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91–848 2015

21ST CENTURY CURES: INCORPORATING THE 

PATIENT PERSPECTIVE 

HEARING 

BEFORE THE

SUBCOMMITTEE ON HEALTH 

OF THE

COMMITTEE ON ENERGY AND 

COMMERCE 

HOUSE OF REPRESENTATIVES 

ONE HUNDRED THIRTEENTH CONGRESS 

SECOND SESSION 

JULY 11, 2014 

Serial No. 113–158 

( 

Printed for the use of the Committee on Energy and Commerce 

energycommerce.house.gov 

(II) 

COMMITTEE ON ENERGY AND COMMERCE 

FRED UPTON, Michigan 

Chairman 

RALPH M. HALL, Texas 

JOE BARTON, Texas 

Chairman Emeritus 

ED WHITFIELD, Kentucky 

JOHN SHIMKUS, Illinois 

JOSEPH R. PITTS, Pennsylvania 

GREG WALDEN, Oregon 

LEE TERRY, Nebraska 

MIKE ROGERS, Michigan 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 

MARSHA BLACKBURN, Tennessee 

Vice Chairman 

PHIL GINGREY, Georgia 

STEVE SCALISE, Louisiana 

ROBERT E. LATTA, Ohio 

CATHY MCMORRIS RODGERS, Washington 

GREGG HARPER, Mississippi 

LEONARD LANCE, New Jersey 

BILL CASSIDY, Louisiana 

BRETT GUTHRIE, Kentucky 

PETE OLSON, Texas 

DAVID B. MCKINLEY, West Virginia 

CORY GARDNER, Colorado 

MIKE POMPEO, Kansas 

ADAM KINZINGER, Illinois 

H. MORGAN GRIFFITH, Virginia 

GUS M. BILIRAKIS, Florida 

BILL JOHNSON, Missouri 

BILLY LONG, Missouri 

RENEE L. ELLMERS, North Carolina 

HENRY A. WAXMAN, California 

Ranking Member 

JOHN D. DINGELL, Michigan 

Chairman Emeritus 

FRANK PALLONE, JR., New Jersey 

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 

JIM MATHESON, Utah 

G.K. BUTTERFIELD, North Carolina 

JOHN BARROW, Georgia 

DORIS O. MATSUI, California 

DONNA M. CHRISTENSEN, Virgin Islands 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

JERRY MCNERNEY, California 

BRUCE L. BRALEY, Iowa 

PETER WELCH, Vermont 

BEN RAY LUJAN, New Mexico 

PAUL TONKO, New York 

JOHN A. YARMUTH, Kentucky 

(III) 

SUBCOMMITTEE ON HEALTH

JOSEPH R. PITTS, Pennsylvania 

Chairman 

MICHAEL C. BURGESS, Texas 

Vice Chairman 

ED WHITFIELD, Kentucky 

JOHN SHIMKUS, Illinois 

MIKE ROGERS, Michigan 

TIM MURPHY, Pennsylvania 

MARSHA BLACKBURN, Tennessee 

PHIL GINGREY, Georgia 

CATHY MCMORRIS RODGERS, Washington 

LEONARD LANCE, New Jersey 

BILL CASSIDY, Louisiana 

BRETT GUTHRIE, Kentucky 

H. MORGAN GRIFFITH, Virginia 

GUS M. BILIRAKIS, Florida 

RENEE L. ELLMERS, North Carolina 

JOE BARTON, Texas 

FRED UPTON, Michigan (ex officio) 

FRANK PALLONE, JR., New Jersey 

Ranking Member 

JOHN D. DINGELL, Michigan 

ELIOT L. ENGEL, New York 

LOIS CAPPS, California 

JANICE D. SCHAKOWSKY, Illinois 

JIM MATHESON, Utah 

GENE GREEN, Texas 

G.K. BUTTERFIELD, North Carolina 

JOHN BARROW, Georgia 

DONNA M. CHRISTENSEN, Virgin Islands 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

HENRY A. WAXMAN, California (ex officio) 

(V) 

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. Henry A. Waxman, a Representative in Congress from the State of 

California, opening statement ............................................................................. 3 

Hon. Fred Upton, a Representative in Congress from the State of Michigan, 

prepared statement .............................................................................................. 5 

WITNESSES

Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research, 

U.S. Food and Drug Administration ................................................................... 6 

Prepared statement .......................................................................................... 8 

Answers to submitted questions ...................................................................... 123 

Pat Furlong, Founding President and CEO, Parent Project Muscular Dystrophy .................................................................................................................... 53 

Prepared statement .......................................................................................... 55 

Answers to submitted questions ...................................................................... 132 

Robert J. Beall, Ph.D., President and CEO, Cystic Fibrosis Foundation ........... 58 

Prepared statement .......................................................................................... 60 

Answers to submitted questions ...................................................................... 137 

Richard F. Pops, Chairman and CEO, Alkermes .................................................. 68 

Prepared statement .......................................................................................... 70 

J. Leonard Lichtenfeld, M.D., Deputy Chief Medical Officer, American Cancer 

Society ................................................................................................................... 77 

Prepared statement .......................................................................................... 79 

Marshall Summar, M.D., Director, Scientific Advisory Committee, National 

Organization for Rare Disorders ......................................................................... 85 

Prepared statement .......................................................................................... 88 

SUBMITTED MATERIAL

Statement of the National Health Council, submitted by Mr. Pitts ................... 114 

(1) 

21ST CENTURY CURES: INCORPORATING THE 

PATIENT PERSPECTIVE 

FRIDAY, JULY 11, 2014 

HOUSE OF REPRESENTATIVES, 

SUBCOMMITTEE ON HEALTH, 

COMMITTEE ON ENERGY AND COMMERCE, 

Washington, DC. 

The subcommittee met, pursuant to call, at 9:00 a.m., in room 

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

Present: Representatives Pitts, Burgess, Shimkus, Murphy, 

Blackburn, Gingrey, Lance, Cassidy, Guthrie, Griffith, Bilirakis, 

Ellmers, Upton (ex officio), Pallone, Engel, Capps, Schakowsky, 

Green, Barrow, Castor, Sarbanes, and Waxman (ex officio). 

Also present: Representative DeGette. 

Staff Present: Clay Alspach, Chief Counsel, Health; Gary Andres, 

Staff Director; Mike Bloomquist, General Counsel; Sean Bonyun, 

Communications Director; Leighton Brown, Press Assistant; Paul 

Edattel, Professional Staff Member, Health; Brad Grantz, Policy 

Coordinator, O&I; Sydne Harwick, Legislative Clerk; Robert Horne, 

Professional Staff Member, Health; Carly McWilliams, Professional 

Staff Member, Health; Macey Sevcik, Press Assistant; Heidi Stirrup, Health Policy Coordinator; John Stone, Counsel, Health; Jean 

Woodrow, Director, Information Technology; Ziky Ababiya, Minority Staff Assistant; Eric Flamm, Minority FDA Detailee; Karen 

Lightfoot, Minority Communications Director and Senior Policy Advisor; and Rachel Sher, Minority Senior Counsel. 

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

OF PENNSYLVANIA 

Mr. PITTS. The subcommittee will come to order. We are going 

to have early votes, so we are going to have to start. We understand the minority members are on their way. 

The chair will recognize himself for an opening statement. Today’s hearing provides us with an opportunity to examine perhaps 

one of the most important aspects of the 21st Century Cures Initiative. What does medical innovation or faster cures mean for patients? Keeping our work centered on the patient and understanding the patient perspective will bring much needed focus on 

results for patients who may lack adequate treatment options. Remember, there are only effective treatments for 500 of the 7,000 

known diseases impacting patients today. 

2 

While FDA has developed an enhanced structured approach to 

benefit risk assessment in regulatory decisionmaking for human 

drug device and biologic products, the committee recognizes the 

value of considering patients in decisionmaking about therapy development and access. Assessment of a drug or device’s benefits 

and risk includes an analysis of the severity of the condition treated and the current treatment options available, and getting the patient’s unique perspective should be a part of that assessment. 

One of our witnesses today, Pat Furlong of the Parent Project 

Muscular Dystrophy, PPMD—and I must say Pat is accompanied 

by Mary Bono Mack, a distinguished former member of this committee. Welcome, Mary. And Pat will explain how this organization 

was founded to create opportunities for families waiting for therapies to stop Duchenne muscular dystrophy from claiming young 

lives. To quote Pat Furlong, ‘‘Patient-focused drug development acknowledges the need to gather input from patients and their caregivers to create a more complete assessment of the benefit-risk 

equation, encouraging predictability, and increased flexibility within the review process. The clock is ticking for patients who need 

and deserve access to promising therapies.’’ 

I would like to applaud her tireless work drafting guidance 

PPMD recently released that actually quantifies patient priorities 

and preferences. This guidance will serve the Duchenne community 

and every other patient community because it provides a path for 

other patient groups to follow. This was an enormous undertaking, 

and I am confident it will make a substantial contribution to the 

entire medical community. 

I would like to welcome our witnesses today and look forward to 

learning more about the assessment of benefits and risks central 

to medical product development, regulations, and healthcare decisionmaking and the tradeoffs between desired benefits and tolerable risk. Thank you. 

Any member on the majority side seeking recognition? 

[The prepared statement of Mr. Pitts follows:] 

PREPARED STATEMENT OF HON. JOSEPH R. PITTS

The Subcommittee will come to order. 

The Chair will recognize himself for an opening statement. 

Today’s hearing provides us with an opportunity to examine perhaps one of the 

most important aspects of the 21st Century Cures Initiative: what does medical innovation or faster cures mean for patients. Keeping our work centered on the patient and understanding the patient perspective will bring much needed focus on results for patients who may lack adequate treatment options. Remember, there are 

only effective treatments for 500 of the 7,000 known diseases impacting patients 

today. 

While FDA has developed an enhanced structured approach to benefit-risk assessment in regulatory decision-making for human drug, device and biologic products, 

the Committee recognizes the value of considering patients in decision-making about 

therapy development and access. Assessment of a drug or device’s benefits and risks 

includes an analysis of the severity of the condition treated and the current treatment options available and getting the patient’s unique perspective should be a part 

of that assessment. 

One of our witnesses today, Pat Furlong of Parent Project Muscular Dystrophy— 

PPMD—will explain how this organization was founded to create opportunities for 

families waiting for therapies to stop Duchene muscular dystrophy from claiming 

young lives. To quote Pat Furlong, ‘‘Patient focused drug development acknowledges 

the need to gather input from patients and their caregivers to create a more complete assessment of the benefit-risk equation, encouraging predictability and inVerDate Nov 24 2008 11:05 Feb 05, 2015 Jkt 037690 PO 00000 Frm 00008 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-158 CHRIS
3 

creased flexibility within the review process. The clock is ticking for patients who 

need and deserve access to promising therapies.’’ I would like to applaud her tireless 

work drafting guidance PPMD recently released that actually quantifies patient priorities and preferences. This guidance will serve the Duchene community and every 

other patient community because it provides a path for other patient groups to follow. This was an enormous undertaking, and I am confident it will make a substantial contribution to the entire medical community. 

I want to welcome our witnesses today and look forward to learning more about 

the assessment of benefits and risks central to medical product development, regulations, and healthcare decision-making, and the tradeoffs between desired benefits 

and tolerable risks. 

Thank you, and I yield the remainder of my time to ——————————————————————. 

Mr. PITTS. The chair recognizes the vice chairman, Dr. Burgess 

for the remainder of time. 

Mr. BURGESS. And thank you, Mr. Chairman. 

And Dr. Woodcock, thank you for joining us again. It is always 

good to see you, always good to have you as a witness. You always 

provide valuable testimony. And our second panel representatives. 

I also want to acknowledge just as the chairman did, many of the 

patient organizations that you represent have worked well with our 

office and myself over the last several years. 

Mr. Chairman, the laudable goals of the 21st Century Cures Initiative, and they are indeed laudable, but we got to remember, at 

the end of the day, it is all about patients. Doctors want to heal. 

We want to cure. That is why we entered the profession. No doctor 

ever wants to tell a patient there is nothing more we can do. The 

good news is that the golden age of medicine is really right around 

the corner. The doctors of tomorrow will have tools at their disposal 

unlike any before in human history. The ability of tomorrow’s doctor to alleviate human suffering is going to be unparalleled and unmatched in history. Yet every day that goes by where these tools 

are not realized is a day that patients and their families have to 

struggle through the pain and suffering of their condition. 

Every day counts for these Americans and for their families. For 

those who struggle with rare diseases, their struggle is only compounded by the lack of biomedical research. For those patients, it 

is difficult to see over the horizon. We have much work to do on 

this committee, and we have done a lot in the past. We particularly 

celebrate the 2-year anniversary of the Food and Drug Reauthorization Act that was just a few days ago. That was a good template. 

It was a good method for moving forward, and I appreciate that the 

Cures initiative is following that template, but there is no doubt 

that we can do much more. 

I welcome the testimony of our witnesses, and I will yield back 

my time. 

Mr. PITTS. The chair thanks the gentleman. 

And I now recognize the ranking member of the full committee, 

Mr. Waxman, 5 minutes for opening statement. 

OPENING STATEMENT OF HON. HENRY A. WAXMAN, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF CALIFORNIA 

Mr. WAXMAN. Thank you very much, Mr. Chairman. 

This hearing is a fitting followup to Wednesday’s hearing on clinical trials. After all, it is patients who live with the diseases and 

4 

conditions for which treatments are being sought, and this hearing, 

which is called ‘‘21st Century Cures: Incorporating the Patient Perspective,’’ illustrates that we should take every opportunity to understand their experience. 

Congress has a long history of listening to concerns of patients. 

That is what I did in 1983 when I wrote the Orphan Drug Act. 

That law came up when I heard from a constituent, Adam Seligman, who had a rare disease called Tourette’s Syndrome. Adam 

was forced to take a drug that he could only get from Canada because, at that time, there were no effective treatments available in 

the United States. When his drugs were ceased at the border, his 

mother made a desperate call to my office begging me to do something. 

I set out to figure out why there were no drugs in the U.S. for 

Adam’s condition. We discovered that Adam was not alone. There 

are 134 drugs for rare diseases but only 10 had come to market 

solely as a result of industry. We knew we had a problem on our 

hands, and we set out to solve it. 

The Orphan Drug Act has been a resounding success. Today, 

there are over 400 drugs for rare diseases, and I want to welcome 

the National Organization for Rare Disorders here today and look 

forward to their testimony. 

I am telling this story about the Orphan Drug Act not only as 

an example of how Congress has listened to the concerns of patients and acted on them, I tell it because it is an example of appropriate use of legislation. In the case of rare diseases in the early 

1980s, there was very clear evidence of a market failure in need 

of congressional action. 

In the context of the 21st Century Cures Initiative, we need to 

assure that both FDA and the drug and device companies are listening to the concerns of the patients. FDA has a long history of 

engaging with patients, both in the context of advisory committees 

and in its review of drugs and devices. In the 2012 FDA Safety and 

Innovation Act, Congress pushed FDA to do even more to hear patients’ concerns, and I look forward to hearing more from FDA 

today. 

From what I can tell, the agency has taken that mandate seriously and is engaged extensively with the patient community. We 

should ask today whether FDA has adequate resources to continue 

to do this work. 

As I mentioned on Wednesday when we had our last hearing, 

when it comes to legislating in complicated scientific areas, like the 

conduct of clinical trials, we need to proceed with great caution. 

For example, one issue in the area of clinical trials that is likely 

to come up today is how to incorporate so-called patient reported 

outcomes. As I understand it, this is an area that is multifaceted 

and scientifically complex. Congress should ensure that FDA has 

the flexibility and authority to make use of these outcomes but not 

dictate how and when that occurs. 

I hope FDA will tell us about how it is applying other novel approaches to clinical trials in their regulation of drugs and devices. 

I would also like to know whether the agency believes it has the 

authorities necessary to adopt new approaches and whether other 

new statutory powers are necessary. 

5 

Mr. Chairman, thank you for holding this hearing. I look forward 

to the witnesses’ testimony. I must say in advance that there is another subcommittee scheduled at the very same time as this one, 

so I will try to be back and forth to participate in both of them. 

Thank you, and yield back my time. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize the chairman of the full committee, Mr. Upton, 

5 minutes for opening statement. 

Mr. UPTON. I yield back my time. I will just submit my statement in order to—— 

[The prepared statement of Mr. Upton follows:] 

PREPARED STATEMENT OF HON. FRED UPTON

The entire purpose of our 21st Century Cures initiative is to accelerate the discovery, development, and delivery of safe and effective treatments to America’s patients. We are here today to better understand how we can incorporate the most 

important perspective—that of patients and their families—into the conversation. 

Patients should and need to play a key part of this process if we are to be successful. As one of our witnesses, Dr. Beall has noted, ‘‘Congress should work to ensure 

patients have a seat at the table, because no one understands a disease better than 

the people who suffer and fight every day.’’ 

I would like to issue a special welcome to Pat Furlong who has continued to fight 

after losing her two boys, Christopher and Patrick, to Duchene. We are very humbled that you are here to help with the cures initiative. I’d also like to welcome to 

Dr. Marshall Summer—a parent of a child with Down’s Syndrome. Parents are tireless advocates of their children and we are pleased that you are here today. Thank 

you and all of our witnesses for being here today. 

