirely gone, has diminished in the more than seven 

years that the reauthorization for the Act's programs has not occurred. The reach and scope of 

current activities suffer significantly from under-funding at the national level, with only a 

maximum of 9-10 treatments centers/sites were ever funded. 

H.R.l807 

While work is still progressing, the HRSA grant programs need to be reauthorized to assure 

program stability and allow for a broader reach into areas of the country where people with sickle 

cell disease are not being adequately served. HR 1807 will also authorize a much needed 

surveillance program in SCD and other outreach and education efforts through the CDC. 

Key Points: 

• New name "The Sickle Cell Disease Research, Surveillance, Prevention, and Treatment 

Act of2013" 

• Allows states to receive federal funding for patient counseling, educational initiatives and 

community outreach programs 

• Sets the groundwork for the development of up to 25 sickle cell treatment centers located 

across the country and establishes a National Coordinating Center for Sickle Cell Disease. 

• Supports the continuance of a National Coordinating and Evaluation Center and (6) 

community-based demonstration sites that provide SCD follow-up and other services to 

support comprehensive care for newborns diagnosed with SCD. 

• Expands the development of transition services for adolescents to adult health care 

• Seeks authorization of Centers for Disease Control (CDC) to continue and establish a 

Hemoglobinopathies Surveillance System program and SCD public health promotion 

initiatives. 
33 


22416.020

Testimony of Sonja L. Banks 

President, Sickle Cell Disease Association of America 

"Examining Legislation to Improve Public Health" 

Energy & Commerce Subcommittee on Health 

September 8, 2016 

Chairman Pitts and Ranking Member Green, on behalf of the Sickle Cell 

Disease Association of America, Inc. (SCDAA) it 45 member organizations, over 

35 affiliate partners and 100,000 plus patients and families we represent I want to 

thank you for holding this hearing and allowing me the opportunity to testifY in 

support ofH.R. 1807, the Sickle Cell Disease Research, Surveillance, Prevention, 

and Treatment Act. I also would like to thank Dr. Burgess, the lead Republican 

cosponsor of this legislation, and Rep. Butterfield, for their leadership on this 

important legislation. We would also be remiss if we did not recognize Rep. 

Danny Davis for not only leading this important legislation but remaining one of 

the sickle cell community's key champions. 

I am here today representing the nation's only organization working full 

time on a national level to resolve issues surrounding sickle cell disease and sickle 

cell trait. Since 1971, the SCDAA has been on the forefront of improving the 

quality of health, life and services for individuals, families and communities 

impacted by sickle cell disease. Additionally, SCDAA has been and remains 
34 


22416.021

instrumental in promoting policies and research facilitating the continued search 

for a "universal" cure for sickle cell disease. 

Sickle cell disease is an inherited blood disorder affecting approximately 

100,000 Americans. The disease causes the destruction and deformation of red 

blood cells due to the presence of abnormal hemoglobin, which results in anemia 

and vasa-occlusion, which essentially means the blocking of blood vessels due to 

sticky, inflexible, sickled- shaped red blood cells. One in every 400 African 

American newborns has sickle cell disease, as does one in every 1,200 newborns of 

Hispanic descent. 

The consequences and complications of this disease arc extreme. Common 

complications include early childhood death from infection, stroke in young 

children and adults, lung problems similar to pneumonia, chronic damage to organs 

including the kidney, leading to kidney failure, and to the lungs causing pulmonary 

hypertension, and severe painful episodes. In fact pain episodes are a hallmark of 

sickle cell disease. They arc unpredictable in many ways, both the timing of when 

they occur, how severe they will be and how long they will last. For those with the 

disease, these devastating pain episodes can start as early as six months of age and 

can span a lifetime, impacting school attendance and participation in the 

workforce. In fact, these pain crises contribute significantly to the 200,000 

emergency room visits collectively made by sufferers of sickle cell disease each 
35 


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year in our country. A typical crisis will result in a hospital stay of seven to ten 

days. Due to the lack of effective treatment options to treat the disease, patients 

are given medication to ensure hydration and opioids to manage the pain. 

Although we have known for over five decades the gene responsible for sickle cell 

disease, we have no medications to alter the course of a pain episode. Sadly, 

people with the most severe form of the disease may have a two to three decade 

shorter life expectancy. 

In 2004, the Congress recognized the need to do more to improve the 

treatment and prevention of sickle cell disease. Specifically, as part of the 

American Jobs Creation Act, a Sickle Cell Disease Prevention and Treatment 

Demonstration Program was enacted. The demonstration program required the 

Health Resources and Services Administration to make grants to up to 40 eligible 

entities tor the purposes of developing and establishing systemic mechanisms to 

improve the prevention and treatment of sickle cell disease. Specifically, grantees 

were charged with improving the coordination of service delivery for patients; 

providing genetic counseling; training health professionals; working to ensure 

greater newborn screening; and working with state health departments to ensure 

access to care, among other things. To ensure this was done most effectively and 

comprehensively, the grants were to be awarded to heighten geographic diversity 

and grantee collaboration with sickle cell disease treatment centers. Additionally, 
36 


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the legislation called for the creation of a National Coordinating Center for the 

demonstration program, and this Center is administered by the National Institute 

for Children's Health Quality. 

While the enactment of the Sickle Cell Treatment and Prevention 

Demonstration Program was a major step forward for the individuals and families 

suffering with this disease, since that time the program has not been fully-funded 

and opportunities for advancement have been lost. As stated, while the law 

envisioned the creation of 40 eligible entities to develop and establish systemic 

mechanisms to improve the prevention and treatment of sickle cell disease, to date 

only nine grants have been given to eligible entities. 

H.R. 1807 reauthorizes and improves upon the HRSA demonstration 

program. The treatment and prevention component reauthorization, contained 

within section 4 of the bill, sets a more realistic number of eligible entities which 

can be funded. The original law specified 40 eligible entities, H.R. 1807 sets that 

number at 25 eligible entities. 

Importantly, a major advancement made in H.R. 1807 would place a duty on 

these grantees to "expand, coordinate, and implement transition services for 

adolescents with sickle cell disease making the transition to adult-focused health 

care." Today, so many young people fall through the cracks as they transition 
37 


22416.024

from childhood to adolescence and young adulthood. This very important change 

would make it a requirement for grantees to adopt strategies to ensure that these 

individuals transition appropriately, minimizing the disruption of care and resulting 

in better health outcomes. 

Additionally, H.R. 1807 establishes a new surveillance grant program for 

states, wherein grants would be authorized to up to 20 states representing a 

majority of the sickle cell disease patient population. The current surveillance 

conducted by the CDC is limited to the state of California and the data collected is 

general in nature. The data which would be accumulated under this grant program 

authorized by HR 1807 would cover associated health outcomes, complications 

and treatments, and would result in public health initiatives and strategies which 

would improve current estimates about the incidence and prevalence of the disease, 

would identifY health disparities, would assess the utilization therapies and 

strategies to prevent complications from the disease, and would evaluate the 

impact of genetic, environmental, behavioral and other risk factors that may impact 

health outcomes. 

SCDAA has been working in partnership with the American Society of 

Hematology, the world's largest society of professional Hematologists, in support 

ofthe reauthorization ofthe sickle cell disease programs in HR 1807 and on other 

legislative and regulatory initiatives to better meet the needs of this underserved 
38 


22416.025

community. Earlier this week ASH released a report supported by more than 20 

organizations evaluating and making recommendations related to multiple aspects 

of sickle cell disease, including access to care in the US, training and professional 

education, research and clinical trials, and global health. Together, private sector 

organizations along with federal and state government agencies working in a 

coalition will make a real difference in improving the lives of people with sickle 

cell disease. 

Finally, SCDAA wants to recognize the important advancements being 

made to not only treat the symptoms of those suffering from sickle cell, but to treat 

the disease itself. It's been decades since the FDA has approved new treatments 

for the benefit of patients with sickle cell disease. During the debate on the 21st 

Century Cures Act, Rep. Rush noted the historic underreprcscntation of minority 

communities in clinical trials, and the fact that the nation needs to do a better job of 

supporting institutions that educate medical professionals with an interest in 

working in minority communities. By doing this, we could make advancements in 

the development of therapies for diseases like sickle cell which primarily impact 

minority populations. 
39 


22416.026

Nearly 80% of hospitalizations for sickle cell disease occur among those 

covered by public payers Medicaid and Medicare." The good news is, as it relates 

specifically to sickle cell disease, we are on the precipice of exciting new therapies 

which hold the promise of lessening patient suffering and reliance on expensive 

acute care. These therapies could be approved by FDA as soon as 2018. More 

importantly these therapies may improve the quality of life of those living with 

sickle cell disease. The SCDAA would like to work with this committee and the 

Congress to ensure that when these therapies, which will alleviate so much pain 

and suffering for patients with sickle cell disease, are approved, there are 

incentives for hospitals to provide these drugs to patients. We cannot have a 

situation develop where a breakthrough sickle cell disease drug is approved for the 

benefit of patients in crisis, but there are disincentives for hospitals to use these 

new therapies. There are government programs, such as the New Technology 

Add-On Payment program in Medicare, which attempts to ensure that new 

therapies are available for needy, hospitalized patients, but only a handful of drug 

therapies have ever been approved for this program. And some have found that the 

program doesn't do enough to incentivize hospitals to use the new therapies. And 

no such program exists under the Medicaid program, which provides coverage for 

so many sickle cell disease patients. The presence of new, breakthrough therapies 
40 


22416.027

is a tremendous advance, but we must ensure that disease sufferers have access to 

these therapies when they are in need. 

Thank you very much for allowing me to testifY before you today. 
41 

Mr. PITTS. The chair thanks the gentlelady and now recognizes 

Dr. Morrison 5 minutes for your summary. 

STATEMENT OF R. SEAN MORRISON, M.D. 

Dr. MORRISON. Chairman Pitts, Ranking Member Green, and 

members of the Energy and Commerce Health subcommittee, good 

morning, and thank you for the opportunity to address H.R. 3119, 

the Palliative Care and Hospice Education and Training Act. My 

name is Sean Morrison, and I am professor and vice chair of geriatrics and palliative medicine and director of Palliative Care at the 

Mount Sinai Health System New York City. I am a former president of the American Academy of Hospice and Palliative Medicine, 

and I am here today representing the Patient Quality of Life Coalition, a group of over 40 patient, provider, and health systems focused on improving the quality of life for persons living with serious illness and their families. I would also like to thank Representative Engel, a fellow New Yorker, for his continued leadership, and 

the 33 other bipartisan members of this committee who have 

signed this bill. 

As a practicing physician, health services researcher, and teacher, I am acutely aware of the challenges faced by the seriously ill 

in this country. Multiple studies have demonstrated that inadequately treated systems, fragmented care systems, poor communication between patients, families and their physicians, strains on 

caregivers, and escalating healthcare use all characterize the experience of living with a serious illness in this country. Five percent 

of seriously ill Medicare beneficiaries account for over 50 percent 

of spending, and contrary to the popular perception, only 11 percent of these persons are in the last year of life. The majority live 

for many years with progressively debilitating illness that interferes with their quality of life and ability to work and live independently. 

Palliative care is team-based care: doctors, nurses, social workers, and chaplains, focused on relief of pain and other symptoms 

and support for the best quality of life for patients and families in 

the setting of a serious illness. It should be provided at any age at 

the time of diagnosis of a serious illness and concurrently with all 

other appropriate medical treatment, including those directed at 

cure and life prolongation. Palliative care has been shown to enhance quality of life, doctor-patient family communication, satisfaction with care, reduce healthcare costs, and in cancer, improve survival. Over 95 percent of mid- to large-size hospitals now have palliative care teams, and palliative care is being rapidly integrated 

into the nonhospital settings. 

Yet three major challenges remain if palliative care is to become 

universally accessible and, indeed, inserted into the genome of 

American medicine. First, based on a recent national survey, over 

three-quarters of patients and families who could benefit from palliative care don’t know what it is and thus cannot request it when 

it would be most beneficial. Yet when read a definition of palliative 

care, more than 90 percent said they would want it for themselves 

or their family members and that it should be universally available 

throughout the country. Targeted educational efforts to increase 

42 

patient, family, and provider awareness about palliative care and 

its benefits are appropriately recommended in H.R. 3119. 

Workforce shortages prevent patients from accessing palliative 

care. There are simply too few palliative care specialists to meet 

the needs of the population. The 134 existing palliative medicine 

fellowship programs graduate fewer than 300 new doctors a year, 

less than a 10th of what is needed. Because palliative care was recognized as a subspecialty, after the Balanced Budget Act of 1997 

limited the number of Medicare-supported residency spots, training 

in palliative medicine is now supported only by private sector philanthropy and variable and inconsistent institutional support. 

H.R. 3119 would support specialist training in palliative care, 

palliative care education for students and trainees, and mid-career 

training in the core palliative care knowledge and skills for nonpalliative-care practicing healthcare professionals. 

Finally, the knowledge base to support palliative care is inadequate. Treatment for symptoms, such as breathlessness, fatigue, 

itching, and pain, are primitive compared to the science underlying 

most disease treatments. Despite four reports from the Institute of 

Medicine calling for major Federal investment in palliative care research, a recent study found that less than 1/100th of a percent of 

the NIH budget is focused on improving quality of life in the setting of serious illness. 

I struggle daily with the fact that opioids with all of their attendant risks remain the most effective treatment for my patients in severe pain. H.R. 3119 would require the Director of the National Institutes of Health to expand and intensify research specific to palliative care. 

To close, H.R. 3119, the Palliative Care and Hospice Education 

and Training Act will help address the barriers preventing all 

Americans from enjoying the highest quality of life in the setting 

of serious illness. I would like to again express my sincere thanks 

for the opportunity to address this important issue and legislation 

on behalf of the Patient Quality of Life Coalition with you this 

morning. Thank you again. 

[The prepared statement of Dr. Morrison follows:] 

43 


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Statement by 

R. Sean Morrison 

Professor and Vice-Chair of Geriatrics and Palliative Medicine 

Director of the Hertzberg Palliative Care Institute 

lcahn School of Medicine at Mount Sinai, New York NY 

Before the Subcommittee on Health 

Energy and Commerce Committee 

U.S. House of Representatives 

September 8, 2016 

Hearing: Examining Legislation to Improve Public Health 

Chairman Pitts, Ranking Member Green and Members of the Energy and 

Commerce Committee Health Subcommittee. Good morning and thank you for the 

opportunity to address the Subcommittee. My name is Sean Morrison and I am a 

physician, Professor and Vice-Chair of Geriatrics and Palliative Medicine, and Director of 

the Hertzberg Palliative Care Institute at the lcahn School of Medicine at Mount Sinai. 

also direct the National Palliative Care Research Center in New York City- a 

philanthropically funded organization dedicated to improving the evidence base for the 

care of persons living with serious illness and their families. I am a former President of 

the American Academy of Hospice and Palliative Medicine and am here today 

representing the Patient Quality of Life Coalition; a group of over 40 patient, provider 

and health system organizations including the Academy. The Patient Quality of Life 

Coalition was established in 2013 and is focused on improving quality of life for patients 

with serious illness and their families. Thank you for the opportunity to testify before 

the Subcommittee in support of H.R. 3119, the Palliative Care and Hospice Education 
44 


22416.029

and Training Act. I'd also like to thank Representative Engel, particularly, for his 

leadership and sponsorship of this important legislation, as well as note the strong 

bipartisan support the legislation has from over half of the full committee Membership 

of the Energy and Commerce Committee signed on as co-sponsors. 

Palliative care is a relatively new medical and team-based specialty devoted to 

improving the quality of life- through expert pain and symptom management; skilled 

communication about what matters most to patients and their families; and wellcoordinated and communicated care over the course of a serious illness. Palliative care 

is delivered at the same time as curative or disease-directed treatments and eligibility is 

based on patient need and not on prognosis. H.R. 3119 would address three important 

public policy issues that have been identified as necessary to provide patients with 

serious illness better access to palliative care services throughout the continuum of their 

care: Public and Professional Education, Workforce Development, and Research. 

Since completing my training in 1995, my research, clinical care, and teaching 

activities have focused exclusively on improving quality of life for persons living with 

serous illness and their families. With my colleague Dr. Diane Meier, I established one of 

the first five palliative care programs at an academic medical center. I have served on 

national committees that have focused on enhancing research and clinical care for the 

seriously ill for the National Institutes of Health, Institute of Medicine, and National 

Quality Forum. I have been continuously funded by the National Institutes of Health for 

the past 20 years and my research has focused exclusively on improving care for persons 
45 


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with serious illness and their families. I am an active clinician caring for seriously ill 

patients and families in both inpatient and outpatient settings. 

The elimination of suffering and the cure of disease are the fundamental goals of 

medicine.1 Although medical advances have transformed previously fatal conditions 

such as cancer and heart disease into illnesses that people can live with for many years, 

they have not been accompanied by corresponding improvements in the quality of life 

for these patients and their families. 2 Living with a serious illness should not mean living 

in pain or experiencing symptoms like shortness of breath, nausea, or fatigue. Yet, 

multiple studies over the past two decades suggest that medical care for patients with 

advanced illness is characterized by inadequately treated pain and other physical 

distress; fragmented care systems; poor communication between doctors, patients, and 

families; enormous strains on family caregiver and support systems and escalating 

health care resource use.2 Five percent of Medicare enrollees account for over 50% of 

Medicare spending and, contrary to popular perception, only 11% of these persons are 

in the last year of life.3 The majority of high cost beneficiaries live for multiple years with 

progressively debilitating illness.3 Over the next decades most health care professionals 

will be caring for seriously ill older adults and their families with multiple chronic 

conditions, multi-year illnesses, and intermittent crises interspersed with periods of 

relative stability.4·s 

What is Palliative Care and Why Is It Needed? 

Palliative care is interdisciplinary team based medical care focused on relief of 

pain and other symptoms and support for the best possible quality of life for patients 
46 


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with serious illness and their families. Palliative care should be initiated starting at point 

of diagnosis of a serious illness and is provided alongside all other appropriate medical 

therapies including those directed at life prolongation and cure throughout the entire 

course of illness.5 Palliative care programs have been shown to reduce symptoms and 

enhance quality of life, improve doctor-patient-family, satisfaction with care, enhance 

the efficiency and effectiveness of hospital services, reduce healthcare costs, and in 

cancer patients, improve survival. 2

•

6•

8 Over 95% of all mid-large size hospitals in the 

United States now have palliative care teams and models of palliative care delivery are 

being rapidly created and disseminated in non-hospital care settings.9 This growth is in 

response to the increasing numbers and needs of Americans living with serious, 

complex, and chronic illnesses, and the realities of the care responsibilities faced by 

their families. 

The development of the specialty of palliative care has been a critical step in 

addressing the unmet needs of patients with serious illness and their families and the 

growth of this field has been remarkable. Nevertheless, challenges remain if care for 

seriously ill patients and their families is to improve in the United States. Most patients 

and families who could benefit from palliative care do not know of its existence or 

equate palliative care with end-of-life care or hospice and thus cannot or do not request 

palliative care when they can most benefit from it: early and throughout the course of a 

serious illness10 Because of lack of investment, and unlike other areas of healthcare, the 

knowledge base to support the core elements of palliative care clinical practice (i.e., 

pain and symptom management, communication skills, spiritual support, practical 
47 


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support for patients and family caregivers, care coordination) is inadequate and care 

models developed to support the needs of patients and families have yet to be 

evaluated. 2 That is, the evidence base to assure high quality clinical care and guide 

appropriate institutional and system benchmarks is lacking. Finally, although progress 

has been made in hospitals such that 95% of mid-large size hospitals have palliative care 

teams and two thirds of all hospitals now provide palliative care, many of these 

programs are understaffed and only able to care for a fraction of eligible patients.9 

Furthermore, the majority of nursing homes and community healthcare settings outside 

of hospitals lack integrated and adequately supported palliative care programs. 

Ensuring Access to High Quality Palliative Care 

A number of key initiatives need to be undertaken for palliative care to be 

accessible to all patients with serious illness. First, there need to be patient and 

provider educational initiatives to increase awareness of the benefits of this care, in the 

setting of a serious illness and the difference between palliative care and end-of-life 

care or hospice. Second, there need to be work force initiatives to ensure sufficient 

numbers of palliative care specialists to teach healthcare trainees and practicing 

clinicians in the core knowledge and skills of palliative care, conduct the needed 

research to enhance the evidence base, and provide appropriate care for the most 

complex population of those with serious illness and their families. Similarly, nonpalliative care specialists need to learn core palliative care knowledge and skills given 

that there will never be enough specialists to provide this type of care. Third, research 

initiatives are necessary to augment the current inadequate evidence base. H.R. 3119, 
48 


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the Palliative Care Hospice Education and Training Act would address all three of these 

important policy changes, and allow for greater patient access to palliative care services 

for patients and their families. 

Public and professional misperceptions 

A major issue impeding access to palliative care is the perceptions among 

doctors and other healthcare professionals that palliative care is appropriate only at the 

end of life, that palliative care is synonymous with hospice, and that patients will react 

negatively and lose all hope if palliative care referral is discussed.11·12 This is perhaps not 

surprising given that clinicians, particularly those trained more than 10 years ago, 

received little to no training in the core knowledge and skills of palliative care nor were 

they exposed to modern palliative care teams during their educational training. While 

many physicians have misperceptions about palliative care, most patients do not know 

anything about it. A recent survey showed that almost 90% of adults in the United 

States had either no knowledge or limited knowledge of palliative care. However, when 

read a definition for palliative care, more than 90% of the respondents stated that they 

would want palliative care for themselves or their family member and that it should be 

universally available. 10 Targeted social marketing and educational efforts must be 

directed both to the public and to medical professionals. A national educational 

campaign to increase public and professional awareness about palliative care and its 

quality of life, family, and survival benefits, as called for in H.R. 3119, is critically needed. 

Such a campaign would define palliative care as appropriate care for persons with any 

serious illness throughout the course of their disease, encourage patients and families 
49 


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to seek high quality palliative care early in the course of illness, and educate healthcare 

professionals as to the appropriate role of palliative care in the care of their patients. 

Workforce Initiatives 

Workforce shortages also prevent many patients from accessing or using 

palliative care services. The number of palliative care specialists falls far short of what is 

necessary to serve the current population in need despite the fact that the American 

Academy of Hospice and Palliative Medicine and the Hospice and Palliative Nurses 

Association are among the fastest growing professional membership organizations in 

medicine and nursing respectively. A 2010 study estimated that over 12,000 full time 

palliative care physicians were needed at that time just to meet current demand for 

palliative care services in the United States.B The demand for palliative care 

professionals in 2016 is even greater. Similar shortages exist across the other core 

palliative care disciplines of nursing, social work, and chaplaincy. The demand for the 

expansion of palliative care services in community care settings that was created by 

incentives under the Affordable Care Act, the Joint Commission Advanced Certification 

for Palliative Care, and the increasing palliative care infrastructure in both public and 

private sectors of healthcare is further straining the limited specialist-level palliative 

care workforce. 

A major reason for this shortage is the "newness of the field". Palliative 

medicine was recognized as a subspecialty by the American Board of Medical Specialties 

only in 2008. In 2016, only 119 fellowship programs accredited by Accreditation Council 

for Graduate Medical Education and 15 fellowship programs accredited by the American 
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Osteopathic Association existed in this country, together graduating a total of 296 new 

palliative care physicians each year (personal communication, Steven Smith, American 

Academy of Hospice and Palliative Medicine). Furthermore, because the Balanced 

Budget Act of 1997 placed a limit on the number of Medicare-supported residency slots 

before palliative medicine was formally recognized as a medical subspecialty by the 

American Board of Medical Specialties, specialty training in palliative medicine is 

entirely dependent on private sector philanthropy or variable and inconsistent 

institutional support and not by Medicare funding, as is the case with all other medical 

training in this country. Palliative care specialists are critically needed to teach and 

mentor healthcare trainees and practicing clinicians in the core knowledge and skills of 

palliative care, conduct the needed research to enhance the evidence base in order to 

provide the highest quality care to patients with serious illness and their families, and 

provide appropriate care for the sickest and most complex population of those with 

serious illness and their families. 

Creating a specialist workforce is not enough, however, to ensure that patients 

with serious illness and their families receive the care that they deserve. As noted 

above, the specialist workforce will never be large enough to meet the needs of those 

Americans with serious illness and their families. Enhancing the palliative care 

knowledge and skills of ALL front-line clinicians must occur if care for the seriously ill is 

to improve. Expanding core palliative care knowledge and skills of all clinicians will be a 

key step toward resolving the shortage in the palliative care specialist workforce. The 

core palliative care competencies of skilled communication, expert pain and symptom 
51 


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management, and psychosocial assessment remain, at best, a small part of most 

medical school and residency training programs. The vast majority of practicing 

physicians and trainees have either rudimentary or no skills in these areas, which 

negatively affects patient and family outcomes. Indeed, after 10 years of graduate and 

post-graduate medical education from 1986-1996 at the University of Chicago, the New 

York Hospital Cornell Medical Center, and the Mount Sinai School of Medicine, I had 

received only a thirty-minute lecture on pain management- it occurred in my first year 

pharmacology course and focused on how opioids bind to nerve cells, are metabolized 

in the liver, and excreted in the kidney. How to approach and treat the patient in pain 

or how to effectively communicate to patients a serious diagnosis, discuss goals of care, 

address prognosis, or facilitate complicated decision making in the setting of serious 

illness was never covered during my education. From research conducted over the past 

ten years, we now have a body of evidence that demonstrates that these skills 

(particularly communication skills) can be effectively learned and developed and are 

associated with improved outcomes. 14-

16 Strategies to expand training in core palliative 

care knowledge and skills to all clinicians- those in training and those already in 

practice are needed. H.R. 3119 would establish an education and training program 

modeled after the successful geriatric education and training programs created a 

number of years ago, that allow for support of palliative care curriculum development in 

medical schools and training programs in palliative care for all key healthcare 

professionals required to provide palliative care- doctors, nurses, social workers and 

certified healthcare chaplains. 
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Knowledge and Evidence Base 

Reports from the Institute of Medicine in 1997, 2001, 2003, 20154

•

17-19 have 

consistently called for major federal investments in palliative care research and yet to 

date, these calls have remained unanswered. Unlike other areas of medical research 

traditionally funded by the NIH, the knowledge and evidence base to support core 

elements of palliative care clinical practice (i.e., pain and symptom management, 

communication skills, care coordination) is inadequate. The reasons for this distressing 

state of affairs are many, but almost all stem from a philosophy of medical research that 

has traditionally viewed symptoms and suffering as unimportant in themselves and 

interesting only insofar as they guide the physician to a correct diagnosis. 20 The 

prevailing philosophy dictates that once the diagnosis is made (e.g., cancer) and the 

disease is treated (e.g., chemotherapy), the symptoms (e.g., breathlessness, pain) will 

dissipate. What is left unsaid is what happens when the disease can't be cured, or is only 

partially treated or managed, or the treatment itself results in temporary or permanent 

distress and disability.20 It is not surprising, perhaps, that a comprehensive review of 

research in palliative medicine supported by the National Institutes of Health (NIH) 

revealed the data that should guide the treatment of human suffering associated with 

serious illness of all kinds are not only inadequate but in many instances are completely 

absent. As a result, current clinical practice regarding symptoms is driven not by 

evidence but by extrapolation from other situations, small and underpowered or single 

site studies, and is often anecdotal or based on hearsay and therefore not based on 

valid science.20 
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Key research needs to be funded and performed if palliative care is going to 

achieve its potential to enhance value throughout the health care system. First, 

important gaps in clinical evidence need to be addressed so that persons with serious 

illness can receive the best available care. For example, because the mechanisms 

underlying symptoms are poorly understood, treatments for symptoms such as 

breathlessness, fatigue, itching, delirium, anxiety, and even pain are primitive compared 

the science underlying many disease treatments. Indeed, it is almost beyond 

comprehension that the most effective treatment for severe pain in the setting of most 

serious illnesses remains opioids with all of their attendant complications and risks- a 

fact that has not changed since the 1600s. Treatment for nerve (neuropathic) pain, a 

common complication of diabetes and cancer, is even less effective. Fewer then 2/3rds 

of patients with severe neuropathy have responses to the best available treatment and 

only 20% of these patients report fifty percent improvement in pain relief. 21 Although a 

recent report points to the success of non-pharmacologic measures in the treatment of 

chronic pain (such as low back pain or migraines), none of these treatments act 

immediately and most are ineffective for the most severe pain syndromes associated 

with a serious illness like cancer. It is hard to imagine that we would tolerate this state 

of affairs if we were considering conditions of high blood pressure or diabetes or 

cholesterol, rather than pain and other sources of human suffering. 

Second, the needs of older adults with serious illness and their caregivers and 

the long term and changing nature of those needs are not well described. In particular, 

the complex care needs of patients with multiple coexisting conditions and functional 
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dependency must be investigated. The ability to identify and support the population at 

risk requires moving beyond prognosis and diagnoses to include powerful predictive 

factors such as needing another person to get through the day, and prior need for 

institutional care. 22 

Third, data to guide care for seriously ill children are needed. Although the 

numbers of children living with serious illness are notably smaller than those of adults, 

in 2010, 45,000 children died in the United States, over 25,000 thousand children are 

living with a serious illness at any given time, and nearly 17 million adults are serving as 

caregivers to a seriously ill child. 19•

23 ·

24 Despite the need, the evidence on how best to 

deliver palliative care for seriously ill children is almost non-existent. Finally, the 

development and evaluation of palliative care-delivery models outside hospitals (in 

people's homes, nursing homes, office practices) is essential. To achieve this goal 

research funding for palliative care will need to be increased beyond the 0.01% of the 

National institutes of Health budget that currently supports research on palliative 

care. 25 H.R. 3119 would require the director of the National institutes of Health to 

expand and intensify research on palliative care, and pain and symptom management 

across institutes at the NIH. 

Conclusion 

Research has conclusively demonstrated that most seriously ill Americans 

experience treatable suffering and many are impoverished because of uncompensated 

medical care. 26 At the same time, rising government healthcare expenditures threaten 

to bankrupt Medicare. 27 Palliative care offers a rational solution to this problem by 
55 


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improving quality of life and quality of care and in so doing substantially reducing need 

for costly crisis care for the highest risk and highest need patient population. The 

evolution and growth of palliative care in the United States has resulted from the 

combined investments of both the public and the private sectors. Substantial privatesector contributions exceeding $300 million in the last twenty-five years28 have created 

the new field of palliative care and are reflected in the growth of hospital palliative care 

services, education and training for health professionals, and formal recognition of 

subspecialty status for physicians and nurses. Commercial and integrated health plans 

are experimenting with creative benefit design supporting palliative care delivery 

resulting in better value.29'

32 The combined and sustained commitment of both the 

private and the public sectors will be necessary to bring the palliative care innovation to 

scale in the United States. As Hubert H. Humphrey said at the dedication of the 

Humphrey Building in 1977 "the moral test of government is how that government 

treats those who are in the dawn of life, the children; those who are in the twilight of 

life, the elderly; and those who are in the shadows of life; the sick, the needy and the 

handicapped." 33 The provisions in the bill under consideration, H.R. 3119, the Palliative 

Care and Hospice Education and Training Act will contribute significantly to addressing 

the barriers preventing all Americans from enjoying the highest quality of life in the 

setting of serious illness. In closing, I'd like to express again my thanks to Chairman Pitts 

and Ranking Member Green, as well as to all of you on the sub-committee for allowing 

me to address this issue with you today. 
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References 

1. Cassell EJ. The nature of suffering and the goals of medicine. N Eng/ J Med. 

1982;306(11):639-645. 

2. Kelley AS, Morrison RS. Palliative Care for the Seriously Ill. N Eng/ J Med. 

2015 ;373(8) :74 7-755. 

3. Aldridge MDKAS. Epidemiology of Serious Illness and High Utilization of 

Healthcare; Prepared For: Institute of Medicine Committee on Approaching 

Death: Addressing Key End of Life Issues. 2014. 

4. Field M, Cassel C, eds. Approaching Death: Improving Care at the End of Life. 

Washington DC: Institute of Medicine; 1997. 

5. National Consensus Project for Quality Palliative Care. Clinical Practice Guidelines 

for Quality Palliative Care 3rd edition 2013. New York2013. 

6. Temel JS, Greer JA, Muzikansky A, et al. Early palliative care for patients with 

metastatic non-small-cell lung cancer. N Eng/ J Med. 2010;363(8):733-742. 