I also would like to thank Dr. Woodcock for testifying today. Unfortunately, prior 

obligations prevented her from coming to Wednesday’s hearing so today she will provide her expertise on both modernizing clinical trials and incorporating the perspective of patients. 

As I’m sure Dr. Woodcock will explain, FDA has taken steps to incorporate the 

perspective of patients in the drug development process. FDA’s work with Parents 

Project Muscular Dystrophy is a good example of collaboration, and we look forward 

to hearing about next steps on the guidance they put together. 

However, much work remains. We would like to learn how we can leverage the 

successful examples of agency-patient collaboration and what other steps we can 

take to ensure the patient’s perspective on the benefit-risk framework is thoroughly 

considered and incorporated throughout the cycle of the drug review process. 

At our first 21st Century Cures roundtable, we learned that there are treatments 

for only 500 of the more than 7,000 known diseases affecting our nation’s patients. 

Our work will not be done until we can close this gap in cures. I look forward to 

hearing how we can incorporate the voice of patients in this process. 

I yield the balance of my time to ————————————————. 

Mr. PITTS. The chair thanks the gentleman. 

We have two panels today. 

On our first panel, we have Dr. Janet Woodcock, director, Center 

for Drug Evaluation and Research, U.S. Food and Drug Administration. 

Thank you for coming again today. And you will have 5 minutes 

to summarize your testimony. Written testimony will be placed in 

the record. 

So, at this time, the chair recognizes Dr. Woodcock 5 minutes for 

opening statement. 

6 

STATEMENT OF JANET WOODCOCK, M.D., DIRECTOR, CENTER 

FOR DRUG EVALUATION AND RESEARCH, U.S. FOOD AND 

DRUG ADMINISTRATION 

Dr. WOODCOCK. Thank you. We are here to discuss how drug development better meets the needs of patients. Decades ago, 

healthcare was very physician-centric and actually very paternalistic. We all recognize that today. It was kind of ‘‘The doctor knows 

best; don’t ask any questions.’’ 

Today, the model is collaboration between the patient and the 

healthcare team. These changes, though, have evolved slowly in 

our society, and the thinking and drug development has slowly 

changed in parallel. 

The FDA Safety and Innovation Act of 2 years ago took significant steps in this direction of patient-centric development. It contained agreements under PDUFA that FDA would sponsor at least 

20 patient-focused disease-focused meetings over 5 years. Eight of 

these meetings have been held to date, and they have been very 

impactful. The first one we held on chronic fatigue syndrome, we 

have issued a draft guidance on drug development in this area of 

very serious unmet medical need. Also, under PDUFA, were agreements to advance the development and use of patient-reported outcome measures. These are measures that the patient can fill out 

to say from their point of view how well they are feeling, how well 

the treatment is working, what adverse events they are experiencing. 

We are having an expert meeting next week and will continue to 

work in collaboration consortiums to try and advance the science 

of patient reported outcomes. This is very important to really scientifically incorporate the patient’s perspective into clinical trials. 

Additionally, under FDASIA, FDA was to advance the development and use of a structured benefit risk framework in drug approval decision, and this work is under way, and it really explicitly 

provides for considering the burden of disease, the impact of current therapies, both for good and for ill, and the tolerance of risk 

from the patient’s point of view, and this is an extremely important 

set of factors that need to go into the benefit-risk decision, but we 

need to do this in a scientific manner and a structured manner and 

we are rolling out the structured benefit risk framework. 

Now, we know that for people with very serious diseases who 

may lack good therapy or actually lack any therapy, access to new 

treatments is their number one priority, and that is why expediting 

drug development programs in these areas is so important. If you 

look at the diagram that we have here that was provided, these 

data and the diagram were actually developed by the Pharma organization, talks about, shows the drug development process, and it 

is starting on the left, it shows you start with many thousands of 

compounds, up to 10,000 compounds at one end, the beginning, and 

after 9 to 13 years, you may end up with one safe and effective 

drug on the market. 

The clinical development phase, which is the gray phase, the 

middle phase on this diagram, is the longest and by far the most 

expensive phase. 

In contrast, the FDA review phase, of which much attention has 

been paid to, is actually the very small slice there, the white slice 

7 

toward the end of the process, right before the drug gets on the 

market and is typically at this time less than a year in duration. 

So FDA has made strenuous efforts, really, to help reform and 

modernize the clinical development phase of drug development because that is the major bottleneck. Not only is it expensive and 

long, many products fail in this phase, and there is a tremendous 

opportunity cost there where other treatments could have been developed. 

Now, the FDASIA included several innovations to this process 

and the most striking being the breakthrough therapy designation 

program, so if we could have the next diagram. Thank you. This 

was mandated by Congress and was specifically directed at that 

clinical development phase, so that we could help when therapies 

were particularly promising and were designated, we could help 

move them through that phase more quickly. The BT designation 

has been enthusiastically subscribed. We have had over 160 requests in the 2 years since the legislation was passed. We have actually—and this is the really surprising part, we have granted 52 

designations. So what Dr. Burgess said about we are on the verge 

of a new era in therapeutics, I think, is reflected by this. We would 

not have seen this a decade ago, and we have approved six products: four new products and two new indications. 

Now, it is too early to judge really the impact of the breakthrough designation program; is it really going to be able to speed 

up drug development? However, I will say the four products we approved, their clinical development time was 4.5 years, so much 

shorter than what I showed in the earlier diagram. 

Also, in FDASIA were clarifications of the application accelerated 

approval, and we issued a final expedited draft guidance in May 

that includes, in response to stakeholders’ requests, examples of 

rare diseases and includes more information on the use in rare disease. However, it is clear much more needs to be done to modernize 

the clinical trial process. That is the big bottleneck now in getting 

discoveries to patients. This can’t be done, though, by FDA alone. 

We don’t execute this process. All the stakeholders need to participate, and I think the series of hearings that have been held and 

the 21st Century Initiative can help provide the framework for significant reform in this process. 

Mr. PITTS. The chair thanks the gentlelady. 

[The prepared statement of Dr. Woodcock follows:] 

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Mr. PITTS. I will begin the questioning, recognize myself 5 minutes for that purpose. 

Dr. Woodcock, what is FDA’s plan to advance biomarkers and the 

use of patient reported outcomes data during the drug development 

process and post-market setting? 

Dr. WOODCOCK. Many years ago, a decade ago, we recognized 

that there was no structured scientific process to provide the evidentiary basis for use of a biomarker in decisionmaking, and so 

doctors and biomedical researchers would float new biomarkers, 

but there was no rigorous process by which they could be evaluated 

to see if they were really useful. So we actually established a process for this. It is not really in our mission, but we established it, 

and it is called the Biomarker Qualification Process. And we also 

work with the European medicine agencies and the Japanese regulators so that this would be a worldwide activity. And consortia can 

come into the FDA and propose a biomarker, a new biomarker, and 

we give them advice on what needs to be done, and then—and also 

for patient-reported outcomes. And if, in fact, that evidence is developed, then we will publish a letter that is public, and so will the 

EMA if they accept it and so forth, and then any developer can use 

that biomarker or measure in a development program and will rely 

on it for the context of use. 

We have 79 projects by different consortia in different phases of 

this process right now. 

Mr. PITTS. Good. Describe your plans for implementation of the 

structured benefit-risk framework you mentioned that—transparent to the public and the sponsor so that the assessment of data 

from clinicals trials and other studies by FDA reviewers can be better understood and acted upon. 

Dr. WOODCOCK. Yes. Well, this is an iterative process. We have 

had public meetings. Then we went back, and we are piloting this 

in the different drug review divisions and having the medical officers work through this framework that we have developed and see 

what the results are. Then when we have those results, we will go 

back through a public discussion and get input on how this can be 

improved, so this is not something that can happen overnight. 

It is a scientific process, and actually, we feel that we don’t have 

the tools right now. They exist out in society in science, but we 

haven’t applied them, these rigorous analytical tools to the benefitrisk decision, and so we have had workshops on this, various scientists come in and advise us, so we will have a public process once 

we have gathered more experience. 

Mr. PITTS. I have been hearing a lot about FDA’s efforts to improve the quality of pharmaceutical manufacturing. Where do U.S. 

drug manufacturers currently stand when it comes to producing 

quality medicines? Can you tell me a little bit about your plans in 

this area? 

Dr. WOODCOCK. Well, I think the major problem here is that 

many of our essential drugs are not made in the United States, and 

they are made all around the world, and sometimes they may only 

come from a single source, and this is, I think, a real vulnerability 

to medicine. And in addition, we used to be a manufacturing powerhouse in drug manufacturing, but those jobs have moved offshore. And I think now we have an opportunity, with new modern 

30 

manufacturing methods, such as continuous manufacturing, to actually build a high-tech industry in the United States that actually 

will make the drugs we need here in this country. And FDA has 

been collaborating with this manufacturing community to help 

bring this about, and we are very interested in seeing this happen. 

Mr. PITTS. Now, we have recently heard a lot about Lung-MAP, 

the Lung Cancer Master Protocol trial. There are other examples 

of similar innovative trial designs, like I–SPY for breast cancer. 

What else needs to happen before these types of trials are no 

longer front-page stories? 

Dr. WOODCOCK. That is a good question. We also have been advocating for this for many years, and it is wonderful to see it start 

to become a reality. The concept, I think, in drug development 

needs to be turned on its head in clinical drug development, and 

instead of, for each investigational drug, there is a whole clinical 

trial program developed with different clinical trials that take a 

very long time, as you heard on Wednesday, to get started and so 

forth, that there are networks that are available that investigational drugs can be plugged into. This will provide independence of 

an assessment but also really decrease the time and expense of assessing whether these drugs are safe and effective. 

But what needs to happen, I think, is we need to expand this to 

more diseases. The NIH is very interested in antimicrobials in setting up a network, and other groups are looking into this, and I 

think you may hear today from some patient groups say Cystic Fibrosis has really successfully set up the infrastructure to have cystic fibrosis drugs rapidly evaluated once they reach the clinic. 

Mr. PITTS. The chair thanks the gentlelady. 

Now recognizes the ranking member of the subcommittee, Mr. 

Pallone, 5 minutes for questions. 

Mr. PALLONE. I am going to have to—since I just got here. 

Mr. PITTS. OK. You want to yield to Green? 

Mr. PALLONE. Yes. 

Mr. PITTS. All right. Mr. Green. 

Mr. GREEN. Thank you, Mr. Chairman. 

Dr. Woodcock, welcome back. I want to thank our chairman, 

ranking member, and Dr. Woodcock for testifying. In a time where 

revolutionary science and technological development, we have an 

opportunity to target specific patient populations, advance personalized medicine, and transform how we approach the prevention 

and treatment of disease, one of the goals I think is particularly 

worthy of exploration is the idea of personalized medicine, in which 

a patient may be able to receive more tailored drugs and treatment 

suited to his or her specific condition. 

Our understanding of the human genome is the key to that goal. 

Academics and researchers tell me, another piece is the potential 

for researchers and developers to discuss these drug and device innovations with patients during the development phase. 

Dr. Woodcock, can you give us your views on the upsides and 

downsides of any increasing permissibility of communication between patients and developers during the clinical trial phase of development? 

Dr. WOODCOCK. It is a very interesting question. We have seen 

from the 1990s, where only 5 percent of drugs were targeted; in 

31 

2013, 45 percent of the drugs we approved were targeted in some 

way. There are barriers to locating patients and joining up patients 

who have specific conditions, subsets with appropriate investigational therapy, and these diseases are fragmented into smaller and 

smaller subsets. It is harder and harder to find these people who 

might be eligible for a given therapy. 

The Lung-MAP trial is one way of doing that where it has multiple investigational arms in one trial, so people can come in, and 

they can be spread out. But there is great interest, of course, with 

more patient activism in using social media and other ways to actually match up the right patients with the right investigational 

drugs, and I think this is one of the challenges right now of the 

clinical trial enterprise. 

Mr. GREEN. Well, increasing patient involvement in FDA’s decisionmaking surrounding drugs, devices is a significant yet challenging endeavor. Can you provide your suggestions on how mechanisms need to be developed to accurately measure what meaningful 

outcomes for patients are, both in the clinical outcomes and the 

quality of life? Can we do that? 

Dr. WOODCOCK. Yes. That is what I was referring to with Chairman Pitts is that there is a science of measurement, and patientreported outcomes is one science. How do you measure how a patient feels from their point of view? And there are ways to do this, 

but these measurements have to be developed. We approved many 

drugs based on their impact on quality of life, so that is completely 

possible, but what needs to be done is this science needs to be developed, and we are participating in that. As I said, we have an 

expert meeting next week on patient-reported outcomes. 

Mr. GREEN. Well, and the patient involvement process has to be 

data driven and improve the overall efficiency of drug development 

and maintain FDA standards of safety and effectiveness. How can 

Congress support the FDA in incorporating patient perspectives in 

regulatory decisionmaking in a way that helps deliver that innovative, safe, and effective medicines to the patients sooner? 

Dr. WOODCOCK. Well—— 

Mr. GREEN. Do you need statutory authority, or do you think you 

already have it? 

Dr. WOODCOCK. To my knowledge, we have the authority to do 

this, and I think you will hear from the next panel, for example, 

how patient groups can develop draft guidance, submit it to the 

FDA. They can run processes that actually incorporate all their 

points of view and those of the expert scientists, so more of that 

needs to be done, but I don’t know that it needs more statutory authority. 

Mr. GREEN. Can you do it within current resources, because 

again, you are specializing, instead of a broad brush—and I assume 

it costs more when you do an individual? 

Dr. WOODCOCK. Yes. Well, when you have 7,000 diseases that 

need good treatments and most of them don’t have them, it would 

be very difficult for FDA alone to develop the standards for patient 

reported outcomes in each one of those diseases, much less the clinical outcomes. So much more participation of the medical and patient community is needed in drug development, and we need to 

32 

find better ways to do that, but I am not sure that is through legislation. 

Mr. GREEN. OK. And without a doubt, our greater resources, but 

again, our committee has worked over the years to try and provide 

those resources—— 

Dr. WOODCOCK. Yes. 

Mr. GREEN [continuing]. To the FDA and look forward to working 

with you. 

Thank you, Mr. Chairman. I yield back. 

Mr. BURGESS [presiding]. The gentleman yields back. 

I recognize myself for 5 minutes for the purposes of question. 

Dr. Woodcock, again, good to see you, good to have you back in 

the committee. So you have talked about how the FDA routinely 

works with sponsors to apply flexibility, including the use of biomarkers, surrogate endpoints, and nontraditional trial designs, and 

other available tools to expedite the development of products to 

treat both common and rare diseases. 

With respect to the common diseases, how is the FDA working 

with sponsors to apply these innovative development and review 

methods? 

Dr. WOODCOCK. Well, for example, hypertension is a common disease. We approve drugs for hypertension based on a surrogate 

measure, blood pressure, that is very well accepted, and for a number of years ago, we looked at automated blood pressure monitoring, and we decided it was unbiased, and so we decided that you 

really didn’t need a control group in the same way that you would 

for most other diseases because you have an unbiased measure, 

and so we issued new approaches to studying hypertensive medicines. So that is an example. 

Mr. BURGESS. What could happen so that the FDA could use this 

more frequently? 

Dr. WOODCOCK. Well, 45 percent of the drugs that we approved 

over the last several years used a surrogate endpoint. So we do use 

that when it is appropriate and it is available. For many diseases, 

we don’t know what the right surrogate is, and that is why many 

of the accelerated approvals have been confined say to cancer and 

HIV is because the science, a great deal of science has been driven 

in those conditions, and we understand the biomarkers. But for 

other diseases, there needs to be more scientific development, and 

that is why we are using this, for example, biomarker qualification 

process to try and get more biomarkers developed that we can use, 

but we can’t just dream them up and use them. 

Mr. BURGESS. I thought that was your job. Well, let me ask you 

this. Are there situations where a majority of the scientific or research community believes that a certain biomarker sufficiently 

predicts the clinical outcome, but the FDA has yet to accept that? 

Dr. WOODCOCK. There may be. I think there is a lot of controversy around use of these. You heard some of that on Wednesday. There are two sides to this. If you rely upon a surrogate, often, 

especially when it isn’t well validated, there is more uncertainty 

about whether or not the drug is actually going to work or not, and 

so there are different points of view. And as we have all been saying, the community, the patient community really ought to have— 

and treating community ought to have—a lot of input into how 

33 

much uncertainty should be tolerated, given the circumstances of 

that disease. 

So there are situations where there is disagreement amongst 

various parties, external and internal, about the use of a surrogate. 

Mr. BURGESS. Are you able to give us any examples of that, of 

a surrogate that the FDA may not right now be willing to accept? 

Dr. WOODCOCK. Well, for example, raising good cholesterol, all 

right. We had a series of trials on that. Everybody thought raising 

good cholesterol would be really good, and in fact, it turned out to 

be either neutral, or in one case, it actually increased mortality, so 

we no longer accept that surrogate. That is the kind of example 

where—and there are many others like that. 

In bone density, for osteoporosis, estrogens do a very good job 

and they decrease fractures. Although they have other liabilities. 

But some other agents were tried, and actually, they increased 

bone density, but they also increased fractures, and so we have to 

be careful when we use these surrogates to make sure that we are 

getting the intended results, clinical results. 

Mr. BURGESS. Thank you for that. 

Let me ask you a question that is a follow-up from when we visited in April. 

Do you have an update on the status of the FDA’s guidance on 

biosimilars naming and when that guidance will become final? 

Dr. WOODCOCK. Well, I certainly would like to get that guidance 

out as soon as possible. We are working diligently on that, and I 

don’t have any further update. 

Mr. BURGESS. But that was submitted as a question in April, and 

we are awaiting an answer. 

Now, also, along with that, I asked if anyone in the administration, outside of the FDA, had provided the agency with suggestions 

or recommendations with respect to this guidance. 

Can you, if the answer to that is yes, can you provide us with 

the name or names of those individuals? 

Dr. WOODCOCK. We would have to get back to you on that. 

Mr. BURGESS. And again, we anxiously await your answer. 

My time is expired. 

I will recognize the gentleman from New Jersey, the ranking 

member, 5 minutes for questions. 

Mr. PALLONE. Thank you. 

Mr. Chairman, you asked a lot of my questions, so I am going 

to have to move on to other things. 

But Dr. Woodcock, we heard a lot at Wednesday’s hearing about 

the accelerated approval program at FDA, and as you know, the 

program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need, and the drugs are approved 

on the basis of surrogate endpoints which we also learned about on 

Wednesday, and of course, a critical requirement of the system is 

that companies conduct studies to confirm the clinical benefits suggested by the surrogate endpoint, and these studies are called 

phase 4 confirmatory trials. So a critical part—I want to ask about 

the phase 4 trials. What challenges has FDA faced with respect to 

phase 4 trials? Do sponsors complete in a timely manner? 

Dr. WOODCOCK. Well, it is sometimes difficult to complete these 

trials, and the reason is that if you had a serious and life-threatVerDate Nov 24 2008 11:05 Feb 05, 2015 Jkt 037690 PO 00000 Frm 00039 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-158 CHRIS
34 

ening disease and we approved a treatment for it, you probably 

would be somewhat reluctant to enter a trial where you had a 

maybe 50 percent chance of not getting the treatment. So what we 

often do is ask that trials be conducted in a different stage of disease or something where it actually hasn’t been studied yet, so 

then we can get the results since that might take time. 

So I think in the early years of the program, we didn’t track this 

as well as we should, and we did have a lot of trouble getting these 

trials completed. But in the current era, we are on top of this, and 

generally speaking, the sponsors are diligent in trying to get them 

completed, generally, but they have difficulty sometimes enrolling 

patients in these trials. 

Mr. PALLONE. Another important component of the program is 

that when the surrogate endpoints do not ultimately show the anticipated clinical benefit, FDA could be faced with needing to remove the indication or take the drug off the market, and I imagine 

that is also no easy task. 

Can you describe what is involved with removing the indication 

or taking a drug off the market and what challenges does the FDA 

face there? 

Dr. WOODCOCK. Yes. Generally, if the confirmatory trial failed to 

show benefit, the first thing we ask is the sponsor to voluntarily 

withdraw the drug or the indication from the market. It is only if 

the sponsor does not agree to do that, then we go into a long administrative process, which includes hearings and formal findings 

and so forth, and this can take a long time if the sponsor can test 

our finding that the drug isn’t effective. 

Mr. PALLONE. Now, just a couple of years ago, we included some 

provisions to improve upon the accelerated approval program, and 

the FDA Safety Innovation Act of 2012. For example, the law made 

it clear that FDA could rely upon evidence developed using biomarkers or other scientific methods or tools when assessing surrogate endpoints. Can you describe what impact those legislative 

changes had on the program, and are there any other changes that 

you feel are necessary to allow you to make full use of the most 

recent scientific developments with respect to surrogate endpoints? 

Dr. WOODCOCK. I think the legislation was very helpful. We have 

taken it quite seriously. We have issued guidance, final guidance 

on expedited programs, and probably the biggest change that the 

legislation brought about was its focus on intermediate clinical end 

points, and we had to have quite an internal discussion about what 

that means, and I think you will see us approving more products 

under accelerated approval based on these intermediate clinical 

endpoints. 

Mr. PALLONE. All right. Well, thanks. 

Again, it is clear to me that this is an extremely complicated 

area and one that is not necessarily conducive to further legislation, but I wanted to ask last about the master protocol. 

At the hearing on Wednesday, some panelists described some of 

the inefficiencies that exist in the way that clinical trials are currently conducted, and one of the suggestions for addressing those 

inefficiencies is to create a master protocol. So I just wanted to ask, 

first, can you tell us more about this, what is a master protocol? 

How would it help to improve the way we conduct clinical trials? 

35 

Has FDA been involved in the development of a master protocol, 

and are there particular diseases that the master protocol is more 

appropriate for than others, and if so, which ones, and are there 

other areas where it might be expanded? 

Dr. WOODCOCK. Well, master protocol is one version of using clinical trial networks or standing clinical trials to evaluate investigational therapies where the drug development program isn’t just for 

one therapy. It is for any therapy for that disease. So master protocol, though, has to be somewhat disease specific. You can’t just 

have a general overall master protocol, right. It has to be focused 

on one disease. 

For example, the Lung-MAP trial is on squamous cell cancer of 

the lung that is advanced but five different agents right now are 

being studied all at once within that protocol, and that is a huge 

efficiency. But there are other versions of standing trials or trial 

networks that also could be used in other diseases. And as I said, 

the Cystic Fibrosis Foundation has a kind of network of clinical excellence where they actually sequenced the genome of all their patients, and so they are ready when a targeted therapy comes along. 

They are ready. They can put those patients into the protocol, and 

that tremendously improves the efficiency. 

So it is a long conversation that probably can’t be had in 5 minutes, but I have long advanced this concept and tried to push this 

concept because the current clinical trial paradigm is not sustainable. 

Mr. PALLONE. All right. Thank you very much. 

Thank you. 

Mr. BURGESS. The gentleman’s time is expired. 

The chair recognizes the gentleman from Pennsylvania, Dr. Murphy, 5 minutes for questions, please. 

Mr. MURPHY. Thank you. 

And good morning, Doctor. It is always good to have you here. 

Let me start out by asking about it is important for the medications and research to advance those but also for those that are already approved, and so let me ask you, we had passed the PDUFA 

laws a while ago, certainly that was supposed to help us get more 

generic drugs in the queue, but what has happened is we got 1.5 

billion authorized over 5 years, what has happened is approval 

times have gone up, and there are fewer approvals, even though 

the law was supposed to reduce all those. 

Can you give me some indication of what is going on and what 

FDA is going to do about that? 

Dr. WOODCOCK. Certainly. We are well aware of these issues. In 

June, we received 625, I believe, generic drug applications, so the 

rate of submission is well above what was projected in the negotiations that we held. 

However, on October 1, the deadlines kick in for timelines for review of generic drugs, and we are fully prepared to meet those 

timelines as well as deal with this large backlog of pending. 

We had to hire a large number of people and totally revise our 

processes, reorganize the generic drug review offices and conduct 

many other changes, and that is what we have done over the past 

2 years in preparation for the deadlines coming into effect on October 1. 

36 

Mr. MURPHY. Thank you. Another question here about some labeling issues. The abbreviated new drug application that would 

allow generic manufacturers—this is a proposal for FDA to change 

a label without FDA’s prior approval but then come back later on, 

and the FDA itself has recognized, and say, quote, ‘‘consistent labeling will assure physicians help professionals and consumers that 

a generic drug is as safe and effective as its brand name counterpart,’’ unquote. But there is a concern out there that allowing these 

changes take place and then go backfill them later on can cause a 

lot of confusion in studies that have asked pharmacists and physicians this, so I am wondering where this issue stands in clarifying 

this. 

Dr. WOODCOCK. Well, we have received comments on the proposed rule. It was a proposed rule, and we received many comments. We are analyzing the comments, and subsequent to that, we 

will have to go forward with a rulemaking process. 

The proposed rule contemplated that we would actually have less 

disparities of labels in the marketplace on this because of this proposal because we would put up a Web site, and we would also require conformance of labels, which we cannot carry through right 

now, given the current systems. 

Mr. MURPHY. A lot of us back in January asked to meet with 

Commissioner Hamburg and others about this, and I am not sure 

those things have taken place yet, so I hope this gets expedited and 

that these issues are addressed because I think it still leads to 

some confusion. So I am not clear yet in understanding even why 

this proposed rule was set up there to allow individuals to change 

the label and then come back later and ask permission. 

Dr. WOODCOCK. Well, currently, generic labels do not always 

match the innovator and they do not change their label in a timely 

manner, and so there will be labels out there for quite a bit of time, 

even with serious safety issues like new box warnings that don’t 

conform to the innovator label, so we are trying to address this situation. And also, as generics are now 85 percent of all drugs dispensed to consumers—that they should have the opportunity, since 

their drugs are the ones that people are being exposed to, to submit 

their findings of adverse events and suggest label changes, proposed label changes and actually execute them. 

Mr. MURPHY. Well, I just hope that you will meet with the committee staff members and the companies to help clarify this because it still is not clear to me why this would be allowed, and I 

think it would end up confusing. 

I want to bring up one last thing just while you are here. I had 

sent a letter a few weeks ago to Dr. Hamburg. I am sure you didn’t 

see this, but one of the things that is out there, too, is complications that are oftentimes reported in the media about caffeine, 

whether it is—and sometimes toxic levels people take. 

Dr. WOODCOCK. Yes. 

Mr. MURPHY. Through over-the-counter things, pure caffeine or 

some of these supplements out there for athletes, et cetera, and yet 

it is also in everything from chocolate to coffee and other things we 

promote all the time, so I am hoping, at some point, FDA can also 

give some recommendations in terms of individual levels per drink, 

37 

per dose, per day, per male, female, the genders, for weight, age, 

whatever that is. 

Dr. WOODCOCK. Yes. 

Mr. MURPHY. Because it is still pretty confusing, whatever those 

products are that they can be beneficial, but I hope you will expedite that. 

Dr. WOODCOCK. Thank you. 

Mr. MURPHY. Thank you. 

I yield back. 

Mr. BURGESS. The gentleman’s time is expired. 

The chair recognizes the gentlelady from California, Mrs. Capps 

for 5 minutes for questions, please. 

Mrs. CAPPS. Thank you for holding this hearing, to our chairman 

and ranking member. 

Thank you, Dr. Woodcock, for your testimony. 

This is an issue very dear to me, and as you know, I am incredibly concerned about our Nation’s history of excluding minority 

groups, especially women, from all levels of medical research, from 

the lab rats to the most advanced clinical trials. And reports have 

shown that even when these groups are included in trials, there 

are often too few participants in the groups to analyze the effects 

on them or the analyses are simply not run or reported. 

I am sure you are familiar with the case of Ambien, commonly 

prescribed medication that recently had its label changed because 

it metabolizes differently in women than men, meaning that 

women had been receiving an inappropriately high does of this 

drug for over 20 years. 

In addition, in spring, a report entitled ‘‘Sex-Specific Medical Research Why Women’s Health Can’t Wait’’ was released, which provides evidence for the further inclusion of sex and gender in scientific research. And the FDA’s own August 2013 report, which 

was initiated by the inclusion of My Heart for Women Act in the 

FDASIA legislation, showed that there is still much work to be 

done to make sure that women are fully represented in clinical 

trials and that the safety and effectiveness of the information is 

readily available. 

I know the FDA is continuing to work on an action plan to address these disparities, so Dr. Woodcock, can you give us an update 

on where the agency is on this? 

Dr. WOODCOCK. Certainly. I would expect that that would be released, we would be timely in its release. I believe there was a statutory deadline or not, or there is some expectation, so we are working diligently on the action plan, yes. 

But I will say for drug development, which is what I am discussing here, that we did a study, for example, the class of 2010, 

the product that we approved, we found that about 45 percent of 

the participants were male, all right. 

And we found that almost all the submissions included the required gender analysis, which has been required for drugs for 20 

years, because I oversaw that when I first joined the Center for 

Drugs in 1994. So it is by regulation, so we do have these, but I 

think the transparency of the information is the problem, and we 

are working on that, and I really am committed to making that inVerDate Nov 24 2008 11:05 Feb 05, 2015 Jkt 037690 PO 00000 Frm 00043 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-158 CHRIS
38 

formation more transparent so people understand what we know 

and what we don’t know. 

Mrs. CAPPS. I think you put your finger on something, and I 

want to highlight a bipartisan letter I led, signed by the women of 

the House of Representatives, urging this agency to include clear 

and actionable strategies. And I think what you said about transparency and the reporting in the action plan is a way to address 

this issue once and for all. 

At Wednesday’s hearing this week, I also asked the panel about 

the tools FDA is developing that could supplement our knowledge 

base, especially in the light of less robust clinical trial designs. The 

FDA Sentinel system, which I understand is making progress, if 

slowly, to conduct post-market passage surveillance of drugs and 

devices, could help spot issues like adverse drug interactions more 

quickly. I believe the Sentinel program holds great promise, and 

that is why I worked to get the Sentinel Assurance for Effective 

Devices Act included in FDASIA to continue progress on the program and ensure the design for both drugs and devices. Could you 

update us on the development of the Sentinel program, please, and 

what other resources or authorities do you need to get the system 

up and running to protect consumers more effectively and expeditiously. 

Dr. WOODCOCK. Well, I think use of electronic health data, which 

is rapidly becoming available, and the electronic health records and 

so forth, has tremendous promise for actually finding out what 

happens in the real world for medical products, both that are approved recently and those that have been on the market a long 

time, and that is what the Sentinel system is intended to do. 

We have run a mini-Sentinel network for 5 years, and that was 

between drugs and biologics. We paid for that out of our money 

that we have, and we are recompeting that to put up the Sentinel 

system, so that contract proposal is out on the street, and we hope 

to establish the real Sentinel system, which will be a large-scale 

system for surveillance. 

Now, as far as medical devices, we require a unique identifier or 

some kind of identifier in the medical record electronically so that 

we are able to capture that because the Sentinel system uses those 

electronic records to get the information, and I will repeat for everyone that it does not take any personnel information and move 

it to some central database. It strictly runs those analyses within 

the healthcare system and then the results only are combined. 

So that has tremendous promise. We feel very good about that. 

We actually are piloting running active surveillance on there, so 

when we approve a drug and we have a question about it, we can 

watch over time and see what actually happens. And as more and 

more people get on electronic health records, we can really have 

more insight in what is happening. 

So that is where we are with that, and it is resource limited. I 

have to pull resources from other activities to fund that, but I believe very strongly that this is the future. 

Mrs. CAPPS. Thank you. I appreciate that. 

Mr. PITTS [presiding]. The chair thanks the gentlelady. 

Now recognizes the gentleman from Georgia, Dr. Gingrey, 5 minutes for questions. 

39 

Mr. GINGREY. Mr. Chairman, thank you. 

Dr. Woodcock, thank you for appearing. It is always good to see 

you. 

I understand that a number of the challenges that have led to 

the duration and cost of conducting clinical trials in the U.S. to increase essentially are outside of FDA’s purview. That being said, 

clinical trials are conducted to generate evidence used in the application for FDA approval, so my first question, how early do you 

typically communicate with these pharmaceutical companies, to 

discuss their trial design before the investigational new drug application is submitted? 

Dr. WOODCOCK. Well, we have agreements under PDUFA, that 

prescription drug user fee program, and for novel products or novel 

indications, say they are testing a disease that really doesn’t have 

any treatment, companies can come in and have a pre-IND meeting. That meeting is before they start their clinical trials, their first 

in human studies, and we talk about that development program so 

they can start thinking about how that is going to be done. 

We do have information, it is preliminary, but looking at our information, it seems that companies that have more interactions 

with the FDA are able to get their products through more quickly, 

through the entire clinical trial process than companies that 

haven’t had interaction with the FDA during the development process. But there are formal meetings that are held at different times 

under the user fee program, and those minutes are tracked, and we 

track the meetings and so forth, so there is quite a process for 

interaction during drug development. 

Mr. GINGREY. So you, as a manager, would be, maybe at that 

particular time, you make sure that your reviewers are not requesting overly burdensome data that really is not necessary so 

that the process can be speeded up? 

Dr. WOODCOCK. Well, there is always a push and pull. Scientists 

of all stripes always want more data, and that is scientists in the 

companies and scientists in the FDA, and so we have to walk that 

path between getting more data and actually the cost that is generated. And we have made a number of efforts under the CITI collaboration that we do with Duke University and many, many, 

many other partners to try and figure out how to streamline clinical trials as far as data collection, for example. But it is difficult. 

We have 1,600 meetings a year under the PDUFA, and when we 

meet with companies, the supervisors are there, the senior medical 

officials are also at these meetings. 

Mr. GINGREY. Well, that is the whole purpose of 21st Century 

Cures, of course, and as we get to the second panel and we hear 

about the associations and from the families, I am sure they are 

going to talk about how we can speed this process up. 