7. May P, Garrido MM, Cassel JB, et al. Palliative Care Teams' Cost-Saving Effect Is 

Larger For Cancer Patients With Higher Numbers Of Comorbidities. Health Aft 

(Millwood). 2016;35(1):44-53. 

8. Morrison RS, Penrod JD, Cassel JB, et al. Cost savings associated with US hospital 

palliative care consultation programs. Arch Intern Med. 2008;168(16):1783-1790. 

9. Dumanovsky T, Augustin R, Rogers M, Lettang K, Meier DE, Morrison RS. The 

Growth of Palliative Care in U.S. Hospitals: A Status Report. J Palliat Med. 2015. 
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10. Center to Advance Palliative Care. 2011 Public Opinion Research on Palliative 

Care: A Report Based on Research by Public Opinion Strategies Research. 2011; 

http://www.capc.org/tools-for-palliative-care-programs/marketing/publicopinion-research/2011-public-opin ion-research-on-palliative-care .pdf. 

11. Schenker Y, Crowley-Matoka M, Dohan D, et al. Oncologist factors that influence 

referrals to subspecialty palliative care clinics. J Oneal Pract. 2014;10(2):e37-44. 

12. Hanratty B, Hibbert D, Mair F, et al. Doctors' understanding of palliative care. 

Palliative medicine. 2006;20(5):493-497. 

13. Lupu D. Estimate of current hospice and palliative medicine physician workforce 

shortage. J Pain Symptom Manage. 2010;40(6):899-911. 

14. Back AL, Arnold RM, Tulsky JA, Baile WF, Fryer-Edwards KA. Teaching 

communication skills to medical oncology fellows. J C/in Oneal. 

2003;21(12):2433-2436. 

15. Au E, Loprinzi CL, Dhodapkar M, et al. Regular use of a verbal pain scale improves 

the understanding of oncology inpatient pain intensity. J Clin Oneal. 

1994;12(12):2751-2755. 

16. Tulsky JA, Arnold RM, Alexander SC, et al. Enhancing communication between 

oncologists and patients with a computer-based training program: a randomized 

trial. Ann Intern Med. 2011;155(9):593-601. 

17. Institute of Medicine. Dying in America: Improving Quality and Honoring 

Individual Preferences Near the End of Life,. Washington, DC: Institute of 

Medicine;2015. 
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18. Institute of Medicine. Improving Palliative Care for Cancer. Washington DC: 

National Academy Press; 2001. 

19. Field MJ, Behrman RE, eds. When children die: Improving palliative and end-oflife care for children and their families. Washington, D.C.: National Academy 

Press; 2003. 

20. von Gunten CF. Interventions to manage symptoms at the end of life. 1 Palliat 

Med. 2005;8 Suppll:S88-94. 

21. Parsons B, Emir B, Clair A. Temporal analysis of pain responders and common 

adverse events: when do these first appear following treatment with pregabalin. 

1 Pain Res. 2015;8:303-309. 

22. Kelley AS. Epidemiology of care for patients with serious illness. Journal of 

palliative medicine. 2013;16(7):730-733. 

23. Hoyert DL, Xu J. Deaths: Preliminary data for 2011. In: Statistics NCfH, ed. 

National vital statistics reports. Vol 61. Hyattsville, MD: U.S. Department of 

Health and Human Services; 2012. 

24. American Cancer Society. Cancer Facts & Figures 2013. Atlanta;2013. 

25. Gelfman LP, Du Q, Morrison RS. An update: NIH research funding for palliative 

medicine 2006 to 2010.1 Pal/iat Med. 2013;16(2):125-129. 

26. Himmelstein DU, Warren E, ThorneD, Wool handlerS. Illness and injury as 

contributors to bankruptcy. Health Aff (Millwood). 2005;Suppl Web 

Exclusives:W5-63-W65-73. 
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27. Siska A, et al. Health Spending Projections Through 2018: Recession Effects Add 

Uncertainty to The Outlook. Health Affairs. 2009;28(2):w346-w357 .. 

28. Meier DE, Isaacs S, Hughes R, eds. Palliative Care: Transforming the Care of 

Serious Illness. Jossey-Bass; 2010. Robert Wood Johnson Foundation Health 

Policy Series. 

29. Brumley R, Enguidanos S, Cherin D. Effectiveness of a home-based palliative care 

program for end-of-life. J Palliat Med. 2003;6(5):715-724. 

30. Brumley R, Enguidanos S, Jamison P, et al. Increased satisfaction with care and 

lower costs: results of a randomized trial of in-home palliative care. JAm Geriatr 

Soc. 2007;55(7):993-1000. 

31. Spettell CM, Rawlins WS, Krakauer R, et al. A comprehensive case management 

program to improve palliative care. J Palliat Med. 2009;12{9):827-832. 

32. Krakauer R, Spettell CM, Reisman L, Wade MJ. Opportunities to improve the 

quality of care for advanced illness. Health Aff (Millwood). 2009;28(5):1357-

1359. 

33. Humphrey HH. Remarks at the dedication of the Hubert H. Humphrey Building. 

Congressional Record. 1977;123:37287. 
60 

Mr. PITTS. The chair thanks the gentleman and now recognizes 

Dr. Marino 5 minutes for your summary. 

STATEMENT OF BRAD MARINO, M.D., MPP, MSCE 

Dr. MARINO. Good morning. My name is Brad Marino. I am a pediatric cardiologist at Ann & Robert H. Lurie Children’s Hospital 

of Chicago. I am a professor of pediatrics at Northwestern University Feinberg School of Medicine. I am the chair of the Medical Advisory Board for the Pediatric Congenital Heart Association, and I 

currently chair the Council for Cardiovascular Disease in the 

Young for the American Heart Association. 

Thank you very much for the opportunity to offer testimony 

today in support of H.R. 3952, the Congenital Heart Futures Reauthorization Act of 2015. I wish to thank Chairman Pitts and Ranking Member Green for holding this hearing and Representative 

Bilirakis and Representative Schiff and the dozens of congressional 

cosponsors for the bipartisan effort to build upon existing programs 

which promote lifelong research, track epidemiology, and raise 

awareness for congenital heart disease, or CHD, the most common 

birth defect. 

On behalf of Lurie Children’s, the Pediatric Congenital Heart Association, the American Heart Association, the Children’s Heart 

Foundation, and the Adult Congenital Heart Association, and the 

millions of individuals with CHD, I want to offer my strongest support for this very important legislation. Lurie Children’s, the sixth 

ranked children’s hospital nationally by U.S. News and World Report, is the largest provider of pediatric specialty care in Illinois, 

as well as serving children from all 50 States and 46 countries, 

many of whom have congenital heart disease. 

As a practicing pediatric cardiac intensivist, epidemiologist, and 

outcomes researcher, for more than 20 years, I have borne witness 

to the catastrophic results of CHD on affected children and their 

families that last a lifetime. Critical information about the epidemiology of CHD, the effectiveness of treatments, and lifelong outcomes is seriously lacking at best and nonexistent in specific areas 

such as secondary sequelae of CHD. 

Over the last several decades, tremendous advances in care have 

dramatically reduced mortality rates for children with the most 

complex congenital heart disease and increased life expectancy of 

adults with CHD. In the absence of U.S. data, extrapolation of Canadian data suggests that there are currently more than 2.4 million individuals living in the United States with CHD, half of 

whom are adults. However, while survival has improved, the reality is that complex CHD and its treatments may result in significant cardiovascular complications and organ-specific comorbidities, 

including kidney and liver disease and brain injuries, that significantly impact health status, physical, and psychosocial functioning, 

and quality of life. 

Early intervention for CHD is not a cure, underscoring the need 

for those with CHD to have lifelong care by expert providers. We 

need to better understand and improve the transition from pediatric to specialized adult cardiovascular care. Estimates suggest 

that less than 25 percent, one out of four, adults with complex congenital heart disease are receiving appropriate subspecialty care. 

61 

People born with CHD require lifelong, costly specialized cardiac 

care. As a result, healthcare utilization among the CHD population 

is disproportionately higher than the general population. 

It is estimated that compared to the medical costs of care for the 

general population, the medical costs for individuals with CHD are 

10 to 20 times greater. Around half of all dollars spent on pediatric 

CHD-related inpatient admissions is paid by Medicaid. 

To improve care and reduce costs, it is essential that Congress 

enacts legislation supporting increased understanding of CHD 

across the lifespan. The Congenital Heart Futures Reauthorization 

Act of 2015 calls for the robust public health research and surveillance that will help us better understand and improve long-term 

outcomes for the more than 40,000 babies born each year with 

CHD. 

Since the enactment of the Congenital Heart Futures Act of 

2010, Congress has appropriated nearly $15 million to support 

CHD activities at the CDC, which has led to the standardization 

of research methods and an increased understanding of the public 

health burden that CHD poses. Continued Federal investment is 

desperately needed to better understand CHD across the lifespan, 

improve outcomes, and reduce costs. The Congenital Heart Futures 

Reauthorization Act of 2015 does just that. This legislation assesses the current state of biomedical research for CHD across the 

lifespan by directing the NIH to provide a status report on its current research on CHD. This will improve an understanding of the 

causes of CHD and drive innovation and effective treatments for 

CHD and related disease processes. H.R. 3952 expands public 

health research on CHD by directing the CDC to plan, develop, and 

implement a representative cohort study. The data from this cohort 

will help to describe basic U.S. demographics of CHD, assess 

healthcare utilization, and develop evidence-based practices and 

guidelines for CHD care, eliminating our reliance on statistics from 

Canada to describe the burden of CHD in the United States. 

This bill also directs the CDC to establish and implement an outreach, education, and awareness campaign, and ensuring that 

those with CHD receive appropriate care across their lifespan. 

With this critical education campaign, individuals with CHD and 

their families will better understand their lifelong healthcare needs 

and the necessity of receiving appropriate lifelong specialized cardiac care. 

Congenital heart disease is common. It is costly, and it is a critical public health issue. In enclosing, on behalf of the Ann & Robert 

H. Lurie Children’s Hospital of Chicago, the Pediatric Congenital 

Heart Association, the American Heart Association, the Children’s 

Heart Foundation, and the Adult Congenital Heart Association, I 

urge you to take swift action to enact H.R. 3952, the Congenital 

Heart Futures Reauthorization Act of 2015. It is essential that 

Congress pass this legislation to provide improved care, outcomes, 

and quality of life for the millions of individuals in the United 

States with CHD. 

Thank you for your time and consideration. 

[The prepared statement of Dr. Marino follows:] 

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Statement for the Record 

In support of 

H.R. 3952 

"The Congenital Heart Futures Reauthorization Act of 2015" 

Submitted to the Subcommittee on Health 

House Committee on Energy and Commerce 

Submitted by: 

Bradley S. Marino, MD, MPP, MSCE 

Professor Pediatrics and Medical Social Sciences 

Northwestern University Feinberg School of Medicine 

Director, Center for Cardiovascular Innovation 

Director, Cardiovascular Bridge Programs 

Co-Director, Research and Academic Affairs 

Co-Director, Neo-Heart Developmental Support Program 

Divisions of Cardiology and Critical Care Medicine 

Ann & Robert H. Lurie Children's Hospital of Chicago, IL 

September 8, 2016 

1 
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On behalf of the Ann & Robert H. Lurie Children's Hospital of Chicago, the millions of 

people with congenital heart disease, or CHD, the health professionals who care for 

them, and the Pediatric Congenital Heart Association, Children's Heart Foundation, 

American Heart Association and Adult Congenital Heart Association that represent 

them, I want to offer my strong support for H.R. 3952, the Congenital Heart Futures 

Reauthorization Act of 2015. 

I wish to thank Representative Bilirakis and Representative Schiff for their steadfast 

leadership as sponsors of this bipartisan effort to build upon existing programs which 

track the epidemiology, raise the awareness of and promote lifelong research on the 

most common birth defect. Thank you, also, to the dozens of congressional cosponsors 

committed to this goal. I also want to express my appreciation to Chairman Pitts and 

Ranking Member Green for holding this hearing, providing the opportunity to move this 

important bill toward enactment. 

Lurie Children's, the 61

h ranked Children's Hospital nationally by US News and World 

Report, is the largest provider of pediatric specialty care in Illinois, as well as serving 

children from all fifty states and 46 countries. The Heart Center at Lurie Children's 

provides the highest quality care for patients with the most complex and serious heart 

conditions. As the region's largest children's heart center, we care for more patients with 

cardiac conditions than any other hospital in the state of Illinois, treating more than 

10,000 patients with congenital and acquired heart disease each year. Lurie Children's 

also provides more pediatric Medicaid services than any other hospital in the State of 

2 
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Illinois. On average, 55 to 60% of the beds in our hospital hold a child that is insured by 

Medicaid. 

As a practicing pediatric cardiac intensivist, epidemiologist, and outcomes researcher 

for more than 20 years, I have borne witness to the catastrophic results of CHD on 

affected children and their families. As the national Chair for the American Heart 

Association Council for Cardiovascular Disease in the Young, Chair for the Pediatric 

Congenital Heart Association Medical Advisory Board and the Chair for the 36 institution 

Cardiac Neurodevelopmental Outcome Collaborative, I can attest that CHD has a 

lifelong impact. However, critical information about the epidemiology of CHD, the 

effectiveness of treatments, and lifelong outcomes is seriously lacking, at best, and nonexistent in specific areas such as secondary sequelae of CHD. 

Over the last several decades, advances in surgical techniques, intensive care, 

imaging, and medical therapies have dramatically lowered mortality rates for children 

with the most complex CHD and increased the life expectancy of adults with CHD. 

Survival is now expected, with long-term survival rates (>20 years) estimated at 80% in 

the current era. In the absence of US data, which the Congenital Heart Futures 

Reauthorization Act will address, extrapolation of Canadian data suggests that there are 

currently more than 1.3 million adults with CHD in the US. This prevalence is expected 

to increase 5% annually, resulting in a burgeoning population of CHD survivors who 

need life-long specialized cardiac care. Although the growing population of individuals 

with CHD is a testament to important innovations in CHD care, the reality is that 

3 
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complex CHD and its treatments may result in important complications including 

persistent valvular or heart muscle issues that may require further surgery, catheterbased intervention, or heart transplantation, abnormal heart rhythms that may require 

the placement of a pacemaker, and important kidney, liver, and neurodevelopmental 

problems. In short, the interventions children with CHD receive are not cures, 

underscoring the need for children and adults with CHD to have lifelong care by expert 

providers to avoid health complications later in life. 

One of the biggest issues we face is transitioning care for individuals with CHD from 

pediatric to specialized adult cardiovascular care. As the Director of the Cardiovascular 

Bridge Programs at Lurie Children's and Northwestern Memorial Hospital, I see firsthand, each day, the lack of infrastructure, systems, providers and payors required to 

care for the ever increasing adult congenital heart disease population. Adding to this 

challenge are those patients who no longer seek care believing their childhood 

intervention has "cured" them, or that they are well enough to no longer need 

specialized cardiac care. Estimates suggest less than 25% of adults with CHD are 

receiving appropriate subspecialty care. This population of patients who are lost to 

follow-up care present a critical public health issue, which will be addressed by this 

important legislation. 

The incidence and prevalence of heart failure in the CHD survivor population is 

dramatically increasing with a dearth of well-tested and approved heart failure 

medications, mechanical ventricular assist devices, devices specifically sized and tested 

4 
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for use in the cardiac catheterization laboratory, and organs for heart transplantation. In 

addition, congenital heart survivors can face debilitating, ongoing neurodevelopmental 

issues. Cardiac surgery and perioperative treatments in the infant put the developing 

brain at tremendous risk for injury and long-term neurodevelopmental complications. 

Survivors often suffer injury to the brain due to chronic or intermittent low oxygen levels, 

decreased brain blood flow, and/or reperfusion injury related to the abnormalities of their 

circulatory systems and the medical and surgical therapies they have received. These 

brain injuries result in worse neurodevelopmental, psychosocial, and physical 

functioning, and can significantly negatively impact the individual's quality of life. 

In fact, recent studies have shown that individuals with complex CHD have an increased 

risk for neurodevelopmental impairment across a broad range of domains, including 

intelligence, school achievement, language, visual processing, memory, attention, 

executive functioning (organization, planning, and task-management), and fine and 

gross motor skills. In addition, a disproportionate number of these patients have 

significant behavioral or emotional problems, including ADHD, anxiety, depression, and 

post-traumatic stress symptomatology. Many school-age survivors of infant cardiac 

surgery require remedial services including tutoring, special education, and physical, 

occupational, and speech therapy. Up to 1/3 of complex CHD survivors will require 

some form of special education or learning supports. As adults, individuals with CHD 

are less likely to be employed and reach economic self-sufficiency than the general 

population. 

5 
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People born with CHD require lifelong, costly, specialized cardiac care, and face an 

ongoing risk of permanent disability and premature death. As a result, healthcare 

utilization among the CHD population is disproportionately higher than the general 

population. It is estimated that compared to medical costs of care for their peers, the 

medical costs for individuals with CHD are 10 to 20 times as great. Inpatient care costs 

(not including costs of physician care) for patients< 21 years old with CHD alone totaled 

more than $5.6 billion in 2009, representing 15 percent of hospitalization costs for all 

patients in this age range. Around half of all dollars spent on pediatric CHD related 

inpatient stays is paid by Medicaid. 

Hospital admissions for adults with CHD roughly doubled between 1998 and 2005. 

Nearly 20% of these admissions were for cardiac surgery or catheter-based 

intervention. Healthcare utilization and costs continue to rise, due to hospital 

admissions, cardiac and non-cardiac surgery, and emergency room visits. For example, 

with improved longevity to childbearing age, the number of high risk annual births in 

women with CHD is increasing. Childbearing women with CHD are fourteen times as 

likely to experience cardiovascular complications during pregnancy and are eighteen 

times as likely to die from such complications as are women without CHD. They also 

have longer hospital stays and incur higher hospital charges. 

With disproportionate medical costs, it is critical for us to understand the life-course of 

those living with CHD, their health care utilization and potential cost reduction 

strategies. 

6 
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22416.051

Before us, we have a reasonable and meaningful solution. To improve care and reduce 

costs, it is essential that Congress enacts legislation supporting improved 

understanding of CHD across the lifespan. The Congenital Heart Futures 

Reauthorization Act of 2015 (CHFRA) calls for improved public health research and 

surveillance that will help us better understand and improve long-term outcomes for the 

more than 40,000 babies born with CHD each year. 

Previous Congressional investment for CHD activities supported by the Centers for 

Disease Control and Prevention's (CDC's) National Center on Birth Defects and 

Developmental Disabilities (NCBDDD) has funded the development of innovative 

surveillance strategies among children, adolescents and adults with CHD. This has led 

to the standardization of research methods and an increased understanding of the 

public health burden of this condition. Since the enactment of the Congenital Heart 

Futures Act in 2010, Congress has appropriated nearly $15 million to NCBDDD for 

these activities. Continued federal investment is necessary to provide rigorous 

epidemiological and longitudinal public health surveillance and public health research 

on individuals across the lifespan to better understand CHD at every age, improve 

outcomes and reduce costs. 

The Congenital Heart Futures Reauthorization Act of 2015 (CHFRA) continues and 

builds upon these important activities. 

7 
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22416.052

The reauthorization bill assesses the current bio-medical research needs and 

projects related to CHD across the lifespan. It directs the National Institutes of 

Health (NIH) to provide a report on its current research into CHD. This will provide a 

better understanding of the state of bio-medical research to improve understanding of 

causes and drive innovative and effective treatments for CHD and related disease 

processes. 

The Congenital Heart Futures Reauthorization Act fills a critical gap and expands 

public health research on CHD. The legislation directs the CDC to plan, develop and 

implement a representative cohort study to help describe basic U.S. demographics of 

the disease, assess healthcare utilization, and develop evidence-based practices and 

guidelines for CHD care. Once this data is collected, we will no longer have to rely on 

statistics from Canada to describe the burden of CHD in the United States. 

The Congenital Heart Futures Reauthorization Act raises awareness about the 

specialized cardiac care needs of those with CHD across the lifespan. The bill 

directs the CDC to establish and implement an awareness, outreach and education 

campaign aimed at ensuring that those with CHD receive appropriate care across the 

lifespan. With this critical educational campaign, individuals with CHD and their families 

will better understand their life-long healthcare needs and the necessity of receiving 

appropriate lifelong specialized cardiac care. 

8 
70 


22416.053

Congenital heart disease is common, costly and a critical public health issue. On 

behalf of Ann & Robert H. Lurie Children's Hospital of Chicago, the Pediatric 

Congenital Heart Association, the Children's Heart Foundation, the American Heart 

Association, and the Adult Congenital Heart Association, I want to thank the many 

members of Congress who join me in support of H.R. 3952, the Congenital Heart 

Futures Reauthorization Act 2015. It is essential that Congress pass this legislation, 

to provide improved care, outcomes and quality of life for the millions of individuals 

with CHD. 

Thank you for your time and consideration. 

9 
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22416.054

Summary of Written Testimony for Dr. Bradley Marino: 

Congenital heart disease (CHD) is the most common birth defect, for which there is no 

cure. Advances in early detection and intervention are resulting in increased survival. 

However, individuals born with CHD require lifelong, costly, specialized cardiac care, 

and face an ongoing risk of permanent disability and premature death. 

With disproportionate medical costs and nearly half of all dollars spent on inpatient 

stays paid by Medicaid, we must take measures to understand the life-course of CHD, 

health care utilization and cost management opportunities. 

Before us, we have a reasonable and meaningful solution. This legislation will support 

improved understanding of CHD across the lifespan, which will improve care and 

reduce costs. 

The Congenital Heart Futures Act Reauthorization builds upon previous Federal 

investment to: 

1. Assess current bio-medical research needs and projects related to CHD across 

the lifespan at the NIH; 

2. Authorize rigorous epidemiological and longitudinal surveillance of people with 

CHD throughout the lifespan at the CDC; 

3. Lead an education and awareness campaign to raise attention to the public 

health burden of CHD across the lifespan. 

CHD is common, costly and a critical public health issue. It is essential that Congress 

pass H.R. 3952, the Congenital Heart Futures Reauthorization Act 2015, to provide 

improved care, outcomes and quality of life for the millions of individuals with CHD. 

10 
72 

Mr. PITTS. The chair thanks the gentleman. 

And I will begin the questioning and recognize myself for 5 minutes for that purpose. 

Dr. Leffert, we will just go down the line. Thank you for highlighting the impact that metabolic diseases, such as diabetes, have 

on our healthcare system. Can you talk specifically about some of 

the issues or problems with the way the Federal Government currently administers programs for diabetes and related diseases that 

the commission is intended to address? 

Dr. LEFFERT. Thank you, Mr. Chairman. 

We have over 30 agencies across the Federal Government landscape that are engaged in either diabetes research or clinical care. 

And over the 5-year timeframe from 2007 to 2012, there has been 

a 48 percent increase in the money spent on diabetes care and lost 

productivity due to diabetes. The commission will recommend programs and activities to affect the quality of life, productivity, cost 

to society of patients with diabetes, prediabetes, and related conditions. Additionally, there are examples of inconsistent and sometimes counterproductive policies emanating from Federal agencies 

that reflect a lack of communication and coordination in the administration of Federal diabetes activities. The commission is intended 

to provide a venue that brings these agencies to the table on a consistent basis to improve upon those issues. 

Mr. PITTS. Thank you. 

General Dean, in your written testimony, you state that the incidence of underage drinking has been going down in each year 

among 8th, 10th, and 12th graders. If this is true, why do we need 

to continue investment in this STOP Act, and what work is still 

left to be done? 

Mr. DEAN. In fact, it is true, but there is much work that needs 

to be done to address underage drinking in the country. And we believe that the STOP Act has been a catalyst for these improvements, and without it, we don’t believe these improvements will 

continue. But we need to keep the pressure up to maintain these 

efforts. Despite our progress, in response to our 2015 survey of coalitions, alcohol continues to be the number one problem they face 

in their communities, and also we know that students that are underage in universities are significantly abusing these as well. The 

restructuring of the STOP Act will cause our coalitions to work directly with higher institutions of education to address these problems as well. 

Mr. PITTS. Thank you. 

Ms. Banks, can you elaborate on the biggest barriers to quality 

health care for those suffering from sickle cell disease? 

Ms. BANKS. Well, I think some of the largest barriers for individuals with sickle cell disease, first and foremost is the lack of access 

to care. As mentioned, there is a shortage of primary care physicians, and therefore, our patients do not have a medical home, 

which means that they frequent the emergency room often, so that 

is a huge deficit for us. Also, there is no comprehensive model of 

care and what our patients lack and what we do not have in our 

community is a care coordination program where someone is actually providing care coordination with our patients. Our patients are 

born with sickle cell disease, so we know throughout the lifespan 

73 

that they are going to have it. There is no cure for it. So the goal 

would be for us to coordinate their care throughout the lifespan, 

and that is what is really missing in the sickle cell community. 

Mr. PITTS. Thank you. 

Dr. Morrison, how does palliative care specifically help those individuals and families of those who are suffering from a serious but 

not necessarily terminal illness? 

Dr. MORRISON. Living with a serious illness in this country, such 

as congestive heart failure, chronic obstructive pulmonary disease, 

cancer, Alzheimer’s disease, is associated with a number of distressing symptoms—pain, breathlessness, fatigue, nausea, anxiety—which people live with on a daily basis. It provides an enormous strain on family caregivers, who often give up their jobs, 

their work, to care for a seriously ill older relative. Palliative care 

addresses these needs by providing an added layer of support to 

patients, their families, and doctors. It treats the pain and symptoms of a distressing illness. It helps facilitate communication and 

provides support to patients and families. It addresses psychological, emotional, and spiritual needs, and it allows them to obtain 

the best quality of life possible in the setting of a serious illness. 

And it is absent from our American healthcare system at this 

point. 

Mr. PITTS. Thank you. 

Dr. Marino, why is it so difficult to retain patients in followup 

care for their congenital heart disease, and what does H.R. 3952 do 

to help change that? 

Dr. MARINO. So one of the programs that I have been spearheading at Northwestern is something called the cardiovascular 

bridge programs. So typically in the U.S. today, when you are 18 

years and 364 days, your doctor will say: It has been great taking 

care of you. Here is the name of a doctor in the city. Have him call 

for the records. Good luck. 

Only one out of four patients that need ongoing cardiovascular 

care actually get that cardiovascular care. What this bill is going 

to do is create awareness among patients and parents that when 

your child has surgery as a baby, it is not curative. There are ongoing specific cardiac issues. There are developmental issues, kidney 

and liver issues, that have to be dealt with as that child ages and 

then gets transitioned into adulthood. 

In our bridge programs at Northwestern, we literally have a 

team of adult and pediatric providers, social workers, and advanced 

practice nurses that basically work with patients 16 to 26 to allow 

these patients to have a graded transition instead of an abrupt 

transition at 18 that will keep them in care. 

Beside the awareness, by having the cohort study that is put in 

3952, that the CDC would put together, we would know much more 

specifically which patients are at the highest risk for not following 

up, which patients are at the highest risk for having complications. 

That will then tell us, of those patients that we know we need to 

follow up, which are the most critical to make sure they stay in 

care. 

And then, lastly, with the NIH putting forth a status report on 

the biomedical research, there is so much research that still needs 

to be done on how best to care for these patients, what interVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00077 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
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ventional procedures might result in a better quality of life as they 

transition from an adolescent to an adult. By having that new research and that priority for that research in place with NIH, we 

will be able to put new care models in place and new treatment 

models that will help these patients transition more effectively 

from adolescence into adulthood. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize Mr. Green 5 minutes for questions. 

Mr. GREEN. Thank you, Mr. Chairman. 

Dr. Leffert, diabetes can be effectively managed through evidence-based treatments, as well as through behavioral changes, including changes in diet, increasing physical activity. Some patients 

still experience devastating complications from diabetes, including 

blindness, kidney failure, and limb amputation. Why do these complications occur in spite of the availability of the treatments we 

have? 

Dr. LEFFERT. Representative Green, the issue really is that we 

have a limited number of endocrinologists who are able to take care 

of patients with diabetes. We have a primary care base of physicians who take care of diabetes, but oftentimes, they are not all 

given the tools or the experience to be able to take care of these 

patients in the appropriate way. They need a lot of help, and our 

commission bill would do that. 

In addition, our patients need education. Education is the key because this is a self-managed disease, and this bill would also help 

that in relationship to many of the programs that are currently 

being projected by the National Diabetes Education Program, 

which gives patients education towards diabetes. 

Mr. GREEN. Thank you. It sounds like this commission would 

help us explaining to physicians how we can treat diabetes, again, 

with medication. And I always tell people it is much better to have 

prediabetes than diabetes so that you can manage it much better. 

Thank you. 

The STOP Act became law in 2006, and almost 30 percent of the 

underage individuals who were alcohol users and 19 percent were 

binge users that year. This legislation marked the first national 

comprehensive effort to combat underage drinking. And, again, I 

want to recognize my colleague and classmate, Congresswoman 

Roybal-Allard, for her diligent effort. And like I said—before you 

were here, Lucille—she has worried me about this bill for a number of months. 

General Dean, can you talk about the progress you have made 

since 2006 and why reauthorization of these programs is so important? 

Mr. DEAN. Thank you very much, Ranking Member Green, for 

your interest, your leadership, and your support. We have made 

tremendous progress. Monitoring the Future cites that underage 

drinking percentages are down. They are the lowest they have been 

for years, but we still need to continue to work diligently. And 

what this reauthorization is going to do is not only will we be able 

to provide enhancement grants to community-based coalitions that 

have been trained and understand how to tackle and resolve problems in their communities, but it is also going to give them a few 

dollars, allow them to work with higher education, colleges and 

75 

universities as well, where we know there is a serious problem 

there too. And we think there are too many losses of life. Certainly, 

we can prevent that. We also can continue to tackle the violence 

and the unfortunate incidents that are taking place on our universities as a result of drinking, so there is still much work needed 

to be done, and we believe the reauthorization and the way it has 

been restructured will allow us to continue to make progress 

around this serious underage drinking problem. 

Mr. GREEN. Thank you. Thank you for your effort on that. 

Palliative care is a critically important aspect of healthcare system. It does not always garner the attention that it warrants. 

Dr. Morrison, can you help this committee understand that palliative care, both from its impact on a patient’s quality of life and 

the workforce involved, and how does this legislation improve the 

palliative care? 

Dr. MORRISON. Thank you for the question. Palliative care is a 

relatively new specialty. It began in the 1990s when a number of 

us said: Why do you have to be dying in order to have a good quality of life? And back before palliative care, the only real area that 

focused on improving patient’s quality of life was hospice. And as 

we all know, you have to have a prognosis of 6 months or less to 

be able to access hospice in this country. 

So it is a relatively new specialty, and it is one of the fastest 

growing specialties in the United States, but we still have a workforce issue. We have one palliative medicine physician for every 

13,000 people with serious illness, and this bill addresses this in 

three ways. 

First of all, it does create a specialist workforce that will provide 

the research, the teaching, and take care of the most complex patients and families. But it also provides the core knowledge and 

skills of palliative care to those in training and those in practice. 

I spent 4 years at the University of Chicago, 3 years at New York 

Hospital Cornell Medical Center, another 3 years at Mount Sinai, 

and in that entire 10 years of education, had a 30-minute lecture 

about pain management that happened in my first year pharmacology course. And it dealt with how drugs like morphine are broken down in the liver and excreted in the kidneys. That was the 

extent of my education in how to treat distressing symptoms, and 

we have a generation of healthcare providers with that base fund 

of knowledge. 

So this bill will address that as well by training those doctors, 

nurses, social workers, chaplains, who care for the seriously ill in 

the core knowledge and skills of palliative care: pain and symptom 

management, communication, care coordination. 

And, finally, it addresses the evidence gap. We have all seen the 

problem in this country of inappropriate prescribing of opioids because we have a generation of doctors who do not know how to assess pain, how to manage pain, how to appropriately use opioids, 

and how to identify the problems of addiction. I have never had a 

patient come to me in serious pain and say: I would like my pain 

treated and, oh, by the way, I would like to be addicted to the 

medication afterwards. That can be addressed through outreach, 

and it can be addressed through appropriate knowledge and teaching. And that is what this bill addresses as well. 

76 

Mr. GREEN. Mr. Chairman, thank you. And I have some questions we will submit. But our committee and the subcommittee has 

actually passed a number of opioid bills. And I think you are correct. We need to have training for the physicians who are actually 

prescribing, and hopefully, this bill along with the package of bills 

we passed out. 

Thank you, Mr. Chairman. I yield back. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize the vice chair of the subcommittee, the gentleman from Kentucky, Mr. Guthrie, 5 minutes for questions. 

Mr. GUTHRIE. Thank you very. I am going to try to get as many 

questions, so if answers could be kind of brief. 

But first, Ms. Banks, I have a friend of mine whose son has sickle cell. And I didn’t really know that much about it. Dr. Burgess 

has been helping me with it sitting here. But we hired him so he 

would be gainfully employed. And just working with his schedule 

was about my only experience with it. 

So, with this bill, are there any other efforts existing within the 

sickle cell community that would complement this bill and allow it 

to be more expansive to the patient population. 

Ms. BANKS. Absolutely. Currently, we are in the process—and 

when I say ‘‘we,’’ I mean SCDAA and many office treatment centers 

or hospitals—are working with community health workers. So we 

have actually instituted what is called a community health worker 

program. What we feel like in the sickle cell community is it is 

going to take a concerted effort throughout the community. So we 

are utilizing not only the providers but also community-based organizations and utilizing community health workers to actually go 

into the field, find these patients, because a lot of our patients are 

lost, meaning they haven’t been to a physician in a year or so. And 

that is not good for them and their health. And then not only are 

they finding these individuals, but they are getting them into a 

medical home. So the goal is that you find the patient, but then 

you enroll the patient or you help that patient find medical care. 

In addition, SCDAA has launched a national patient registry, 

which is the first ever of its kind. It is a patient-powered registry. 

We know that it is long overdue in the sickle cell community. We 

know that it is going to help us to collect the data that we need. 

But also, along with collecting that data, this is patient-powered. 

So it allows the patient to communicate with the physicians. It allows the patient to actually manage their care via technology. And 

also it allows the patient to be up-to-date on treatments and research that is going on in the community for sickle cell patients. 

Mr. GUTHRIE. OK. Thank you very much. 

Dr. Marino, great to see that Northwestern is doing good work. 

I am moving my daughter there Monday. So she will be on the 

Evanston campus. So, in your testimony, you mentioned the use of 

Canadian data. What type of U.S. data do we have, and why do 

have to use the Canadian data? I would just let you expand on 

that. 

Dr. MARINO. Mr. Guthrie, first, congratulations to your daughter. 

Northwestern is a very, very tough school to get into. So congratulations to her. 

77 

There is no data in the U.S. What we have is single-center data 

of very small numbers of individuals that we can’t extrapolate to 

national data. And because the data is collected very differently at 

the different centers, with variable definitions that don’t match, 

you can’t take 10 or 15 centers and put the data together. Because 

the Canadians have a national health system, they actually have 

a national data set that allows them in a very specific way, like 

Denmark as well, to gather this longitudinal date on the congenital 

heart disease patients. So 3952 would specifically have the CDC 

create a cohort study in the United States that would follow this 

very high-risk complex CHD population over time to collect that 

similar data. We don’t know if what we have in the U.S. is different than Canada or if it is the same. There is just no data. 

Mr. GUTHRIE. OK. Well, maybe on one of my visits over the next 

little while, I will be able to see what you guys are doing. That 

would be interesting to see. 

So, General, I just want to ask you a question. And congratulations on your service and obviously reaching one of the top ranks 

in the military. That says a lot about your ability. Why is training 

pediatric healthcare providers in screening—let me start over. Why 

is training pediatric healthcare providers in screening, brief intervention, and referral to treatment important? And why is the provision on this being added to the STOP Act? And can you discuss 

how this new provision’s authorization will work within the overall 

authorization for the bill? 

Mr. DEAN. Thank you for your question. It is a very important 

question. It is a, we believe, significant change in the restructure 

of this reauthorization. And we are very excited about the inclusion 

of—we call it SBIRT for pediatric healthcare providers. It is a 

strong complement to the universal prevention as it allows youth 

who have been misusing substances to be identified more readily 

and to get effective intervention in a larger number of community 

settings. We also know it is effective. And a 6-month followup with 

SBIRT participants found that heavy alcohol consumption was 39 

percent lower among individuals who initially screened positive for 

hazardous drugs and alcohol use. So screening early, training pediatric providers to do this, we have found already it is reducing the 

consumption by young people. 

Mr. GUTHRIE. OK. Thank you. 

And, Dr. Leffert, what are the expectations that the clinical care 

commission can establish within the next 3 years? I guess you have 

3 seconds to tell me that. So I apologize. 

Dr. LEFFERT. Well, as you know—thank you for the question, 

Representative Guthrie. As you know, diabetes is a big issue, and 

there are a lot of issues going along with it but. But we would hope 

that the process would allow for federally funded research initiatives from the bench to the bedside so that patients could have access to 21st century cures and innovations, and that these would 

become more coherent and synergistic, and that there would be better communication and coordination among the agencies, specifically NIH, FDA, and CMS. The commission would also help focus 

the efforts of the government research community toward improving clinical care for people with diabetes and to slow the incremental rise in diabetes and its associated complications. 

78 

Mr. GUTHRIE. Thank you very much. 

My time has expired, so I yield back. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize the ranking member of the full committee, Mr. 

Pallone, 5 minutes for questions. 

Mr. PALLONE. Thank you, Mr. Chairman. 

I wanted to ask Dr. Morrison to focus on the palliative care legislation, you know, both the access to treatment but also the services 

available to those individuals to cope with their conditions. First, 

from diagnosis, what is the most common diagnosis for individuals 

who receive palliative care services? 

Dr. MORRISON. The common diagnoses are what we consider to 

be serious illness. So it is cancer, heart disease, advanced lung disease, Alzheimer’s disease and related dementias, and neuromuscular diseases such as Lou Gehrig’s disease or amyotrophic lateral sclerosis. In children, it is really two large groups of diseases. 

One is obviously cancer, and the other is congenital or genetic diseases. And because of advances in pediatrics, we have a whole population now of kids who are living long, long periods of time. And 

that is wonderful. However, they are living with multiple symptoms and high caregiver burden on their families. 

Mr. PALLONE. Well, you mention symptoms. What are the most 

common symptoms that are treated with palliative care? 

Dr. MORRISON. They are what you would anticipate: pain, breathlessness, fatigue, nausea, anxiety. In children, it is primarily fatigue. And it is the one symptom for which we have really no effective treatments as of yet because of the lack of the evidence base 

and the lack of the research science. 

Mr. PALLONE. All right. Now, let me ask a couple questions about 

access. Do most people battling serious health conditions, such as 

cancer, have access to palliative care services, and what are some 

of the consequences for individuals with serious illness who don’t 

have access? 

Dr. MORRISON. It is a very good question. What we know from 

a study actually we did earlier this year was that now 97 percent 

of mid- to large-size hospitals in the United States now have palliative care teams. And if we look at the Medicare population, about 

75 percent of all Medicare beneficiaries who die live in an area 

where they could potentially access palliative care. The problem is 

that those hospital teams are relatively small and, because they 

are still understaffed, see only a small proportion of the number of 

patients and families who could truly benefit. What we have seen 

in the past 3 years also, however, is the expansion of palliative care 

into the community, particularly in Medicare Advantage plans and 

commercial plans, which aren’t limited by the fee-for-service structure of the traditional Medicare program. So we are seeing some 

very, very new and exciting models of care happening in the community, particularly in Tennessee and Nashville, as was said earlier. 

Mr. PALLONE. Well, in terms of increasing access to important 

services, how can we increase it? How does this bill help? 

Dr. MORRISON. I think it helps in three ways. The first is that, 

because palliative care is a relatively new specialty, most people 

don’t know what it is. Again, a survey that we did with the AmerVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00082 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
79 

ican Cancer Society several years ago showed that about 80 percent 

of a national representative sample had never heard of palliative 

care and didn’t know what it was. And yet, when we read a definition to them, over 90 percent said that this is what they would 

want for themselves and their families. Providers too don’t understand palliative care and too often confuse it with hospice and endof-life care, when the reality is palliative care is for everybody. It 

is not dependent on prognosis. And in fact, we provide it to people 

we expect to be cured. So there is an educational campaign awareness that needs to happen. 

And the second issue is we need to address the workforce gap. 

Every single clinician in this country who cares for somebody with 

serious illness needs to be able to treat pain appropriately, manage 

breathlessness. Talk to them about how a serious illness—I have 

probably as much training, sir, as you in how to talk to somebody 

and break bad news that you have cancer. When I finished medical 

school, we had the same amount of training in terms of how to 

have that conversation. And we need to address that through our 

medical schools and our training programs and to physicians like 

me who are in practice. This bill will do this as well. 

Mr. PALLONE. Well, thank you. I am just trying to get in one 

question for General Dean about strategies for preventing underage drinking. I just want to learn more about the methods to prevent underage drinking. General Dean, what types of strategies 

and programs work best to prevent underage drinking, and what 

evidence is available to prove that these interventions work? 

Mr. DEAN. OK. Thank you, Congressman. It is a great question. 

We believe that by mobilizing the entire community—what I mean 

by that is all of the sectors in the community: parents, teachers, 

youth, the police, business providers, faith community, civic business leaders all coming together. And we have built over the last 

25 years a strategy, an academy type approach, to train the members of the community how to identify their problems, how to address their problems, and how to implement evidence-based strategies to reduce their problems. There have been evaluations done by 

the Office of National Drug Control Policy independent of CADCA 

that show that when these communities have been trained, the results are significant. And we have great examples. In the interest 

of time, I will not cover them with you. But I do have several examples here where communities have reduced their underage 

drinking by large percentages, 20, 30, 40, 50 percent, using these 

methods. 

Mr. PALLONE. Well, through the chairman, if maybe we could ask 

him in writing to follow up with and give us those examples. Mr. 

Chairman, with your permission, he mentioned that he doesn’t 

have the time to give some examples. So, with your permission, 

maybe we could have him follow up in writing and give us that information. 

Mr. PITTS. I am sorry. I was talking. 

Mr. PALLONE. No, I know. That is all right. He wanted to give 

me some examples, but in the interest of time, he is not doing it. 

I was going to ask if he could do it in writing. 

Mr. PITTS. Yes. We will submit that to you in writing and ask 

you to please respond. 

80 

Mr. DEAN. I will be glad to. I have several examples of communities that have made significant progress, some exceeding 50 percent reduction using these community-based strategies to reduce 

underage drinking all across the country, both rural, urban, as well 

as suburban. 

Mr. PALLONE. Thank you. 

Mr. PITTS. Very good. Thank you. 

The chair now recognizes Dr. Burgess 5 minutes for questions. 

Mr. BURGESS. Well, thank you, Mr. Chairman. 

And thanks to the panel for being here. 

It has been a very interesting morning listening to you all. 

Ms. Banks, let me just ask you, and I was struck in your testimony, both the written testimony and your testimony here this 

morning, you say it has been decades since there has been a new 

FDA-approved treatment for sickle cell. Is that correct? 

Ms. BANKS. Correct. 

Mr. BURGESS. So my recollection of Parkland Hospital in the 

1970s is actually pretty much current therapy. Is that right? 

Ms. BANKS. Correct. 

Mr. BURGESS. I referenced this in my opening statement, this 

committee has spent a lot of time on a bill, H.R. 6, called Cures 

for the 21st Century. And although sickle cell—and we tried not to 

have disease-specific parts of the bill, I mean, this just strikes me 

as one of those areas where the type of translational research that 

would go across the National Institute of Health or would give the 

National Institute of Health Director much more discretion as to 

what they researched and what they funded, that this would be one 

of those areas. 

And I just went on clinicaltrials.gov to look for the current clinical trials in sickle cell, and there weren’t as many as I thought 

there ought to be for a disease of this magnitude that is so pervasive in the community. I mean, is that a reasonable assumption I 

have made looking at clinicaltrials.gov? 

Ms. BANKS. Actually, you are correct. We are always wanting 

more research for sickle cell disease. I will say this. 

Mr. PITTS. Poke your microphone. 

Ms. BANKS. Oh, I am sorry. I will say this. About 7 years ago, 

when I started the Sickle Cell Disease Association of America, we 

literally had about two pharmaceutical companies in the space for 

sickle cell disease, and today we with about 16. So it is looking up 

for sickle cell disease. Of course, we still have to get over that hurdle of getting individuals in those trials and going through that. 

But it is hopeful. 

But I totally agree with you. Sickle cell disease has long been forgotten. And over 100 years—this is probably one of the oldest diseases for its discovery out there—for there only to be one drug for 

treatment—and by the way, that drug is hydroxyurea. That drug 

was not approved for sickle cell disease. It is an actual cancer drug. 

So, really, when you look at those kinds of issues, it is long overdue 

in the sickle cell community. 

Mr. BURGESS. Yes. And I appreciate your comments on that. And 

it is something we will keep an eye on in this committee because, 

of course, I am going to be optimistic that we are going to get 

Cures for the 21st Century done in this Congress. But there will 

81 

also be an FDA reauthorization that takes place in the next Congress. And that is another appropriate place to focus on this. 

General Dean, I want to ask you a question. It is probably not 

fair because it is not on the bill that you came to testify on. But 

in your role as the CEO of Community Anti-Drug Coalitions of 

America, I got asked a question by a constituent, and I didn’t know 

the answer. And I was a little bit embarrassed that I didn’t know 

the answer. And if you don’t know the answer, it is OK. You don’t 

need to be embarrassed. Perhaps you can point me in the direction 

that I need to go. There is a woman who came into my office. She 

had lost her son in a—he was a pedestrian struck by a vehicle. He 

was in a crosswalk. The individual who was driving the vehicle was 

not issued a ticket or a citation. He did have alcohol in his system, 

but it was under the .08 limit in the State of Texas. But he also 

had a positive qualitative test for the active ingredient in marijuana. OK. It seems to me that—and obviously, this would be a 

state law, but does your group look at, now that there are more 

and more states that are providing a legal avenue for consumption 

of marijuana, does your group look at the additive multiplicative effects of drugs and alcohol? Do states need to perhaps reconsider 

what their limits are? This just struck me—of course, it is a very 

tragic and unfortunate case. But that just seemed like it was one 

of those things that cried out for something in addition to be done. 

Now, law enforcement made, their position was, as far as laws 

of the State of Texas, we don’t prosecute for having small amounts 

of marijuana in your blood. And this was a qualitative test, not a 

quantitative test, so we don’t even know to what degree of intoxication there might have occurred from that, but from the alcohol 

standpoint, under the legal limit of intoxication. 

Mr. DEAN. To answer your question, Congressman, we do care 

about this issue very much. We do watch and observe what is happening in states that have, first, through citizen votes decreed that 

marijuana is medicine. It obviously has not gone through the FDA 

process for that to be done. So we watch that carefully. We also are 

watching the states where they have—through citizens have passed 

it for recreational use. And we have seen, looking at data coming 

out of States like Colorado, Washington, and others, that there is 

a substantial increase in citizens, both young and old, driving 

under the influence of drugs versus alcohol. And in some cases, 

there are more impaired drivers on the streets these days in those 

locations from drugs than there are from alcohol. 

So the law enforcement challenge is having the appropriate instruments and tests to test for it. It is not as simple as it is for 

alcohol. And, therefore, it becomes challenging for them to do that. 

So the answer is we are seeing the results. We are seeing the impact. We are concerned about it, and the law enforcement community is extremely concerned about it. 

Mr. BURGESS. Well, I will have my office follow up with you. We 

may have further discussion about this. But you are the first person who has come to this committee who might know something 

about this. And I do want to follow up with you. And I would appreciate the opportunity to do so. 

Mr. DEAN. It would be our pleasure. 

82 

Mr. BURGESS. And, Mr. Chairman, I also would ask unanimous 

consent, I have a 2013 article, but it is the most recent one I could 

find, ‘‘Current Management of Sickle Cell Anemia,’’ and I would 

like to submit this for the record. 

Mr. PITTS. Without objection, so ordered. 

[The information appears at the conclusion of the hearing.] 

Mr. PITTS. The gentleman yields back. 

The chair now recognizes the gentleman from North Carolina, 

Judge Butterfield, 5 minutes for questions. 

Mr. BUTTERFIELD. Thank you, Mr. Chairman. 

Ms. Banks, I want to come back to you for just a couple of minutes, if you don’t mind. Ms. Banks, can you describe for me how 

diminishing Federal funding, including lack of funding for treatment centers, has made it more difficult for people with sickle cell 

disease to get the care that they need? 

Ms. BANKS. Sure. Of course, with every disease, especially with 

the complications in sickle cell disease, it is a challenge when you 

do not have the funding in place. Eventually, when the bill was 

first passed, because this is a reauthorization, there was a place in 

the bill where it said 40 treatment centers. To my knowledge, sickle cell only received 10 of those treatment centers or 10 of those 

treatment centers were funded. Because of that, our patients basically do not have a medical home. That is the reason why a lot of 

them frequent the emergency room. So, when you talk about diminishing Federal funding, you are looking at, where do our patients 

go for access to care? That is extremely, extremely important. 

In this bill, we ask for 25 treatment centers, because we wanted 

to be very realistic in our ask and we wanted those 25 centers to 

be in areas where there are high populations of individuals with 

sickle cell disease. We are hoping that that would provide some 

coverage. We know it will not for 100,000 patients, but it would 

provide some coverage for our patients and somewhere to go. 

It is very interesting, in comparable diseases, for instance, with 

cystic fibrosis, they have over 100 treatment centers. With hemophilia, I think they have over 40, 42. So, with sickle cell disease, 

only having 10 funded, you can see with 100,000 patients where we 

are at a huge deficit. 

Mr. BUTTERFIELD. Is it true that African American children have 

higher rates of disease in trait? Is that an accurate statement? 

Ms. BANKS. Say that again. 

Mr. BUTTERFIELD. That black children, African American children, have higher rates of disease. 

Ms. BANKS. Of sickle cell disease? 

Mr. BUTTERFIELD. Yes, of sickle cell disease. 

Ms. BANKS. Yes. 

Mr. BUTTERFIELD. Yes. And what proportion would you say of African American babies are screened for this disease at birth? 

Ms. BANKS. Well, actually, it is mandatory in every state. So, 

right now, every state screens for sickle cell disease when you are 

born. 

Mr. BUTTERFIELD. It is a Federal mandate or a state mandate? 

Ms. BANKS. It is Federal. 

Mr. BUTTERFIELD. Yes. All right. Are there any barriers that prevent babies from being screened for sickle cell? Are there any barVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00086 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
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riers that would prevent that from happening at birth? Or is it 

completely uniform across the board? 

Ms. BANKS. To my knowledge, it is completely uniform across the 

board. 

Mr. BUTTERFIELD. All right. 

Ms. BANKS. Our issue, Congressman Butterfield, is that, years 

ago, when sickle cell was very prevalent and people heard about it, 

it was because babies were dying. And so, because of treatments, 

because of the newborn screening, babies are living well into adolescence. Our issue now is transition, and where do you go after 

you are 14, 15 and you begin to transition into young adult care? 

That is where we are having the shortage of adult hematologists 

or adult primary care physicians for those individuals. So now the 

challenge in sickle cell disease is where our babies are getting better, they are living through teens and young adult, and they don’t 

have a place to go. So when you go to college, when you get to that 

college age, where do you start? I was talking to my chief medical 

officer, and it is interesting because there are individuals 24 and 

25 years of age still going to pediatric physicians, a hematologist, 

because they do not have an adult physician that would treat their 

disease in a system in managing their disease. 

Mr. BUTTERFIELD. Would you discuss the barriers, if any, facing 

African Americans from being screened or receiving treatment for 

sickle cell disease? 

Ms. BANKS. I think the barriers for African Americans, particularly—or anyone with the disease pretty much is, again, the lack 

of a medical home. Our patients have been stigmatized mainly because the key issue is pain. And if you are frequenting the emergency room for pain, what are you going to be classified as? For 

most, our patients feel as if they are ostracized because when they 

go in, they are going for drugs. And it is because we do not have 

any drugs for treatment of the disease that we are treating our patients with opioids. So that is a huge barrier in the African American community. But it is a huge barrier in the sickle cell community as a whole. 

Mr. BUTTERFIELD. Thank you. You are very kind. 

I yield back, Mr. Chairman. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize the chair emeritus of the full committee, Mr. 

Barton, 5 minutes for questions. 

Mr. BARTON. Mr. Chairman, I don’t have any questions, and I am 

late arriving. So I am going to yield to the members who have been 

here. 

Mr. PITTS. I recognize the gentleman from Florida. You are recognized for 5 minutes. 

Mr. BILIRAKIS. Thank you, Mr. Chairman. 

I appreciate it. Thank you, Mr. Chairman. 

Again, I want to thank Chairman Pitts and Ranking Member 

Green for holding this very important hearing and including my 

bill, the Congenital Heart Futures Reauthorization Act. I appreciate it so very much. This legislation provided a 5-year reauthorization to the underlying law that I coauthored back in 2009. The 

Congenital Heart Futures Reauthorization Act will continue the 

CDC surveillance program, continue to provide NIH grants for furVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00087 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
84 

ther congenital heart disease research, and require NIH to report 

on their ongoing research efforts. Congenital heart disease is the 

number one cause of birth defects related deaths. Twenty-five percent of children born with a congenital heart defect will need heart 

surgery or other interventions to survive. An estimated 2 to 3 million people are living with CHD. And individuals with CHD have 

an ER visitation rate of three to four times higher than the general 

population. The Congenital Heart Futures Reauthorization Act will 

continue our commitment to monitoring and increasing the available research and helping people born with a congenital heart defect. I would like to ask unanimous consent, Mr. Chairman, to introduce these letters of support: the Pediatric Congenital Heart Association, a letter from the Adult Congenital Heart Association, and 

a letter from the American Society of Echocardiography. I would 

like to ask unanimous consent. 

Mr. PITTS. Without objection, so ordered. 

[The information appears at the conclusion of the hearing.] 

Mr. BILIRAKIS. Thank you very much. 

And I have a couple questions for Dr. Marino. Dr. Marino, what 

are the biggest challenges facing children and adults with CHDs as 

they age, and how will the Congenital Heart Futures Reauthorization Act help meet these challenges? 

Thank you again, sir, for testifying today. 

Dr. MARINO. Congressman, thank you very much. We greatly appreciate your support. 

It is a great question. Simply put, when you have your surgery 

as a baby, you are not cured. And we know that these patients 

have specific neurodevelopmental issues that come from brain injury from when they had their surgery as a baby. We know that 

they have specific cardiovascular complications. Many of them go 

on to heart failure and need heart transplantation. They might 

need a ventricular assist device, mechanical support device. They 

often have abnormal heart rhythms. They often have decreased exercise function. These patients will also—many of them have kidney or liver injury from their original therapies when they were a 

baby. 

So, with this complex medical milieu of multisystem organ failure, they then now need to transition from an adolescent care 

model to an adult care model. You have heard from several of the 

other witnesses who are testifying today that there is lack of adult 

providers who can care for these types of patients that survive the 

childhood illness and then now move on to adult care. Adult congenital heart disease physicians are in—we have the same dearth 

of care providers. You have heard about palliative care and sickle 

cell disease. While there is now an accreditation for adult congenital heart disease care, there are still very few adult congenital 

heart disease care certified individuals nationally. 

And then probably the last part which we discussed a little bit 

earlier was that, right now, there is no specific means by which we 

transfer these high-risk complex patients into adult care. And if 

you just hand off a patient at age 18, it is very unlikely, actually, 

that they are going to get followup care. And what we know—and 

I want to focus on cost for a second—if you get patients into appropriate followup care and you minimize secondary complications as 

85 

adult congenital patients, you will lower overall costs to the system. And given that more than 50 percent of these patients will be 

cared for through Medicaid as children and then Medicare as 

adults, if we can find ways to transition them better, not lose three 

out of four patients during that transition process, it will likely 

lower the overall costs for these 2.4 million individuals nationally 

who survive with adult congenital heart disease. 

Mr. BILIRAKIS. A very good point. Thank you so much. 

Next question, the Congenital Heart Futures Reauthorization Act 

specifically calls for a cohort study. How does this differ from current surveillance techniques being used to study CHD, and why is 

this needed? 

Dr. MARINO. So, if you look over the last 20 years, there are lots 

of individual center studies trying to benchmark how these patients 

are doing. They don’t talk to each other relative to a data standpoint. When I was at Harvard, working at Boston Children’s Hospital or Children’s Hospital Philadelphia or Cincinnati Children’s 

or now at Lurie Children’s during periods of my training and now 

my faculty positions, the data that is collected for these individual 

studies are all quite different. The definitions used to codify the patients and codify the complications that we have talked about are 

very, very different. So I can’t take 10 different studies from 10 different centers and pull them together into a cohesive whole. 

What 3952 will allow is for the CDC for the first time in the 

United States—and by the way, this has been done in at least a 

dozen other countries around the world—take the U.S. population, 

start collecting data at dozens of centers with the same variables, 

the same data set, and then follow them longitudinally, not just 

over 2 years, hopefully over decades, for us to get a much better 

sense for, what is the true incidence and prevalence of these complications that I have alluded to? What are the impacts of treatments that we then bring into the care models for these patients 

nationally? And then, more importantly, when it comes to transition, how do we best predict who is not going to have effective transition, who will, and then get the supports in place in adolescence 

to make sure we don’t lose three out of four of these patients. Because I can tell you, in my work in Northwestern, there are literally dozens and dozens of patients each year that walk into us 

at Northwestern Memorial Hospital, who have been out of care, 

cardiac care, for 5 years, 10 years, who literally are near death. 

And I know for a fact if they had actually had appropriate transition and actually stayed in care, they likely would have survived 

multiple more decades with a great quality of life. 

Mr. BILIRAKIS. Well, thank you very much, doctor. 

I want to thank the entire panel for their testimony. 

And I yield back. I appreciate it. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize the gentlelady from Illinois, Ms. Schakowsky, 5 

minutes for questions. 

Ms. SCHAKOWSKY. Well, first, let me just apologize. It is hard to 

be everywhere at once. I was looking forward to hearing from you. 

And I want to thank you so much for being here, all of you. 

I want to say I am proud to see that Dr. Marino is here from 

Lurie Children’s Hospital, and I just want to acknowledge the unVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00089 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
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paralleled care that you provide for not just the children of Chicago 

but many who come to the hospital. 

I have really focused for much of my public career, both in the 

Illinois legislature and now here, on improving senior citizens’ access to health care. Along with my colleague Doris Matsui, I have 

served as the co-chair of the House Seniors Task Force. Given that 

10,000 people turn 65 every day, it is imperative that we really 

work to address the specific health needs of seniors. 

So, Dr. Morrison, let me focus on that. I am interested in hearing 

how the aging of our population is going to affect the need for palliative care services moving forward, and by the way, you might 

want to distinguish between hospice and palliative care as you 

talk. Specifically, I am interested in hearing more about a statement you included in your written testimony that says: ‘‘Over the 

next decade, most healthcare professionals will be caring for seriously ill older adults and their families with multiple chronic conditions, multiyear illnesses, and intermittent crises interspersed with 

periods of relative stability.’’ So how is this going to affect the need 

for palliative care services among the population, this population, 

as well as a workforce trained in palliative care? 

Dr. MORRISON. Thank you, Congresswoman. 

And as a geriatrician, thank you so much for your work for older 

adults in this country. 

Let me take this in two ways. First, let me clearly differentiate 

between hospice and palliative care. Hospice was started in this 

country in the 1970s really as an alternative to life-prolonging curative treatment when it was recognized that many people near the 

end of life were experiencing distressing symptoms and very high 

care needs. And it has been a wonderful system of care since 1972, 

and even more so since Medicare covered hospice in 1982. 

The problem with hospice is that you have to be dying to access 

it. And you to have a predictable prognosis of 6 months or less. And 

for those of us who began in palliative care, the question was, why 

should you be dying to have efforts focused on enhancing your 

quality of life? 

When we look at the aging of the population, as you pointed out, 

it is the fastest growing segment in the United States. And for 

most of us, the time after the age of 65 or 70 is going to be many, 

many years of a very good quality of life. It will be time to integrate our work and life experiences. It will be time spent with our 

children and our grandchildren. But most of us, those of us who 

aren’t killed crossing the street or have a sudden death, will develop a series of chronic ongoing progressive illnesses: heart disease, lung disease, even cancer, for which we have transitioned 

many cancers into chronic illnesses. And as we age, we will have 

more and more of those: diabetes, frailty, multiple chronic conditions. And the data that we have now suggests that most of us will 

spend at least 7 years of our life in that state. And the data that 

we have nationally suggests that 70 percent of older Americans 

with a serious illness have three or more distressing symptoms on 

a daily basis. And we can do better. 

The last years of our life, the last 5, 10 years, should not mean 

living with daily symptoms. It should not mean tremendous burdens on our children and our grandchildren to care for us. And it 

87 

should not mean bankrupting Medicare to care for those. And palliative care, as a relatively new specialty, has demonstrated that it 

meets all those needs. 

First of all, we have a wealth of data that palliative care teams 

improve symptoms. They make people feel better and their quality 

of life better. Secondly, it improves caregiver well-being and reduces burdens on caregivers. And, thirdly, by really providing the 

right care to the right people at the right time, it reduces costs 

largely by providing that added layer of support in caring for people where they want to be cared for, in the home. In New York 

City, if my 85-year-old patient falls in the middle of the night and 

his wife can’t get him up and he is struggling to breathe because 

of heart failure, right now, she calls a doctor’s office. And when you 

call the doctor’s office in New York City, what do you get? If this 

is a medical emergency, please call 911. And maybe you will get 

a voice at the end of the phone. If you call our palliative care team, 

you get a real person at the end of the phone. You may get somebody to come into the home. And you will have in place a plan to 

deal with predictable crises for older adults. That is the added 

layer of support that palliative care can provide to our healthcare 

system. 

Ms. SCHAKOWSKY. Perfect. Thank you. 

Dr. MORRISON. Thank you. 

Mr. PITTS. The gentlelady yields back. 

The chair now recognizes the gentleman from Missouri, Mr. 

Long, 5 minutes for questions. 

Mr. LONG. Thank you, Mr. Chairman. 

And, Dr. Morrison, how long has palliative care been around? 

Dr. MORRISON. Palliative care really developed as a specialty in 

about the mid-1990s, as I said, when we had this lightbulb go off 

that said: You don’t have to be dying to have good quality of life. 

But it only became a sub-specialty in 2008. So it has really only 

been since 2008 that the American Board of Medical Specialties 

has recognized palliative care as a specialty. And so it is a very 

young field. 

Mr. LONG. My mom passed away in 2009. And I remember that 

when they came in and said, ‘‘We need to talk about palliative 

care,’’ that was the first that I had really heard about it. 

My series of questions are for you, Dr. Morrison. They focus on 

the care and support needs of individuals with Alzheimer’s disease 

and other dementias. Could you elaborate on how palliative care 

could benefit people with Alzheimer’s? 

Dr. MORRISON. Yes. Absolutely. As you know, the prevalence of 

Alzheimer’s disease is increasing rapidly in the United States, 

largely as we have made tremendous progress in treating other diseases. Alzheimer’s disease fits in many respects perfectly within 

the paradigm of palliative care. It is a multiyear illness. Families, 

as cognitive status declines, patients are more and more dependent 

upon their families. It is a disease that is associated with a tremendous symptom burden. All of the diseases that people had before 

Alzheimer’s disease, their osteoarthritis, their heart disease that 

causes breathlessness, their lung disease, don’t go away in the setting of Alzheimer’s disease. What happens, though, is people can’t 

tell you that they are breathless. They can’t tell you that they are 

88 

in pain. They can’t tell you that they are hungry because of cognitive impairment. And so the suffering continues, but the suffering continues silently. 

It is also a disease that has periods of stability where people will 

be the same for long periods of time and then there will be a crisis, 

an infection, a pneumonia, a urinary infection, a pressure ulcer. 

And so it doesn’t fit well within our current model of hospice because people with Alzheimer’s disease aren’t dying quickly. They 

are actually living for a long period of time. And what they do is 

they need support, and they need then crisis intervention, which 

palliative care can provide, and then ongoing support after that 

throughout the course of that illness. And, as importantly, Alzheimer’s disease is not just a disease that affects the patient. All 

of us who have had a loved one with dementia or know somebody 

know that it extends to the family as well, and that the burden on 

families is almost as great as the patient itself, and that, as a specialty, palliative care focuses on both the patient and the family as 

the unit of care. 

Mr. LONG. OK. Thank you. My aunt passed away from Alzheimer’s about 6 weeks ago. So I can relate to everything you are 

saying there. 

How easy is it for individuals to gain access to palliative services 

today? Second part, are there enough providers offering these services across different settings? And are there enough new providers 

being trained in this space to meet patient needs? 