The last question. At our first 21st Century Cures hearing, we 

heard that only 19 drugs, outside of cancer and HIV space, have 

been approved by the accelerated approval pathway since 1992, 

and I understand that you wrote a blog post after that hearing 

about how a number of drugs that were being considered under accelerated approval ultimately received traditional approval, so 

these statistics, according to your blog, were somewhat misleading. 

40 

Can you provide some examples of when that occurred as well as 

the process involved? 

Dr. WOODCOCK. Certainly. Well, for certain rare diseases, we 

may decide, for example, that the surrogate is fine, OK, and it correlates with clinical benefit. Then the term ‘‘accelerated approval’’ 

is a little misleading. It sounds like it is faster than regular approval, but actually, if we give regular approval on a surrogate, it 

is just as fast as accelerated approval, but you don’t have to do confirmatory studies afterward because we already believe the surrogate. 

So, for a lot of, say, rare deficiency diseases where there is something missing, you may be able to show that you actually, when 

you replace that protein in the body, you give the activity back to 

the person, right, and so you may not have to show clinical outcomes. It is still a surrogate, but we feel it is good enough because 

we understand the problem that something is missing, and you deliver an active drug to the site of action of where the problem is, 

and that would be enough. 

So, in many cases, we are able to do traditional approval with 

the surrogate; that means that the patients and the sponsor don’t 

have to go through all these confirmatory trials. I described the difficulties of that when you have a serious disease; you have approved a drug; and then you ask people to be randomized after approval. 

Mr. GINGREY. Dr. Woodcock, thank you. 

And my time is expired. Mr. Chairman, thank you, and I yield 

back. 

Mr. PITTS. The chair thanks the gentleman. 

I recognize the gentlelady from Florida, Ms. Castor, 5 minutes 

for questions. 

Ms. CASTOR. Well, thank you very much. 

Thank you, Dr. Woodcock, for everything you are doing to ensure 

safe and effective drugs are available for the American public and 

those with health challenges. 

This is a hearing about the patient involvement in FDA drug approvals, and I think we can agree, they deserve a seat at the table 

when companies are developing drugs and medical devices within 

the clinical trial process. I have long been a supporter of the Department of Defense’s Congressionally Directed Medical Research 

Programs known as the CDMRP. CDMRP funds peer-reviewed research into breast cancer, autism, ovarian cancer, prostate cancer, 

and other diseases. And since 1993, the patients have been involved and have been a part of CDMRP, and they have a consumer 

reviewer as part of a peer-review panel to represent the stakeholder community, and it has been very successful in combining patient perspectives and needs with scientific research and bringing 

those perspectives together. 

Has FDA, as you begin to consider improving patient involvement, have you looked at CDMRP to see if there is anything you 

can borrow from that in the drug approval process? 

Dr. WOODCOCK. We have not, and that is a good suggestion, so 

we would be happy to do that. 

Ms. CASTOR. OK. You mentioned previously that the Patient-Focused Drug Development Initiative that was included in PDUFA 

41 

was designed to allow FDA to hear from patients on how a disease 

impacts their life, and I understand you are scheduled to hold 20 

public hearings. Share with us who FDA has met with so far. Have 

you started those hearings? If so, what have you learned already? 

Dr. WOODCOCK. Well, we have learned the devastating impact, I 

think, of the diseases, of these different diseases on people’s lives 

it just incredible. We had one on chronic fatigue syndrome—that 

was our first one—HIV, lung cancer, narcolepsy, sickle-cell disease, 

fibromyalgia, pulmonary arterial hypertension, and inborn errors of 

metabolism, and we plan to have 16 of these meetings completed 

by the end of 2015, but we recognize this is just a drop in the bucket of what people suffer from. 

So what we are trying to do is really model how people can do 

this, and hopefully, it could be done more—not put on by the FDA 

but by the patient groups themselves in the medical community 

that serves them so that they can assemble more of this information and kind of multiply the effect of this, and we are already seeing some of that. NORD, for example, has offered to help with rare 

diseases, for example, to have more input that way because our resources are limited. We are not going to be able to cover all the different diseases. 

Ms. CASTOR. Good. So I expect we will hear from the patient organizations later today and their view on how they can be helpful 

and we can be effective. 

I think the wave of the future really is the information we will 

be able to gather through the electronic health record, so it is interesting to hear what you have done already with the Sentinel initiative. I heard from research institutes back home that are doing so 

much in genomics and personalized medicine that they think these 

larger networks are the wave of the future. You say you don’t need 

additional legislation to continue, but you are having to borrow resources from this and that. 

Dr. WOODCOCK. Yes. 

Ms. CASTOR. So is your advice to the committee that we need to 

do more in technology when it comes to improving timelines on 

clinical trials by focusing on these networks and the electronic 

health record? 

Dr. WOODCOCK. The electronic networks have much promise in 

doing clinical trials. 

If we could move clinical trials more out into the community and 

have people out in the community, like most cancer patients in the 

U.S. who have diseases that are untreatable don’t get into trials 

because they are being treated at places that aren’t running trials. 

So we need to move this out into the community, make those folks 

eligible. 

And I am on the Steering Committee of the Lung-MAP trial, and 

I really urged that we make sure that we are out there in the community so that anyone who has lung cancer has an opportunity to 

participate in this research and perhaps have a more effective therapy. 

So I think the electronic health records, that is a huge different 

area that we are working on in how to do clinical trials utilizing 

that infrastructure that is emerging. 

Ms. CASTOR. Great. Thank you very much. 

42 

Mr. PITTS. The chair thanks the gentlelady. 

I now recognize the gentleman from New Jersey, Mr. Lance, 5 

minutes for questions. 

Mr. LANCE. Thank you very much, Mr. Chairman. 

A portion of your testimony has focused on the FDA’s efforts on 

patient engagement. It is my understanding that ClinicalTrials.gov 

was intended to be a resource that provides clinical study information for patients, for healthcare providers, and for researchers. But 

it seems to me that the site lacks considerable information and has 

proven to be difficult to navigate. 

Dr. Woodcock, would you please comment on the current utility 

of the ClinicalTrials.gov. 

Dr. WOODCOCK. Well, I think that it has provided, along with the 

requirement of the medical editors of the journals that things be 

registered before they are going to be published—provided tremendously more transparency into what clinical trials are ongoing in 

the United States. 

And that has been a big achievement. All right? So we know, the 

issue of publication bias and everything is minimized because we 

know what trials have been done. 

However, I agree that, certainly for patients, I think that initiation of trials and understanding where there might be a trial that 

might be ongoing that might be available to them has also been effective, although, as you said, there are technological issues that 

remain. So it has made tremendous progress in transparency. 

Mr. LANCE. Is there a way that you and we can work together 

to improve it? And I am not suggesting that you are in any way 

responsible for the challenges that remain. But moving forward for 

the better health of the American people, how together can we improve it? 

Dr. WOODCOCK. Well, the FDA Amendment Act required that 

regulations be issued around the results—— 

Mr. LANCE. Yes. 

Dr. WOODCOCK [continuing]. Section of this and that they consider whether to require the submission of clinical trial results for 

unapproved products, because much of the lag in getting results in 

there is that the products still are not on the market. 

So NIH is the lead for this rulemaking and I think they would 

be in the best position, and, also, they operate the infrastructure 

for this database. 

Mr. LANCE. Thank you. 

In another area, in the past several hearings, we have discussed 

the difficulty of various institutions communicating one with another and a lack of coordination often leads to inefficiencies. 

What methods are currently in place to reduce redundancies in 

clinical trials? And what steps can we take together to ensure that 

we are not doubling up on research or making the same mistakes 

over and over? 

Dr. WOODCOCK. Hopefully, most things would eventually come 

out and be published. But certainly in the drug development area, 

there is interest in more sharing of earlier data and sharing of failures. 

But this has proven to be very a intractable area—— 

Mr. LANCE. Yes. 

43 

Dr. WOODCOCK [continuing]. For transparency. All right? 

Mr. LANCE. Yes. 

Dr. WOODCOCK. But we have continued to work on that. 

Mr. LANCE. Yes. 

Dr. WOODCOCK. As far as some of the things that were referred 

to in the prior hearing, which I was able to listen to some of, they 

were talking about some of the inefficiencies, say, of IRBs, where 

you might have to have 100 IRBs that looked at—— 

Mr. LANCE. Yes. 

Dr. WOODCOCK. And I believe that there are efforts to try and 

address this. It is not an FDA issue. But, really, we came out a 

number of years ago in saying that central IRBs would really be 

preferable in these large multi-center trials. 

And then the contractual agreements that take so long to set up 

with each specific site is something that has been taken on. They 

have tried to develop model agreements and so forth. 

But that is something that the standing trial addresses because 

you sign this contractual agreement once and then you can do multiple investigational agents. 

Mr. LANCE. Are we moving in the direction of central IRBs, in 

your judgment? 

Dr. WOODCOCK. Yes. There is certainly a consensus, I think, in 

the clinical trial investigator community that that is desirable, but 

various universities, naturally, are legally concerned about their 

own—— 

Mr. LANCE. Of course. 

Dr. WOODCOCK [continuing]. Liabilities and so forth. And so 

there is a push and pull about that. 

Mr. LANCE. I think this is an area that we should engage in further investigation to make sure that we move forward in a manner 

that does not result in redundancies. 

Thank you, Mr. Chairman. I yield back the balance of my time. 

Mr. PITTS. Chair thanks the gentleman. 

Now recognize the gentlelady from Illinois, Ms. Schakowsky, for 

5 minutes for questions. 

Ms. SCHAKOWSKY. Thank you, Mr. Chairman. 

Thank you, Dr. Woodcock. I think you are really an excellent witness. I appreciate your answers. 

Dr. WOODCOCK. Thank you. 

Ms. SCHAKOWSKY. I wanted to go a little bit further on the problem that Congresswoman Capps raised about the underrepresentation of women. 

I know you said that you found that, actually, women were overrepresented, but recently the Congressional Caucus for Women’s 

Issues sponsored a meeting with leading women heart experts— 

both clinical and research experts, physicians. 

Those experts raised concerns that the lack of representation 

from women in clinical trials is limiting our ability to effectively 

treat women with heart disease. They were focusing in on heart 

disease. 

And according to those experts, for the last 50 years, women’s 

heart treatment has largely been based on medical research about 

men. 

44 

And even today, despite that fact, what they said is that women 

make up more than 50 percent of the U.S. population, that women 

comprise only 24 percent of participants in all heart-related studies. 

And, additionally, scientists from the Women’s Health Research 

Institute at Northwestern—that is in my district—have raised concerns about the disproportionate number of adverse drug effects 

that occur in women due to the lack of sex-based clinical research. 

And, as you know, the biological, physiological, hormonal differences in males and females impact the rate of drug absorption, 

distribution, metabolism, elimination and, ultimately, affect the 

drug’s effectiveness. 

According to those experts, the lack of requirement for drug manufacturers to take this into account and document any sex variability early in the drug development pipeline before a drug has 

been released places consumers, especially female patients, at an 

increased risk of adverse drug effects. 

So I want you to respond to that. 

Dr. WOODCOCK. Certainly. 

Ms. SCHAKOWSKY. Many of us were really left with a very disturbing feeling because heart disease is the major killer of women 

right now. 

Dr. WOODCOCK. Right. Well, I think we have to—what are the 

facts on the ground. All right? One of the reasons for the disparities that they are mentioning is actually the fact that men suffer 

heart disease earlier in life than women. 

Ms. SCHAKOWSKY. Although, let me just point out, they also said 

that the growing number, even though it is lower—— 

Dr. WOODCOCK. Yes. 

Ms. SCHAKOWSKY [continuing]. Is younger women getting heart 

attacks and heart diseases. 

Dr. WOODCOCK. Yes. Yes. So that the reason for maybe maldistribution in the trials is because there is an age cutoff, and 

there always has been. 

In our survey, we found that there were—19 percent of the people in the trial in these 147 studies we looked at were over 65, 

which is more than in the general population, obviously, but of sick 

people, that is still low representation, right—to save people with 

heart disease. 

Generally speaking, there is often a cutoff—age 75—and we are 

trying to eliminate those cutoffs for age and concomitant conditions 

so that the population will be more representative. 

But to your original point, we have always required male and female animals in the toxicology studies. All right? We require what 

we call population pharmacokinetics, PK/PD, early in drug development. 

And our clinical pharmacologists look at blood levels and exposure in men and women and we understand that, usually, and that 

is modern drug development. 

So there are multiple trials that are done that look at exposures, 

in other words, achieve blood level by gender and other factors, 

liver failure, kidney failure and so forth. 

45 

And we can look at the phase 3 trials to see if they are—there 

has been a requirement in the regulations since, I think, 1994 that 

sponsors submit a gender analysis with their application. 

Ms. SCHAKOWSKY. Is this incorrect, then? It says women comprise only 24 percent of participants in all heart-related studies. 

Dr. WOODCOCK. Well, that may be true. And that may also include medical devices. It also may have to do with this age disparity when onset of disease. 

Ms. SCHAKOWSKY. I really hope that you will look at that because 

it is a great concern. It is a growing problem for women. 

And let me just give you an example of what—she said women, 

because we have different symptoms of heart disease—she said, if 

you have some of these symptoms of nausea, dizziness, go to the 

emergency room, but say, ‘‘I am having chest pains’’ because, without that, you may not get an electrocardiogram and you may be 

misdiagnosed. We need to help women. 

Thank you. I yield back. 

Mr. PITTS. Chair thanks the gentlelady. 

Now recognize the gentleman from Louisiana, Dr. Cassidy, 5 

minutes for questions. 

Mr. CASSIDY. Hello, Dr. Woodcock. I always enjoy your testimony. 

Dr. WOODCOCK. Thank you. 

Mr. CASSIDY. I mean that as a big compliment. 

So, next, real quickly—because I want to talk about something 

else—but does FDA—you mentioned that some institutions may be 

nervous about their liability if they refer their IRB activity to a 

centralized IRB. 

Dr. WOODCOCK. Correct. 

Mr. CASSIDY. Except so many do, we know that is a false argument. 

Is there any way FDA can reassure those institutions? Because 

the gentleman from Mayo suggested it is a cultural issue. He didn’t 

mention anything about legal. Thoughts? 

Dr. WOODCOCK. Right. Yes. I heard his testimony. 

In my experience, that there are legal—there are concerns of 

the—counsel of the various—— 

Mr. CASSIDY. Attorneys are always nervous. Right? I mean, they 

don’t make money if they are not nervous. I hate to be cynical, 

but—— 

Dr. WOODCOCK. Yes. 

Mr. CASSIDY. Is there any way FDA can send reassurances regarding that? 

Dr. WOODCOCK. Well, we have tried. In guidance and so forth, we 

have encouraged this. And in the city initiative, we had a whole 

discussion and dissemination of information about central IRBs. 

But possibly there is more that we can do to encourage this. 

Mr. CASSIDY. OK. Let me then bring on—you mentioned something intriguing earlier, that there may be some at high risk for 

disease; so, therefore they will be more risk-tolerant. 

Dr. WOODCOCK. Yes. 

Mr. CASSIDY. Now, I have a family member, a nephew, with 

Down syndrome. And I am looking on the alzheimers.org Web site, 

and they mention how virtually 100 percent of adults with Down 

46 

syndrome by age 40 will have evidence of the tangles associated 

with Alzheimer’s. 

Dr. WOODCOCK. Yes. 

Mr. CASSIDY. Now, what are the issues regarding—wow. This is 

a group of adults who are 100 percent at risk for a terrible condition. 

Dr. WOODCOCK. Right. 

Mr. CASSIDY. But there are other issues involved as well. 

What are your thoughts about this? How do we make stuff available for folks incredibly at risk for such a terrible disease? 

Dr. WOODCOCK. Yes. Well, with Alzheimer’s, there are a number 

of problems. The basic problem is we still don’t understand the disease well enough and the interventions that have been tried, which 

have been in late-stage disease when people are already demented, 

have failed to work. 

Mr. CASSIDY. Now, as I gather, though, the problem is predicting 

at an earlier stage those at risk. Correct? 

Dr. WOODCOCK. That is correct. If you want to intervene early. 

We recently issued a draft guidance saying that, OK, if you want 

to intervene earlier, we would accept an end point that is subtle 

cognitive testing. 

Mr. CASSIDY. I accept that. 

But how do you decide which population is at such high risk? Because, if you have a control group—you follow what I am saying— 

only 10 percent are really going to be at risk. 

Dr. WOODCOCK. Right. 

Mr. CASSIDY. You with me? This is a really expensive study. 

Dr. WOODCOCK. That is right. And so we advocate techniques 

called enrichment, which you try to use biomarkers or other tests 

to figure out. There are genetic conditions that increase your risk 

for Alzheimer’s disease. 

Mr. CASSIDY. So speaking of Down syndrome as one example? 

Dr. WOODCOCK. That would be one example. Yes. There are others. 

Mr. CASSIDY. And can you give us the progress of that. So if you 

accept these, are people now using these? 

Dr. WOODCOCK. Well, we need agents to use them in. So that is 

part—— 

Mr. CASSIDY. And I am sorry. ‘‘Agents,’’ you mean as in—— 

Dr. WOODCOCK. I am sorry. Investigational interventions that we 

can test in the people. 