Dr. MORRISON. Moderately easy, no, and no. And let me elaborate. 

Mr. LONG. OK. 

Dr. MORRISON. The first is that we have built over the past 20 

years, largely because of private sector philanthropy and investment in infrastructure to support the development of palliative 

care. So, right now, 95 percent of our mid- to large-size hospitals 

have palliative care teams. And over two-thirds of all American 

hospitals have that infrastructure in place. 

The issue is, as you pointed out, it is the workforce, that we actually don’t have enough providers to be able to provide those services to everyone in need. And we really need two things. First of 

all, we need a specialist workforce not to take care of everybody 

with serious illness. That will never happen, and that should not 

be our goal. We need specialists to teach, to do the necessary research, and take care of the most complex patients and their families. And that is what the provisions of 3119 provide. 

But, as importantly, we need to train every clinician who cares 

with somebody with serious illness in the core knowledge and skills 

of palliative care so that every doctor in this country knows how 

to treat pain effectively, every nurse knows how to communicate 

serious illness to somebody, and we have a care system that can 

provide that added layer of support for that very small but very expensive and very vulnerable patient population. 

Mr. LONG. Lastly, I would like to know, how do the needs differ 

of the older patients from the needs of younger patients as relates 

to providing palliative services, and do current training opportunities address these differences? 

89 

Dr. MORRISON. It is a very good question. For most younger 

adults, most younger adults are typically living with a single illness. Is it cancer? Heart disease? For children, cystic fibrosis. For 

older adults, it is much more complex, because most of us, when 

we age, will develop multiple chronic conditions that all intersect 

and all affect our quality of life. So it is not just cancer. It is cancer. It is heart disease. It is debilitating arthritis. It is diabetes. 

And it is both cognitive impairment, Alzheimer’s disease, and often 

functional impairment, difficulty walking. So it is a much more 

complex population in many respects. I think what we have done 

very well within our field is the collaboration with geriatrics. The 

recognition that we will never have enough geriatricians, we will 

never have enough palliative care physicians to treat the population, the older adult population that need, and it requires a collaboration and for us to break out beyond specialist-level care to 

think about population-related care. And that is one of the reasons 

that 3119, the bill before you, is modeled after the very successful 

Geriatric Academic Career Awards. Because that was such a good 

model for improving access to care for—— 

Mr. LONG. Speaking of 3119, that is how many minutes I am 

past time. So I yield back. 

Dr. MORRISON. I apologize, sir. 

Mr. PITTS. The chair thanks the gentleman and now recognizes 

the gentleman from California, Mr. Cardenas, 5 minutes for questions. 

Mr. CARDENAS. Thank you very much, Mr. Chairman. And thank 

you for having this important hearing. First, I would like to recognize and thank my colleague Lucille Roybal-Allard for championing 

the legislation H.R. 1717, which is part of this hearing today, the 

Sober Truth in Preventing Underage Drinking Reauthorization Act. 

It is unfortunate that, being on Appropriations, you are not allowed 

to be on Energy and Commerce. 

But thank you, colleague Lucille Roybal-Allard, for being here 

and for introducing that great bipartisan legislation. 

My questions today are based on diabetes. And I would like to 

ask some questions to Dr. Leffert. If you don’t mind explaining to 

us what prediabetes is and how it increases the risk of an individual developing type 2 diabetes. 

Dr. LEFFERT. Thank you, Congressman. 

Prediabetes is the process of developing diabetes but before that 

happens. So the genetic and environmental process results in a situation where what we call glucose intolerance or impaired fasting 

glucose. Both of those conditions are what we now have termed 

prediabetes. The issue with prediabetes is that prediabetes is a 

surrogate for cardiovascular disease. So, in people who have 

prediabetes, the risk for cardiovascular disease goes up, and then 

the disease process then may progress on to type 2 diabetes. So it 

has both an effect in and of itself and also as a progenitor towards 

type 2 diabetes. The data, I think, is that about a third of the patients will go on to type 2 diabetes, about a third of the patients 

will remain prediabetic, and about a third of the patients will regress if they start with diet and exercise. And so the main issue 

in prediabetes for our population and why it is such a huge issue 

is because of the fact that it is associated with obesity and genetic 

90 

factors, particularly among populations, like African Americans 

and Hispanics, in our country. 

Mr. CARDENAS. And with proper education and cooperation with 

their health providers, et cetera, a person can decrease their 

chance of going from prediabetic to developing type 2 diabetes? 

Dr. LEFFERT. That is absolutely correct. And I think that should 

be a big push of what we are doing in our healthcare prevention, 

meaning keeping people at the level of prediabetes or moving backward would be the most important aspect of what we do. And we 

can do that through a recognized approach toward nutrition therapy, toward dietary therapy, toward exercise. And there have been 

programs that have been done through the diabetes prevention program that have been successful in doing that particular thing itself. 

Mr. CARDENAS. OK. How can the commission improve our ability 

to reduce the development of diabetes among individuals with 

prediabetes? 

Dr. LEFFERT. Well, I think, again, it is a coordination issue. I 

think our Federal Government has a number of different agencies 

that are all working somewhat in silos. And I think our commission 

would allow us to have all of the organizations, including the private sector, industry, and other organizations together, to be able 

to coordinate that effort and prevent the onset of diabetes if we 

have people who have prediabetes. 

Mr. CARDENAS. Now, obviously, there is a quality-of-life issue for 

somebody to not develop into having type 2 diabetes. But what 

quantifiable numbers when it comes to dollars would be saved if we 

were more successful in our efforts and coordinated better like you 

just described? Are we talking just a few million dollars a year to 

our economy, or are we talking billions of dollars? 

Dr. LEFFERT. I think we are talking more like billions of dollars 

because I think the issue, when we go from prediabetes to diabetes 

and the hospitalizations that are associated with diabetes, the complications of diabetes related to kidney disease, heart disease, eye 

disease, is astronomical, and I think we could save a large amount 

of money of our Federal budget related to that. 

Mr. CARDENAS. So diabetes-related illnesses like you just described I would imagine over lifetimes of tens and hundreds and 

millions of people would actually cost us trillions of dollars. 

Wouldn’t it? 

Dr. LEFFERT. Well, I think, right now, as Chairman Pitts said in 

his opening statement, one in three dollars of the Medicare budget 

is spent in diabetes. 

Mr. CARDENAS. So it is in the trillions. 

Dr. LEFFERT. It is a very, very significant amount of money that 

is being utilized in that regard. 

Mr. CARDENAS. Thank you. I yield back. 

Mr. PITTS. The chair thanks the gentleman. 

And I now recognize the gentlelady from Indiana, Mrs. Brooks, 

5 minutes for questions. 

Mrs. BROOKS. Thank you, Mr. Chairman. 

In my home State of Indiana, over 750,000 Hoosiers have type 

1 or type 2, and nearly 2 million of about 6 1⁄2 to 7 million Hoosiers 

have prediabetes. And so we know it is taking an immense toll on 

our State and on the Nation’s healthcare system. But yet you 

91 

talked about some innovations, continuing glucose monitoring and 

artificial pancreases. Can you tell me, Dr. Leffert, how the new 

treatments, new devices that are on the horizon that can help bend 

the curve on both the incidence and the cost of the disease and help 

patients better manage their disease, how is this commission going 

to have a role in expediting patient access to these innovations? 

Dr. LEFFERT. So you told us about a very big problem. And we 

talked about that already. I think the issue for us in terms of getting the treatments to the patients is really one, to some extent, 

of making sure that the cost is available, that we have treatments 

that are cost-effective and not so expensive that patients can afford 

them, but in addition, the commission will allow us to utilize the 

resources of multiple different agencies working together to be able 

to first move these new technologies forward—the artificial pancreas being one of them, particularly in type 1 diabetes—and then 

also help us with prioritization of the ability to educate the physician workforce. 

We have 5,000 endocrinologists in the United States. That is not 

nearly enough to take care of diabetes. We have to educate and 

maintain that workforce at the primary care level and with all clinicians who are taking care of people with diabetes. So it is a tremendous opportunity here to utilize this commission to then focus 

our efforts at the level of the clinical physician and associated 

healthcare providers to be able to give these patients the best care. 

Mrs. BROOKS. And is that being done? Because I am a huge believer in public/private partnerships, especially when it comes to 

commissions and government commission work, and so is involving 

the private sector clinicians on the commission the manner in 

which you are going to educate the agencies? 

Dr. LEFFERT. To some extent I think that is exactly the reason 

for our bill. This bill started within our organization in being interested in trying to make sure that the flow of dollars coming from 

the Federal Government was adequately being utilized to take care 

of patients. Our organization, the American Association of Clinical 

Endocrinologists, are the physicians on the ground who take care 

of patients on a daily basis. We have the ability to see, though, not 

all of the patients that have diabetes. We focus, to a large extent, 

our efforts on complicated patients. And we want to be able to 

translate our knowledge and information throughout the system. 

And so that allows us to give primary care physicians the information and education, and we need the Federal Government to be 

able to push that through to the whole sector of physicians who 

take care of patients. 

Mrs. BROOKS. Thank you. 

Dr. Morrison, in your opening statement you talked about your 

concern, and there has been a lot of attention on opioids, and we 

just passed and had signed into law a very significant piece of 

opioid legislation. My involvement with that involved, actually, a 

task force focused on the prescribing practices of physicians. And 

can you please speak to the impact of that legislation or what your 

concern is? And we did add pain specialists to our task force because we want to ensure that patients who need opioids get them. 

But can you talk about that balance when we have an opioid epidemic happening in the country? 

92 

Dr. MORRISON. It is a challenge. And I recognize in many respects and I am envious I am not in your position about how to 

address it. But let me address a couple things. 

First of all, there are really two populations of patients to think 

about when we talk about pain. First, there are the people who live 

with chronic pain and pain is their only symptom. The much larger 

population, and the one that we are focusing on here, is the patient 

population with serious illness where pain is just one of a number 

of distressing symptoms, so, for most people, pain, breathlessness, 

fatigue, anxiety. And it is a constellation. And yet the prescribing 

practices that we need to teach are very similar within both populations. And I often hear about: Well, we are going to carve out people with cancer or people at the end of life, and they are not going 

to be part of the legislation. The reality is, though, that that is only 

a small fragment of those with serious illness, that people are 

going to live for many years with pain, distressing symptoms, and 

they are not going to be treated by specialists. They are going to 

be treated by primary care physicians, cancer doctors. 

And so what we need to do is we need an aggressive effort that 

is going to focus on teaching appropriate opioid prescribing. But, as 

importantly, it still strikes me as almost unimaginable that the 

drug we have for pain has not changed since the 1600s and that 

we need major investment in alternatives to a drug that we know 

has not only significant side effects but significant complications. 

So we need to teach appropriate opioid prescribing, appropriate recognition of the signs and symptoms of addiction, appropriate training into what are opioid-responsive pain syndromes and what are 

not, and we really need a critical investment in research to give us 

an alternative to opioids to treat pain and other symptoms. 

Mrs. BROOKS. Thank you very much. 

I yield back. 

Mr. PITTS. The chair thanks the gentlelady and now recognizes 

the gentleman from New York, Mr. Engel, 5 minutes for questions. 

Mr. ENGEL. Thank you, Mr. Chairman and Mr. Green, for convening this morning hearing. I am so pleased to have an opportunity to discuss H.R. 3119, the Palliative Care and Hospice Education and Training Act, a bill that I introduced with Congressman 

Tom Reed, my colleague from New York. Every one of us has been 

touched by serious illness, whether we have been affected personally or stood by a loved one grappling with critical illness. We all 

know how physically and emotionally trying situations can be for 

all those involved. Palliative care aims to relieve these stresses. 

And thank you, Dr. Morrison, for everything you have been saying. 

I am from New York City as well. Palliative care complements 

efforts to treat or cure illness by focusing on patients’ quality of 

life. Palliative care is appropriate for patients with serious illness, 

starting at the point of diagnosis through treatment and onward 

through hospice and the end of life. It involves capable communication with patients and their families to coordinate care, determine 

preference, and help with medical decisionmaking throughout the 

care continuum. Despite the benefits of palliative care, many Americans aren’t aware of the supports available to them. In addition, 

93 

there is a shortage of educated providers who can offer quality palliative care. 

So my bill, H.R. 3119, aims to remedy these issues. My bill would 

expand opportunities for training in palliative and hospice care and 

offer incentives to attract and retain providers. In addition, 

through existing programs, my bill would create a national campaign to educate patients, families, and health professionals about 

the benefits of palliative care. And, finally, H.R. 3191 would expand 

critically needed research on palliative care at the National Institutes of Health. 

I want to thank Chairman Upton, Chairman Pitts, Ranking 

Member Pallone, Ranking Member Green, for considering this important bill. And I would also like to thank the 200 Members, colleagues of House, who have cosponsored it, including several members of this committee. And I hope today’s discussion, as it has 

been doing, will bring us one step closer to enacting this legislation 

and extraordinarily improvingpatients’ quality of life. 

So let me, Dr. Morrison, thank you again for being here today. 

During your testimony, you noted that palliative care has the potential to bring about long-term savings for the healthcare system. 

Would you explain exactly how improved access to palliative care 

and, specifically, this bill would produce these savings? And have 

there been studies that actually conclude that there are real savings? 

Dr. MORRISON. Before answering your question, Mr. Engel, thank 

you, on behalf of the patients and families that I take care of. I live 

just south of your district. 

Mr. ENGEL. Move on up. 

Dr. MORRISON. Thank you for sponsoring this legislation. The 

question is, will palliative care provide savings to the healthcare 

system? And the answer is, yes, it will. When we look at the population that palliative care provides for, it is the 5 percent of Medicare beneficiaries that are accounting for over 50 percent of spending. And what palliative care does is it provides the added layer of 

support that reduces the misutilization for that population. How 

does it do that? First of all, it provides a safe environment at home, 

so in the setting after crisis in the middle of the night, on a weekend, or any time that is not Monday to Friday, 9 to 5, it provides 

the added layer of support at home so that somebody doesn’t have 

to go to the emergency department for care. Our modern-day hospitals are designed for the 95 percent of people who don’t need palliative care, and it is a mismatch, a tremendous mismatch for 

somebody with multiple chronic conditions, cognitive impairment, 

frailty, in our modern hospitals. And what palliative care teams do 

is they provide that added layer of support and make the hospitals 

friendly to people with chronic illness. They address pain and other 

symptoms. They sit with patients and families and identify: What 

are their values? What are their goals for care? What are they hoping to accomplish? And then we match treatments to meet those 

goals, and in doing so, we reduce unnecessary and unwanted 

healthcare utilization. And conversely to hospice, we do this at the 

same time as all other appropriate life-prolonging treatments. 

The question about cost and cost savings is an important one. We 

now have studies in general hospitals within the Medicaid popuVerDate Nov 24 2008 12:00 Jan 25, 2017 Jkt 037690 PO 00000 Frm 00097 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 114\114-165 CHRIS
94 

lation, within the Veterans Administration, all that demonstrate 

that when palliative care is provided at the same time as other appropriate treatments, costs are dramatically reduced, and importantly, quality of life goes up, and survival is exactly the same, if 

not longer. 

Mr. ENGEL. I think the point about survival certainly the same, 

if not longer, how do efforts to better patients’ quality of life simultaneously enhance patients’ clinical outcomes, you know, the tie-in 

between the two? 

Dr. MORRISON. How does palliative care enhance clinical outcomes? We don’t know for sure, but we have a very strong hypothesis why. First of all, we know that people living in pain, people 

who are depressed, people who are anxious, all contribute to increased medical complications. Pain is associated with delirium and 

confusion. Pain means that you can’t get out of bed and walk, so 

you lose muscle mass. Pain prevents you from eating because you 

just don’t feel hungry. Nausea does the same. So palliative care, by 

specifically focusing on distressing symptoms, allows people to get 

better. 

The best example I can give is my 35-year-old who had a very 

aggressive lymphoma but because of the palliative care she received, she made every single one of her chemotherapy appointments on time because she wasn’t too nauseated, too sick, too distressed, and subsequently, she made every single radiotherapy on 

time and completed her treatment, so the palliative care she provided allowed her to complete her curative treatments. 

Mr. ENGEL. Thank you. 

Mr. Chairman, can I please ask unanimous consent to enter into 

the record statements in support of H.R. 3119 from the Alzheimer’s 

Association, the American Academy of Hospice and Palliative Medicine, the National Hospice and Palliative Care Organization, and 

the Oncology Nursing Society, as well as a letter of support from 

the 45 organizations on record in support of the bill? 

And I want to thank Dr. Morrison for being the most eloquent 

speaker on this that I have heard. Thank you. 

Mr. PITTS. Without objection, so ordered. 

[The information appears at the conclusion of the hearing.] 

Mr. ENGEL. Thank you, Mr. Chairman. 

Mr. PITTS. The chair thanks the gentleman. That concludes the 

questions of the members of the committee present. We will have 

some followup questions, questions in writing. We will provide 

those to you. We ask that you please respond promptly. 

I remind members that they have 10 business days to submit 

questions for the record, so members should submit their questions 

by the close of business on Thursday, September 22. 

Very interesting, very important, very informative hearing. 

Thank you very much for your testimony. 

And, with that, this hearing stands adjourned. 

[Whereupon, at 12:05 p.m., the subcommittee was adjourned.] 

[Material submitted for inclusion in the record follows:] 

95 


22416.055

September 8, 2016 

The Honorable Fred Upton 

Chairman 

House Energy & Commerce Committee 

Washington, D.C. 20515 

The Honorable Joe Pitts 

Chairman 

Health Subcommittee 

Washington, D.C. 20515 

The Honorable Pete Olson 

U.S House of Representatives 

2133 Rayburn House Office Bldg. 

Washington, D.C. 20515 

The Honorable Frank Pallone 

Ranking Member 

House Energy and Commerce Committee 

Washington, D.C. 20515 

The Honorable Gene Green 

Ranking Member 

Health Subcommittee 

Washington, D.C. 20515 

The Honorable Dave Loebsack 

U.S. House of Representatives 

1527 Longworth House Office Bldg. 

Washington, D.C. 20515 

Dear Representatives Upton, Pallone, Pitts, Green, Olson and Loebsack, 

The undersigned organizations, representing physicians, allied health professionals, patients, community health 

organizations and industry, commend you for holding a hearing on September 8th regarding H.R. 1192, "The 

National Diabetes Clinical Care Commission Act." We urge passage by the Energy and Commerce Committee as 

soon as possible to facilitate expedited approval by the House and subsequent approval by the Senate this year. 

We are very hopeful that the Committee's action will allow for enactment of H.R. 1192 this year and help the 

nation pivot from continuing the status quo to instead undertaking more proactive and innovative approaches 

to metabolic, autoimmune and insulin resistant diseases, which include the escalating diabetes epidemic and 

the chronic disease complications of diabetes. The United States spends $322 billion annually controlling the 

high blood glucose levels that characterize diabetes or the cardiac, nerve, kidney and newly recognized cancerrelated complications of the disease. At this level of investment, the nation cannot afford to be complacent and 

accept inefficiencies and inconsistent policies as part of the federal response to a disease that continues to grow 

unchecked. 

As you know, H.R. 1192 creates a commission which would include leaders of the federal government as well as 

several private sector experts in diabetes and its disease complications. The commission would review the 

myriad of ways the government currently spends money on activities related to the diagnosis and treatment of 

diabetes and other metabolic, autoimmune and insulin resistant diseases, and make recommendations on how 

these funds could be utilized more effectively. 

Through innovation, collaboration, and application of advances in care called for in this important legislation, 

H.R. 1192 provides the opportunity to begin to reduce the staggering impact of diabetes on health care 

spending, while simultaneously improving care for the tens of millions of Americans living with diabetes, and the 

many more tens of millions of Americans impacted by diabetes. 

We thank you and the Members of the Energy and Commerce Committee for taking action to advance H.R. 1192 

and we are gratified by the support of the 220 co-sponsors of this bill. We look forward to working with you to 

secure its passage by the House of Representatives and the Senate this year. 

Sincerely, 
96 


22416.056

Abbott 

Academy of Nutrition and Dietetics 

American Academy of Family Physicians 

American Academy of Ophthalmology 

American Association of Clinical Endocrinologists 

American Association of Diabetes Educators 

American Association of Kidney Patients 

American Clinical Laboratory Association 

American College of Cardiology 

American Diabetes Association 

American Medical Association 

American Optometric Association 

American Pharmacists Association 

American Society for Metabolic and Bariatric Surgery 

American Society of Endocrine Physician Assistants 

American Society of Nephrology 

AstraZeneca 

Becton Dickinson and Company 

Board for Certification of Nutrition Specialists 

Children with Diabetes 

Dexcom, Inc. 

Diabetes Hands Foundation 

Diabetes Patient Advocacy Coalition 

diaTribe Foundation 

Eli Lilly and Company 

Endocrine Society 

Healthcare Leadership Council 

Health Monitor Network 

Johnson & Johnson 

JDRF 

Medtronic 

National Kidney Foundation 

Novo Nordisk, Inc. 

Obesity Action Coalition 

Obesity Medicine Association 

Omada Health, Inc. 

Renal Physicians Association 

Roche Diabetes Care, Inc. 

Sanofi 

The Obesity Society 

Vivus, Inc. 

Weight Watchers International 

YMCA of the USA 
97 


22416.057

September 8, 20 16 

The Honorable Fred Upton 

Chairman 

House Energy & Commerce Committee 

Washington, D.C. 20515 

The Honorable Joe Pitts 

Chairman 

Health Subcommittee 

Washington, D.C. 20515 

The llonorable Pete Olson 

U.S House of Representatives 

2133 Rayburn House Office Bldg. 

Washington, D.C. 20515 

eat• AcademyofNutrition 

right. and Dietetics www.eattight.org 

120 South Riverside Plaza 

Suite2000 

Chicago, Illinois 60606-6995 

800.877.1600 

1120 Connecticut Avenue NW 

Sutte460 

Washington, D.C. 20036 

The Honorable Frank Pallone 

Ranking Member 

House Energy and Commerce Committee 

Washington, D.C. 20515 

The Honorable Gene Green 

Ranking Member 

Health Subcommittee 

Washington, D.C. 20515 

The Honorable Dave Loebsack 

U.S. !-louse of Representatives 

1527 Longworth House Office Bldg. 

Washington, D.C. 20515 

Dear Representatives Upton, Pallone, Pitts, Green, Olson and Loebsack: 

The Academy of Nutrition and Dietetics commends you for holding a hearing on September 8, 2016, regarding 

H.R. 1192, ''The National Diabetes Clinical Care Commission Act." Representing more than 100,000 registered 

dietitian nutritionists (RDNs), nutrition and dietetic technicians, registered (NDTRs) and advanced-degree 

nutritionists, the Academy is the largest association offood and nutrition professionals in the United States. 

Academy members play an important role in the prevention and treatment of diabetes and we support this 

important legislation. We urge passage by the Energy and Commerce Committee as soon as possible to facilitate 

expedited approval by the House and subsequent approval by the Senate this year. 

The Academy is hopeful the Committee's action will allow for enactment ofH.R. 1192 this year and help the 

nation pivot from continuing the status quo to instead undertaking more proactive and innovative approaches to 

metabolic, autoimmune and insulin resistant diseases, which include the escalating diabetes epidemic and the 

chronic disease complications of diabetes. The total cost of diabetes to the United States has increased nearly 50 

percent in five years, totally $322 billion annually. This cost includes not only diabetes care, but also the myriad 

of complications that occur with diabetes. Furthermore, there are 29.1 million people in the U.S. with diabetes and 

86 million with prediabetes. As the Medicare population continues to grow at a rapid pace, it is critical that 

Congress act now to ensure our health care dollars are invested in the best treatment and prevention options for 

people with prediabetes and diabetes. 

As you know, H.R. 1192 creates a commission that includes leaders ofthe federal government, as well as private 

sector health care professionals, including registered dietitian nutritionists (RDNs) who provide care for patients 

with prediabetes and diabetes. The commission would review the myriad of ways the government currently 

spends money on activities related to the diagnosis and treatment of diabetes and other metabolic, autoimmune 

and insulin resistant diseases and make recommendations on how these funds could be utilized more effectively. 

Through innovation, collaboration and application of advances in care called for in this important legislation, H.R. 

1192 provides the opportunity to address the enormous economic and human toll caused by diabetes and 

complications of the disease. 
98 


22416.058

The Academy of Nutrition and Dietetics thanks you and the Members of the Energy and Commerce Committee 

for taking action to advance H.R. 1192 and we again offer the Academy's support for this critica11egislation. We 

look forward to working with you to secure its passage by the House of Representatives and the Senate this year. 

Sincerely, 

Lucille Besclcr, MS, RDN, LDN, CDE, FAND 

President 2016-2017 

Academy of Nutrition and Dietetics 
99 


22416.059

novo nordislt 

Statement of Novo Nordisk 

In Support of H.R.1192, the National Diabetes Clinical Care Commission Act 

To the Subcommittee on Health 

Committee on Energy and Commerce 

United States House of Representatives 

Hearing on "Examining Legislation to Improve Public Health" 

September 8, 2016 

Thank you for giving us the opportunity to provide a statement in support of H.R. 1192, the 

National Diabetes Clinical Care Commission Act. Novo Nordisk applauds the House Energy 

and Commerce Committee for taking up this important legislation, which could help improve 

care for people living with diabetes. 

Novo Nordisk is a healthcare company with more than 90 years of history in innovation and 

achievement in diabetes care. In addition to diabetes care, Novo Nordisk is a leader in obesity, 

hemostasis management, growth hormone therapy, and hormone therapy for women. Novo 

Nordisk's business is driven by our Triple Bottom Line: a commitment to economic success, 

environmental soundness, and social responsibility to our employees and customers. Our global 

headquarters are in Denmark and our US headquarters is in Princeton, New Jersey. We conduct 

research and manufacturing in the US, and have over 6,000 US-based employees in all 50 states. 

Here in the US, Novo Nordisk is also proud to be a Co-Chair of the Diabetes Advocacy Alliance, 

a coalition of 20 members representing patients, professional and trade associations, other 

nonprofit organizations, and corporations, all united in the desire to change the way diabetes is 

viewed and treated in America. 

We agree that it is time to look at how the Federal Government can best manage its programs 

and spending on diabetes, so that we can meaningfully impact the numbers of people living with 

these diseases, and also improve the care and quality of life for patients with chronic conditions. 

The epidemic of diabetes is devastating our healthcare system and economy. Today, more than 

29 million people in the U.S. are living with diabetes, and another 86 million are at high risk for 

the disease.' During the past 30 years, the percentage of Americans diagnosed with diabetes has 

more than doubled; and by 2050, one in three will be living with diabetes.ii In the Medicare 

population, the statistics are just as concerning. Over one quarter of the Medicare-eligible 

population (nearly 11 million seniors) have diabetes. iii Furthermore, the longer people live with 

diabetes, the more likely it is they will suffer from related complications, including heart disease, 

blindness, lower limb amputations, and kidney failure, among others. 1 These largely preventable 

problems will add a human and economic burden not only to the individual and their families, 

but also to the federal government. 
100 


22416.060

This chronic disease continues to be one of the largest healthcare threats to our nation's 

economy. Currently, one in three Medicare dollars is spent on people with diabetes, and the cost 

of diabetes has continued to increase significantly, with the true total cost of diabetes rising to 

$322 billion per year in 2012, up 48 percent ($1 00 billion) in just five years from $218 billion in 

2007.tv For individuals, the financial burden is taking a toll. People diagnosed with diabetes 

have healthcare costs 2.3 times higher than if they didn't have the disease.1 

We have the real potential to bend the Medicare and Medicaid cost curve if we can begin to 

develop effective strategies to reduce the onset of diabetes and the costly but preventable 

complications of the disease. Currently there are 35 different agencies that have programs or 

policies related to diabetes. However, they are largely uncoordinated and likely redundant. This 

bill would create a commission for the purpose of improving the implementation and 

coordination of clinical care for patients with pre-diabetes, diabetes, and the chronic diseases and 

conditions that result from diabetes. The Commission, representing a partnership between 

private sector experts and specialists in the Federal agencies most active in clinical care, will 

issue recommendations to Congress and to the Secretary ofHHS on new approaches to improve 

patient care, such as getting information and resources to clinicians on best practices for 

delivering high quality care, and effectively deploying new treatments and technologies, such as 

the artificial pancreas. The bill requires no new money because the legislation specifies that the 

Commission meetings will be supported through existing HHS funds. 

Several states have already recognized the toll of diabetes on their state budgets and have passed 

Diabetes Action Plans in their state to address this epidemic. We believe strongly there should be 

a similar response at the federal level and that is why we urge for passage of the National 

Diabetes Clinical Care Commission Act. 

According to the Novo Nordisk Way, our contribution to society comes from having a patientcentered approach. Given this bill's potential to make a difference in the Jives of millions of 

people with diabetes and prediabetes, we strongly support its passage and look forward to 

working with you as this bill progresses. 

1 Centers for Disease Contro1 and Prevention. National Diabetes Statistics Report 2014. Accessed June 10. 2015. 

"Centers for Disease Control and Prevention. Projection of the year 2050 burden of diabetes in the US adult population: dynamic mcxleling of 

incidence, mortality, and prediabetes prevalence Report 2010. Accessed June 10, ZO!S. 

!»Center for Medicare: and Medicaid Services, Chronic Conditions Among Medicare Beneficiaries Chartbook: 2012 Edition. lv ADA Cost of Diabetes in 2012. 
101 


22416.061

September 6, 2016 

The Honorable Fred Upton 

Chairman 

U.S. House of Representatives 

Energy and Commerce Committee 

Washington, D.C. 20515 

The Honorable Joe Pitts 

Chairman 

U.S. House of Representatives 

Health Subcommittee 

Washington, D.C. 20515 

The Honorable Pete Olson 

U.S House of Representatives 

2133 Rayburn House Office Bldg. 

Washington, D.C. 20515 

The Honorable Frank Pallone 

Ranking Member 

U.S. House of Representatives 

Energy and Commerce Committee 

Washington, D.C. 20515 

The Honorable Gene Green 

Ranking Member 

U.S. House of Representatives 

Health Subcommittee 

Washington, D.C. 20515 

The Honorable Dave Loebsack 

U.S. House of Representatives 

1527 Longworth House Office Bldg. 

Washington, D.C. 20515 

Dear Representatives Upton, Pallone, Pitts, Green, Olson and Loebsack: 

Diabetes 

Advocacy 

Alliance~ 

The Diabetes Advocacy AllianceTM (DAA) would like to express our support and appreciation for the public 

health hearing the House Energy and Commerce Committee's Subcommittee on Health has scheduled for 

September 8th, 2016 regarding H.R. 1192, the "National Diabetes Clinical Care Commission Act." The DAA 

strongly supports this legislation and urges the Energy and Commerce Committee to pass the legislation as 

soon as possible so it can move expeditiously to the House and Senate floors for approval. 

The DAA is a coalition of 21 diverse member organizations, representing patient, professional and trade 

associations, other non-profit organizations, and corporations, all united in the desire to change the way 

diabetes is viewed and treated in America. Since 2010, the DAA has worked to increase awareness of, and 

action on, the diabetes epidemic among legislators and policymakers. The organizations that comprise the 

DAA share a common goal of elevating diabetes on the national agenda so we may ultimately defeat diabetes. 

The "Notional Diabetes Clinical Core Commission Act" creates a commission comprised of federal government 

leaders and private sector experts in diabetes and its disease complications to review how the government is 

currently spending resources on activities related to the diagnosis and treatment of diabetes and other 

metabolic, autoimmune and insulin resistant diseases. The commission would then make critically important 

recommendations on how to utilize these funds more effectively. The legislation has the potential to reduce 

the overwhelming impact diabetes has on health spending while at the same time improving care for some of 

the nearly 30 million Americans living with diabetes and the many more tens of millions of Americans 

www.diabetesadvocacyalliance.org 
102 


22416.062

impacted by the disease. 

Again, we thank you and the Members of the Energy and Commerce Committee for taking action to advance 

H.R. 1192 which has very strong, bipartisan support. The DAA looks forward to working with you to secure its 

passage by the House of Representatives and Senate this year. 