And that is part of the problem. The science of understanding 

what causes Alzheimer’s and what you can intervene in that would 

actually delay or prevent the disease is not mature enough. 

And we have approved a couple imaging agents for Alzheimer’s, 

but they aren’t 100 percent. And you would maybe be kind of advanced—— 

Mr. CASSIDY. But, for example, I know hyperinsulinemia is 

thought to be a potential risk factor. 

Dr. WOODCOCK. Yes. I know that. 

Mr. CASSIDY. And I think there are some studies suggesting that 

Actos might give some benefit. 

Dr. WOODCOCK. Yes. 

47 

Mr. CASSIDY. Presumably, it would be at an earlier stage, not a 

later stage, would be a non-metabolic syndrome indication for the 

use of Actos. Fair statement? 

Dr. WOODCOCK. Yes. 

Mr. CASSIDY. So there is at least some of that. I guess I pose that 

to ask the degree to which that has been, again, the current state. 

I will go back to what is the current state of using that sort of 

thing? 

Dr. WOODCOCK. Right. So the current state, if someone decided 

to do a trial—and I believe there have been some intervention 

trials, not of Actos, but an earlier intervention at high-risk—in 

higher-risk people—they might identify people they felt were high 

risk for one reason or another, randomize them to this intervention 

or not, and then we would allow use of neurocognitive testing even 

before they had symptoms, if they had subtle changes, and if the 

treatment group did better than the placebo group, we could give 

accelerated approval. 

Mr. CASSIDY. So I guess you have got the framework. It is just 

a question of someone coming forward to take advantage of it. 

But how long would such a study, do you imagine, take to complete its course? Twenty years? 

Dr. WOODCOCK. No. No. But we need to have better measurements that stick to these biomarkers and other measurements, like 

of subtle cognitive function, where we—the NIH and us and others 

are working on this. 

Because the earlier you can intervene—if you have a very targeted test that can identify people early, they don’t have any symptoms, but you can tell their brain isn’t working as well as it should, 

and then it will decrease over time. So that is kind of the rate-limiting step. 

But I agree. Prevention is very difficult because there you want 

to intervene on people who are well and treat them for a long time 

and expose them to something with the hope that, at the end of the 

day, they are not going to get whatever bad outcome. 

Mr. CASSIDY. We are out of time. Thank you very much. 

Mr. PITTS. Chair thanks the gentleman. 

Now recognize the gentleman from Virginia, Mr. Griffith, 5 minutes for questioning. 

Mr. GRIFFITH. Thank you very much, Mr. Chairman. 

Dr. Woodcock, as others have said today and, also, what I have 

heard in some of our informal conversations is that you not only 

do a good job as a witness, but that you are doing a good job overall. 

Dr. WOODCOCK. Thank you. 

Mr. GRIFFITH. And so I appreciate that, and thank you so much 

for being here today. 

You and Dr. Cassidy had a little conversation about lawyers. 

Some lawyers are always nervous. Other lawyers are always looking for a way to solve the problem. 

And so maybe we need to get some of those lawyers on your team 

and some of the corporate teams to solve the problem, figure out 

how we can make these things work, because I do think it is important. 

48 

As you probably know, I am one of those who advocates that we 

try to move a little quicker in those areas where we have problems 

that we don’t have solutions for currently and, also, favor what is 

known in some State laws as right to try when you have a situation where doctors have tried everything and folks are given a diagnosis they have got months to live or their condition is going to 

be fatal. 

I am one of those people who believes that we ought to let them 

go ahead and try whatever it is they are willing to pay to do because the FDA can’t protect you if you are going to die from something that might kill you. 

Dr. WOODCOCK. Right. 

Mr. GRIFFITH. I mean, it is going to happen one way or the other. 

You might as well have the right to try something. 

That being said, I know there are a lot of issues surrounding 

that. I am not sure we have time for for that discussion today. 

And I know that there is another panel, and I want to hear from 

the patients as well because they are involved in this process. 

So respecting you greatly, I yield back my time. 

Mr. PITTS. Chair thanks the gentleman. 

Now recognizes gentlelady from North Carolina, Mrs. Ellmers, 5 

minutes for questions. 

Mrs. ELLMERS. Thank you, Mr. Chairman. 

And thank you, Dr. Woodcock, for being with us again today. 

This is such an important issue. As you know, we had our panel 

on Wednesday. And it seemed to me that it was a general consensus that everyone is looking for ways to expedite this and to 

make it more efficient and get those drugs to market sooner so that 

we can be taking care of our patients more effectively. 

In your testimony and in the discussions that we have had today, 

you have touched on the biomarkers and targeted drug development to benefit disease populations, obviously. 

As all of our representatives here, we all have constituents with 

rare diseases, heart-breaking. Especially right now in my community, I have a very good friend with ALS. And as I am learning 

more and realizing, we have had a number of members of our community diagnosed with ALS. So this is something that is very important to me right now. 

And I am just looking at the idea—as far as the target approval 

process being appropriated and applied through the FDA, it seems 

to be that we are looking at cancer and HIV. Where do some of 

those rare diseases fall within that? 

And you had mentioned and there was discussion about the master protocol and that seems to be applied more to cancer or HIV. 

Where can some of the rare diseases fall in there? And what can 

we do to help make that happen? 

Dr. WOODCOCK. Well, any rare disease would be a great candidate for a standing network, a network of experts—and I think 

you may hear more about this from the next panel—where they are 

ready to evaluate any therapy that advances through the early, the 

nonhuman, stages. 

So they could pick that up right away and test it quickly. In the 

meantime, until that happens, they can get what we call natural 

history, which I know sounds very wonky. 

49 

But people are asking—just now Mr. Cassidy—like how long does 

Alzheimer’s progress from presymptomatic to symptomatic. Well, 

we need to know that so that we can design the trial correctly. 

In rare diseases, even more difficult because nobody knows. And, 

usually, they get experts together and say, ‘‘Well, in my opinion, it 

takes this long.’’ Right? And they are usually wrong because they 

have only seen a few people. 

So we are encouraging these natural history studies, these networks. First, they look at the people and they can look at the biomarkers, too. 

So what changes in ALS? What can we measure? Could we measure something that gives us indication that treatment might be 

working? Right? 

And then, as soon as a therapy becomes available, then you can 

rapidly get people into a trial and there would be no delays because 

there is no delaying an ALS. 

Mrs. ELLMERS. Right. Exactly. 

And that is obviously part of the concern. And certainly I agree 

with my colleague in talking about right to try. This would be a 

perfect example of decisions that families and patients can make. 

I do want to talk about—you had also mentioned listening carefully to patients and families. 

Dr. WOODCOCK. Yes. 

Mrs. ELLMERS. And do you consider and give more weight—that 

is one of our questions, is how much weight are you giving to the 

patients and families? And there again, from our perspective in 

Congress, what can we do? 

You know, as we have heard everyone agreeing that we need to 

make a difference here and we can move things forward, how open 

is the FDA to this possibility? And what can we do right now to 

make this happen? 

Dr. WOODCOCK. Well, as I said in my testimony in the beginning, 

medical culture has changed over the years. It used to be very doctor-centric and now it is patient-centric. And the FDA culture and 

drugs is a medical culture. And so that has changed at the same 

time, but slowly. 

So we have been working, though, very diligently with patient 

groups and so forth to try to get the patient point of view more central to the evaluation of benefit and risk and what it means to the 

person who actually has the disease, is going to take the drug. 

To answer your question what can be done, I think a lot of this 

needs to be done out in the community. The patient groups need 

to get organized and develop these. Some of them are working with 

PCORI and trying to use that mechanism to get more information 

available and so forth. 

We have gotten draft guidances from different groups, including 

Muscular Dystrophy, that really are a statement of, ‘‘This is what 

we care about. This is what we value. This is what we want you 

to look at.’’ And we will pay extremely close attention to those, and 

those are extremely valuable. 

Mrs. ELLMERS. Thank you, Dr. Woodcock. 

And I yield back the remainder of my time. 

Mr. PITTS. The chair thanks the gentlelady. 

50 

We are voting on the floor. We have 10 minutes left in the vote. 

We have three more questioners. 

Mr. Guthrie recognized for 5 minutes for questioning. 

Mr. GUTHRIE. Thank you, Mr. Chairman. I will try to be brief. 

I will echo what the others said about your testimony. Appreciate 

it. 

But since we started this 21st Century Cures and—everybody’s 

excited. Both sides are trying to see how we can do this better. 

And I have heard from a lot of groups and I have heard several 

times that the oncology division seems to be one people really like 

to work with and it works well. Some of the other divisions in expedition is not as well to work with. 

And I have always believed—Jack Kemp used to say, ‘‘Don’t 

study failures and point out the problems. Let’s look at successes 

and see how it can be replicated.’’ 

So within your own agency, you are having wonderfully successful programs, at least according to the feedback I have gotten, and 

some not as fun as the ability to work with. 

So I guess my question is: Is there any impediment to saying, 

‘‘Hey, this’’—the oncology is what we hear about more, not that the 

others aren’t, but we hear more—is there any impediment to taking what is happening there and transferring to other agencies? Is 

there something Congress can do to make it easier or is it just 

learning and moving forward? 

Dr. WOODCOCK. Let me tell you that 10 years ago, I heard a lot 

of negative comments about our oncology group. All right? And now 

we have therapies that are so effective. They are really on fire. 

They see that, for their patients they took care of—they are all 

oncologists, hem onc doctors—that these new treatments would 

really have made a difference for those people. And so they are 

doing everything they can to get those treatments out. 

And I think what we need, we need the same kind of inspiring 

therapies in these other areas. And I do think the doctors—they 

are doctors. They are physicians. They care about patients in their 

disease area. 

And this breakthrough—I don’t know whether you can see it 

here, but you see that other disease areas are coming up and we 

are designating—in neurology and anti-infectives and psychiatry, 

we are designating potential breakthroughs. And so this type of 

thing will really help. 

But, also, of course, we try to have a management structure, 

multiple mechanisms whereby we have consistency and uniformity 

of our approach and our procedures, and I think we do quite well 

in our procedures. 

But I think the attitude may have something to do with the underlying science. We had a war on cancer. It is starting to pay off. 

And we need to really expedite that. 

Mr. GUTHRIE. Well, thanks. 

And I have a bill particularly to put the same professional budget judgment status for Alzheimer’s, which we are going to spend in 

2050 $1 trillion. This is not loss of income, loss of productivity. 

Dr. WOODCOCK. Right. 

Mr. GUTHRIE. That is $1 trillion spent on that disease. 

51 

That is when I am 86. So that is when my children and our 

grandchildren will be taking care of us. So, hopefully, we can have 

the same inspiration and do that, particularly in Alzheimer’s. 

Dr. WOODCOCK. I can assure you that, if they were promising 

treatments for Alzheimer’s, we would jump right on them. 

Mr. GUTHRIE. Appreciate that. Thank you very much. 

And I will yield back. 

Mr. PITTS. Chair thanks the gentleman. 

Recognizes the gentlelady from Tennessee, Mrs. Blackburn, 5 

minutes for questions. 

Mrs. BLACKBURN. Thank you so much, Dr. Woodcock. I have basically one question that I do want to get to. 

Looking at the QIDP and the moving forward of that, it can give 

up to 5 years of additional data exclusivity. Bipartisan effort. We 

were all for it. 

What I want to know from you is: How many QIDPs has the 

FDA designated to date? How many products have actually been 

approved to date? And do you believe that the QIDP is an important designation? 

Dr. WOODCOCK. It is absolutely important. 

Mrs. BLACKBURN. OK. 

Dr. WOODCOCK. We have granted 50 designations for 34 unique 

molecules. And in the last several weeks, we have approved the 

first two medications that are designated, the first two 

antimicrobials. 

Mrs. BLACKBURN. Excellent. 

Dr. WOODCOCK. So that is making a difference. We do feel, 

though, that probably more needs to be done. 

Mrs. BLACKBURN. And in that ‘‘more needs to be done,’’ give me 

a couple of examples of what you think the next step should be. I 

would be interested in that. 

Dr. WOODCOCK. Well, we are very interested in the pathway that 

people call limited population antibacterial drugs or other streamlined pathways for development that would be matched with some 

sort of symbol or logo that would enable doctors and other prescribers to recognize that it was from a limited program. We think 

that would also allow us to streamline the development program. 

Mrs. BLACKBURN. Excellent. 

And for the second panel, I want to welcome a fellow 

Tennesseean, Dr. Marshall Summar, who is going to be speaking 

on behalf of the National Organization of Rare Diseases. 

So welcome. We are delighted you are here. 

And I would yield back. 

Mr. PITTS. Chair thanks the gentlelady. 

Now recognizes the gentleman from Florida, Mr. Bilirakis, 5 minutes for questions. 

Mr. BILIRAKIS. Thank you, Mr. Chairman. I appreciate it. 

And thank you for your testimony, Dr. Woodcock. 

I asked these questions a few months ago and I didn’t get a response. So I am going to see if I can get a response this time. Appreciate it if you can answer. 

Can you tell me how many treatments were approved with novel 

biomarkers used for the first time? 

Dr. WOODCOCK. No. I don’t have that in the—— 

52 

Mr. BILIRAKIS. Can you get that information to me as soon as 

possible? 

Dr. WOODCOCK. I would be happy to. It is a very interesting 

question. Yes. 

Mr. BILIRAKIS. And then next question: Have any accelerated approval occurred within novel biomarker in never-before-treated disease? 

Dr. WOODCOCK. Oh, yes. All the time. And I can get that for you. 

I don’t have it, again. 

Mr. BILIRAKIS. Please. 

How many new biomarkers did the FDA accept for a first-time 

use in the last 5 years? 

Dr. WOODCOCK. Certainly. 

Mr. BILIRAKIS. Can you get that for me? 

Dr. WOODCOCK. Absolutely. 

Mr. BILIRAKIS. OK. Very good. Thank you very much. 

I know we don’t have a lot of time; so, I will yield back. Thank 

you, Mr. Chairman. 

Mr. PITTS. Thank you. 

There is 2 minutes left in the vote on the floor; so, we are going 

to recess. There are two votes. As soon as we have the second vote, 

we will come back and reconvene with our second panel. 

Again, Dr. Woodcock, thank you for coming. You have been a terrific witness. 

Dr. WOODCOCK. Thank you. 

Mr. PITTS. Members will have follow-up questions. We will send 

them to you. We would ask that you please respond. 

Thank you. And thank you for your patience. 

The subcommittee stands in recess. 

[Recess.] 

Mr. PITTS. Time of recess having expired, we will reconvene the 

subcommittee on Health and introduce our second panel. 

In our second panel, we have five witnesses. I will introduce 

them in order of their presentation. First, Ms. Pat Furlong, Founding President and CEO of the Parent Project Muscular Dystrophy; 

second one, Mr. Robert Beall, President and CEO of Cystic Fibrosis 

Foundation; third, Mr. Richard Pops, Chairman and CEO of 

Alkermes; fourthly, Dr. Leonard Lichtenfeld, Deputy Chief Medical 

Officer of American Cancer Society; finally, Dr. Marshall Summar, 

Director of Scientific Advisory Committee, National Organization 

for Rare Disorders. 

Thank you all for coming. You will each be given 5 minutes to 

summarize your testimony. Your written testimony will be placed 

in the record. 

Ms. Furlong, we will start with you. You are recognized for 5 

minutes for your opening statement. 

53 

STATEMENTS OF PAT FURLONG, FOUNDING PRESIDENT AND 

CEO, PARENT PROJECT MUSCULAR DYSTROPHY; RICHARD 

F. POPS, CHAIRMAN AND CEO, ALKERMES; MARSHALL 

SUMMAR, M.D., DIRECTOR, SCIENTIFIC ADVISORY COMMITTEE, NATIONAL ORGANIZATION FOR RARE DISORDERS; 

ROBERT J. BEALL, PH.D., PRESIDENT AND CEO, CYSTIC FIBROSIS FOUNDATION; AND J. LEONARD LICHTENFELD, M.D., 

DEPUTY CHIEF MEDICAL OFFICER, AMERICAN CANCER SOCIETY 

STATEMENT OF PAT FURLONG 

Ms. FURLONG. Thank you. 

Good morning, Chairman Pitts and members of the committee. 

My name is Pat Furlong. Twenty years ago I joined other parents 

to form Parent Project Muscular Dystrophy to end Duchenne, one 

of the many forms of muscular dystrophy and the most common lethal genetic disorder diagnosed in childhood. 

In 1984, I received the horrific diagnosis on my two sons, Christopher and Patrick, and both of my sons are gone now. I wage this 

crusade in their honor. 

Much has happened over the past 15 years to transform the 

Duchenne clinical and research landscapes, and much of this is a 

direct result of the actions by Congress and this committee, notably 

the enactment of the Childs’ Health Act in 2000, and the Muscular 

Dystrophy CARE Act 1 year later. Since the MD CARE Act was enacted, we have seen about 10 years added to the lifespan of patients with Duchenne. 

There has been an improvement in quality of life driven largely 

by the development and dissemination of care standards so that all 

patients can be diagnosed accurately and as early as possible and 

provided with the highest quality of care. 

The MD CARE Act also transformed the Duchenne research 

landscape. What was just 12 years ago a near-barren field has 

evolved into a robust area of research where multiple potential 

therapies are in clinical testing and several others are in early 

stages of development. 