Sincerely, 

Karin Gillespie 

Novo Nordisk 

DAA Co-chair 

Meghan Riley 

American Diabetes Association 

DAA Co-chair 

www.diabetesadvocacyalliance.org 

Henry Rodriguez, MD 

Pediatric Endocrine Society 

DAA Co-chair 
103 


22416.063

AMERICAN SOCIETY OF HEMATOLOGY 

2021 L Street, NW, Suile 900, Washington, DC 20036 ph 202.776.0544 tax 202.776.0545 o·moll ASH@hematology.org 

American Society of Hematology 

Statement to the 

Committee on Energy and Commerce 

Subcommittee on Health 

Hearing on Examining Legislation to Improve Public Health 

September 8, 2016 

Chairman Pitts, Ranking Member Green, and members of the Energy and Commerce 

Subcommittee on Health, the American Society of Hematology (ASH) is extremely grateful for 

the opportunity to provide written testimony for the record on H.R. 1807, the Sickle Cell Disease 

Research, Surveillance, Prevention, and Treatment Act of2015, which was introduced last year 

by Representatives Danny Davis and Michael Burgess. We applaud their efforts along with 

those of members of the Congressional Sickle Cell Disease Caucus. 

ASH represents more than 16,000 physicians, researchers, and medical trainees committed to the 

study and treatment of blood and blood-related diseases such as leukemia, lymphoma, and 

myeloma; non-malignant conditions, including anemia and hemophilia; and congenital disorders, 

such as thalassemia. ASH members also include clinicians who specialize in treating children 

and adults with sickle cell disease (SCD) and researchers who investigate the causes and 

potential treatments of sickle cell disease manifestations. 

SCD is an inherited, lifelong blood disorder that causes individuals to produce abnormal 

hemoglobin, causing their red blood cells to become rigid and sickle-shaped. These sickled cells 

have a shortened lifespan, resulting in a constant shortage of red blood cells. When these sickled 

cells travel through the blood, they often get stuck in the smaller blood vessels, blocking other 

oxygen-rich red blood cells from freely flowing throughout the body. This leads to 

complications ranging in severity, including severe pain, acute chest syndrome (a condition that 

lowers the level of oxygen in the blood), stroke, organ damage, and even premature death. In the 

United States, approximately 100,000 individuals have SCD. The Centers for Disease Control 

and Prevention (CDC) estimates that SCD affects 1 out of every 365 black or African-American 

births, and 1 out of every 16,300 Hispanic-American births. 

Although the molecular basis of SCD was established five decades ago, it has been challenging 

to translate this knowledge into the development of novel targeted therapies. Many important 

discoveries have been made, and some treatments developed. These discoveries have identified 

innumerable questions and opportunities to better understand and treat this complex disease. 

Yet, many basic scientific processes are still not fully understood, too few treatments have been 

developed, and care that could improve the duration and quality oflife for individuals with SCD 

is inconsistent in the United States and wholly absent in large parts of the world. 

Currently, Hydroxyurea, the only FDA-approved drug for adults with SCD (often used off-label 

in children), improves the course of SCD and might lead to significant health care cost 

reductions. In a two year pediatric study, overall health care costs for children on hydroxyurea 

were $1.8 million, compared with $2.5 million for those who did not receive this treatment. 
104 


22416.064

Unfortunately, hydroxyurea is not regularly prescribed and adherence to the therapy is poor. In 

addition to hydroxyurea, blood transfusions can help to manage SCD, but they can lead to 

abnormally high levels of iron in the blood, which can cause long-term organ damage and 

reactions due to a mismatch between the donors and recipients. There has also been early 

success in curing SCD through bone marrow transplant, but this process is costly, dangerous, and 

is not widely available. 

In an effort to identify unmet medical needs for people with SCD, ASH, along with other groups, 

earlier this week issued the State o[Sickle Cell Disease: 2016 Report, evaluating the disease in 

four priority areas- access to care, training and professional education, research and clinical 

trials, and global health. The report shows that significant improvements are needed across all 

areas and that, though patients are living longer, the system of care needs to change to ensure a 

better quality of life. To address these challenges, ASH launched the Sickle Cell Disease 

Coalition along with more than 20 other organizations who are issuing a call to action that will 

amplify the voice of the SCD community, promote SCD awareness, and transform SCD care 

both in the United States and around the globe. 

ASH thanks the Subcommittee for focusing needed attention on SCD and supports The Sickle 

Cell Disease Research, Surveillance, Prevention, and Treatment Act of 2015 (H.R. 1807). 

However, the Society believes there is the need for additional legislation to enhance current 

federal activities in SCD research, training and services. 

Strengthening and expanding current efforts will help ensure that individuals living with this 

disease receive adequate care and treatment. In fact, a multi-pronged approach by key Federal 

agencies in collaboration with private sector sickle cell stakeholder groups could more 

efficiently, effectively and economically improve the comorbidities and health disparities faced 

by individuals with SCD and their families. Specifically, ASH believes the following priorities 

are important components of any legislative package addressing SCD: 

• Authorization of the Department of Health and Human Services' Interagency Working 

Group on SCD to coordinate efforts among federal agencies. 

• Enhancement of the Centers for Disease Control and Prevention's SCD outreach and 

education programs on SCD and SCD trait for patients and providers. 

• Improvement of access to high quality care via demonstration projects at CMS and 

development of best practices. 

• Provision of incentives for drug development for SCD within the Food and Drug 

Administration (H.R. 1537, Advancing Hope Act of2015). 

The status quo is unacceptable, and we must improve the circumstances under which care for 

individuals with SCD is provided. Now is the perfect time- today, we have opportunities to 

improve overall treatment, care, and quality of life for millions of people, especially young 

children. We are calling for action in sickle cell. 
105 


22416.065

Thank you again for the opportunity to submit testimony. Please contact Stephanie Kaplan, ASH 

Senior Manager of Government Relations and Public Health, at 202-776-0544 or 

skaplan@hematology.org, or Tracy Roades, ASH Legislative Advocacy Manager, at 202-776-

0544 or troades@hematologv.org. if you have any questions or need further information. 
106 


22416.066

22416.067

The organizations that endorsE the 

State of Sickle Cell Disease: 2016 Report include: 

CJAPHr\ • 

Inc. 

®1 

eSTl'IOl'I'Gffi'H•" 

I=· .... --·- a 

Suf!"mary 

Summary 

Over the past century, great advances have been made in the unders

and treatment of sickle eel! disease (SCD}. This first "molecular diseas

by a stng!e gene mutation, has advanced the field of modern human 

biology. Many tmportant discovenes have been made, and some trea

developed. These discoveries have identified innumerable questions 

opportunittes to better understand and treat this complex disease. 

basic scientific processes are still not fully understood, too few treat

been developed, and most of the people who have SCD do not hav

w these treatments which could improve the duration and quality o

With profound concern about the unmet scientific opportunit1es and 

of access to care, a group of SCD stakeholders has conve

is eager to f1nd remedies

document outlines their 

and details a comprehen

to address these issues. 

The plan encompasses f

areas- Access to Care in 

States, Training and Pro

Education, Research and 

Tnals, and Global Issues 

to SCD- which will be the focus of our collective efforts toward ultima

advancing SCD care, early diagnosis. treatment, and research. 

tor a detatled list of strategies to address these iSSues VISit scdc

22416.068

Sickle eel! d.sease {SCD), whtch causes 

a wtde range of severe and even life· 

tnreatemng consequences, IS caused by a 

single mtsspelhng 1n the )NA instructtons 

for hemoglobin, a protem v.tal for carrying 

oxyge:l m the blood As a result of this 

mutatton, indivtduals with SCD expenence 

!tfelong comphcattons mduding anemia, 

mfections, stroke, t1ssue damage, organ 

failUre, intense pamfu! eptsodes, 

and premature death. These debilitating 

symptoms and the complex treatment 

needs of people living with SCD often Hm1t 

thetr education, career opportunities, and 

quality of life. 

The many comp!icattons of SCD can make 

every stage of life extremely chal!engmg 

for indivtduals with the disease. For 

example, approximately 10 percent of 

chtldn;f"l wtth SCD will have a symptomatic 

stroke, whiCh can cause learntng problems 

and lifelong disabilittes. Patn is the most 

common clinical manifestation of SCD and results 1n tremendous suffering, 

prolonged absences from school, and 

dtfficulty maintalnmg full employment. 

lndlvtdua!s with SCD experience chrontc 

pain compounded with acute pain 

episodes. In a six-month study of adults 

With SCD, ha!f of the respondents 

reported experiencmg some level of pa;n 

for half of the days, wh1le nearly a th1rd 

noted hav:ng pa1n the majonty of the 

t1me.' Acute patn eptsodes can occur 

multiple mnes per year and may result tn 

long hospital stays, leading to a complete 

disruption of the person's life and hts or 

herfami!y'shfe. 

SCD is the first known molecular 

disorder. However, advances in 

treatment have been limited compared 

with later discovered molecular dtseases 

-such as cystic ftbrosts- where multiple 

treatments have benefited pat1ent 

populations. Over the past century, 

several important discoveries have 

been made m the understanding and 

treatment ofSCD. Yet, many very basic 

scientific processes are not understood, 

and far too few treatments have been 

developed. ln addttion, most people Wltn 

SCD do not have access to treatments 

tnat could improve the duration and 

quahtyofthetrlives. 

We know that more can be done for 

people w1th SCD to provtde tmproved 

access to h1gh quality care. Whtfe a cure 

currently extsts, It is available to only a 

small portion of the pattent population 

and limited primarily to developed 

countries. The health outcomes and 

treatment disparities related to SCD 

make 1t a public health pnority both in 

the United States and globally. 

There ts enormous opportunity to 

improve the state of SCD. There are 

actions we can take today to address 

unmet needs- both tn the Un!ted States 

and around the world. 

lntheUrutei'!Stato:s itiSe'S!Jm<:ote.:J:that 

About11n13 

Afncilr.Arr.enl:<ln 

Stddecelltralt(SCT) 

22416.069

We must address these disparities 

by mcreasing access to d1agnost1c 

and therapeutic interventions, and 

provid1ng comprehensive care and 

appropnate support services to 

There is enormous opportunity to 

improve the state of SCD. There are 

actions we can take today to address 

unmet needs -both in the United 

people living with SCD. 

Emerging therapies 

and promising 

new insights m the 

treatment of SCD 

represent a sigmficant 

States and around the world. step toward impco,;ng 

outcomes and 

reducing the treatment burden for 

affected ch1ldren and adults. 

Alleviating the pam and suffering 

caused by this disease, as well as the 

socioeconomic costs, is entwely withm 

our grasp. Although conquering 

SCD is doable, It requires a wellIntroduction orchestrated plan and a coordinated effort from a range of partners including government agencies, patient advocacy organizations, health care providers, public health organizations, researchers, foundat1ons, pharmaceutiCal and biotech companies, and other stakeholders. !nan effort to advance a forward-looking and comprehensive agenda that will make a significant difference, a group of SCD researchers, clinicians, individuals with the disease, and po!ic:ymakers have come together to develop an organized approach to Improving outcomes for people w1th SCD. From these meetings, four priority areas were identified to focus our collective efforts toward ultimately advancing SCD care, early diagnosis, treatment, and research. 

Globally Jt I!> est1mated that 

l 

;JCDisJPOSfpf"l!V'ak-ntlnyrtalarla 

pnffiarl!y AfriG>, ti'le Middle East; 

and$outhAsla 

lmtoduct:on 

THE FOUR PRIORITIES 

to improving outcomes for people with sickle cell disease 

Access to Care in the U.S. 

rn 

Research and Clinical Trials 
110 


22416.070

22416.071

AmencanSooetyofHelll<ltology 

llJRRINISIAll 

0 

'·'";) .. 31% 

<:;>""'ii4os'/y- .~/;1£7.; / ';'' ; ;? 

/ 

SlJteofSickleC..,UO!sease A[CE!~Sto(are More than 75 percent of adults with SCD with frequent 

pain crises fail to get hydroxyurea, which is the 

recommended treatment.1 

Desprte un1versal newborn screening for SCD in the United States, 

one study found that long·term follow-up after diagnosis was not 

performed in nearly one-third (30.8%) of cases.l 

SCD is also associated with h1gh treatment costs. For an average 

person with SCD reaching age 45, total lifetime health care costs 

were estimated to be nearly $1 million, with annual costs ranging 

from over $10,000 for children to over $30,000 for adults.~ Hydroxyurea, the only FDA-approved drug for adults with SCD 

(often used off-label in children), improves the course ofSCD 

and might lead to significant health care cost reductions. In a 

two year pediatric study, overall health care costs for children 

on hydroxyurea were $1.8 million, compared with $2.5 million 

for those who did not receive this treatment.' Unfortunately, 

despite the National Heart, LUng. and Blood Institute's (NHLB!} 

recommendations, hydroxyurea is not regularly prescribed and 

adherence to the therapy is poor. 

------; 1111 ll!lc'------~4 Sctlh<nb«~-Jor l>uMud••fJIIOr.<bytll~ Pt"''l~of~tkGbydlf/t-1 n~i~==~ Hetl'kkpro\lldes~f~Mformal ~o!:>st!!WsfOUtttot<lPM.«<Fm des<;dpiJ¢0ofsld4eceianemta, ofsid<leQbloodandnamesttla(!lse~ r~a•pow.,.~.an4 "ssd<ktced.mwa.• elongated'llloodC<!UfrQIDa Cl>!alt:odental:ltullel'!l A<:;ces~toCare .J't-~ StateofSKkleCell D1se~se AmencanSocoetyofHem

Though the mortahty rate for children with SCD decreased by 3 percent each year in a study from 1979 to 2005, the mortality rate for adults w1th SCD mer eased by 1 percent each year during the same period." People with SCD in the emergency department for pain experienced longer delays to administration of the initial analgesic compared With control patients, despite higher arrival pain scores and triage acuity levels.? favailablespedahzedprov•ders(e.g .. hemo r~e !nan _o~'::reliance e~e.~g:~cy dep Many children with SCD do not receive the necessary services to prevent serious complications from the disease. • Only one-third of children With SCD receive appropnate monltonng for strokensk. • 25 percen( of Children w1th SCD do not receiVe pneumooxcal vacc;nation, wh1ch !S recomm~mded for all children younger than five years of age.,. ~$5~~~--- Wl!l'llt<!stedtodrwngv.shbet'Neens.ldde 1111 ltJlo\emphi5,Ttnn.,2,SOOAf~Amerk<lm -----~--t cdtr;ut~nd!ildde«l!dfs.em 

22416.072

Today, individuals with SCD are living longer, 

but the system of care needs to change to 

ensure a better quality of life. 

1940 

In the Un1ted States, access to 

appropriate care is mfiuenced by a 

number of factors, 1ndud1ng health 

Insurance, the ava1lab1hty of health care 

proVlders, and provider expenence 

treat1ng people w1th SCD.Indiv1duals 

with SCD reported experiencing poor 

communication netween pnmary-care 

phySICians and hematologists, providers 

With lim1ted knowledge and expen<:>nce 

w1th pa1n management. d1ff1culty m 

scheduling urgent appointments, and lack of follow-up after hospltahzat1on:' 

People with SCD and their fam1hes 

nave expressed a need for better 

information, shared dedsion-making, 

and strengthened communication With 

those prov1d1ng the:r medical care. 

Other factors, such as geography, 

economic status, and co-ex1stmg condit.ons have an impact on access 

to care. Those liVIng in or around 

metropolitan areas are more likely to 

conditions related to SCD such as 

strokes or cognitive impairment struggle 

tonavigatethehealthcaresystem 

without assistance. 

The transition from ped1atnc to adult 

care particularly challeng1ng as 

the focus of care differs. In the Umted 

States, children With SCD are more likely 

than adults to rece1ve care 1r1 academiC 

med1cal centers, which have SCDspeof•c expertise. Most of these ch1!dren 

are seen by primary-care providerspediatncians, family mediCine doctors, 

and nurse practitioners- and many 

have access to ped1atnc hematologists. '1 

In contrast, adulrs With SCD are more 

likely to receive care in community hosp1ta!s, where SCD-spec1fic expert1se 

Js much less common.'! As more people 

with SCO are livmg 1nto adulthood, 

d1sease management needs to shift 

from acute care of complications to 

a chron1c care model that focuses on 

have access to knowledgeable prov1ders prevention of crises, and intervention 

than those l•v1ng in rural communities; and relief for common complications. A 

however, people with access to SCD coordinated heallh care delivery system 

treatment centers may have difficulty 

affording frequent and expensive 

treatments. Additionally, people with 

for adults wtth SCD is a step to 

improve health outcomes,14 

114$ 

GDAlSFDRTHEFUTURE 

Ensure implementation of existing Improve the pediatric-to-adult care 

standard-of-care guidelines and best transition by ensuring there are 

practices in disease management qualified physicians in both ped1atric 

as well as develop new resources and adult care, through creatwn of 

to help a!l physiCians make the best training incentives and retention of 

deCISions m treatmg people With SCD prov1ders who treat SCD. 

Implement coordinated care models Improve access to evidence-based care 

that Incorporate community health through innovative health care delivery 

workers, ensunng more equal qual1ty of models and incorporate SCD care 1nto 

care regardless of where an md1vidual the health care system and delivery 

with SCD receives care, 

Rwd more about the goals for the future at scdcoalition.org 

1!53 
113 


22416.073

22416.074

Amencan Sooety ofH~matology lllRIINTSIAII Trall',ngJr-c?rof€Ss•onaiEduc<Wor; 

Currently, there are not enough health care 

providers with the comprehensive knowledge 

and expertise to care for people with SCD. 

This knowledge gap not only exists within 

speCialty (e.g., hematologists) and pnmary 

care, but also among other proVIder groups, 

such as dmicians who provide hospital-based 

and outpatient care. This contributes to an 

enormous unmet medica! need, especially for 

adults with the d1sease who need coordinated 

chronic care and ongoing pain management. 

The unprediCtable and often pers1stent nature 

of the pain and other complications associated 

w1th SCD poses a diff1cult challenge for 

providers, especially for those unfamiliar with 

treat1ng people w1th this disease. 

Although comprehens1ve programs exist for 

some children with SCD, many adults do not 

have access to preventive and comprehensive 

care. Their health care providers may be 

inexperienced in treating SCD and the 

coordination of their care may be insufficient. 