Despite these advancements, Duchenne remains a fatal disease 

without any FDA-approved therapies. Most boys end up in wheelchairs by their mid-teens, and only a few live beyond their late 20s. 

Our community needs therapies and we need them fast to. To 

achieve this goal, PPMD has led groundbreaking efforts over the 

past year to address two major impediments in our request to end 

Duchenne. 

One is a lack of regulatory understanding of patient and parent 

perspectives on benefit-risk; and, two, a lack of clear guidance or 

direction to the biopharmaceutical companies designing these clinical trials. 

PPMD partnered with Johns Hopkins University to conduct the 

first-ever scientific survey on benefit-risk perspectives. The survey 

involved 120 parents of Duchenne children. It validated what we 

have known anecdotally for years. 

Because Duchenne is 100 percent fatal at a young age, many patients and families are willing to accept higher levels of risk in return for the prospect of potential benefit. 

54 

The data has been shared with the FDA and was recently published in an academic journal. Now the FDA must ensure its reviewers apply this evidence to their decisionmaking process. 

Another impediment to drug development, particularly in rare 

diseases, is the absence of a clear guidance from FDA when it 

comes to designing clinical trials. Small patient populations, limited knowledge about the condition and a lack of accepted or validated biomarkers are some of these challenges. 

At the invitation of the FDA, PPMD led a comprehensive 6- 

month effort to convene key stakeholders—patients, parents, clinicians, researchers and industry—to write a draft guidance document that would address trial design and many other issues. This 

was submitted to the FDA last month, marking the first time a patient group has led the development of such a product. 

Now the FDA must step up promptly to review the draft, gather 

stakeholder input and issue a guidance document under the Agency’s name. 

While each of these projects is focused on Duchenne, each also 

offers a template or a model that could be applied to other diseases 

or other conditions, particularly rare diseases, and I hope other organizations will take on similar programs. 

So what can Congress and Federal agencies do moving ahead? 

First, you can make sure that the patient perspective on benefitrisk and other issues is considered by reviewers of the FDA. 

One way to do so could be by establishing a nonburdensome step 

where reviewers would disclose how they did or did not take such 

information into account making their decisions on a drug application. This would shed light on for what many considered a mysterious process and could be done in a very simple manner. 

Second, I suggest an even greater focus on regulatory science so 

the FDA keeps pace with the breakneck speed of innovation. Specifically, NIH could bolster support for regulatory science research 

and infuse that into clinical and translational awards. Incorporating a regulatory perspective earlier in the pipeline can maximize the likelihood that candidate therapies will be ready for the 

rigor of the FDA. 

Finally, I would encourage greater flexibility in clinical trials, 

particularly rare fatal conditions like Duchenne that have small 

populations. Business-as-usual trial designs simply do not hit the 

mark when working with these populations. 

The Duchenne community has traveled a great distance over the 

past 15 years, thanks in significant part to the leadership of this 

very committee, leadership that will continue on Monday with action by the full committee on the MD CARE Act amendments. 

For far too many families, my own included, this journey has not 

been fast enough. We stand ready to work with your committee to 

make sure the 21st Century Cures Initiative ends Duchenne and 

so many other rare diseases. 

Thank you. 

Mr. PITTS. Chair thanks the gentlelady. 

[The statement of Ms. Furlong follows:] 

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Mr. PITTS. Mr. Beall, you are recognized for 5 minutes for your 

opening statement. 

STATEMENT OF ROBERT J. BEALL, PH.D. 

Mr. BEALL. Thank you very much for this invitation to present 

this testimony. 

The story of cystic fibrosis is clearly a story of determination of 

hope and optimism. The progress that we have documented in our 

submission really shows what is possible when a system works 

well, when patients, when stakeholders and the regulatory agencies collaborate to develop life-changing treatments. 

Cystic fibrosis is clearly a life-threatening genetic disease that affects about 30,000 individuals in the United States. There has been 

tremendous progress in life expectancy over the decades. 

In the 1950s, people with cystic fibrosis barely lived to elementary school. But there are people that are living today with cystic 

fibrosis in their 30s and 40s, and some are even going beyond. 

But we still lose too many patients at very young ages. The increase in life expectancy is due in large part to groundbreaking advancements and treatments made possible because of the Cystic Fibrosis Foundation, our patient community and our industry collaborators. 

Two years ago the FDA approved Kalydeco, the first drug to 

treat the underlying causes of cystic fibrosis in a small subset of 

people with the disease. Hailed as a game-changer, it has transformed the lives of those taking this drug. 

It is a perfect example of personalized medicine. I might mention 

that the FDA approved this drug in near record time, 3 months before the prescribed PDUFA date and months before the EMEA. 

Just 2 weeks ago we saw another breakthrough in cystic fibrosis. 

It happened when—the positive data from a phase 3 clinical trial 

for a new therapy that is targeted at 40 percent of the CF population. 

This data was released by Vertex Pharmaceuticals Company. 

These products would not have been possible without the breakthroughs that have taken place in basic research, in all the efforts 

that our foundation has made over the years. 

The CF community was thrilled to learn that the trial participants showed a significant improvement in lung function, weight 

gain, and 30 to 40 percent reduction in exacerbations. That is the 

time that they would have to go to the hospitals or have IV infections. 

So this is clearly a game-changer for these patients. Obviously, 

Vertex plans to submit the new drug application to the FDA by the 

end of the year for this treatment. 

What is exciting about this progress is that these drugs would 

not have been possible were it not for the Foundation and our patient community. Our commitment to scientific discovery and drug 

development is at the root of our success, but it hasn’t been easy 

and it hasn’t occurred overnight. 

In 1965, we created the first patient registry in the United 

States, now a model for chronic disease. Because of this registry, 

we have a documented natural history of cystic fibrosis. 

59 

We have the mutation analysis on most of these patients, as Dr. 

Woodcock referred to this morning, and we have the ability to have 

post-marketing phase 4 follow-up on these new drugs as they are 

introduced to the community. 

The same year, 1965, we created a care center network. 90 percent of all patients seen in the United States are seen at these 

CFF-accredited and funded care centers. 

In 1989, through our support, the CF gene was discovered, 12 

years before the human genome was completed. 

In 1998, we established a Clinical Trials Network, the first Clinical Trials Network founded solely by a nonprofit organization like 

the Foundation. It is a critical component of our ability to conduct 

CF clinical trials efficiently and effectively. 

In 1999, the CF Foundation pioneered a successful venture philanthropy model to derisk companies from investing in CF research 

drug development. 

It was our initial investment of $42 million in a small biotech 

company in San Diego that ultimately led to Kalydeco. Vertex 

would not have had Kalydeco and the other drugs announced last 

week were it not for the Cystic Fibrosis Foundation. 

The CF Foundation spearheads collaboration across all sectors, 

and this same collaborative spirit extends to the Foundation’s 

strong partnership with the Food and Drug Administration. 

With the FDA, we are committed to collaboration and bringing 

strong data to the table. As often has been stated, the CF Foundation comes with data, not demands. 

Just last week we met with FDA officials to discuss strategies for 

clinical research design that may not occur until 5 years from now. 

However, curing a disease is never easy, and even more risky is 

the approval of drugs without sufficient data to assure efficacy and 

safety. 

If this happens, you place patients immediately at risk and you 

risk losing the opportunity to test drugs that could have a real impact and beneficial effect. 

So, in closing, what can Congress do for us? Congress should 

make sure that patients have a seat at the table, as was just referred to. 

Congress must provide the necessary resources so that the FDA 

can attract the best and the brightest. And Congress must provide 

the NIH and FDA sufficient resources for regulatory sciences, as 

also mentioned. 

But, finally, Congress may also encourage that they look at the 

CTSA program, a network of care centers that are funded by the 

NIH, and see how they might use these to be able to facilitate Clinical Trial Network and the development of patient registries in 

other rare diseases. 

So, once again, thank you for this opportunity to add the CF 

community’s perspective to this important discussion. 

Mr. PITTS. Chair thanks the gentleman. 

[The statement of Mr. Beall follows:] 

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Mr. PITTS. Mr. Pops is recognized 5 minutes for an opening statement. 

STATEMENT OF RICHARD F. POPS 

Mr. POPS. Thank you very much. 

I would like to thank you, Mr. Chairman, Ranking Member 

Pallone and all the members of the committee for inviting me to 

testify. 

I just want to thank Chairman Upton and Congressman DeGette 

for spearheading the 21st Century Cures Initiative. 

I would also like to express my respect for and appreciation for 

the folks on this panel and for Dr. Woodcock. We are all partners 

in this together, and it is an credibly important mission. 

The simple and powerful concept of incorporating insights from 

patients is centrally important to the future of the Nation’s 

healthcare system. And it is also one of the great opportunities for 

us all to have a transformative impact. 

I have served as the CEO of Alkermes for over 20 years. Our 

company develops medicines for people living with chronic debilitating diseases, such as opioid addiction, schizophrenia, and depression. Our approach is entirely dependent upon considering the 

patient perspective early and consistently throughout the drug development process. 

I also serve on the Boards of both BIO and PhRMA and was 

deeply involved in the PDUFA V negotiations, as well as the preparations ongoing for PDUFA VI, where elevating the patient voice 

has already emerged as a key theme for that initiative. 

Today I would like to propose a new framework for patient involvement in developing new medicines, which requires engagement from all three of the major parties involved, innovative biopharmaceutical companies like ours, FDA, and the patients who 

stand to benefit from these medicines. 

And the framework is based on three core principles. 

First is that interactions must be data-driven, based on science 

and separate from powerful and passionate advocacy messages that 

patient groups otherwise deliver. 

Second, the engagement framework should be actionable, not 

theoretical. It should improve the overall efficiency of the process 

rather than adding new steps in a process that is already incredibly complicated. This is particularly important for young biotechnology companies who are developing their first drugs on limited resources. 

Third, the approach should preserve and enhance FDA’s gold 

standard of safety and efficacy, which is really one of our great national treasures. I believe deeply, personally, that increased patient 

input can coexist with efficiency and the highest level of scientific 

rigor. 

So from industry’s perspective, there is clearly no consistent way 

to incorporate patient-generated input. This input would have a 

really meaningful impact on a range of critical decisions we and 

our researchers make specific to particular product candidates and 

certainly to the way we design clinical trials and implement them 

around the world. This is an important missing link. 

69 

As Dr. Woodcock mentioned and the FDA, patient engagement is 

not a new concept. Several provisions included in PDUFA as well 

as FDASIA have resulted in meaningful new expansions in patient 

engagement. 

FDA has also been open to and has taken initial steps to include 

patient input into their reviews, and we can build on this. The proposed framework I am considering would build on all of these 

things. 

The historical paradigm of drug regulation as a bilateral process 

between FDA and the industry is outdated. Science and society 

have continued to advance. Patients are organizing in new ways, 

and their critical role in driving innovation is becoming more the 

rule than the exception. We have 20th-century regulatory framework for 21st-century drug development. 

To tackle these increasingly complex scientific and regulatory 

issues as we look to treat and cure complex diseases, all three parties can work together to develop improvements to their existing 

regulatory framework. 

These would include new clinical trial designs, more efficient 

clinical trial enrollment methods, advancing FDA’s evaluation of 

risk and benefit, and more sophisticated post-market data collection. These are incredibly exciting areas for future consideration. 

We would need to evolve the way we work together, all the different parties, recognizing our shared responsibility to improve the 

efficiency of the development process and our accountability to assure the medicines are safe and efficacious for patients. 

There will be a number of challenges to this as we move in, and 

these could include establishing a common threshold for data and 

scientific rigor that is shared by patient industry groups and FDA, 

modifying existing regulations to accommodate this new framework, protecting intellectual property and data, which is essential 

to enabling innovation and maintaining this gold standard of safety 

and efficacy. 

As next steps, I propose that Congress, industry, FDA and patient groups come together to develop and implement this new 

framework, building on existing patient-focused provisions of 

PDUFA and FDASIA. We should also analyze existing statues and 

regulations to identify impediments and opportunities. 

In conclusion, the concept of a new and comprehensive patientinclusive framework is both ambitious and, at the same time, it is 

quite modest. 

It is ambitious as it could result in a dramatic change in the way 

we discover and develop medicines. It is modest because it is not 

a new regulatory pathway or authority, but it builds on an existing 

foundation. 

And we at Alkermes and all of our colleagues in the biopharmaceutical industry are standing by to help you in that effort. We 

really thank you very much for your leadership. 

Mr. PITTS. Chair thanks the gentleman. 

[The statement of Mr. Pops follows:] 

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Mr. PITTS. Dr. Lichtenfeld, you are recognized for 5 minutes for 

your opening statement. 

STATEMENT OF J. LEONARD LICHTENFELD, M.D. 

Dr, LICHTENFELD. Good morning, Chairman Pitts, Ranking Member Pallone, and members of the subcommittee. 

I am Dr. Len Lichtenfeld, and I am Deputy Chief Medical Officer 

for the American Cancer Society and truly appreciate the opportunity to be with you today to testify. The American Cancer Society 

is pleased to contribute to the dialogue around the committee’s 21st 

Century Cures Initiative. 

Today I would like to focus on three critical areas for the committee’s consideration. One is the need for greater investment in research; secondly, expedited approval processes that continue to ensure safety and efficacy of approved drugs; and, third, making patients active partners in all aspects of research development and 

regulation of new therapies. 

We are fortunate and blessed that today we have 14 million cancer survivors in the United States. It is a remarkable number, and 

it is due to more effective treatments and improved screening tools 

that have been made possible through research. 

We must continue and expand our steadfast commitment to research, and we must continue to support researchers working on 

finding the next generation of cures. 

Just as important, we must ensure that expedited approval processes for drugs and devices are appropriately safe, effective and accessible to patients. The goal of the 21st Century Cures Initiative 

is to accelerate the development and approval of new medical treatments. 

There are a few other areas that can match the research and development activity in the field of cancer. It is, in fact, and has been 

a model of innovation. 

The FDA’s Office of Hematology Oncology Products has aggressively used the tools provided by Congress to speed new drugs to 

patients and has encouraged drug companies to be innovative in 

clinical trials. 

In the past 8 months, three cancer drugs have been approved 

using the accelerated pathway. One approval was based on a trial 

of 111 patients, an example of research approvals happening faster 

and with smaller clinical trials as has been the case in the past. 

Small-sized trials and accelerated approval do have drawbacks. 

They may not include a diverse population, which may yield an incomplete picture of how a drug might work in a broader population. 

Small trials and accelerated approvals also tend to be seen in deadlier cancers where there are no other good therapeutic options. 

And I want to stress that the risk-benefit tolerance of a cancer 

patient facing a poor prognosis may be much different than for 

those with other available treatment options. 

And, therefore, the same acceptance of reduced data on which to 

base FDA approval may not be appropriate in other fields or for 

other diseases. 

Finally, I want to stress the importance of researchers, pharmaceutical companies and the FDA in engaging widely and meaningfully with patients. 

78 

The Food and Drug Administration Safety and Innovation Act requires greater patient involvement throughout the drug and device 

approval process. ACS CAN championed provisions to expand the 

FDA’s patient representative program to maximize patient input 

during the drug development process. 

We need to continue to build on that progress. Patients can provide important perspectives at various stages of medical product 

development and regulation. 

They know more than anyone what is most important to patients, to themselves: Symptom reduction, risk tolerance and design elements that might affect trial recruitment or retention. 

This kind of patient involvement should be reinforced and supported and, to this end, the FDASIA provisions requiring FDA to 

address challenges that have hindered patient involvement must be 

fully implemented. 

We urge the committee to consider examining opportunities for 

providing greater funding to support the FDA patient representative program as well as broader continued engagement with the patient community. 

Another important way patients’ perspectives can inform development of therapies is through the design and use of patient-reported outcomes. 

Measures of cancer therapy effectiveness sometimes include functional status, pain or quality-of-life measures, but these may be reported by the physician rather than by the patient. 

Quality-of-life measures like pain or nausea should come from 

patients themselves, and patients should help prioritize the importance of these side effects in the overall response to a disease and 

the associated treatments. 

When quality-of-life outcomes are vigorously measured and supported by the FDA, they should be included in a drug’s labeling 

and they should be considered for a drug’s approval. 

The FDA should also be encouraged to work with industry and 

researchers to incorporate self-reported symptom measurements as 

a regular part of clinical trials. 

In closing, we appreciate the opportunity to contribute to the dialogue around the committee’s 21st Century Cures Initiative and 

look forward to working with the subcommittee and its staff. I am 

happy to take any questions. 

Thank you very much for this opportunity. 

Mr. PITTS. Chair thanks the gentleman. 

[The statement of Dr. Lichtenfeld follows:] 

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Mr. PITTS. Dr. Summar, you are recognized for 5 minutes for 

your opening statement. 

STATEMENT OF MARSHALL SUMMAR, M.D. 

Dr. SUMMAR. Thank you, sir. 

Good morning, Chairman Pitts, Ranking Member Pallone and 

members of the subcommittee. And thank you for inviting me 

today. 

My name is Marshall Summar. I have the good fortune to be the 

Chief of Genetics and Metabolism at Children’s National Medical 

Center here in Washington, D.C. 

I have been working in the field of rare diseases for the last 29 

years, and I am here today in my capacity as a member of the 

Board of Directors of the National Organization of Rare Disease 

and Chair of the Scientific Advisory Committee of NORD. 

On behalf of the estimated 30 million individuals with rare diseases, NORD thanks you in the Energy and Commerce Committee 

for your continued strong support of the rare disease community. 

You have made a huge difference for us. 

NORD’s a unique federation of over 200 patient advocacy groups, 

clinicians, researchers, dedicated to helping people with rare diseases. 

NORD provides resources, research advocacy, education, community and infrastructure support to the rare-disease community that 

small individual organizations cannot. It is the nature of rare diseases. They are small. 

NORD was founded in 1983 and played an active role in the passage of the Orphan Drug Act, which is a successful model of how 

to incentivize the development of treatment that saves lives. 

Data show that years of life lost to rare diseases declined at an 

annual rate of 3.3 percent after the Orphan Drug Act due to the 

development of new treatments. 

Without these new drugs, if you take them out of the equation, 

the number of years of lives lost should have increased at about a 

1 percent rate per year. So it has made a real impact on our patients. 

Speaking personally, without these treatments, many of my patients would not be here. I thank you for what you have already 

done. 

These efforts represent a good beginning, but there is much more 

we can do to improve the lives of our patients, and NORD views 

the 21st Century Cures Initiative as a great way to do this. 

NORD’s long advocated increased involvement of patients in the 

drug development process. We appreciate the commitment by many 

at the FDA to increase patient involvement, but believe much more 

needs to be done to make patients feel they are partners in the 

process. NORD will continue to work with the FDA to advance the 

patient role in the development and approval process. 

We have developed a series of recommendations that we believe 

will advance not only the development of new orphan drugs and devices, but non-orphan ones as well. We look forward to discussing 

these ideas with the committee as the 21st Century Cures Initiative continues. 

Permit me to focus on two of our recommendations. 

86 

First, we support the establishment of a commission and national 

plan to determine priorities, methods, resource needs and a consistent agenda on rare-disease registries and natural history studies. 

They have got a lot of variation. They tend to be all over the 

map. To assess the drug’s efficacy, we need the information on the 

existence, frequency, and severity of clinical findings. This information is needed before a clinical trial can begin. 

We encourage the creation and maintenance of programs to create, curate, and standardize registries and national history studies 

which can generate this needed data. 

This could be one of the most important accelerators of the treatment development and monitoring process. These registries can 

also be used in the post-approval process as well. 

This is an area where patients can have a major and cost-saving 

impact on the process. Patient-entered data has been shown to be 

accurate and useful when collected properly. 

Creative hybrids using physician-, patient- and other health professional-collected data can greatly speed the understanding, discovery, approval and monitoring process. 

In collaboration with the NIH and FDA, NORD has built and is 

in the process of testing a rare-disease patient-driven registry national history program. The NIH’s Rare Disease Clinical Research 

Network has already demonstrated the benefits of this approach. 

In a registry I have been involved with, we have had approval 

of three drugs over a 10-year period with only 700 patients. So it 

definitely has accelerated the approval process for us. 

The Patient Centered Outcomes Research Institute is developing 

these statistical methods and models to use data from rare-disease 

patient studies that will further refine this process. 

They are also involved in patient-driven registries through 

PCORnet and will begin working with NORD on our rare diseasefocused registry program. So we should have good input from multiple agencies. 

All of these efforts will help our patients, but a national plan and 

standards would help prevent duplicated effort and resources. This 

is what we truly need. 

The other thing we advocate is significant reform to the Institutional Review Board system. I have been working with this system 

for the last 30 years; so, I am pretty familiar with all of its manifestations. 

Currently, all clinical trials for new treatments, whether a drug, 

biologic or medical device, must receive approval from an IRB. 

Each institution and study site typically requires approval and 

protocol adjustment by its own IRB. With a large number of sites 

needed for rare-disease study, this is one of the greatest impediments and cost to clinical trials. 

NORD recommends that Congress develop legislation that would 

de-risk the process and foster the creation of an IRB system that 

is portable across institutions. 

The de-risking of the IRB process and the encouragement requirement of reliance agreements between institutions receiving 

Federal funding would save cost and time while accelerating the 

clinical trials and clinical research process greatly. This will signifiVerDate Nov 24 2008 11:05 Feb 05, 2015 Jkt 037690 PO 00000 Frm 00092 Fmt 6633 Sfmt 6633 F:\MY DOCS\HEARINGS 113\113-158 CHRIS
87 

cantly increase the pool of study sites and allow greater patient 

participation. 

These are just two of our recommendations. My written testimony includes the rest. 

And I on behalf of NORD, I thank the committee for allowing us 

to testify today. 

Mr. PITTS. Chair thanks the gentleman. 

[The statement of Dr. Summar follows:] 

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Mr. PITTS. Thanks to all the witnesses for your opening statements. And we will now begin questioning. 

I will recognize myself 5 minutes for that purpose. 

Ms. Furlong, we will start with you. 

Do you believe your guidance collaboration with industry is a 

scaleable model that can be used in other conditions, specifically, 

where there are unique factors that make Duchenne muscular dystrophy guidance a special case in the multi-stakeholder effort that 

you led with encouragement from the FDA? 

Ms. FURLONG. Thank you, Chairman Pitts, for the question. 

I certainly think that this methodology and process is exportable 

to other rare conditions. How we started the guidance or initiated 

the guidance was to develop a steering committee that was representative of the stakeholders, which included patients, academia, 

as well as industry. 

From there, the steering committee identified several areas, 

seven working groups, actually, of things that they felt were relevant, to include diagnosis, biomarkers, clinical trial design, natural history, and benefit-risk. 

And then we further developed a CAB, which is the Community 

Advisory Board, so that would be—incorporate the entire patient 

voice and any individual or patient group that wanted to contribute 

to the development of the guidance. 

The standardization for the guidance was that it would be a reference document and that it would include documented evidence 

that was published or accepted for publication by the end of July. 

So we felt—and we are writing up the methodology—that this 

methodology is exportable. It was certainly an investigation and a 

thorough, thoughtful, reasoned look at the community and the nuances of Duchenne. 

But I believe that most rare diseases could do the same. Their 

issues may be slightly different and their progress to date might 

be slightly different, but it is certainly exportable. 

Mr. PITTS. Thank you. 

Dr. Beall, communication with patients to make sure they can 

make informed decisions about clinical trial participation is critical. 

How does the cystic fibrosis community communicate with patients about the various options? And how do you think we can best 

translate your good practices into the Cures Initiative? 

Mr. BEALL. Thank you very much. 

First of all, because 90 percent of all of our CF patients are seen 

in a network of care centers and that we also have a Clinical Trials 

Network, there is a very close relationship between our physicians 

and the patients that are involved. 

And that is critical for the recruitment of patients in the clinical 

trials. It is critically important for showing them the value and the 

risk of participating in clinical trials. 

And it is that close association between the physician and the patient and the recruitment process in a very closed network that is 

critical. That is why I think Clinical Trials Networks are critically 

important. 

So we also have established within our Clinical Trials Network 

a data safety monitoring board independent of the Cystic Fibrosis 

Foundation, but it is made up of experts. 

98 

And that provides a degree of assurance to every single patient 

that there is somebody looking out for their continuing interest and 

for any risk that may be inherent in any single trial. 

So I think all of these things, plus we have worked very hard to 

try to create a culture of participation and a responsibility that 

each patient, when you have a small patient population, needs to 

participate in the process. So I think it is that reassurance that is 

so important. 

Mr. PITTS. Thank you. 

Mr. Pops, what stage of drug development could most use the assistance of patient insight about benefit expectations and risk tolerance? 

Mr. POPS. Thank you for the question. 

It is actually the most exciting part of the whole opportunity, 

that it is every stage, actually from identification of new drug candidates, all the way through to determination of the value of the 

medicine after the completion of the pivotal clinical trials. 

And I think that is the whole idea of this framework, is creating 

a structure where we can get that input on a continuous basis, and 

I think it could fundamentally transform the way we approach 

these development programs. 

Mr. PITTS. Thank you. 

Dr. Lichtenfeld, you have discussed examples of cancer drugs 

that have recently been successfully approved by FDA through an 

accelerated approval process. 

Are there best practices that we can learn from cancer and how 

FDA is expediting the approval process for particular drugs? 

Dr. LICHTENFELD. Thank you, Mr. Chairman. 

When we talk about best practices, I think the question really 

came up with Dr. Woodcock earlier today: What is the oncology 

community doing that is different than other communities? 

Let’s understand it is a complex process in the sense that we 

have research that has been building literally for decades that has 

produced very exciting results that is actionable and companies are 

standing up to create drugs for the targets that we are finding for 

the new immunotherapies for genetic disease, what have you, genetic markers. So we are, in a sense, at an interesting and turningpoint kind of place. 

But important, relevant to your question, the Office of Hematology Oncology Products has also stepped up to the plate. And as 

was mentioned earlier, the oncology community appreciates the efforts of the FDA staff to reach out to the patient community, to 

reach out to the pharmaceutical community, to reach out to those 

who do clinical trials, to be active participants, to be at the table. 

Lung-MAP was cited several times. The American Cancer Society 

was grateful to be able to have contributed to that effort, among 

many other organizations. 

But the FDA has become an active partner with the process. And 

so I don’t know if that is a best practice or a best example. But 

it is that source of communication. 

But let’s not forget it is also the opportunity because we are now 

in a place that we only dreamed of just a short while ago. 

Mr. PITTS. The chair thanks the gentleman. 

My time has expired. 

99 

The chair recognizes the ranking member, Mr. Pallone, 5 minutes for questions. 

Mr. PALLONE. Thank you, Mr. Chairman. 

My questions are of Mr. Beall. 

The Cystic Fibrosis Foundation has done some great collaborative work that has resulted not only in successful marketing of 

Kalydeco, but also the recent positive test results of a complementary drug that may extend treatment to nearly half of all patients 

with CF. And, of course, I commend you for your efforts. 

But I wanted to ask you about a couple of points in your testimony. In your remarks, you spoke about the CF Foundation’s 

strong relationship with the FDA and the importance of bringing 

good data to the table when consulting with the FDA, which I 

know is true. And I would like to hear more about that relationship. 

Obviously, we are hearing a lot today about the need for FDA to 

do more to seek and incorporate patient input into its review process. 

So the basic question, Mr. Beall, is: Can you tell us more about 

the CF Foundation’s interaction with the FDA? And are there any 

lessons that can be learned by other disease groups? 

Mr. BEALL. Well, I can give you a perfect example because, on 

Wednesday of this week, we had three officials, including Dr. Robert Temple, who is in the drug division at our offices, talking about 

the development of clinical trial protocols of drugs that might not 

enter into clinical trials until 3 to 5 years from now. 

So that is a perfect example of this open discussion. Because we 

have a natural history of the disease. We know that the drugs that 

we have tested are treating the basic defect. We know the mechanism of action. We have a safety profile. 

And now we can start to talk about the future. And I think it 

is that kind of example. And that goes back many, many years. 

Soon after we discovered the CF gene, we talked about gene therapy and we had extensive dialogues with the FDA, not only with 

manufacturers, but with the FDA and the Foundation. 

So I could just say that we have always had a wonderful collaboration. We have data. We have natural history of the disease because of the patient registry. 

And, again, we come with data and we come with experience and 

we come with the networks that can make these things happen. 

So I just gave you an example. That was the example. 

Mr. PALLONE. No. That is fine. 

We are hearing a lot about the various expedited drug review 

processes at FDA, and it is clearly a push by many to get the Agency to use these pathways more frequently and in more disease 

areas. And I share the goal of speeding the therapies to patient at 

the earliest possible time. 

But I think we need to be cognizant of the risks that could accompany that speed, and we especially need to be concerned about 

such risks if we are ultimately thinking about somehow requiring 

more frequent use of these expedited pathways through legislation. 

And I know you share that concern. 

Your testimony mentions the health risk that could result from 

approving therapies based on early data that needs more vigorous 

100 

study, but you also describe the possibility that these kinds of approvals could endanger progress toward the development of other 

treatments. 

Can you just elaborate on both of those concerns, if you would. 

Mr. BEALL. Well, certainly, one of the things when you are dealing with a small population—and now we are talking about personalized medicine where you may only have 25 patients with a certain genotype that may be approachable or therapeutic opportunities for that particular drug—if those patients were introduced to 

a drug that was less than effective, what happens when the next 

drug that could be effective—how do you do the clinical trial? 

So I think that is really very critical because we want to make 

sure that our first introduction is drugs that are efficacious, and 

then we move forward to the next level. Because then you really 

are depriving, if you don’t have safe drugs, of developing good 

drugs and effective drugs that could move us above the therapeutic 

options that we have. 

So I think that that is the critical thing that we always face. 

There is always the risk. But now we are dealing with small populations, personalized medicine. Maybe there is only going to be 6, 

10, 1,000 patients. 

So I think you have to be particularly critical on that issue with 

rare diseases. 

Mr. PALLONE. OK. Yes. I just wanted to echo another point you 

made in your testimony about the importance of resources. 

And I couldn’t agree more, that, as you say, FDA needs resources 

to ensure that they can rely on the best regulatory science available and they need adequate resources to enable them basically to 

meaningfully engage with the patient community. 

And we have this 21st Century Cures Initiative, which is progressing now. We have had some sort of larger meetings and now 

some hearings. And my colleagues always ask what can Congress 

do. 

And I think that the most effective thing we can do is provide 

adequate resources to make sure that FDA, as well as NIH, have 

the resources to fulfill the expectations we have for both agencies. 

And I hear not only from the agencies, but, also, from my constituents, that they don’t have enough resources. So I just wanted 

to echo again what you said. 

Thank you, Mr. Chairman. 

Mr. PITTS. The chair thanks the gentleman. 

I now recognize the vice chairman of the subcommittee, Dr. Burgess, 5 minutes for questions. 

Mr. BURGESS. Just before my time starts to run, could I make 

a unanimous consent request? 

Mr. PITTS. You may proceed. 

Mr. BURGESS. I would like to move that the committee make people aware that, if someone wishes to contact or communicate with 

the Cures Initiative, it is cures@mail.house.gov. 

I know there are many people watching who think, ‘‘I would like 

to interact with the committee staff.’’ So that is the way to do it. 

Mr. PITTS. Thank you. 

Without objection, so ordered. 

Mr. BURGESS. Very well. I knew they wouldn’t deny me. 

101 

Let me ask Ms. Furlong. You were kind enough to mention the 

work on the MD CARE Act, and thank you for that. As you know, 

we will likely be marking that up next week. So that is a big milestone. 

Can you talk about how the MD CARE Act needs updating and 

the type of updating that this committee has pursued? 

Ms. FURLONG. Certainly. And thank you for the question, Dr. 

Burgess. 

The MD CARE Act was the solid foundation that set Duchenne 

and the muscular dystrophies—really galvanized their progress. 

So the MD CARE Act was enacted in 2001 and reauthorized in 

2008. And right now the amendments are really to look at what we 

have learned in the meantime. 

So the cardiac issues in Duchenne muscular dystrophy are real 

and they have to be tackled in order to answer the question. As you 

look at these potential therapies that were hopeful to be approved 

in the next months and years, they extend function. Will they protect or have a negative effect on the heart? 

So it is the gaps that we need to really look at with the amendments, in addition to the fact that, when this legislation was enacted in 2001, young boys with Duchenne didn’t live to be adults. 

So now we have an adult population and we need to really address those adults in terms of their medical care and, also, to 

incentivize and understand how to treat them, how to encourage 

them to have long and independent lives as they become adults and 

reach for their dreams. 

So I think that the MD CARE Act is now looking with the muscular dystrophy committee from the NIH and other agencies. Their 

research plan has to be updated and these amendments to be incorporated so that we are really achieving the full effect that the MD 

CARE Act was initiated for. 

Mr. BURGESS. Thank you. 

And of course, Mr. Beall will also acknowledge that the population of patients is changing because of some of the successes that 

have happened over the past several years. 

And in both of those illnesses, both cystic fibrosis and muscular 

dystrophy, it is important that we keep pace with the way the patient population is changing. 

We want people to live longer and fuller lives with their conditions and, at the same time, we don’t want the legislation then to 

stymie that. So it is, in my opinion, an important step forward. 

Dr. Beall, we talked—or you talked about the development of 

mutation-specific therapies and the next evolution in precision 

medicine and you could see the cystic fibrosis example impacting 

the way we address other serious illnesses. 

Is there something more you would like to add to that? 

Mr. BEALL. Well, again, we are clearly in the age of personalized 

medicine. Fortunately, with the completion of the human genome, 

we understand the genetics of so many more diseases and genetic 

diseases that it is a very critical time for us. 

Mr. PITTS. Microphone. 

Mr. BEALL. Not on? OK. 

I just saw Dr. Collins downstairs, and he is excited because he 

was one of the discoverers of our CF gene. 

102 

So we live in a very unique age, and I think more and more 

therapies are going to be directed towards specific mutations. 

And that is one of the reasons that we have to have these kinds 

of patient registries, so we can start to identify those mutations. 