And while there has been increased access 

to health insurance in the last few years, 

the number of providers with SCD expertise 

has not increased. Consequently, health 

~~~~~~~~Er: 

..... "" ______ __, an Sl<.l~e of St{kle D•S<oase AmencanSocteryofHem

outcomes for people w1th the disease have not improved.'a The issues with access often relate to training and comfort with providing care. The number of hematologists trained and wilhng to care for ch1ldren with SCD has ina eased, but still fewer are providing care for the more complex adults with SCD." In fact, a survey of prominent pedtatric SCD centers reported that the largest barrier to transition and continuity of care was the ability to identify an adult proV1der- specifically a hematologist- with the training. expertise, and comfort in treating this population.'" As a result much of the care of adults with SCD rakes place in emergency departments or other non-spectalty settings.6 Some people wtth SCD rely exclusively on the hospital or emergency department for care, due to a lack of SCD proVIders, which can greatly disrupt the continuity of care that they receive. In fact. nearly three-quarters of hospitalization for SCD onginates in the emergency department.'~ Frequency of emergency department visits is substantially higher for adults With SCO than for children. This is potentially problematic because people wtth SCO often warrant a comprehensive assessment related to their other medical complications (Le., comorbidities). However, the emergency care system is not designed to care for the chronic problems of an individual with SCD. Re!ymg on uncoordinated ep1sodic care also leads to increased health care costs and the potential for Inadequate or inappropriate treatment.9 Primary<are providers, such as family physicians, often provide care to individuals with SCD; however, they report feeling that they do not have adequate backgrounds in SCD management due to their lack of experience treating these indiV1duals. Factors currently associated wrt:h primary· care physicians' comfort !eve! in treating SCO is whether or not they typically see people With this d1sease, or from their previous tratning expenencesP Greater availability of clinical decision support tools tsu E11~n~~~~;~~--+ 
115 


22416.075
116 


22416.076

22416.077

CURRENT STATE 

@ 

(} 

ln tb.e United States, there are currently 36 clina:<:ll tnals under.vay 

related to SCD to evaluate novel agents and approaches. This 

marrow 

1980 

tm 

The majority of available treatments manage symptoms 

of the disease, rather than treating the underlying 

cause of sickling. 

Although the mo!ecdar bas1s of SCD was 

established more than half a century ago, 

1t has been d:a!leng1ng to translate thts 

research 1nto the development of nove! 

targeted therapies. wh;Je new approaches 

1n manag:ng th•S disease have 1m proved 

diagnosis and supportive care over the 

last few decades, people w1th SCD stl!i 

have severe complicatiOns to overcome. 

There !s no Widely available cure for SCD 

and few etfect1ve Treatment 

for those w1th SCD focuses on d1sease 

and pa1n management, treatment of 

complications, and acute care dunng 

sickl!ng cnses, whiCh occurs when red 

blood cei!s become ng1d and st1cky and block the flow through small blood 

vessels. Only one therapy, hydroxyurea, 

1s approved by the U.S. Food and Drug 

Adm1n1strat1on (FDA), but research 

shows that It is underused by health care 

providers and not taken wns1stently by 

md1viduals W1th SCD. 

22416.078

Regular blood transfusions are 

somet!mes used as a preventive 

measure; however, they can lead to 

abnormally high levels of 1ron m the 

blood, which can cause long-term organ 

damage and reactions due to a mismatch 

between the donors and reciptents. 

Currently there are a number of clinical 

trials underway; however, the maJonty 

are single agent trials sponsored by 

the pharmaceuticaltndustry and 1t 

will be years before they will translate 

into widely available and affordable 

theraptes. Now more than ever there 

is a need to develop a clinical trial network that could increase the number 

of patients recrUited and allow for 

assessment of combinatton therapies. 

Furthermore, there is great need for 

mcreased pain research and exploratton 

of additional curat1ve therapies. 

While bone marrow and stem cell 

transplantation are potentially curanve, 

their use is limited due to high cost, the 

difficulty of locat1ng matched donors, 

and the high level of nsk assoCiated 

With the procedure. While stiH man 

exploratory stage, gene therapy and 

genome ed1ting hold the promtse of a 

future approach for cunng SCD and other 

hemoglobin diSOrders. 

Today, there is an extraordmary 

opportumty to link research and care 

more closely. One way to achieve this 

IS through a dlnkal research network. 

Some of the most pressing research 

quest1ons that link to patient care include 

how to improve dosing and better 

anttdpate response to treatments hke 

hydroxyurea, mvestigating potentia! 

combinations of treatments, better 

defmtng the indications for transfusions 

m SCD, and improving the safety of 

transfusions by adopting accurate, stateof·the-art blood matChing protocols. 

While chm<:al trial research helps to move 

the needle on improving SCD care, often 

these trials stagnate due to insufficient 

patient enrollment."' n Therefore, it is 

1mperat1ve that individuals have a more 

aatve role in the clinical research process. 

90percent 

GOAlSFORTHEfUTURE 

Identify predictors of the severity 

of the disease. includmg optimiZing 

dosing and treatment response 

predictors for hydroxyurea, biomarkers 

for SCD cns1s and prognosts, and SCD 

dtagnostiC methods. 

Optimize the use of existing 

therapies. better defining the 

indications for red blood ce!! 

transfusions, and identifying prinop!es 

for accurate blood matchmg and 

developing longitudinal stud1es to determine long-term effectiveness of 

transfusions and hydroxyurea. 

Develop novel therapies, including 

combination therapies, new drugdelivery modes, and new agents that 

<:an be used in combinatiOn with 

hydroxyurea. 

Develop clinical trial networks 

to increase enrollment 1n climcal tnals 

and share the data with Interested 

stakeholders. 

Strengthen curative therapies, such as 

bone marrow and stem cell transplantation, 

and support funding for research in SCD 

gene therapy and genome editing methods 

Enhance pain research for improved 

outcomes such as pain, fatigue, and 

infertility, and create a validated SCDspedflc functional assessment tool for pain. 

Enhance the participation of individuals with SCD in setting the 

research agenda and increase patlent 

participation in clinical research 

Read more about the goofs for the future at scdcoa/ition.org 

For the ASH PnontHJsforSCD and Sickle Ce!ITra,t, Ciickhe<e 

uu "" _______
119 


22416.079

22416.080

CIJRR!NTSTAJI 

SJ 

<7 

---------r lOll --------> 

Organ,zations sucn as the World Health 

Organ•zat!on (WHO) and Umted Nations 

(UN} have recogniLeCl SCD as a global 

health ISSue. !n 2006, the World Health 

Assembly passed a resoi<JMn recognrz1ng 

SCD as a public health pr•ority and called 

on countries to tackie the drsease. Th1s 

resoluuon was also adopted by the 

Unrteo Natrons in 2009. 

The global picture of SCD IS Slmtlar to that 

of the disease in the Umted States before 

Children, stmlar to otber d1seases like 

malana and Hlv/ AIDS. In resource-poor 

countf"ies, 90 percent of children With 

SCD do noc survtve to adulthood. And the 

problem IS growing; by 2050 the number of 

people w<th SCD IS expected to rrocrease by 

about 30 percent globally."' 

Some middle-mcome countnes are 

rnakmg advancements 1n both early 

diagnoSIS and management of SCD. 8raz1l, 

for example, has shown remarkable 

1970. In countries w1th poor public heaith progress over the past two decades with 

s;stems and htgh levels of poverty, SCD newborn screemng programs being n~mams a maJor ki!ler of mfants and offered across the countr;." 

1011 

22416.081

A 10-year study in Rio de janeiro 

showed that d1agnosis through 

newborn screening programs and 

treiltment was associated with 

improved surviva! and quality of life of 

Brazilian children with SCD. Moreover, 

in Brad, health care mmntenance for 

SCD 1s seen as an essential component 

of pnmary care, and the government 

supplies hydroxyurea free of charge. 

This type of program has resulted in 

mcreased awareness and education 

for SCD, although there have been 

only modest improvements in 

mortality rates.jamatca represents 

another example of a middle-income 

cout"Jtry With SCD screentng and 

treatment approaches that have 

resulted 1n remarkable Improvements 

in the median !ife expectancy. 36 

As newborn screening and treatment 

efforts are Implemented around 

the world, h1gher surv1va! rates will 

increase the need for 1mproved 

treatmer>t opt1ons for adults with 

SCD and ongoing care. !n developmg 

our goals for the future, one of our greatest pnorfties IS des1gning, testmg, 

and Implementing sustainable care 

and pain management approaches for 

countries With limited resources. 

99%1 

SURVIVAl OF CHILDREN WITH SCD 94% UK I 

USA 1 

2109 288! 2811 

GOAlSfORTHEfUTURE 

lne,ee ec!ee<itlDn of governments 

and p!-ulanthroplCgroups about the 

1mportance of screening and canng 

for 1nd1vidua!s With SCD in heavily 

burdened countries. 

pn.wfd€!rs, such as increasing the 

number of health care providers 

that apply to specific, low-resource 

areas globally mcluding SCD adult care 

and enhanced use of community-based 

organizations. addressmg pam m low-resource settings 

Read more about the goofs for the future at scdcoalition.org 

2812 

22416.082

Conclusion 

Why IS now the nght t!me to focus on 

m:sd1sease? 

SCDisachromcdJsea<>ethathas 

been neglected for far too long. Those 

affected by this disease are among the 

most vulnerable and underserved, and 

the d1sease has a profound 1mpact on theirhves. 

Currently, the only approved drug 

for adults With SCD - hydroxyurea -

reduces the seventy and frequency 

of pa1nful episodes and is used for 

stroke prevention, but may not prevent 

acute complications or reverse organ 

damage that can result m early death or otPer health problems wh1ch affect 

the1r quality of hfe (i.e., comorb1dit1es) 

There is a need for the development 

of new treatments and a w1dely available and affordable cure for this disease. Ex1St1ng treatments and cures, 

1nduding bone marrow and stem cell 

transplantation, are underutt!tzed and 

do not reach the majonty of mdiv1dua\s 

-especially adults w1th SCD who could 

benefit from them. Increased strateg1es 

to educate prov1ders and people !ivmg 

With SCD are also needed to address 

1ssues of fragmented care. There IS a 

tremendous disparity m SCD o:;tcomes 

between ind,vlduals m low- and middle· 

mcome countnes compared to highincome countnes 

The status quo IS unacceptable. It JS 

tmperat1ve that we vastly Improve 

the Circumstances under wh1ch care 

Is provided. There IS opportunity to 

address these dispant;es and alter the coL<rse ofSCD, by 1mprov1ng overaH 

treatment, care, and quality of life for 

millions of people, espeoally young 

children, A number of orgar\!Zatlons are 

commg together to address this issue and each has important assets to he!p advance a comprehensive SCD action 

plan. To real change for those 

affected by SCD, we need partK!patlon from many different stakeholdersmduding government agencies, patient 

advocacy orgamzatlons, health care 

providers, researchers, foundations, and 

the pnvate sector. Those with SCD are 

wa1t1ng, and their well-bemg depends on 

our he!p. Their future IS in our hands. 

The time is now to change the course of the disease. 

Will you ;oin us? For more mformot!On VIS If scdcoalition.org 

1115 1115 2111 -'"-------t 

ASHhoststheASHSCI.)S<.snrMACallto 

prpgra:mJtopn:Mdert\edltcllassistal\<:ef«Sj":D 

"""""' 

References 
123 


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124 


22416.121

9/8J2016 Current Management of Sickle Cell Anemia 

~ Cold Spring Harbor 

~ Perspectives in Medicine 

Cold sm Harb Perspect Mrut 2013 Aug; 3{8): aQ11817. 

doi: 10.1101/glhpersped.a0118H 

Current Management of Sickle Cell Anemia 

Patrick I. McGann,1·

2 A!ecja C Nero,3 and Busse!! E. Ware1

•

2 

1 Te)(8S Children's Center for Global Health, Houston, Texas 77030 

Zrexas Chik:lren's Hematology Center, Baylor College of Medidne, Houston, Texas 770"30 

3university of Texas Southwestern Medica! Center, Danes, Texas 75390 

Correspondence:Emaij; re'!Ej![§@bgn.adu 

~@ 2013 Cold Sprin{j Harbor Laboratory Press; au rld'J.ts reserved 

This article has been~ other articles In PMC. 

PMCID: PMC3721270 

Go to: 

Proper management of sickle cell anemia (SCA) begins with establishing the correct diagnosis early in life, ideally 

during the newborn period. The identification of affected infants by neonatal screening programs allows early 

initiation of prophylactic penicillin and pneumococcal immunizations, which help prevent overwhelming sepsis. 

Ongoing education of families promotes the early recognition ofdisease-re]eased complications, which aUows 

prompt and appropriate medical evaluation and therapeutic intervention. Periodic evaluation by trained specialists 

helps provide comprehensive care, including transcranial Doppler examinations to identify children at risk for 

primary stroke, plus assessments for other parenchymal organ damage as patients become teens and adults. 

Treatment approaches that previously highlighted acute vaso-occlusive events are now evolving to the concept of 

preventive therapy. Liberalized use of blood transfusions and early consideration of hydroxyurea treatment 

represent a new treatment paradigm for SCA management. 

The natural history of untreated sickle cell anemia (SCA) is well described and documents serious morbidity and 

early mortality (powars 1975.; Platt ct al 1994; Se,cjeant 1995; Powarn et al 200~). Hemolytic anemia, acute vaseocclusive events (VOEs), and chronic end~organ damage begin early in life, and complications accumulate 

throughout childhood. Without early identification or specific interventions, many patients with SCA have poor 

quality of life, and most die as young adults ofSCA-related complications(~; Rogers et al 1978). 

Fortunately great strides have occurred over the past 40 years, and better management strategies have altered this 

previously bleak outlook. Despite the complexity and multifactorial pathophysiology ofvaso-occlusion <Will:!:. 

2illili!), relatively straightforward measures have greatly improved outcomes for children with SCA: (I) early 

identification by neonatal screening programs; (2) education of parents and patients about medical complications 

and early recognition; (3) preventive measures with prophylactic penicillin and pneumococcal immunizations; (4) 

aggressive treatment of acute VOEs including hydration, analgesics, antibiotics, and transfusions; (5) screening 

programs for early signs of organ damage, especially primary stroke risk using transcranial Doppler (TCD) 

examinations; and (6) therapeutic intervention with transfusions, hydroxyurea, or stem cell transplantation. For 

children receiving medical care at comprehensive care programs, 95%-99% survival rates into adulthood are 

documented Cfelter et al 2007; Quinn eta! 20 I 0). For adults with SCA, screening programs and anticipatory 

guidance are less standardized but still critical, and the benefits of preventive therapy using hydroxyurea are even 

more compelling. 

http:llwww.ncbi.nlm.nih.gov/pmc/artides/PMC3721270/ i/17 
125 


22416.122

91812016 Current Management of Sickle Cell Anemia 

Here we will focus primarily on the management ofSCA (HbSS or HbSiiJ0 -thalassemia). We emphasize and 

summarize general principles of care and rnanagernen~ rather than discussing details of pathophysiology or 

mechanisms of disease. The management of specific examples of acute VOEs will be highlighted. 

EARLY IDENTIFICATION Go to: . --- -----~~~ 

Perhaps the most critical aspect of optimizing SCA management is early identification of affected patients, before 

the onset of signs and symptoms of disease. Without early diagnosis and intervention, SCA often acts as a swift and 

invisible killer, with many infants dying suddenly of bacterial sepsis or acute splenic sequestration crisis (ASSC) 

within the first few years of life (Pearson eta! I 969, .l9n; Rogers eta! 1978; Powars ct a! 1981). Sometimes futal 

complications oecur even before families or medical providers are aware the infants have SCA. 

Witl1 the recognition that infants with SCA have greatly increased risk of bacterial sepsis, the landmark multicenter 

double-blinded placebo-controlled PROPS trial proved that penicillin prophylaxis significantly lowered the risk of 

bacteremia and death (Gaston et al. I 986). This simple intervention provided the justification needed for newborn 

screening of SCA, to identifY affected infunts soon after birth and to allow lifesaving prophylactic antibiotic 

therapy. Although a 1987 Nll! Consensus Conference recommended newborn screening for SCA, universal 

screening was not accomplished in ail U.S. states and tetTitories until 2006. 

Newborn screening programs in the United States, Jamaica, and Europe have documented the utility of early 

identification of SCA, with a marked reduction in motbidity and mortality, especially in the first 5 years of life 

(Rogers et al 1978; Yichinsky et al 1988; Almeida et al ?QQI; Bardakdjjan-Mjchan eta!. 2001). :Eiilw:llJ. 

illustrates that early identification ofSCA through neonatal screening programs has contributed to the improved 

survival rates (Quinn et al 20 I Q). 

EiiDu:U.. 

Survival curves of infants with SCA in the United States and Jamaica, by 

era. This research was originally published in Blood. (From~ 

2lll.Q; reprinted, with permission,© American Society ofHematology.) 

Testing of newborns in the United States for SCA began with targeted screening, which involves selecting at-risk 

populations to screen, such as babies whose parents arc AfiicanwAmerican. Such an approach is problematic in 

several ways, and has evolved now to universal screening for all newborns. In contrast, most European countries 

still perform targeted screening for infants most likely to be affected, such as those of African ancestry. Although 

potentially cost-effective, targeted screening almost certainly misses some babies with SCA, and presents 

difficulties related to both equity and logistics (Grosse et al. 2005). Despite the high burden of disease, newborn 

screening has yet to be implemented systematically in Africa, although pilot studies document a high incidence of 

trait and disease. 

Newborn screening for SCA requires a small dried blood spot (DBS) for analysis, collected from cord blood or the 

infant's heel/toe. Collection technique is important; DBS specimens have variable quality by the amount and 

distribution of blood on the filter paper. Specimens are most easily co11ected in the neonatal period for babies born 

in the hospital, or during initial immunization visits for babies born at home. Testing in the United States is most 

commonly performed by hemoglobin electrophoresis using isoelectric focusing (IEF), which easily distinguishes 

abnormal sickle hemoglobin (HemoglobinS, HbS) from normal hemoglobin (HbA) and fetal hemoglobin (HbF), 

as illustrated in ~· High-performance liquid chromatography (HPLC), capillary electrophoresis (CE) 

techniques, and even DNA-based laboratory diagnosis also can be used for accurate diagnosis. When possible, 

parental studies should also be performed to confmn the diagnosis of SCA. 

Figure2 

lsoelecbic focusing (IBF) electrophoresis technique for identification of 

SCA. Blood specimens from AA, AS, and SS patient controls are shown on 

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EDUCATION 

" ""- --·----~ 

CU"rent Management of Sickle Cell Anemia 

tl1e leji, along with a manufactured Hb AFSC control in tl1e center. 

Newborn samples, typically obtained from ... 

Go to: 

Parental and family sickle cell education should begin once the diagnosis is made and continue throughout 

childhood. Given variable parental education and literacy, education should be provided in written and spoken 

form~ and should be repeated with each visit to ensure information is comprehended. When possible, both parents 

should receive education, plus extended fanlily members and other caregivers, to become knowledgeable about 

SCA. 

Education in the early newborn period should focus on the basics of SCA, including its genetics and inheritance, 

need for penicillin prophylaxis, and benefits of protein-conjugated pneumococcal immunizations. At each visit 

these key points should be repeated to parents and caregivers. The importance of regular medical care should be 

emphasized, especially the need for prompt medical evaluation for fever. Antipyretics should never be given for 

fever at home, because this treatment can mask a serious infection. Education for young patients should also 

include signs and symptoms of ASSC: pallor, fussiness or irritability, and tender splenomegaly, Teaching parents to 

palpate their baby's spleen regularly, ideally several times a day during routine diaper changes, allows early 

diagnosis of ASSC and potentially prompt and lifesaving intervention. 

As the child grows, education should focus more on recognition and early medical intervention for acute vaseocclusive complications such as dactylitis and other painful events, respiratory distress, acute chest syndrome, and 

stroke. Parents should learn to manage ntild pain at home with oral hydration and analgesia. At an early age, 

families should be introduced to possible treatment options including hydroxyurea and transfusions, and even stem 

cell transplantation, if available. As affected children grow up and enter adolescence, it is critical to provide 

ongoing education about SCA and its complications, to provide young patients with the skills necessary to 

understand and advocate for their own medical care. Such self-awareness and investment in their medical care 

becomes critically important on transition from pediatric to adult hematology care. Unfortunately, evidence suggests 

increased morbidity and mortality in late adolescence and early adulthood following this transition of care 

(Brousseau et al 20 I 0; Quinn et al 20 I 0). 

Go to: 

As the result of vascular congestion, intraparenchymal sickling, and hypoxic injury to the spleen, infants with SCA 

have early loss offiltrative splenic function and are susceptible to acute life-threatening infections, particularly from 

encapsulated bacteria such as Streptococcus pneumoniae and Haemophi/us influenzae type b (Wjnkelstejo and 

Drach man 1968; Pearson et al l 969; Pearson 1977). This risk remains increased throughout life, but is most 

significantly increased in the first 5 years, when bacteremia incidence is the highest (Qverturfet al 1977; ~ 

i~L.J.2ll; Gill et a! 1995). 

The PROPS trial showed that prophylactic oral penicillin reduced the frequency of bacterial infection by 84% 

among young children (age 3-36 mo) with SCA (Gaston et al. !986). However, the tollow-up PROPS 2 study was 

unable to show benefit from penici!1in after age 5 yr, primarily owing to their lower incidence of bacteremia 

(Falletta ct al 1995). After introduction of protein~conjugated pneumococcal vaccines, the incidence of invasive 

pneumococcal disease decreased by 93.4% among young children with SCA (Halasa et al 2007). In Africa the 

dangers of pneumococcal sepsis for children with SCA have been questioned, but recent data confu:m its 

prevalence and lethality (Williams et al 2009). 

Based on overwhelming evidence, early pneumococcal prophylaxis is recommended for all infants with SCA. 

Penicillin 125 mg by mouth twice daily should begin by 3-4 mo of age, as a liquid fonnulation or crushed tablet. 

The penicillin dose should be increased to 250 mg by mouth twice a day as the child grows, typically at 3 yr of age. 

Some international programs recommend monthly TM penicillin to help ensure compliance. Oral erythromycin can 

be used as a substitute if penicillin allergy or msh develops, but this is uncommon. 

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91812018 Curren! Management of Sickle Cell Anemia 

Pneumococcal immunization should begin with locally available pneumococcal conjugate vaccines (7, 10, or 13 

valency) and supplemented at age 2 and Sr-7 yr with the 23-valent pneumococcal polysaccharide vaccine 

(Pneumovax). Additional recommended vaccinations include the H. injluenzae type h series, meningococcal 

conjugate vaccine (Menactra}. and yearly influenza. When locally feasible, the published immunization schedule 

for high-risk children as recommended by the American Academy of Pediatrics 

(www2 aap orgflmmunizationi!ZSchcdule html) should be followed. 

Penicillin prophylaxis should continue through age 5, when the risk of invasive bacterial disease is lower. Once the 

inununization series is up to date, including the Pneumovax booster, children with SCA may discontinue penicillin 

prophylaxis. However~ penicillin shou1d be continued indefinitely if a child has had culture-positive sepsis or a 

surgical splenectomy. 

The dramatically increased risk of overwhelming and rapidly fatal infection among young patients with SCA must 

be understood by all caregivers and medical providers. Fever> 3S.5°C is a medical emergency requiring prompt 

medical evaluation, including physical examination with vital signs and splenic palpation, blood culture, complete 

blood count, reticulocytes, urinalysis, and chest X -ray if clinically warranted. Type and crossmatch should be 

obtained if there is extreme pallor, splenomegaly, clinical instability, or acute respiratory or neurologic symptoms. 

After obtaining the blood culture, broad-spectrum antibiotics (e.g., cefuiaxone) should be administered 

intravenously. Addition of another broad-spectrum antibiotic (e.g., vancomycin) should be considered if the child 

appears toxic, has high fever, or suspicion of central nervous system (CNS) infection. 

Hospitalization is recommended if clinical or laboratory indicators suggest sepsis, such as hemodynamic 

compromise including hypotension, child < 1 yr of age, prior history of sepsis, temperature >40°C, WBC > 30 x 

1 09/L, or <3 x 109 fL. concurrent symptoms such as pain or acute anemia, or if close follow·up is not reliable~ 

ct a1,200!). 

Go to: 

Pain Events 

The sudden onset of pain tlmt occurs frequently in patients with SCA results from acnte intravascular sickling, so is 

often referred to as painful VOE or vaso-occlusive "crisis" (VOC). Although many providers and patients use the 

simple phrase "pain crisis," VOE is preferable because it broadly defines the process and avoids stigma about pain 

perception and management. 

The V 0 E results from erythrocyte sickling, microvascular occlusion, and tissue ischemia/reperfusion, and is a 

hallmark clinical feature ofSCA. Pain is the most common cause of acute morbidity ofSCA} and is associated with 

severity of disease and early mortality among young adults Q'latt ct al 1991). Pain often accompanies acute chest 

syndrome (ACS), a serious and potentiaUy life-threatening complication ofSCA, in 72% of cases (Platt ct al 1994; 

Vichinsky et al. 2000). This association usually follows sternal or tluncal pain1 which leads to splinting and poor 

inspiratory effort, and lack of active and complete inspiration following opioid-indnced sedation. The frequency 

and severity of pain in SCA is more than just episodic and acute, however; pain in SCA is often chronic, 

underrecognized and underreported, and therefore undertreated (Solomon 2008). Pain diaries of232 adult patients 

showed that SCA pain is common and often chronic; pain was present on 54.5% of days and 29.3% of patients 

reported pain on >95% of days (Smith eta! 2008). 

The pathophysiology ofvaso-occlusive pain is multifactorial and complex, and includes various blood cells 

including reticulocytes and neutrophils, plus plasma factors and vascular endothelium~). Several factors 

have been identified as triggers of painful VOE, with individual patients often recognizing their own specific 

triggers. The most commonly described triggers include cold temperatures and especially cold water, as well as 

dehydration, overexertion, and menses~~; Rcsar and Oski 1991; Yoong and Tuck 2002). 

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8/812016 CtF.rer.t Management of Sickle Cell Ar'lemia 

A combination approach of nonpharmacologic and phammcologic agents should be used for acute management of 

vase-occlusive palnful events. Nonphatmacologic interventions with shown effectiveness include oral hydration} 

heat. massage, and various cognitive-behavioral and self~reiaxation techniques (R_~s et at 2003; Damoier et al 

2004). Cold packs can increase local sickling and may exacerbate pain, so should be avoided. Phannacologic 

interventions should begin at borne with nonopioid analgesics, including acetaminophen and nonsteroidal antiinflammatory dmgs (NSAlDs); ibuprofen (10 mglkg or 800 mg for adults >40 kg every 6--8 h) is an effective oral 

agent given its potent analgesic and anti-inflammatory properties, Corticosteroids may reduce the duration of 

painful VOE but on discontinuation, are associated witl1 an increased frequency of rebound painful episodes 

requiring readmission (Qrlftin§tfih 1994), and so are relatively contraindicated for routine pain management If 

pain is not controlled with increased hydration, oral analgesia, and other conservative measures~ opioids should be 

used. Oral narcotic therapy such as codeine and its derivatives can often be used effectively at home, in selected 

settings and patients. 

In the event of severe vaso~occlusive pain requiring fotmal medical evaluation, aggressive pain management should 

be implemented promptly with intravenous hydration and opioid (morphine or hydromorphonc) analgesia, and 

adjuvant intravenous NSAID therapy. Historically, tl1e most paillfnl VOEs have been evaluated and treated in the 

local emergency room, but given the relative lack of sickle cell knowledge and familiarity among some heaH.hcare 

providers, such evaluations have delays in initiating appropriate analgesia, poor pain control, and a high rate of 

hospital admission. A sickle cell day hospital approach, which features staffing by experienced sickle cell providers, 

is increasingly used and results in improved pain management. belter patient satisfaction, and decreased rates of 

hospitalization for both adults and children with SCA (Benjamin eta!, 2000: ~eta!. 2008). 

During treatment ofVOE, frequent evaluation of pain is important to assess the degree of relief and potential sirie 

effects of narcotic analgesia. When hospitalization is required, continuous opioid infusion by patient-controlled 

analgesia (PCA) is recommended (van Beers et al. 2007; Jacob et al 2008). Figure 3 illustrates a convenient 

algorithm to consider for management of mild to severe painful VO E, but flexibility should exist for individual 

patient preferences. When opioids are used, an aggressive bowe1 regimen should be used concun-ently to reduce 

gasu·ointestinal complications, especially hypomotility (Q'Btien eta!. 201 Q). Teaching and encouraging frequent 

incentive spirometry with ambulatinn can reduce the risk of developing complications including ACS Clkllllitl!1 

1.222,; Ahmad et al. 2011), 

~ 

Algoritll..m for the management of painful vasa-occlusive events. 

Acute Splenic Sequestration Crisis 

ASSC remalns an important cause of morbidity and mortality for young cbildrcn with SCA. Most ASSC events 

occur in infants or toddlers before age 2 yr. In some cascst ASSC may be the first clinical manifestation ofSCA, 

and hence should be emphasized in the education of families during the first year of life. 

The patl10physiology of ASSC involves erythrocyte sickling and rapid accumulation within the spleen. ASSC is 

clinically defmed as a decrease in baseline hemoglobin concentration by ?::2 g/dL, in the presence of active 

reticulocytosis and splenomegaly; mild thrombocytopenia is common. The acute sequestration event can result in 

severe anemia, occasionally with hypovolemia_ and even can evolve to circuJat01y shock or death (fopiey et al. 

12ill.; Emond eta!. 1985; Powell ct al. 1992). 

Medical management of ASSC begins with ear1y recognition and diagnosis; parents and caregivers must be 

educated about early signs and symptoms and the need to seek urgent medical evaluation. After initial assessment 

including vital signs and physical examination, laboratory studies should include complete blood count with 

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reticulocytes, blood culture if febrile, and type and cross match. Transfusion volumes should not be excessive 

because a transfusion "overshoot" phenomenon can occur when the spleen abruptly unloads trapped erythrocyteS, 

raising the hemoglobin level above the target goal. Small aliquots (from the same unit) should be administered 

every 12-24 h to treat anemia and hypovolemia, while avoiding hyperviscosity following splenic release. 

Recurrent ASSC events are common, occurring in about half of the childten who survive the first episode. Chronic 

transfusions can be implemented after the ftrst episode, but their benefits on reducing recurrent events or avoiding 

splenectomy are limited (Kinney et al. 1990). The benefits of splenectomy for ASSC must be compared to its 

infectious and other postoperative risks; surgery is usually recommended only after one severe or life-threatening 

ASSC event, or after several recurrent ASSC events. Pneumococcal innnunizations should be completed before 

surgery, and then lifelong penicillin prophylaxis is recommended (Ammann ct a! 1977; Peodhar eta! 1993). 

Partial or subtotal splenectomy could potentially preserve some flltrarive and immunological splenic function, but 

published reports in SCA are sparse and anecdotal experiences have been unsuccessful (Rice et al. 2003). 

Acute Chest Syndrome 

ACS is a common cause of morbidity and a leading cause of death among adults with SCA (Castro eta! 1994; Gill 

~; Yichinsky eta! 2000; Powars et nl 2005). First described over 30 years ago (Charache et al 1979), 

ACS has a complex pathophysiology that remains poorly defined. Numerous etiologies have been proposed 

including typical and atypical bacterial pathogens (Miller ct a! 1991; Yichjnsky et al 2000; Neumayr et al '003), 

viral infection (Lowentl1al ct al 1996), fut embolism (Vichinsky et al 1994), intrapulmonary sequestration of 

erythrocytes (Vichinsky eta!. 1994), and nitric oxide-hemoglobin interactions (Gladwin eta!. 1999). 

ACS is defmed as a constellation of signs and symptoms including respiratory distress with tachypnea and dyspnea, 

hypoxemia, fever, elevated WBC coun~ mild anemia, and new infiltrate on chest X-ray (Castro eta! 1994; 

Vichjnsky et al 2000; Bullas et al 2010). ACS is often characterized by rapid clinical decline, so a high index of 

suspicion is needed for early identification and intervention. The onset of A CS may be insidious, often including 

pain, with nearly 50"/o of patients admitted with a different diagnosis. Sternal and n'b pain often leads to splinting 

and poor inspiration, which coupled with mild respiratory depression from opioids, can quickly deteriorate into a 

serious condition requiring aggressive respiratory and hematological supporl To avoid this sequence of events. 

incentive spirometry can reduce the incidence of ACS and should be mandatory for all hospitalized patients with 

SCA (Jk]k!_rul 1995; Ahmad etal. 2011). 

Worsening hypoxemia or tachypnea, and early radiographic changes should lead to aggressive incentive spirometry, 

and oxygen therapy to correct hypoxemia. Close management of fluid status is warranted to prevent fluid overload. 

Although hyperhydration is typically recommended for painful VOE to improve blood flow, intravenous fluids 

should be limited to 50%-75% maintenance in the setting of evolving ACS, to reduce the risks of developing 

pulmonary edema or pleural effusions and worsening respiratory distress. Despite the lack of randomized clinical 

trials investigating the efficacy of antibiotics for ACS (Matti-Cmyajal et al '007), coverage is provided for typical 

community-acquired and atypical pathogens, such as a broad-spectrum third-generation cephalosporin and 

macrolide (LottenhefJ! and Hasse!l20Q1). Bronchodilators are not eflective for all patients but are recommended for 

patients with ACS and concurrent reactive airways disease or asthma (Knight-Madden and Hambleton 2003; 

Knight-Madden eta!. 2005). 

Transfusions are often used for ACS ILanzkowsky et al 1978; Yichinsky eta! 2000), improving botl> anemia and 

oxygen-carrying capacity. Early simple transfusions are beneficial and can avert clinical deterioration that might 

warrant later exchange transfu.'tion. Significant respiratory distress or clinical decompensation, hemoglobin ?:2 g/dL 

below baseline, and oxygen saturation >5% below baseline are all indications for packed red blood cell (PRBC) 

transfusion~). Automated erythrocytapheresis should be used for severe ACS associated with 

significant respiratory distress or hypoxia (Kleinman et al. 1984; Velasquez et al. 2009). There is no current 

evidence that inhaled nitric oxide (NO) has a beneficial role in the current management of ACS (Gladwin et al. 

1999; AI Hajcri ct a!. 2008), but several clinical trials are ongoing. For recurrent ACS, both chronic transfusion 

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91812016 Current Management of Sickle Cell Anemia 

programs (Miller N al 200 l a; Hankins et al 2005a) and hydroxyurea (Steinberg ct a! 2003.; Hankjns eta! 2005h; 

Wan" eta! 201 I) can reduce the frequency and severity of additional ACS events. 

Stroke 

Cerebrovascular accidents are a relatively common and devastating complication ofSCA, with an overt stroke 

incidence rate of I l% by age 20 years and 24% by age 45 (~pqng et al. l99R). Clinical stroke events 

represent only a fraction of the cerebrovascular complications of SCA, which include silent cerebral infarctions 

(Miller eta! 2001 b: Pegelow eta! 200 I; DeBaun et al. 20 !2) and other ncurocognitive deficits (Schatz eta! 2001; 

Thompson eta! 2000). 

In the setting of acute clinical stroke, quickly reestablishing cerebral blood flow is crucial; new-onset weakness or 

aphasia suggests stroke and intervention should never depend on confmnatory radiological imaging. Modest IV 

hydration can help acutely, and should be provided while blood is being crossmatched for transfusion. Particularly 

for children with severe anemia, a simple PRBC transfusion ean rapidly reduce intravascular sludging and help 

improve cerebral blood flow, which is critical to help reverse acute symptoms and prevent stroke progression. 

When available, exchange transfusion should be performed promptly to reduce HbS <30%, with a larget 

hemoglobin concentration of -10 g/dL (Swerdlow 2006). A retrospective analysis of children with overt stroke 

suggested children receiving exchange transfusion had a significantly lower risk of recurrent stroke, compared to 

children receiving only simple transfusion (Hulbert eta! 2006). 

After an initial stroke, the risk of recurrent stroke events is 47o/o-93% without specific treatment (Powars et a1 

.till; Ba!karan eta! 1992; Pegelow ct al 1995). Chronic transfusions provided every 3-4 wk to maintain HbS of 

.-v30% are recommended to prevent recurrent events (Lusher et al 1976; Pege!ow et al 1995; Strater et al 2002: 

~-Once initiated, transfusions should be continued indefmitely, because discontinuation of transfusions is 

associated with increased risk of recurrent events (Wang ej a! 1991 ; Adams and Bramilla 2005). Although 

~-Recent 

efficacious, !Oo/o-20% of patients will develop a second stroke despite transfusions (Pe~low eta! 1995; ~ 

evidence further shows progression of vasculopathy and silent cerebral infarctions among 

chronically transfused patients (Hulbert eta! 20 I I). Hydroxyurea for the prevention of recurrent stroke, coupled 

wid> phlebotomy to remove iron overload, has shown efficacy (Ware et al, 1999, 2004), but in a phase ITI 

randomized clinical trial was inferior to transfusions and chelation therapy (Ware and Helms 2012). In certain 

clinical settings in which chronic transfusions are unsafe or otherwise not feasible, however, hydroxyurea may be a 

viable treatment option (Ali ct al. 20 II). 

Go to: 

Prevention of complications is the ideal way to reduce the morbidity of acute VOE and potentially limit chronic 

organ damage; this is especially true when considering devastating neurologic events like stroke. Because blood 

flow velocity is inversely related to vessel diameter, the measurement of intracerebral arterial blood flow by 

transcranial Doppler ultrasonography (TCD) allows easy and noninvasive identification of large vessel stenosis and 

can accurately identifY children at increased risk of developing primary stroke (Adams et al, I 9n_). Children with 

SCA have increased mean flow velocities when compared to children witl>out anemia (Adams eta! 1989, .lm), 

and markedly elevated TCD values represent a biomarker of cerebrovascular disease and a significant risk factor 

for primary stroke. 

Based on its utility of screening for stroke risk, both with efficacy in clinical trials (Adams eta! 1998) and 

effectiveness in clinical practice (McCarville eta! ?008; Enninfui-Egban eta! 201 0; Bemaudjn eta!, 2011; 

Kwiatkowski eta! 20 II), TCD screening is recommended annually for children with SCA stlrting at the age of2-

3 years,~ illustrates the recommended screening regimen and algorithm for management ofTCD results 

(Platt 2006; McCarville eta! 2008). The risk of first stroke can be nearly eliminated by the initiation of a chronic 

transfusion program to decrease HbS <30% for time-averaged maximum blood flow velocities >200 em/sec in the 

internal carotid or middle cerebral artery (Adams eta! 1998; Lee et al. 2006). Despite its clear efficacy for 

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91812016 Curren! Management of Sickle: Cell Anemla 

prevention of primary stroke, the risks of chronic transfusion therapy are significant and include iron overload, 

alloimmunization, and cost (Rosse et al. I 990; Harmntz ct a! 2000; Wayne eta!. 2000). Hydroxyurea therapy at 

maximum tolerated dose (MTD) also can reduce TCD velocities IZjmmcnnan et al. 2007) and the ongoing 

TWiTCH trial (Clinica!Trials.gov NCTOO! 22980) compares the efficacy of hydroxyurea versus transfusions for 

prevention of primary stroke in children with abnormal TCD velocities. 

~ 

Trnnscranial Doppler screening algorithm for children with SCA. 

ADULT CARE Go to: 

Improved care of the child with SCA has led to increased survival rates into adulthood, and now attention must be 

focused on appropriate management of this older population. The success of neonatal and pediatric care leads to the 

tbrmidablc task oftransitioning patients to the adult healthcare arena, in which providers often are neither trained 

adequately nor prepared to manage their unique needs. Written 'guidelines exist for managing adults with SCA 

(Lottenberg and Hassell 200';) but most of these recommendations do not derive from evidence-based research. 

The upcoming National Heart, Lung, and Blood Institute (NHLBI) guidelines document for SCA care, anticipated 

for release and distribution in 20 J 3, will provide current evidence-based guidelines. 

Adull'i with SCD should receive immunization boosters including pneumococcal, meningococcal, and even 

varicella vaccines based on age-specific recommendations. Management of acute VOEs such as pain, ACS, stroke, 

and priapism are similar for adults as for pediatric patients, but new issues may emerge related to chronic lung 

disease, especially restrictive pattern; leg ulcers that are difficult to manage and heal; and hepatic, endocrine, and 

even cardiac damage related to transfusional iron overload. 

Additional screening for specific organ damage is recommended for teens and adults. Baseline pulse oximetry 

readings will help establish a baseline value and identity chronic hypoxemia. Blood pressure and serum creatinine 

are typically lower than age-related published values; hence, values at the upper limit of normal may indicate renal 

dysfunction. Dipstick urinalysis identifies gross proteinuria, but quantitative testing for microalbuminuria, using a 

24-h urine collection if warranted. detects subclinical renal disease. Screening for hepatitis B, C, and HN is 

warranted for patients who receive chronic blood transfusions; serum ferritin with review of transfusion history may 

identify patients who need iron chelation. Periodic screening for tricuspid regurgitation jet (TR jet) velocities can 

identity patients with pulmonary hypertension and possible early mortality (Gladwin eta! 2004). Ophthalmological 

screening for retinal disease is warranted every l-3 yr with specialist referral for patients with abnonnalities. 

Additional screening tests for the general adult population should also be provided, including strategies to prevent 

or identifY cancer, hyperlipidemia, bone density loss, and even diabetes. (In tl1e latter case, the lack of l:lbA makes 

screening with HbA 1 c innacurate, but serum fructosamine can be used.) 

Go to: 

Urgent erythrocyte transfusions are indicated for many acute complications of SCA including ACS, ASSC, 

transient aplastic crisis owing to parvovirus B 19 infection, and acute stroke. In these settings, transfused blood 

helps to alleviate anemia, improve circulating blood volume~ increase oxygen-carrying capacity, and provide 

erythrocytes that cannot sickle. If the posttransfusion target is high enough, transfhsions also help suppress 

endogenous sickle erythropoiesis. Elective transfusions are often given for preoperative management, to prevent 

perioperative sickle-related complications. Chronic transfusions given on a monthly basis are also highly efficacious 

for primary and secondary stroke prevention. In contrast, transfusions are not indicated for acute painful events or 

anemia per se (recognizing that almost all patients have a baseline steady-state partially compensated hemolytic 

anemia), and have little role in the management of standard VOB (Smith-Whitley and Thompson 2012). 

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In most acute settings, simple transfusions witl1 packed erythrocytes (PRBC) should be administered. PRBC are 

readily available across the United States, and are routinely tested for HIV as well as hepatitis B and C. As a 

general principle, simple transfusions should be given with a target of alleviating anemia or treating the underlying 

condition; whole unils (or halfunils for small pediatric patienls) should be administered whenever possible, instead 

of fiXed volumes (e.g., 10 mL/kg), to help limit foreign antigen exposure. The posttransfusion target hemoglobin 

concentration should not exceed 10-11 g/dL in the untreated patient because hyperviscosity can occur; in 

chronically transfused patienls with low %HbS, however, the postrransfusion target can be raised to help suppress 

endogenous erythrocyte production. It is also important for the Blood Bank to be aware that the patient has SCA, 

because extending red blood cell (RBC) phenotype matching for minor blood group antigens is recommended to 

help prevent alto immunization (Yazdanbakhsh et al 20 12). 

For patients with neurological indications for chronic transfusion therapy such as abnonnal TCD velocities or 

stroke, repeated simple transfusions are effective.in preventing primary and secondary stroke, respectively, but 

ultimately result in transfusional iron overload. For this reason, partial exchange transfusions or isovolemic 

erythrocytapberesis is recommended to minimize iron accumulation. In most patients, intravenous access for 

exchange transfusions is facilitated by the placement of an implantable device. Witl1 chronic transfusions, the goal is 

typically HbS :>30% as a pretransfusion value, which typically requires transfusion every 3-5 weeks depending on 

the type and volume of each transfusion, the patient's own erythropoietic drive, and the response to transfusion 

therapy. Chelation therapy for transfusional iron overload should be considered for all patienls on chronic 

transfusions, but also for teens and adults who have a large cumulative number of episodic or sporadic transfusions. 

HYDROXYUREA Go to: ·-·· - --------

Increased fetal hemoglobin (HbF) levels have been associated with a less severe phenotype ofSCA !Conley et al 

1.2QJ.; !2iwJ.2Ll.; Platt et al 1991, .l22!1) and Hbf induction has become a desired pharmacologic end point for 

SCA therapy (.Chamche I 990; Charnche eta! 1992). Hydroxyurea has been shown to potently increase HbF and 

is currently the most effective disease-modifying therapy for both adulls and children with SCA (Piau eta! 1984; 

Steinberg ct a! 0 003; Zimmcmmn ct a! 2004; Hankins eta! 2005b). The fu·st clinical experience with hydroxyurea 

for SCA was reported nearly 30 years ago in seminal proof-of-principles studies (Platt et al 1984). Subsequently, a 

multicenter phase II study documented laboratory efficacy (increased Hb, %HbF, and MCV; decreased WBC, 

ANC, ARC, and platelets) of hydroxyurea using a dose escalation schedule to MTD (Chamche eta! 1992). The 

Multi-Center Study of Hydroxyurea (MSH) double-blinded, placebo-controlled randomized clinical trial showed 

clinical efficacy of hydroxyurea for adulls with severe SCA, with significantly reduced time to frrst painful even~ 

plus fewer episodes of ACS, transfusions, and hospitalizations IChamche et al 199'). 

In children with SCA, similar laboratory and clinical efficacy have been shown in open-label trials (Kinney et al 

12.22.; Wang el a! 200!; Zimmennan eta! 2004; ~Jll...2.QQ2h; Thombufll et al 2009). In hydroxyurea 

study oflong-tenn effecls (HUSTLE), all pediatric patients with medication adherence had HbF responses, 

although responses were variable and possibly related to differences in drug absorption, pharmacokinetics, and 

pharmacogenetics (Ware ct a! 201 I). The resulls from the double-blinded, placebo-controlled multicenter 

randomized BABY HUG study show the safety and clinical efficacy of hydroxyurea for young infants with SCA, 

regardless of previous clinical severity (Wang et al 20 I l). The primary end point of BABY HUG was the ability 

of hydroxyurea to prevent chronic organ damage (kidney, spleen), and the short-term study resulls were equivocal. 

Anecdotal reporls suggest prevention and even reversal of chronic organ damage with hydroxyurea therapy 

(Zinunennan et al 2007; Hankins ct al 2008; IJJOmburjj et al 2009), so further investigation of the BABY HUG 

cohort is necessary. 

Long-renn follow up from MSH and the Greek Laikon Study of Hydroxyurea in Sickle Cell Syndromes 

documented reduced mortality for adult patients with SCA on hydroxyurea (Steinbefll eta! 20 10· Voskaridou et al. 

;mu)). There is now indisputable evidence that hydroxyurea has laboratory and clinical efficacy for all ages; a 

growing body of evidence also supporls the long-tenn safety of hydroxyurea and the ability of hydroxyurea to 

prevent chronic organ damage and reduce mortality (McGann et al. 20 l I). Whereas hydroxyurea previously has 

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been reserved for older patients with a severe clinical course, hydroxyurea use should be liberalized and offered to 

all adults with SCA. An increasing number of pediatric hematologists believe hydroxyurea should now be 

considered as a treatment option for all children with SCA, regardless of age or previous clinical course. 

Hydroxyurea should be initiated by an experienced clinician familiar with laboratory monitoring and appropriate 

dose escalation to MTD. Hydroxyurea treabnentshould commence at -20 mg!kg/d by mouth, once daily. 

Complete blood count (CBC) should be checked every 4 wk to monitor for myelosuppression, which is typically 

mild and dose dependent, and always reversible by holding the hydroxyurea dose temporarily (Heeney and Ware 

2QJLS). Dose adequacy and medication compliance can be assessed by reviewing changes in CBC parameters and 

reviewing the peripheral blood smear (fiW). To reach MTD, the daily dose should be escalated by -5 mg/kg 

every 8 wk until MTD is mild neutropenia (e.g., ANC of 1500-3000 x 106/L) or reticulocytopenia (ARC of 100-

150 x l 09/L) is reached on a stable dose. Drug toxicity is usually defined by cytopenias such as ANC <1.0 x 

109/L, hemoglobin <7.0 g/dL with low reticulocyte count, ARC <80 x !09/L, andplatelets<80 x !09/L C!i.= 

and Ware 2008). Given the unlikelihood of true hydroxyurea ··nonresponders," efforts must be made to encourage 

medication compliance to reach and maintain a stable and efficacious hydroxyurea MTD (Ware 20 I Ob). 

J:.imnti 

Blood smear changes with hydroxyurea therapy. A illustrates the untreated 

patient with anemia and numerous sickled fom1s; B is after initiation of 

hydroxyurea treatment with macrocytosis and more target cells; and Cis 

after reaching a stable hydroxyurea ••. 

OTHER TREATMENTS ----- ~"-·--- ---·----···- Go to: 

HbF induction can be achieved by a group of short-chain fatty acids that inhibit the enzyme histone deacetylase; 

such HDAC inhibitors, primarily butymte, can alter chromatin structure and induce HbF production by altering the 

transcription of they-globin gene (McCaffrey eta! 1997; ~; Bradnerct al 20 I 0). Clinical experience 

with HDAC inhibitors for SCA is limited but anecdotal reports suggest robust HbF induction in some patients with 

SCA (A.lweh ct a! !999; !lines ct a! 2008). 

Decitabine is a nucleoside analog that induces HbF induction via epigenetic modulation, specifically 

hypomethylation of they-globin gene promoter. Experience with decitabine for SCA is also relatively limited, but 

several reports suggest clinical and laboratory efficacy of subcutaneously administered decitabine in adults who 

were not responsive to hydroxyurea {Creusot eta! 1982; DeSimone et al 2002; Sauntharn.rajah et al 2003, 20.Qft). 

Prospective trials of decitabine are warranted to determine if it has efficacy for a broad spectrum of patients with 

SCA. 

Additional treabnents that target specific pathways of the pathophysiology of SCA are just entering into clinical 

trials. One new promising inhibitor of the Gardos channel was found to have favorable effects on hemolysis and 

RBC survival, yet did not have clinical efficacy in a phase llJ randomized clinical trial (Ataga et al 2011 ). 

CONCLUDING REMARKS Go to: - -~--·--------- ~------------------

Decades of observational studies and therapeutic trials have contributed to a greater understanding of the 

pathophysiology and management of SCA. Based on these results, relatively simple interventions can substantially 

improve the survival ofSCA, especially among children. Newborn screening, early preventive treatments, 

education about complications, and screening programs improve both the morbidity and mortality ofSCA. Going 

forward, attention must focus on the care and management of teens and adults with SCA, and address quality oflife 

as well as medical complications. More aggressive treabnent ofSCA is supported by current evidence, and 

therapeutic options with hydroxyurea should be considered early in life. 

Footnotes Go to: "------·--·~-·---

http:/lwww.ncbi.n!m.nih.gov/pmc/articles/PMC3721270/ 10/17 
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Editors: Davld Weatherall, Alnn N. Schechter, and Dav!d G. Nathan 

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·-----------------·----------:-----·--

Articles from Cold Spring Harbor Perspectives in Medicine are provided here courtesy cf Cold Spring Harbor 

Laboratory Press 

!lttp://www.ncbi.nlm.nih.gov/pmclarllclesiPMC3721270.1 17/17 
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Pediatric Congenital 

Heart Association 

Statement for the Record 

In support of 

H.R. 3952, 

"The Congenital Heart Futures Reauthorization Act of 2015" 

Submitted to the Subcommittee on Health, 

House Committee on Energy and Commerce 

September 8, 2016 

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On behalf of the estimated 2.4 million people living with congenital heart disease (CHD) 

in the United States, and the countless families who have lost loved ones to CHD, the 

Pediatric Congenital Heart Association is writing to offer our strong support for H.R. 

3952, The Congenital Heart Futures Reauthorization Act of 2015, or CHFRA. 

We are extremely grateful to Representative Bilirakis and Representative Schiff for their 

steadfast leadership as sponsors of this bipartisan effort to build upon existing programs 

which track the epidemiology, raise the awareness and promote lifelong research of the 

most common birth defect. Thank you, also, to the dozens of congressional cosponsors 

committed to this goal. We also want to express our appreciation to the leadership of 

the Energy and Commerce Committee and the Health Subcommittee for this 

opportunity to move this important bill toward enactment. 

The Burden of Congenital Heart Disease 

CHD, a structural abnormality of the heart present at birth, is the most common birth 

defect and the leading cause of birth defect-related infant mortality. Nearly 1 in 100 

babies are born with CHD and more than five percent of those with CHD will not live 

to see their first birthday. Even for those who receive successful intervention, it is not 

~· 

However, critical information about the epidemiology of CHD, the effectiveness of 

treatments, and lifelong outcomes is seriously lacking, at best, and non-existent in 

specific areas such as comorbidities that occur later in life. 

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Heart~soelalloo 
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Over the last several decades, advances in treatment have dramatically improved 

survival for children with the most complex CHD and increased the life expectancy of 

adults with CHD. More than 80% of children born with CHD are now expected to 

survive into adulthood. In the absence of US data, which the Congenital Heart Futures 

Reauthorization Act will address, extrapolation of Canadian data suggests that there are 

2.4 million people with congenital heart disease, more than half of which are adults. This 

prevalence is expected to increase 5% annually, resulting in a surge of CHD survivors 

who need life-long specialized cardiac care. Although the growing population of 

individuals with CHD is a testament to important innovations in CHD care, the reality is 

that complex CHD and its treatments may result in chronic complications that may 

require further surgery, intervention, or heart transplantation. In addition to structural 

heart issues, individuals with CHD are at risk for secondary conditions including kidney, 

liver, and neurodevelopmental problems. In short, the interventions children with CHD 

receive are not cures, underscoring the need for lifelong care by expert providers to 

avoid and treat health complications later in life. 

Another challenge faced by those living with CHD is the transition of care from pediatric 

to specialized adult heart care. In part, this is due to the lack of infrastructure, 

providers, and payers required to care for the ever increasing adult CHD population. 

Adding to this challenge are those patients who no longer seek care believing their 

childhood intervention has "cured" them, or that they are well enough to no longer need 

specialized cardiac care. Estimates suggest less than 25% of adults with CHD are 

email. advocacy@conqueringchd org • visit. www.conqueringchd org 

0 

POO!atrlcCoogentla! 

Heart Allso~!ellon 
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receiving appropriate subspecialty care. This population of patients who are lost to 

follow-up care present a critical public health issue, which will be addressed by this 

important legislation. 

Neurological complications related to CHD pose another challenge. Cardiac surgery 

and perioperative treatments in the infant put the developing brain at tremendous risk 

for injury and long-term neurodevelopmental complications. Survivors often suffer injury 

to the brain due to low oxygen levels resulting from their CHD as well as operative and 

perioperative interventions. These brain injuries result in decreased 

neurodevelopmental, psychosocial, and physical functioning, and can significantly 

negatively impact the individual's growth, performance, and quality of life. 

The Cost of Congenital Heart Disease 

People born with CHD require lifelong, costly, specialized cardiac care, and face an 

ongoing risk of permanent disability and premature death. As a result, healthcare 

utilization among the CHD population is disproportionately higher than the general 

population. It is estimated that compared to medical costs of care for their peers, the 

medical costs for individuals with CHD are 10 to 20 times as great. Inpatient care costs 

{not including costs of physician care) for patients< 21 years old with CHD alone totaled 

more than $5.6 billion in 2009, representing 15 percent of hospitalization costs for all 

patients in this age range. Around half of all dollars spent on pediatric CHD related 

inpatient stays is paid by Medicaid. 

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Hc;utAssociatioo 
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Hospital admissions for adults with CHD roughly doubled between 1998 and 2005. 

Nearly 20% of these admissions were for cardiac surgery or catheter-based 

intervention. Healthcare utilization and costs continue to rise, due to hospital 

admissions, surgery and intervention, and emergency room visits. For example, with 

improved longevity to childbearing age, the number of high risk annual births in women 

with CHD is increasing. Childbearing women with CHD are fourteen times as likely to 

experience cardiovascular complications during pregnancy and are eighteen times as 

likely to die from such complications as are women without CHD. They also have longer 

hospital stays and incur higher hospital charges. 

With disproportionate medical costs, it is critical for us to understand the life-course of 

those living with CHD, their health care utilization and potential cost reduction 

strategies. 

Consider the story of Piper, who is one in 100. Piper was born on Independence Day in 

2003 after a seemingly normal pregnancy. However, instead of rosy pink, Piper was 

born blue. Suddenly, the world was flipped upside down for her first-time parents, as 

they learned that there was something critically wrong with Piper's heart. She was 

rushed to the nearby children's hospital to receive life-saving treatment which included 

open heart surgery. Her recovery from 14 hours of surgery was challenging and Piper 

spent the next 6 weeks in the hospital. Imagine that she has since grown to become a 

vibrant adult having completed college and entering the work place. At her annual visit 

she expresses plans to become pregnant, soon. Her physician, after reviewing data, 

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He11rtAssociat1on 
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refers her to a high risk obstetric center, and modifies her medications for heart rhythm 

problems. She delivers a healthy baby boy a year later, without complications for herself 

or her child. But, this data her physician referenced doesn't exist. Instead this current 

teenager is left with questions like can I travel to attend college? Will I able to have 

children? How long am I expected to live? Her life remains extraordinary with ongoing, 

costly, specialized care and concerns about a future where long-term outcomes are 

widely unknown. 

A Critical Public Health Issue 

The Congenital Heart Futures Act of 2015 (CHFRA) offers a reasonable and meaningful 

solution. To improve care and reduce costs, it is essential that Congress enacts 

legislation supporting improved understanding of CHD across the lifespan. The CHFRA 

calls for improved public health research and surveillance of those living with CHD, as 

well as providing for education and awareness, that will help us better understand and 

improve long-term outcomes for Piper and the more than 40,000 babies born with CHD 

each year. 

Previous Congressional investment for CHD activities supported by the Centers for 

Disease Control and Prevention's (CDC's) National Center on Birth Defects and 

Developmental Disabilities (NCBDDD) has funded the development of innovative 

surveillance strategies among children, adolescents and adults with CHD. This has led 

to the standardization of research methods and an increased understanding of the 

public health burden of this condition. Since the enactment of the Congenital Heart 

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HeariAsS<K:Iat•on 
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Futures Act in 2010, Congress has appropriated nearly $15 million to NCBDDD for 

these activities. Continued federal investment is necessary to provide surveillance and 

public health research on individuals across the lifespan to better understand CHD at 

every age, improve outcomes and reduce costs. 

The Congenital Heart Futures Reauthorization Act of 2015 does just that. The 

legislation directs the Centers for Disease Control and Prevention (CDC) to plan, 

develop and implement a representative cohort study to help describe basic U.S. 

demographics of the disease, assess healthcare utilization, and lead to evidence-based 

practices and guidelines for CHD care. The bill also allows for the CDC to establish and 

implement an education, outreach and awareness campaign directed at CHD across 

the lifespan, to ensure that those who have a CHD, and their families, understand their 

life-long healthcare needs and maintain appropriate specialized care. 

The CHFRA also directs the National Institutes of Health (NIH) to assess its current 

research into CHD so we can have a better understanding of the state of bio-medical 

research as it relates to improving understanding of causes and drive innovative and 

effective treatments for CHD and related disease processes. 

Congenital heart disease is common and costly; a critical public health challenge. We 

are thankful for the many members of Congress and colleagues in the community 

who join us in support of support of H.R. 3952, the Congenital Heart Futures 

Reauthorization Act 2015. It is essential for Congress to pass this comprehensive 

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approach to CHD that will address a critical public health issue and lead to better 

care, outcomes and quality of life for the millions of individuals living with CHD. 

The Pediatric Congenital Heart Association's mission is to "Conquer Congenital Heart 

Disease." We are founded on the key purpose to be the resounding voice of the 

pediatric patient population and are accomplishing this through collaboration with 

patients, parents, providers, and partner organizations in order to improve quality and 

outcomes through CHD education, research and awareness. 

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Adult 

Congenital 

Heart• 

Association 

https://www.achaheart.org/ * (888) 921-ACHA 

Testimony of 

Brian Altman 

Adult Congenital Heart Association 

Before the 

Committee on Energy and Commerce; Subcommittee on Health 

on 

"Examining Legislation to Improve Public Health" 

September 8, 2016 

DISCLAIMER: The following testimony outlines my personal position and should not be 

attributed to my employer, the Substance Abuse and Mental Health Services Administration. 

This testimony was prepared on my personal time without using any of my employer's 

resources. 
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Chairman Pitts, Ranking Member Green and distinguished members of the 

Subcommittee. My name is Brian Altman and I am a 41 year old living with congenital heart 

disease (CHD). In addition to being an adult with CHD, I am a former member ofthe Board of 

Directors and Public Policy Chair of the Adult Congenital Heart Association (ACHA). I 

appreciate the opportunity to provide testimony on behalf of ACHA whose mission is to improve 

and extend the lives of the millions born with heart defects through education, advocacy and 

promoting research. 

My Story 

I was born in 1975 with transposition of the greater arties and a ventricular septal defect. 

To my parents, that was a diagnosis that brought tears to their eyes and at the time likely meant 

death for me at a very early age. In the short term, the diagnosis meant that I was a blue baby 

who needed to be rushed out of state for care. We lived in Tulsa, Oklahoma which at the time 

did not have highly skilled pediatric cardiologists. So, at two weeks old I was taken to Texas 

Children's Hospital where I received care until approximately 2003. During those years, I was 

very fortunate compared to many I know with CHD. 

First, I had loving parents with private health insurance. Second, I "only" had three 

surgeries, "only" one of which was an open heart surgery. Third, by all accounts my open heart 

surgery, performed by the world renowned Dr. Denton Cooley, was a success and I was "fixed." 

Finally, at 28 years old as my pediatric cardiologist ensured that I was not lost to care and 

handed me over to a new type of provider, an adult congenital heart specialist. For the last 13 

years, I have been fortunate to continue to receive specialty care at Children's National Medical 

Center and Boston Children's Hospital. 
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However, through ACHA and my doctors I know I was not "fixed" for life, my birth 

defect is a chronic illness and most individuals with my condition born in the 1970s are no 

longer alive. There are also many things which are unknown to me. First, because of a lack of 

research and surveillance about lifelong outcomes for adults with CHD until the Congenital 

Heart Futures Act passed in 20 I 0, no doctor, researcher or epidemiologist can tell me the 

average lifespan of a person with my birth defects. Similarly, no one knows what types of other 

conditions are most prevalent among adults living with a "mustard procedure," the type of open 

heart surgery I had. So, years later when I developed generalized anxiety disorder, atrial 

tachycardia, and a leak in my tricuspid valve, these conditions were not ones that were easily 

predicable, despite CHD being the most common birth defect in America. As a result, I have 

been on costly medications and had a second open heart surgery last year. 

For me, my CI-ID means I try to live each day to the fullest. I've studied hard, engaged in 

a career that advances public health policy and built a family I love with all my being. But it 

also means I have no idea if I will be alive to see my daughter graduate from high school or 

college, if I will get to walk her down the aisle when she gets married, or if I will get to hold 

hands with my husband in a nursing home. 

ACHA 

ACHA has been a life changing organization for me and so many other adults with CHD. 

From its founding, it has been dedicated to supporting individuals and families living with 

congenital heart disease and advancing the care and treatment available to our community. 

ACHA provides resources I did not even realize I needed such as a travel directory of Adult 

Congenital Heart specialty programs. I carry this with me wherever I go so if something 
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happens while away on work travel or vacation, I can be taken to specialists who know my 

conditions and the reason my EKG and heart look and sound the way they do, ACHA listens not 

just to patients or providers, but ensures that we are all working together to foster the best 

research, provide the best care and support each other through surgeries, rehabilitation and 

unfortunately too often death. ACHA thinks strategically about the next steps forward in care 

provision, patient support and advocacy. With this in mind, the ACHA provides webinars aimed 

at providers and patients and hosts a conference with tracks for both, but key sessions that bring 

the providers and patients together. ACHA formed the Heart to Heart Ambassadors program to 

help patients and families have a peer support network. And, ACHA is on the forefront of 

ensuring high quality care by recently launching an accreditation program. This program is 

crucial for advancing and standardizing the quality of care for ACHD. Every person with CHD 

needs access to specialized care regardless of where they live. Finally, ACHA has lead the way 

in advocacy for adults with CHD. 

Congenital Heart Futures Act 

Because of what I have lived through and the many amazing doctors, nurses, individuals 

living with CHD, those I have known and lost and their families, I dedicated many hours to 

ensuring passage of the Congenital Heart Futures Act. The law ensures that the federal 

government dedicates research, surveillance and awareness to CHD not just as a birth defect, but 

as a chronic disease that thankfully individuals live with into their forties, fifties and beyond. 

Even as I fought for passage six years ago, on every visit to offices such as yours I noted the bill 

and the vital programs it authorized were not for me. Rather, the law and hopefully 

reauthorization of the law will ensure that those younger than I will know and understand what 
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steps they and their health care providers can take to ensure they not just live as long as possible, 

but to thrive in their life. 

I am thankful for the efforts the Centers for Disease Control and Prevention and the 

National Institutes for Health have taken so far and know so much more can be learned and so 

many more individuals can be reached. I am appreciative of the funding that Congress has 

provided for the programs authorized by the law and know the funding is vital to keep the 

research going and the registries up to date. 

Conclusion 

I appreciate the key members of Congress who have supported the Congenital Heart 

Futures Act including Representative Bilirakis, Representative Schiff and in the Senate the 

CHD's tireless champion, Senator Richard Durbin. Thank you for holding this legislative 

hearing on the Congenital Heart Futures Reauthorization Act. On behalf of ACHA and its 

members, !look forward to passage of this important bill so that we will have the science, 

surveillance, outreach and education to help individuals with CHD live and thrive. 
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ASE 

June 1, 2016 

Representative Gus Bilirakis 

American Society of 

Echocardiography 

2112 Rayburn House Office Building 

Washington, D.C. 2051 

Representative Adam Schiff 

2112 Rayburn House Office Building 

Washington, D.C. 20515 

Dear Representatives Bilirakis and Schiff: 

On behalf of the American Society of Echocardiography (ASE), I am pleased to have the opportunity to 

offer our strong support for the Congenital Heart Futures Act Reauthorization Act of2015. ASE is an 

organization of nearly 17,000 professionals committed to excellence in cardiovascular ultrasound and its 

application to patient care. Cardiovascular ultrasound is critical to the initial diagnosis and ongoing 

treatment of Congenital Heart Disease (CHD). 

We very much appreciate your leadership in introducing this bipartisan legislation, which would affirm 

the importance of continued CHD research by directing the Centers for Disease Control (CDC) to conduct 

a cohort study examining the CHD epidemiology, authorize a campaign to raise public awareness, and 

promote biomedical research at the National Institutes of Health (NIH) on the diagnosis, treatment, 

prevention and long term outcomes of this serious and prevalent condition. 

While we have made substantial progress in the treatment of heart disease, we appreciate your recognition 

that much work still needs to be done in the area of CHD, which remains the leading cause of infant 

mortality. Nearly I in I 00 babies are born with CHD, and, while life expectancy of many infants born 

with heart defects has been extended, there is no cure. Moreover, those children who are accurately 

diagnosed and treated face life-long risks and potential complications, including heart failure, stroke, 

renal dysfunction, and neurocognitivc problems. The epidemiological studies, research focus, and public 

awareness campaign required by the Congenital Heart Futures Reauthorization Act are desperately 

needed. 

Again, the ASE very much appreciates your leadership on this issue, and looks forward to working with 

you to secure passage of this important legislation. 

Sincerely yours, 

Susan 

~~ 

E. Wiegers, MD, FACC, FASE 

President, American Society of Echocardiography 

Senior Associate Dean of Faculty Affairs 

Professor of Medicine 

Temple University School of Medicine 

3500 N Broad St. MERB llllll-J 

Philadelphia, P A 19140 

Office: 215-707-6920 

{00639836.DOCX! 1 ) 
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United States House of Representatives Committee on Energy and Commerce 

Subcommittee on Health 

Examining Legislation to Improve Public Health 

Statement of the Alzheimer's Association on the Palliative Care and Hospice Education and Training Act 

September 8, 2016 

Thank you for holding today's hearing on legislation to improve public health. We applaud your leadership on issues 

important to Americans with Alzheimer's disease and other dementias and their caregivers. The Alzheimers Association 

proudly supports the Palliative Care and Hospice Education and Training Act (H.R. 3119/S. 2748), which was introduced in 

the House of Representatives by Representative Eliot Engel (D-NY-16) and Representative Tom Reed (R-NY-23) in July 

2015. This legislation, which currently has 200 bipartisan cosponsors in the House of Representatives, will ensure an 

adequate, well-trained palliative care workforce through workforce training, education and awareness, and enhanced 

research. 

Alzheimer's disease is a progressive, neurodegenerative and fatal disease for which there is currently no treatment or cure. 

More than 5 million Americans are currently living with Alzheimer's disease and other dementias. In addition, 15.9 million 

friends and family of those wtth Alzheimers disease are also acting as uncompensated caregivers. In 2015, these 

individuals provided 18.1 billion hours of care, valued at more than $221 billion.' Alzheimer's disease was identified by the 

New England Journal of Medicine as the costliest disease in the United States.' As the baby boom generation continues to 

age, one in nine Amertcans above age 65 will develop Alzheimer's disease or another dementia. By 2050, the number of 

people in the United States with Alzheimer's disease may be as high as 16 million, representing an annual cost to the nation 

of nearly $1.1 trillion (in today's dollars).3 

Palliative and hospice care- with a focus on managing and easing symptoms, reducing pain and stress, and increasing 

comfort- can improve both the quality of care and quality of life for those with advanced dementia. Observational studies 

have shown that as many as half of nursing home residents with advanced dementia have documented pain in the last 

weeks of life- and about a third have difficulty breathing or swallowing, or suffer from agitation. Individuals with advanced 

dementia who are enrolled in hospice have a lower rate of dying in the hospital, a lower rate of hospitalization in the last 30 

1 Alzheimer's Association. (2016). 2016 Alzheimer's Disease Facts and Figures. 

'Michael D. Hurd, Ph.D., Pace Martorell, Ph.D., Adeline Delavande, Ph.D., Kathleen J. Mullen, Ph.D., and Kenneth M. Langa, 

M.D., Ph.D. Monetary Costs of Dementia in the United States. N Eng/ J Med 2013; 368:1326-1334April4, 2013001: 

1 0.10561NEJMsa1204629. 

3 Alzheimer's Association. (2016). 2016 Alzheimer's Disease Facts and Figures. 
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days of life, and better symptom management Additionally, a recent study shows that nursing home residents with 

dementia who receive palliative care at the end of life, compared with those who do not receive such care, are up to 15 

times less likely to die in a hospital, nearly 2.5 times less likely to have a hospitalization in the last 30 days of life, and are up 

to 4.6 times less likely to have an emergency room visit in the last week of life' Families of individuals with dementia who 

are enrolled in hospice also have a greater satisfaction with patient care. 

People with Alzheimer's and other dementias also rely heavily on hospice at the end of life. Of all people living with 

dementia, 18.6 percent are currently in hospice care- a higher percentage than other chronic conditions.s Among seniors in 

hospice care, one in every six has a primary hospice diagnosis of Alzheimer's or other dementia. Neany half of all people 

with dementia die in hospice care. The population of persons with dementia using hospice has grown dramatically in recent 

years6- a trend that is likely to continue as the number of Americans with Alzheimer's disease and other dementias grows.' 

Congress unanimously passed the bipartisan National Alzheimer's Project Act (P.L. 111-375) in 2010. The law instructs the 

Department of Health and Human Services (HHS) to develop a strategic plan to address the rapidly escalating Alzheimer's 

disease crisis. The annually updated National Plan to Address Alzheimer's Disease must be transmitted to Congress each 

year and is to include outcome-driven objectives, recommendations for priority actions and coordination of all federally 

funded programs in Alzheimer's disease research, care and services. The Alzheimer's Association recently convened an 

expert workgroup to develop milestones for the care and support goals under the National Plan to Address Alzheimer's 

Disease. Those milestones include actions that need to be taken by both the federal and state governments in order to 

create a high quality care and support system for individuals with the disease and their families. Specifically, the expert 

workgroup recommends action to outline comprehensive standards of dementia care for palliative and hospice care; to 

develop components of dementia-competent palliative care for persons with Alzheimer's disease and other dementias; to 

modify hospice criterion from end of life estimate to disability severity for persons with Alzheimer's disease and other 

dementias; and to define process components for clinicians to discuss end of life planning with individuals with Alzheimer's 

disease and key caregivers, which would include hospice care8 

The Palliative Care and Hospice Education and Training Act would ensure an adequate, well-trained palliative care 

workforce through workforce training, education and awareness, and enhanced research. Specifically, this legislation would 

establish palliative care and hospice workforce training programs for doctors, nurses, and other health professionals; create 

4 Susan C. Miller et al. "The Effect Of Palliative Care Consults On Acute Care Use For Residents With Dementia In Nursing 

Homes". (November 2015). 

s Unpublished tabulations based on data from the NationalS% Sample Medicare Fee-for-Service Beneficiaries for 2014. 

Prepared under contract by Avalere Health, January 2016. 

'National Hospice and Palliative Care Organization. (2014). NHCPO's Facts and Figures: Hospice Care in America. 7 Alzheimer's Association. (2016). 2016 Alzheimer's Disease Facts and Figures. 

s Soo Borson et al., "Report on Milestones for Care and Support Under the U.S. National Plan to Address Alzheimer's Disease,'' 

Alzheimer's & Dementia 12, no. 3 (March 2016). 
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a national education and awareness campaign to inform patients, families, and health professionals about the benefits of 

palliative care and available services and supports; and enhance research on improving the delivery of palliative care. In 

addition, the legislation is consistent with findings and recommendations made by the Institute of Medicine expert panel on 

advanced dementia, as well recommendations made by the Advisory Council on Alzheimer's Care, Research, and Services. 

Thank you for holding today's hearing and for your continued leadership on issues that are so important to individuals and 

families facing Alzheimer's disease. We look forward to working with you and your colleagues on efforts to improve care and 

support for individuals and families affected by this disease, including the Palliative Care and Hospice Education and 

Training Act 
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American Academy of Hospice and Palliative Medicine 

Testimony for the Record 

Before the House Energy and Commerce Health Subcommittee 

Hearing Entitled 

"Examining Legislation to Improve Public Health" 

Thursday, September 8, 2016 

Chairman Pitts, Ranking Member Green, and members of the Subcommittee, the American 

Academy of Hospice and Palliative Medicine (AAHPM) would like to thank the House Energy and 

Commerce Health Subcommittee for the opportunity to share our Academy's views on H.R. 3119, the 

Palliative Care and Hospice Education and Training Act (PCHETA). AAHPM particularly offers its 

gratitude to Congressman Engel for his strong leadership on this important legislation. 

AAHPM is the professional organization for physicians practicing Hospice and Palliative 

Medicine. AAHPM's nearly 5,000 members also include nurses and other health and spiritual care 

providers who are committed to improving the quality of life of seriously ill patients and their 

families/caregivers. For close to 30 years AAHPM has been dedicated to expanding access of patients 

and families to high-quality palliative care and advancing the discipline of Hospice and Palliative 

Medicine through professional education and training, development of a specialist workforce, support 

for clinical practice standards, research, and public policy. 

This written testimony discusses how Congress can help improve care for the expanding 

population of patients with serious illness or multiple chronic conditions. We encourage the 

Subcommittee to consider the needs of these patients and support PCHETA as part of its efforts to 

improve the nation's health. 

H.R. 3119 would expand opportunities for interdisciplinary education and training in palliative 

care, including through new education centers and career incentive awards for physicians, nurses, 

physician assistants, social workers and other health professionals. The bill would also implement an 
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2 

awareness campaign, to inform patients and health care providers about the benefits of palliative care and 

hospice and the services available to support individuals with serious or life-threatening illness, as well 

as direct funding toward palliative care research to strengthen clinical practice and healthcare delivery. 

AAHPM's leadership stands ready to further discuss how PCHETA can help advance the 

Subcommittee's goals for the healthcare system and to answer any questions the Subcommittee has 

going forward with regard to improving care for patients and families/caregivers through the provision 

of high-quality palliative care. 

BACKGROUND 

Defining the problem 

By 2050, the population aged 65 and over is projected by the U.S. Census Bureau to be 83.7 

million, almost double that in 2012. As the population ages, an increasing number of people will be living 

with serious, complex and chronic illness. According to the Medicare Payment Advisory Commission 

(MedPAC), in 2010 more than two-thirds of Medicare beneficiaries had multiple chronic conditions while 

14 percent had six or more. Treatment of chronic and serious illnesses, such as heart disease and cancer, 

now accounts for nearly 93 percent of Medicare spending. 

Many of the problems of our health care system-high costs, overutilization, lack of 

coordination, preventable transitions between health care institutions, and poor quality-become 

particularly evident during extended chronic and serious illness. But a growing body of medical research 

has documented the benefits of high-quality palliative and hospice care for patients and families, for 

hospitals and payers, and for the health care system as a whole. 

Palliative care is an essential part of the solution 

AAHPM believes that palliative care providers and organizations, including hospices, are integral 

to meeting the "triple aim" of better care for individuals, improved health of populations, and lower 
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growth in health care expenditures. Indeed, the National Priorities Partnership has highlighted palliative 

and end-of-life care as one of six national health priorities that have the potential to create lasting 

change across the U.S. healthcare system. 

3 

Palliative care is an interdisciplinary model of care aimed at preventing and treating the 

debilitating effects of serious and chronic illness, such as cancer, cardiac disease, respiratory disease, 

kidney failure, Alzheimer's, AIDS, ALS, and MS. It can be provided from the time of diagnosis and involves 

the relief of pain and other symptoms that cause discomfort, such as shortness of breath, unrelenting 

nausea, etc. Palliative care can be offered alongside life-prolonging and curative therapies for individuals 

living with serious, complex, and potentially terminal illness and includes hospice care. 

Palliative care focuses on matching treatments to achievable patient goals, in order to maximize 

quality of life from diagnosis to death. In practice, this involves detailed and skilled communication with 

patients and families to elicit goals and preferences, as well as expert assessment and management of 

physical, psychological and other sources of suffering across the multiple settings (hospital, post-acute 

care, ambulatory clinics, home) that patients traverse through the course of a serious illness. Evidence 

and experience show that seriously ill patients and those with multiple chronic conditions and functional 

impairment-many of whom are Medicare's highest need and highest cost beneficiaries-strain these 

systems significantly. 

Recent studies have demonstrated that high-quality palliative care and hospice care not only 

improve quality of life and patient and family satisfaction, but can also prolong survival. •-s Furthermore, 

palliative care achieves these outcomes at a lower cost than usual care, by helping patients to better 

understand and address their needs, choose the most effective interventions, and avoid 

unnecessary/unwanted hospitalizations and interventions. 

However, delivery of high-quality palliative care cannot take place without sufficient numbers of 

healthcare professionals with appropriate skills. By supporting H.R. 3119, AAHPM believes the 

Subcommittee can help build a healthcare workforce more closely aligned with the nation's evolving 
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healthcare needs. PCHETA will help close the large gap between the number of health care professionals 

with palliative care training and the number required to meet the needs of the expanding population of 

patients with serious illness or multiple chronic conditions. 

Workforce Challenges 

4 

The reality today is healthcare providers need better education about pain management 

and palliative care. Students graduating from medical and nursing school have very little, if any, 

training in the core precepts of pain and symptom management, communication skills, and care 

coordination for patients with serious or life-threatening illness. The 2014 Institute of Medicine 

(10M) report Dying in America: Improving Quality and Honoring Individual Preferences Near the End 

of Life noted that "major gains have been made in the knowledge base of palliative care."6 The report 

documented, however, that "these knowledge gains have not necessarily been matched by the transfer of 

knowledge to most clinicians caring for people with advanced serious illnesses." Moreover, the 10M noted 

that an "overall pattern of inattention to palliative and end-of-life care ... still appears to predominate in 

the pediatric world." This lack of healthcare provider knowledge results in too many patients with serious 

illness receiving painful or ineffective treatments that do nothing to prolong or enhance their lives. 

At the same time, despite the growing need for palliative care, the field has been unable to 

meet patient and health system demand because of a significant shortage of specialist providers. The 

current gap between those practicing in the field and the number of physicians required to meet current 

need is likely huge-possibly several thousand physicians. A 2010 article published in the Journal of Pain 

and Symptom Management provides the findings of an AAHPM task force established to assess whether 

a physician shortage existed and to develop an estimate of the optimal number of Hospice and Palliative 

Medicine physicians needed to meet current and future needs. 7 It was determined that an acute 

shortage of hospice and palliative medicine physicians exists, with the current capacity of fellowship 

programs insufficient to fill the gap. 
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As of August 2016, there were a total of 119 Hospice and Palliative Medicine training 

programs accredited by the Accreditation Council for Graduate Medical Education and 15 training 

programs accredited by the American Osteopathic Association. For the 2015-2016 academic year, 

these programs were training just 296 physicians in Hospice and Palliative Medicine. At this rate, 

today's training programs would produce fewer than 6,000 new Hospice and Palliative Medicine 

certified physicians over the next 20 years. 

Yet AAHPM estimated 6,000+ full time equivalents-or 8,000 to 10,000 physicians-would have 

been required to meet the needs in hospice and palliative care programs back in 2010 (most recent 

workforce estimates), with up to 18,000 physicians needed if all hospices and palliative care programs 

were then using exemplary staffing models. These scenarios did not take into account future expansion 

of need due to population growth and aging or expansion of palliative care services into community 

settings such as nursing homes, home care, and office practices, all of which have exacerbated the 

hospice and palliative medicine workforce shortage. 

5 

The current Hospice and Palliative Medicine physician shortage can be attributed in large part to 

faulty Medicare policy. Despite the fact that the majority of patients receiving palliative care and 

hospice services are Medicare beneficiaries, and that palliative care has been repeatedly shown to 

increase value in health care by improving quality while reducing costs compared to usual care, 

Medicare does not invest in the training of Hospice and Palliative Medicine physicians. Instead, Hospice 

and Palliative Medicine specialty training is entirely dependent on private-sector philanthropy or 

institutional support because the Balanced Budget Act of 1997 placed a limit on the number of 

Medicare-supported residency slots before Hospice and Palliative Medicine was formally recognized as a 

medical subspecialty by the American Board of Medical Specialties. Given the instability of such funding, 

this is not a sustainable or rational way to train our nation's Hospice and Palliative Medicine physicians. 

Nonetheless, noting that "hospice and palliative medicine specialists will never be sufficient 

in number to provide regular face-to-face treatment of every person with an advanced serious 
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illness," the IOM report recommends expanding training opportunities to ensure clinicians across 

disciplines and specialties who care for people with serious illness are competent in "basic palliative 

care," including communication skills, interprofessional collaboration, and symptom management. 

6 

Modeled after the existing geriatric education centers, PCHETA would establish Palliative Care 

and Hospice Education Centers to improve the training of interdisciplinary health professionals in 

palliative care, develop and disseminate curricula relating to palliative care; support the training and 

retraining of faculty; support continuing education; provide students with clinical training in appropriate 

sites of care; and provide traineeships for advanced practice nurses. 

H.R. 3119 would also provide for training of physicians who plan to teach palliative medicine, 

academic career awards for junior medical faculty who commit to spend a majority of their funded time 

teaching and developing skills in interdisciplinary education in palliative care, and career incentive awards 

for other eligible health professionals who agree to teach or practice in the field of palliative care. 

Finally, PCHETA would further provide supplemental training for faculty members in medical 

schools and other health professions schools (including pharmacy, nursing, social work, chaplaincy and 

other allied health disciplines in an accredited health professions school or program, such as a physician 

assistant education program) so healthcare providers who do not have formal training in palliative care 

can upgrade their knowledge and skills for the care of individuals with serious or life-threatening illness 

as well as enhance their interdisciplinary teaching skills. 

Expanding Research to Improve Health Care Delivery 

PCHETA also aims to strengthen clinical practice and improve health care delivery for patients 

living with serious or life-threatening illness, as well as their families, by directing funding toward 

palliative care research. Research funding for palliative care and pain and symptom management 

comprises less than 0.1 percent of the National Institutes of Health annual budget. From methods for 

improving communication and decision making to evidence-based treatments for relieving distressing 
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symptoms of serious illness such as fatigue, nausea, shortness of breath, pain, and confusion, PCHETA 

would direct an expansion and intensification of research in these important areas. 

Raising Awareness 

While building the workforce and research base for the field will address key barriers to 

accessing palliative care services, more must be done to ensure patients and providers are aware ofthe 

benefits of palliative care. According to the Institute of Medicine, there is a "need for better 

understanding of the role of palliative care among both the public and professionals across the 

continuum of care so that hospice and palliative care can achieve their full potential for patients and 

their families." PCHETA would direct the implementation of a national education and awareness 

campaign so that patients, families, and health professionals understand the essential role of palliative 

care in ensuring high-quality care for individuals facing serious or life threatening illness. 

**** 

AAHPM urges the Subcommittee to move swiftly to advance H.R. 3119 and improve the care of 

patients with serious or chronic conditions by expanding patient and family access to high-quality 

palliative care. AAHPM looks forward to working with the Subcommittee to improve public health and 

maximizing the contribution of hospice and palliative medicine physicians and the interdisciplinary 

palliative care team in that effort. Thank you again for taking the Academy's written comments into 

consideration. Please contact Jacqueline M. Kocinski, MPP, AAHPM Director of Health Policy and 

Government Relations, at jkocinski@aahpm.org if you have any questions. 

7 
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REFERENCES 

1 Temel JS, Greer JA, Muzikansky A, Gallaher ER, Ad maneS, Jackson VA, Dahlin CM, 

Blinderman CD, Jacobsen J, Pirl WF,Billings JA, Lynch TJ. Early palliative care for patients 

with metastatic non-small cell lung cancer. NEJM 363(8)733-742 (2010). 

2Connor SR, Pyenson B, Fitch K, Spence C, Iwasaki K. Com- paring hospice and nonhospice 

patient survival among patients who die within three-year window. J Pain Symptom 

Manage. 2007;33(3):238-236. 

3 Hanson LC, Usher B, Spragens l, BernardS. Clinical and economic impact of palliative 

care consultation. J Pain Symptom Manage. 2008;35:340-346. 

4Morrison RS, Penrod JD, Cassel JB, et al. Cost savings associated with US hospital palliative 

care consultation programs. Arch Intern Med. 2008;168(16):1783-1790. 

5Penrod JD, Deb P, Dellenbaugh C, et al. Hospital-based palliative care consultation: effects on 

hospital cost. J Palliat Med. 2010;13(8):973-979. 

6 Institute of Medicine. Dying in America: Improving Quality and Honoring Individual Preferences 

Near the End of Life. Washington, DC: The National Academies Press, 2014. 

7Lupu D. Estimate of current hospice and palliative medicine physician workforce shortage. 

J Pain Symptom Manage. 2010;40(6):899-9 

8 
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National Hospice and Palliative 

Organization 

Care ~ ~ 

Statement of Support for the Palliative Care and Hospice Education and Training Act (PCHETA) 

National Hospice and Palliative Care Organization and Hospice Action Network 

Before the House Energy and Commerce Committee 

September 8, 2016 

On behalf of the National Hospice and Palliative Care Organization {NHPCO), our 1,300 member 

organizations serving individuals in more than 3,800 locations nationwide, and the 1.6 million Americans 

served in hospice each year, we commend the Energy and Commerce Committee for its consideration of 

the Palliative Care and Hospice Education and Training Act (PCHETA}. This important legislation will go a 

long way toward training the next generation of physicians and nurses who will care for many of us as 

we cope with serious illness and approach the end of our lives. 

Hospice and palliative care are patient-centered models of care that take into account not only the 

patient's underlying disease state, but his or her emotional and spiritual needs, personal values and 

beliefs, care needs across settings, pain and symptoms, and family and caregiver needs. Hospice and 

palliative care professionals require a multi-dimensional set of skills, attitudes and competencies, 

including the ability to manage the myriad symptoms associated with advanced and comorbid illnesses; 

the ability to facilitate communication and problem solving between patients, their families and their 

care teams; and the ability to coordinate and provide team-based care across a range of settings. 

A 2010 study published in the Journal of Pain and Symptom Management found a significant shortage in 

the number of adequately trained hospice and palliative care physicians and recommended an 

additional6,000-18,000 physicians to meet existing hospice and palliative care needs. In the six years 

that have followed, that need has likely increased significantly. Further, palliative and end-of-life care 

must be better integrated into other specialties. A survey by the American Society of Clinical Oncology 

found that 65% of respondents felt that they had received inadequate education in controlling 

symptoms associated with cancer, and 81% felt they had inadequate mentoring in discussing a poor 

prognosis with their patients and families. 

The Palliative Care and Hospice Education and Training Act will go a long way to correct these problems 

by supporting programs to provide clinical palliative medicine training in a variety of settings, including 

hospice, and developing specific measures to evaluate the competency of trainees. The Palliative 

Medicine and Hospice Academic Career Award program will enable hospice and palliative physicians to 

train members of interdisciplinary teams (lOTs) of healthcare professionals in palliative and hospice care 

techniques. This legislation will further expand the variety of professionals trained to provide hospice 

care, including nurses and clinical social workers. This program is especially important to the hospice 

community because oft he reliance on the !DT to effectively care for patients. 

As our health care delivery system prepares for the aging and decline of the 65 million baby boomers, it 

is imperative that we train physicians, nurses and other health care providers to better manage and 

deliver their care. We strongly support the Palliative Care and Hospice Education and Training Act, and 

encourage you to enact this important and much-needed legislation. 
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Oncology Nursing Society 

Testimony for the Record in Support of 

H.R. 3119, Palliative Care and Hospice Education and Training Act 

Before the House Energy and Commerce Health Subcommittee 

Hearing Titled 

"Examining Legislation to Improve Public Health" 

Thursday, September 8, 2016 

Chairman Pitts, Ranking Member Green, and members of the Subcommittee, the Oncology 

Nursing Society (ONS) would like to thank the House Energy and Commerce Health 

Subcommittee for the opportunity to share ONS' views on H.R. 3119, the "Palliative Care and 

Hospice Education and Training Act" (PCHETA). 

Our written testimony discusses the need to improve palliative care for patients with cancer 

or other serious or life-threatening illnesses. Oncology nurses have a pivotal role in offering 

palliative care for people living with a cancer diagnosis. Oncology nurses listen to what is 

important to patients as they assess how cancer and its treatment impact their physical, emotional, 

spiritual, and social well-being. Oncology nurses work with other health care providers to assist 

patients to manage symptoms, obtain community resources for care at home, and support patients 

in their final days with direct physical care at the end-of-life. 

All patients with cancer can benefit from palliative care. Congress can improve access to 

palliative care by advancing the bipartisan PCHETA legislation introduced in the House by 
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Representative Eliot Engel and Representative Tom Reed and in the Senate by Senator Tammy 

Baldwin. ONS greatly appreciates the many members of the Energy and Commerce Committee 

who have demonstrated their support for this legislation by cosponsoring the bill. 

PCHETA seeks to improve palliative care through education, awareness, and research. Not 

only do we need more trained interdisciplinary palliative care providers, but we need to expose 

more providers in general to palliative care training. The projected shortages of oncology 

providers are real and frightening. A shortage of oncologists will not only result in delays in 

treatment, but also delay conversations about goals of care. Increasingly, oncology nurses and 

primary care providers will need to be prepared to offer appropriate physical and behavioral 

symptom assessment and management and guide patients toward achieving their goals of care with 

effective communication skills and advanced care planning. 

ONS applauds the committee for leading the way to realize the Cancer Moonshot and 

Precision Medicine initiatives through the passage last year of H.R. 6, the 21" Century Cures Ac.t, 

and we hope Congress will soon complete its action on this legislation. However, health 

professionals must be ready to meet these needs. PCHETA will ensure we train more nurses, 

physicians, social workers, pharmacists and others in palliative care. Importantly, the bill follows 

the successful model of the geriatric education programs and ensures the training of teachers and 

that individuals arc drawn to the field and retained. 

Better awareness of palliative care by providers and the public also is necessary. PCHETA 

builds on existing authority at the Agency for Healthcare Research and Quality (AIIRQ) and 

2 
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provides for the establishment of a national campaign to inform patients, families, and health 

professionals about the benefits of palliative care and the services that are available to support 

patients with serious or lite-threatening illness. The awareness campaign would include the 

dissemination of information, resources, and materials about palliative care services in a variety 

offormats. In planning for the campaign, AHRQ is required to consult with relevant stakeholders. 

For most people, cancer will be a chronic illness that will require treatment and support 

through a journey that begins with a single step - usually a biopsy or an abnormal scan. 

Understanding that "wanting everything done" for a loved one will also need to include expert 

symptom management, emotional and spiritual support and assistance with identifying their "goals 

of care." To do this, there must be shared decision making in which there is a conversation with 

their providers about not only "what's wrong with you" but also "what matters to you"? 

Additional research is needed to determine better ways to relieve suffering throughout the 

cancer care experience and at the end oflife. This is especially true for symptoms such as shortness 

of breath, delirium, and pain control. Research demonstrates that palliative care works and its 

impact is measureable. It improves quality of life, results in less aggressive care at the end-of-life 

with fewer inpatient admissions and less ICU care, lowers emotional distress, and may in some 

cases actually prolong life. PCHETA, using existing authorities and funds, directs the National 

Institutes of Health (NIH) to expand national research programs to improve the delivery of 

palliative care to patients with serious illness. Oncology nurses are very active in palliative care 

research; the National Institute of Nursing Research funds much of the research in this area. 

3 
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ONS views the promotion and improvement of cancer symptom management and pain 

control as a national priority for improving the care of persons with serious illness and their 

families. As both the medical literature and oncology nurses can attest to, far too often patients 

with serious illness experience unnecessary emergency department visits, hospitalization and rehospitalizations, and other medical treatments because they lack the necessary integrated palliative 

care support needed in the setting of a serious illness. 

Palliative care is integral to oncology and indicative of the importance of alleviating 

physical, psychological, social and spiritual pain and suffering. Oncology nurses stand at the front 

lines of cancer care and urge Congress to recognize the importance of better treatment and quality 

of life options, particularly for patients with a diagnosis of cancer. A proactive and integrated 

approach to palliative care, which incorporates a team-based approach that includes multiple health 

care providers, will help improve patients' quality of life across the care continuum. 

The ONS has an important role in educating oncology nurses about palliative care and is 

actively educating its membership about emerging trends in palliative care. 

The ONS is a professional organization of more than 37,000 registered nurses and other 

health care providers dedicated to excellence in patient care, education, research, and 

administration in oncology nursing. The ONS members are a diverse group of professionals 

representing a variety of professional roles, practice settings and subspecialty practice areas. 

Oncology nurses arc leaders in health care committed to continuous learning and leading the 

transformation of cancer care by advocating for high-quality care for people with cancer. 

4 
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The ONS is committed to maximizing the contribution that nurses •• the largest group of 

health care professionals -- have in reducing chronic illness. More importantly, the ONS is 

committed to maximizing the contribution of oncology nurses in palliative care and looks forward 

to working with the Subcommittee to advance this important patient-centered legislation. 

5 
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September 1, 2016 

The Honorable Eliot Engel 

2161 Rayburn House Office Building 

U.S. House of Representatives 

Washington, DC 20515 

The Honorable Tom Reed 

1504 Longworth House Office Building 

U.S. House of Representatives 

Washington, DC 20515 

Dear Representative Engel and Representative Reed: 

The undersigned organizations write to express our support for H.R. 3119, the Palliative Care and Hospice 

Education and Training Act (PCHETA). This legislation will make a difference in the lives of millions of 

patients living with serious or life threatening illness and their caregivers. 

Despite a high intensity of medical treatment, many seriously ill individuals still experience troubling 

symptoms, unmet psychological and personal care needs, fragmented care, poor communication with their 

health care providers, and enormous strains on their family caregivers. However, numerous studies have 

shown that adding palliative care can improve pain and symptom control, quality of life, and patient and 

family satisfaction. 

Palliative care is an interdisciplinary model of care focused on relief of the pain, stress and other 

debilitating symptoms of serious illness, such as cancer, cardiac disease, respiratory disease, kidney 

failure, Alzheimer's, AIDS, ALS, and MS. Its goal is to relieve suffering and provide the best possible 

quality of life for patients and their families. Palliative care can be offered simultaneously with lifeprolonging and curative therapies for persons living with serious, complex, and eventually terminal illness 

and includes hospice care. By its very nature, palliative care is patient-centered care translating patient 

goals to appropriate treatments. 

We appreciate your leadership in recognizing the significant role palliative care and hospice can play in 

creating lasting change across the health care system. With PCHETA's focus on expanding the palliative 

care workforce, promoting awareness of the benefits of palliative care among patient and providers, and 

improving the evidence base for this care, you have demonstrated a strong commitment to addressing key 

barriers to palliative care access for the growing number of Americans with serious or life-threatening 

illness, 

Delivery of high-quality palliative care cannot take place without sufficient numbers of health care 

professionals with appropriate training and skills. Students graduating from medical school today have very 

little, if any, training in the core precepts of pain and symptom management, advance care planning, 

communication skills, and care coordination for patients with serious or life-threatening illness. Further, 

there is a large gap between the number of health care professionals with palliative care training and the 

number required to meet the needs of the expanding population of seriously ill patients. For example, 2010 

estimates by the American Academy of Hospice and Palliative Medicine's Workforce Task Force calls for 

6,000 or more full time equivalents to serve current needs in hospice and palliative care programs. At 

maximum capacity, however, the current system would produce only about 5,300 new hospice and 

palliative medicine certified physicians over the next 20 years. PCHETA would go a long way towards 

bridging this gap by establishing education centers and career incentive awards to improve the training of 

doctors, nurses, physician assistants, social workers and other health professionals in palliative care. 

PCHETA also aims to strengthen clinical practice and improve health care delivery for patients living with 

serious or life-threatening illness, as well as their families, by directing funding toward palliative care 
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PCHET A Support Letter 

September 1, 2016 

Poge2 

research. Research funding for palliative care and pain and symptom management comprises less than 

0.1 percent of the National Institutes of Health annual budget PCHETA would direct an expansion and 

intensification of research in these important areas. 

At the same time, more must be done to ensure patients and providers are aware of the benefits of 

palliative care. According to the Institute of Medicine, there is a "need for better understanding of the role of 

palliative care among both the public and professionals across the continuum of care." PCHETA would 

direct the implementation of a national education and awareness campaign so that patients, families, and 

health professionals understand the essential role of palliative care in ensuring high-quality care for 

individuals facing serious or life threatening illness. 

Thank you again for your support and leadership on this important issue. We look forwand to working with 

you toward the passage of this legislation. 

Sincerely, 

Academy of Integrative Pain Management 

Alzheimer's Association 

American Academy of Hospice and Palliative 

Medicine 

American Academy of Physician Assistants 

American Cancer Society Cancer Action Network 

American College of Surgeons Commission on 

Cancer 

American Geriatrics Society 

American Heart Association I American Stroke 

Association 

American Psychosocial Oncology Society 

American Society of Clinical Oncology 

Association of Oncology Social Work 

Association of Pediatric Hematology/Oncology Nurses 

Association of Professional Chaplains 

C-Change 

California State University Institute for Palliative Care 

Cambia Health Solutions 

Cancer Support Community 

Catholic Health Association of the United States 

Center to Advance Palliative Care 

Children's National Health System 

Coalition for Compassionate Care of California 

Colon Cancer Alliance 

Courageous Parents Network 

HealthCare Chaplaincy Network 

Hospice and Palliative Nurses Association 

Leukemia & Lymphoma Society 

Lung Cancer Alliance 

Motion Picture & Television Fund 

National Alliance for Caregiv·mg 

National Association of Social Workers 

National Coalition for Hospice and Palliative Care 

National Coalition for Cancer Survivorship 

National Hospice and Palliative Care Organization 

National Palliative Care Research Center 

Oncology Nursing Society 

Partnership for Palliative Care 

Pediatric Palliative Care Coalition 

Physician Assistants in Hospice and Palliative 

Medicine 

Social Work Hospice & Palliative Care Network 

Society of Palliative Care Pharmacists 

St. Baldrick's Foundation 

Susan G. Kamen 

Supportive Care Coalition 

Trinity Health 

Visiting Nurse Associations of America 
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American Cancer Society 

Cancer Action Network 

555 11th Street, NW 

Suite 300 

Washington, DC 20004 

202.661.5700 

www.acscan.org 

Statement for the Record on Behalf of the 

American Cancer Society Cancer Action Network 

United States House of Representatives 

Energy and Commerce Committee 

Subcommittee on Health 

Legislative Hearing: Examining Legislation to Improve Public Health 

September 8, 2016 

The American Cancer Society Cancer Action Network (ACS CAN) would like to thank 

Chairman Upton, Ranking Member Pallone, Chairman Pitts, Ranking Member Green, and 

Members of the Energy and Commerce Health Subcommittee for holding a hearing on 

Examining Legislation to Improve Public Health, that includes H.R. 3119, the Palliative Care 

Hospice Education and Training Act (PCHET A). We would also like to thank Congressman 

Eliot Engel and Tom Reed for their leadership and commitment as lead sponsors to move this 

important legislation forward. 

ACS CAN, the nonprofit, nonpartisan advocacy affiliate of the American Cancer Society, 

supports evidence-based policy and legislative solutions designed to eliminate cancer as a major 

health problem. As the nation's leading advocate for public policies that are helping to defeat 

cancer, ACS CAN ensures that cancer patients, survivors, and their families have a voice in 

public policy matters at all levels of government. 

During the ll4'h Congress, H.R. 3119 has garnered support from nearly 200 bi-partisan 

cosponsors in the House of Representatives, including over half of the full committee 

membership of the Energy and Commerce Committee. The legislation has also been endorsed 

by the Patient Quality of Life Coalition that was established in 2013 by ACS CAN, and has 

membership of over 40 patient, provider, and health system organizations. 

H.R. 3119 would address three important public policy issues that have been identified as 

necessary to provide patients with serious illness better access to palliative care services 

throughout the continuum of their care: patient and provider education on palliative care as a 

delivery model of care for patients with serious illness; workforce development and training for 

health care providers on core competencies of palliative care; and expanded federal investment 

in palliative care research. 
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The goal of palliative care is to prevent and relieve suffering, and to support the best possible 

quality of life for patients and their families. Research suggests that palliative care should be 

made available to patients with serious illnesses upon diagnosis. 1 

Palliative care focuses on relief of the pain, symptoms, and stress of serious illness and on 

improving communication with patients and families. It is appropriate at any age and at any 

stage in a serious illness. Palliative care is provided by a team of providers that typically 

includes a palliative care doctor, nurse, social worker and a chaplain who work with the patient's 

other doctors to provide an extra layer of support for the patient and their family. 

Evidence based research has concluded that patients with serious illness and their families 

receive poor-quality medical care that is characterized by inadequately treated symptoms, 

fragmented care, poor communication with health care providers, and enormous strains on 

family members or other caregivcrs.2

·

3 By focusing on priorities that matter most to patients and 

their families, palliative care has been shown to improve both quality of care and quality of life 

for patients during and after treatment of a serious illness. In one study, patients with metastatic 

non-small-celllung cancer who received palliative care services shortly after diagnosis enjoyed 

an improved quality oflife compared to those who did not receive palliative care. 4 The 

American Heart Association I American Stroke Association have also stated that palliative care 

can be a helpful complement to current care practices and can improve quality of life for stroke 

patients, caregivers, and providers.5 

Because their needs arc met, patients receiving palliative care avoid crises, spend fewer days in 

the hospital, emergency department and intensive care unit, and have fewer re-admissions.6 In 

fact, recent studies have demonstrated that high-quality palliative care not only improves quality 

of life and patient and family satisfaction, but it can also prolong survival.7·

8

·

9 Palliative care 

achieves these outcomes at a lower cost than usual by helping patients better understand their 

1 Smith, TJ, Temin S, Alesi ER, Abernathy AP, Balboni TA, Basch EM, Ferrell BR, Loscalzo M, Meier DE, Paice JA, 

Peppercorn JM, Somerfield M, Stovall E, Von Roenn JH. American Society of Clinical Oncology Provisional Clinical 

Opinion: The Integration of Palliative Care Into Standard Oncology Care. J Clinical Oneal 2012; 30: 880-887. 

2 Teno JM, Clarridge BR, Casey V, Welch LC, Wetle T, Shield R, Mor V. Family perspectives on end-of-life care at the 

last place of care. JAMA. 2004 Jan 7; 291(1):88-93. 

3 Meier DE. Increased Access to Palliative Care and Hospice Services: Opportunities to Improve Value in Health 

Care. The Milbank Quarterly. 2011;89(3):343-380. doi:10.1111/j.1468·0009.2011.00632.x. 4 Temel JS, Greer JA, Muzikansky A, et al. Early palliative care for patients with metastatic non-small-cell lung 

cancer. N Eng! J Med. 2010;363:733-742. 5 Palliative and End-of-Life Care in Stroke: A Statement for Healthcare Professionals From the American 

Heart Association/American Stroke Association 

http://stroke.ahajournals.org/content/early/2014/03/27/STR.0000000000000015 Mar 14. 

6 Meier DE. Increased Access to Palliative Care and Hospice Services: Opportunities to Improve Value in Health 

Care. The Milbank Quarterly. 2011;89(3):343-380. doi:10.1111/j.1468-0009.2011.00632.x. 7 Temel JS, Greer JA, Muzikansky A, et al. Early palliative care for patients with metastatic non-small-cell lung 

cancer. N Engl J Med. 2010;363:733-742. 8 Bakitas M, Lyons KD, Hegel MT, Balan S, Brokaw FC, Seville J, Hull JG, LiZ, Tosteson TO, Byock IR, Ahles TA 

Effects of a palliative care intervention on clinical outcomes in patients with advanced cancer: the Project ENABLE 

II randomized controlled trial. JAMA. 2009 Aug 19; 302(7):741-9. 

9 Connor SR, Pyenson B, Fitch K, Spence C, Iwasaki KJ Comparing hospice and non-hospice patient survival among 

patients who die within a three-year window. Pain Symptom Manage. 2007 Mar; 33(3):238-46. 

2 
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needs, choose the most effective treatments and avoid unnecessary or unwanted hospitalizations 

and interventions. 

In addition to important benefits to patient well-being and quality of life, palliative care has also 

been shown to reduce overall patient costs or to be cost neutral. One study of patients in Texas 

hospitals found that the provision of palliative care in the first 10 days after admission resulted in 

$9,689 savings per patient for those who died in the hospital and $2,696 savings per patient for 

those who were discharged alive. 10 A study of Medicaid patients in New York hospitals had 

similar findings, as the addition of palliative care resulted in $6,900 savings per patient $7,563 

per patient for those who died in the hospital and $4,098 per patient discharged alive. 11 The cost 

savings associated with palliative care in this study were estimated to save the New York 

Medicaid program an estimated $84-$252 million per year. 12 A study of patients in hospitals 

across multiple states also showed a $4,908 ($374/day) savings per patient for those who die in 

hospital and $1,696 ($279/day) savings per patient for those who were discharged alive.U 

ACS CAN strongly supports H.R. 3119. If enacted the legislation would improve the lives of 

patients with serious illness such as cancer, and provide patients greater access to palliative care 

services that have been proven to provide patients greater quality of life, and positive health 

outcomes. The legislation accomplishes this goal through three main public policy priorities: 

Patient and Provider Education 

Despite the proven benefits of patient access to palliative care, many patients with serious illness 

who could benefit are unaware of the existence of palliative care services, or incorrectly equate 

palliative care with end-of-life or hospice care. H.R. 3119 would direct the Agency for 

Healthcare Research and Quality (AHRQ) to establish a national campaign to inform patients 

with serious illness, their caregivers, and providers about the benefits of palliative care services 

throughout the continuum of their care. A broad based campaign is necessary to appropriately 

define palliative care as care for patients with any serious illness that is made available 

throughout the continuum of their care, encourage patients and families to seck high quality 

palliative care early in the course of illness, and educate healthcare professionals as to the 

appropriate role of palliative care in the care of their patients. 

Provider Education and Training Programs 

Provider education is a critical issue that must be addressed before greater patient access to 

palliative care services can be achieved. There is currently a shortage of palliative care trained 

doctors. Palliative medicine is a relatively new medical subspecialty, recognized by the 

American Board of Medical Specialties only 15 years ago. Also, due to the caps placed on 

Graduate Medical Education in the 1997 Balanced Budget Act, as a practical matter specialty 

training in palliative medicine is essentially entirely dependent on private sector philanthropy. 

10 McCarthy IM, Robinson C, Huq S, Philastre M, Fine RL, Cost savings from palliative care teams and guidance for a 

financially viable palliative care program, Health Serv Res. 2015 Feb;50(1):217-36, Epub 2014 JullS. 

11 Morrison RS, Dietrich J, Ladwig S, Quill T, Sacco J, Tangeman J, Meier DE., Palliative care consultation teams cut 

hospital costs for Medicaid beneficiaries, Health Aff (Millwood). 2011 Mar;30(3):454-63. 

"JQ,_ 

13 Morrison RS, Penrod JD, Cassel JB, Caust-EIIenbogen M, Litke A, Spragens l, Meier DE, Cost savings associated 

with US hospital palliative care consultation programs, Arch Intern Med. 2008 Sep 8; 168(16):1783-90. 

3 
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Thus, palliative care training is not Medicare funded, unlike all other medical training in the 

United States. There is a grave need to train doctors in the medical subspecialty, as well as train 

all health care providers in core competencies of palliative care including pain and symptom 

management, psychosocial assessment and communication. 

H.R. 3119 would establish an education and training program modeled after the successful 

Geriatric Education and Training Programs that currently exist. If enacted, education and 

training programs would support palliative care curriculum development in medical schools, as 

well as training programs in palliative care for all key healthcare professionals required to 

provide palliative care- doctors, nurses, social workers and other health care professionals. 

Research 

H.R. 31 19 would also expand the federal investment in palliative care research at the National 

Institutes of Health (NIH). Historically, research focused on palliative care and symptom 

management has not been a priority across institutes at the NIH. A recent study found that less 

than one-one hundredth of one percent of the NIH budget is focused on improving quality of life 

in the setting of serious illness. 14 H.R. 3119 would address this important issue by requiring the 

Director of the NIH to develop and implement a strategy across all institutes within the NIH to 

expand and intensify research on palliative care and symptom management. 

As the subcommittee examines ways that our current health care system can provide better 

quality, cost efficient care to patients with serious illness, we implore the subcommittee to 

closely examine the policy recommendations in H.R. 3119. On behalf of the millions of cancer 

patients, survivors and their families nationwide, thank you again to Chairman Upton, Ranking 

Member Pallone, Subcommittee Chairman Pitts, and Ranking Member Green for including 

H.R. 3119 in this important hearing on Examining Legislation to Improve Public Health. We 

look forward to continuing to work with the Energy and Commerce Committee to move this 

important legislation forward. 

14 Institute of Medicine. Dying in America: Improving Quality and Honoring Individual Preferences Near the Endof-Life. Washington D.C.: Institute of Medicine, 2015, available at 

http:/lnationalacademies.org/hmd/reports/2014/dying-in-america-improving-quality-and-honoring-individualpreferences-near-the-end-of-life.aspx. 

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