When Vertex felt that they had a drug that might work on a certain mutation, the small drug that came out, G551D, we were willing—we were able to tell them in the United States we have 1,100 

of those patients within 5 minutes after they asked us because of 

a patient registry, because we have a documented history of the 

disease. 

So I think that is why it is very important to have personalized 

medicine, therapies and the options for that, but it is also—we 

have to be able to document the patients that can participate in the 

trials. 

Mr. BURGESS. Yes. It is very powerful. 

And, of course, you referenced to the 1965 registry. In 1965, you 

didn’t know that we were going to know about the sequence of the 

human genome 30 years later. 

Mr. BEALL. Well, but we have been able to document it. Today 

we have 26,000 patients whose data is provided to our patient registry every single year. 

Mr. BURGESS. Let me ask you a question. I am going to run out 

of time pretty quickly. But—and this is either for Mr. Beall or Mr. 

Pops. 

The world is different now and you have people that are perhaps 

lucky enough to enter into a clinical trial and they are likely to perhaps have friends with the same condition. 

So in the old days, a randomized clinical trial, you wouldn’t know 

which arm to which you were randomized, who was getting the target or study drug, who was getting either an older therapy or no 

therapy. But now people communicate. Facebook. Twitter. They are 

likely to be Facebook friends. 

How is that going to impact the ability to have a blind and randomized clinical trial? Are people likely to communicate with each 

other, I mean, look, ‘‘I am getting a lot better on this stuff. How 

about you?’’ ‘‘Wait a minute. I haven’t seen a darn thing’’? 

Mr. POPS. I think it is a real question. It is a real issue because—and you can’t pretend that it is not going to happen. 

This is already happening, particularly as you get large cohorts 

of patients in randomized studies in multiple countries. They are 

all communicating. 

So I think it is very important that we be really rigorous in 

maintaining the blind to the extent that we can. 

Mr. BURGESS. To the extent that we can. But, also, we probably 

need to embrace the fact that the information is out there and 

being communicated and, to the extent that it can further enhance 

what we are doing—— 

Mr. POPS. So let’s take advantage of it. 

Mr. BURGESS. Yes. 

Mr. POPS. Let’s do more in the aftermarket. Let’s approve drugs 

and collect this information and get a more nuanced view of the 

drugs’ use in the real world and turn it to our advantage. 

Mr. BEALL. And, in some cases, it is going to make it easier to 

do clinical trials when you can have large networks that exist out 

103 

there, when they can report patient-reported outcomes and things 

like that. 

So I think sometimes it is looked at as a disadvantage, but we 

ought to turn it—as Mr. Pops just said, we ought to turn it to an 

advantage because I think it can expedite the ability to do clinical 

trials as we move forward with the technology. 

Mr. BURGESS. Great. Thank you. 

Ms. FURLONG. And it should expedite post-hoc analysis so that 

we can see the long-term effects. Because in a clinical trial of 12 

months, for instance, plus or minus, you might not see the full effect of a drug that is multisystemic. So it will enable us to understand the full impact on the patients’ lives. 

Mr. BURGESS. Thank you, Mr. Chairman. I yield back. 

Mr. PITTS. The chair thanks the gentleman. 

Now recognize the gentlelady from Illinois, Ms. Schakowsky, 5 

minutes for questions. 

Ms. SCHAKOWSKY. Thank you very much. 

I had a question for Dr. Lichtenfeld—actually, for anyone on the 

panel that wants to comment on this. 

This is about quality-of-life outcomes. I mean, obviously, if this 

is a known life-threatening disease, you want to do everything you 

can to make sure that the therapies match the disease. 

But there are—you would say, when these quality-of-life outcomes are rigorously measured and supported by the FDA, they 

can and should be included in drugs’ labeling and can by themselves be a basis for a drug’s approval. 

I certainly know people who have suffered so much from side effects of drugs. And I just wonder, in the whole process of drug approvals, how much are these quality-of-life issues really looked at? 

As a basis for approval or just as a basis of whether or not they 

are used? 

Dr. LICHTENFELD. Thank you for your question. 

In fact, it is a work in progress. Let’s understand that quality of 

life is a buzzword today, but it wasn’t a buzzword very recently. 

So as we look at the issues, shall we say, of palliative care, of 

supportive care, quality of life, issues that the American Cancer Society and many others have been involved in, it is relatively new 

to the table. 

Having said that, there have been issues recently with one particular drug where, had the question really centered around was 

the—even though the drug may not have met the FDA standard— 

and this was about 2 years ago—even though it had not met the 

FDA standard, did it meet the quality-of-life standard? Did it improve the quality of life of the—it happened to be a breast cancer 

drug—for the women who took it? 

Because that would have been an important consideration. And, 

unfortunately, the quality of the data measuring quality of life was 

inadequate. 

So going forward, cancer patients have enough on their plates, as 

do everyone represented at this table, as do patients throughout 

this country. We need to be aware that quality of life is an important part of the treatment process, and we need to have tools in 

place. 

104 

They are not uniform yet. They are not as good as we would like. 

But they have to be in place to measure quality of life, and that 

has to be considered. And patient-reported outcomes are very much 

a part of that process. 

Ms. SCHAKOWSKY. Yes. Go ahead, Doctor. 

Mr. POPS. I just wanted to make a comment as it relates to patients with chronic disease as well. We talked a lot about cancer 

and orphan, small diseases. 

We work in the field of chronic disease—schizophrenia, depression, addiction—where patients are taking medicines for long, long 

periods of time. 

And simple things that may seem prosaic to the researcher, like 

nausea—— 

Ms. SCHAKOWSKY. Sure. 

Mr. POPS [continuing]. Fatigue, propensity to get addicted or dependent on the drug, these are really important inputs that we 

want to hear from patients about. 

Ms. SCHAKOWSKY. And is that part of the process? 

Mr. POPS. It is less part of the approval process today than I 

think it will be in the future. It is certainly part of the utilization 

process as patients make a determination, ‘‘Which medicine do I 

want to stay on for years and months?’’ I think that is a critical 

part of it, but it is not really incorporated in the consideration of 

the approval. 

Ms. SCHAKOWSKY. Especially where all things might be equal in 

effectiveness, whether or not something causes nausea, fatigue, 

could be really important. 

Mr. POPS. That is right. Particularly if you are launching a new 

medicine into a large category where there might be an abundance 

of generic drugs that are safe and effective, but might not hit all 

of those parameters for certain subsets of patients for long periods 

of time. 

And we just want people to be sensitive to the fact that, from the 

patient perspective, there are differences between the medicines. 

Ms. SCHAKOWSKY. Right. 

And then, also, Dr. Lichtenfeld, I wanted to ask you about smallsized trials. And you mentioned one of the drawbacks. 

I had talked about the extent to which women aren’t considered. 

And I would just be concerned—I understand the plus. I do. But 

if we rely too heavily on them, isn’t there the real risk of excluding 

important populations? 

Dr. LICHTENFELD. Well, the answer is yes, there is a real risk of 

excluding important populations. 

In fact, when you talked about women and heart disease, I remember back in the early 1990s when the article came out talking 

about the absence of women in clinical trials for the treatment of 

hypertension and heart disease. So this is an issue I am aware of. 

But let’s talk about the other side of the coin, and that is, when 

you are sitting—I have sat in the presentations at ASCO, at oncology meetings—and you see a presentation of 80 patients and you 

see what we call waterfall plots—basically, the responses in survival that occur—and suddenly, 70 percent of those patients are 

having significant responses, In a disease where there was no 

105 

treatment before, I don’t think one asks the question—they ask the 

question in followup, but not at the moment. 

And what has happened and what has been exciting to me is I 

am now sitting in those presentations every June and I see—I 

wrote about it—it took a year for one of the drugs to go from clinical trial to approval because it was that effective in the disease 

where there was no other treatment available. That is pretty spectacular. That is new thinking. That is a new approach. 

Now, we have learned more as time has gone on. Yes. Doesn’t 

mean we have stopped learning, as was mentioned before about 

cystic fibrosis. But when you suddenly see moments like that, no 

one would want to hold back. Develop the data, yes, but don’t hold 

back the opportunity. 

In fact, even a phase 1 trial that was presented at this ASCO 

meeting in June, the company, actually—well, not the company, 

but ASCO in the press release indicated the company was willing 

after a phase 1 trial to put it into compassionate use. And that is 

pretty amazing, a major change in the way that traditionally we 

have seen cancer drugs move through the pipeline. 

Ms. SCHAKOWSKY. Thank you. 

I have overstayed my time. Thank you. 

Mr. PITTS. Sure. Thanks to the gentlelady. 

I now recognize the gentleman from Virginia, Mr. Griffith, 5 minutes for questions. 

Mr. GRIFFITH. Dr. Lichtenfeld, let’s pick up there, because I 

think that that compassionate use is something that—we really 

need to be figuring out how we can make it more effective and how 

we can do it faster at the Federal level. 

You talked about in your testimony that patients needed to be 

involved both on saying what kind of nausea they had and what 

the pain levels were and so forth, and I agree with that. 

But I also think that, particularly when you have no treatment, 

that patients need to be involved in that, too. And as Ms. Furlong 

said earlier, when there isn’t a treatment, you are much more willing to take those risks than you would be if there is some other 

treatment out there that might work, but this might be a little 

more comfortable. 

And I want to give both you and Ms. Furlong an opportunity just 

to address that further. 

Dr. LICHTENFELD. Well, thank you. And I appreciate the question, and I know you mentioned it earlier. 

It is a complex issue. It is not a new issue. As you may well be 

aware, it has been around for some time with the number of drugs 

that have gone through the pipeline, which seem to show some opportunities. Various state legislatures are involved. 

And I am sitting here today both as a representative of the Society, but also as an individual, and understand that there are discussions on both sides of that issue and they are complicated. 

Bottom line is that we need to understand what drugs work 

when they work. We need to understand that patients need to have 

access to promising drugs as soon as possible. 

Companies make those decisions as to how they are going to handle that process. The FDA, as a matter of fact, has approved almost 

all of the applications they received. And we need to have those 

106 

discussions to come to a better resolution about how to address 

that issue. 

Mr. GRIFFITH. And what I would say is that whatever we can 

do—I think I speak for a lot of the members of the committee—I 

am probably a little more out there than some—but whatever we 

can do to help by changing the law to expedite that process, we will 

do. 

Dr. LICHTENFELD. We would be glad to have those discussions on 

behalf of the Society, sir. 

Mr. GRIFFITH. Ms. Furlong, did you want to make another comment about risk assessment? Because, obviously, when your boys 

were sick, you probably would have taken anything that had any 

promise of hope. 

Ms. FURLONG. I think I could tell you stories about looking in 

China to see some tea that you might be appalled about, but that 

was long ago. 

I think it really is up to the companies. FDA has always, to my 

knowledge, at least in the Duchenne and other fields, been willing 

to entertain and talk about compassionate use. 

I think for the rare disease community this really talks about 

and gets us back to trial design. 

In general, trials are designed to test a small subset of patients. 

In the Duchenne community, the 6-minute walk test is the standard primary outcome measure. 

So that means, as a child with Duchenne, you have to walk 6 

minutes and even further, as we learn more about the testing. It 

is a very narrow subset of people within a certain framework of 

that 6-minute walk test, which, as you can imagine, leaves a great 

number of people outside the trial. 

So I think trials have to be designed that are inclusive and welcoming of people that live with the spectrum of the illness, both 

very young as well as adults with very limited functional ability. 

That way, we can test those in those populations. We can have 

labels that are broad and then provide access to all. So I think that 

might be a better solution. 

Mr. GRIFFITH. Thank you. 

I look forward to working with you all. 

And, with that, I yield the remainder of my time to Dr. Burgess. 

Mr. BURGESS. I thank the gentleman. 

Dr. Lichtenfeld, I just wanted to follow up with you because your 

specialty has been involved in this type of activity probably longer 

than any other branch of clinical medicine, going back to 1955 

when the developmental therapeutics program was put into place 

at the National Cancer Institute. 

So with that breadth of experience within your specialty, are 

there things that you want to share with others about what that 

experience has taught you? 

Dr. LICHTENFELD. Well, what we did back in 1965 or whenever 

was a lot different than what we are doing today. I don’t want to 

take the time to really go into it. You may be aware of it. 

But here is the message. It didn’t happen overnight. It took 40 

years of research to get us to the tipping point where we understood the genome and had the opportunity to take advantage of 

that and move forward. 

107 

Immunotherapy, the same story. It has taken us 40 years. That 

was a substantial amount—I don’t want to underestimate the value 

of research investment to get us to the point where we are, where 

suddenly we look like we have so much to offer and to do. 

I also comment, with regard to my co-panelists, that they have 

populations and they have demonstrated that finding the patients 

where they are is critically important. 

We have a substantial amount of work to do to understand not 

only the clinical trial mechanism, but also the medical practice system, so we can make sure that patients and communities—I live 

in a small town in south Georgia—that my friends have opportunities to get these drugs in clinical trials and be part of that process. 

There is a lot of work to do. 

Mr. BURGESS. Thank you, Mr. Chairman. I yield back. 

Mr. PITTS. The chair thanks the gentleman. 

Now recognize the gentleman from New York, Mr. Engel, 5 minutes for questions. 

Mr. ENGEL. Thank you, Mr. Chairman and Mr. Pallone, for holding this hearing. I am pleased to have this opportunity to further 

consider how patient perspectives can best be incorporated into the 

therapeutic development process. 

As the author of the ALS Registry Act and the Paul D. Wellstone 

Muscular Dystrophy Community Assistance Research Education 

Amendments of 2008 and 2013, along with my colleague, Dr. Burgess, I have worked to be a voice with those with rare and orphan 

diseases. 

I am encouraged by the advances we have made into the causes 

and mechanisms of these diseases, as well as our progress toward 

treatments, but, obviously, we still have a long way to go. 

One of the most striking gains we have made is for individuals 

with Duchenne muscular dystrophy. As Ms. Furlong mentioned, 

our efforts have added an average of 10 years to the life expectancy 

of boys with Duchenne. And now, as life expectancy increases, we 

face new challenges in finding effective therapies. 

The patient community brings an important perspective and understanding to this process, and I am interested to see how we can 

best use that knowledge to assist medical researchers with therapy 

developments. 

So, Ms. Furlong, let me ask you this. I am particularly interested 

in the way the Duchenne patient community is engaged with the 

FDA to help inform the benefit-risk determinations made by agency reviewers, as well as the Duchenne community guidance document you referred to in your prepared testimony. 

Could you please comment on how you hope to see these efforts 

affect the therapeutic pipeline and the various stakeholders who 

are part of that pipeline. 

Ms. FURLONG. Yes. Thank you, Mr. Engel, for the question. 

The benefit-risk really originated out of discussions with the 

FDA because, in our early discussions, it was known that we were 

telling anecdotal stories that the equation of benefit-risk was different in, for instance, Duchenne muscular dystrophy than perhaps 

some more common disease. 

108 

And in that the FDA suggested to us that they agreed, but they 

didn’t have anything they could rely on, any quantified evidencebased document that could help them make those decisions. 

So we agreed to go out on benefit-risk and did the pilot with 120 

parents. We learned that their priority is disease stabilization and 

they were willing to accept a great deal of risk. In fact, they are 

living with a great deal of risk, as they know that their child has 

a fatal illness. 

So the FDA has now asked us to expand that study to a greater 

number of patients than 120 patients and, also, to ask these questions of the young men with Duchenne. Our hope is that they will 

incorporate it into the review process and they will demonstrate to 

us how and when they use it and when they don’t and what makes 

sense for them as they make their decisions. 

Mr. ENGEL. Thank you. And thank you for your advocacy and 

hard work. It is very much appreciated. 

Dr. Summar, can you talk about the role you think the patient 

perspectives should play in developing therapies for diseases like 

ALS and muscular dystrophy that have limited treatment options 

and for which quality of life is, obviously, an especially important 

factor to patients. 

How can the FDA best consider the views of patients and families when examining the benefit-risk calculus for these diseases? 

Dr. SUMMAR. Thank you for that question, Mr. Engel. 

This really kind of expands across the entire field of rare diseases, but your question is particularly relevant for those two 

groups. 

Patients often tell us about things that they wish were better 

that we never thought of. One of the things I have run across time 

and time again is, when we go and ask our patients, ‘‘What is the 

worst part of this disease?’’—a lot of times it is parents in the case 

of pediatric patients—they will list some things. And sometimes 

the things we thought were most important are number nine or ten 

on the list. 

So I think, when we look at what our therapeutic targets are, 

what our quality-of-life targets are for these diseases, patient and 

family input is a huge factor, and I think it is something we can 

incorporate a lot better than we have. 

I think during the early stages, particularly when we are designing our pivotal trials, clinical trials, looking at what end points 

are—I think that those are going to become more and more important. 

And the other thing, of course, is the small group sizes with 

these. Many times it is hard to pick one single outcome variable 

that you are going to be able to achieve. 

The smallest study I have been involved with was five patients 

for an approval process. Getting one exact target for that—fortunately, the effect of the drug was massive; so, we were able to do 

it. But if it had been milder, I might have needed more than one 

outcome variable. 

So I think families can help us determine what is important 

there. They can help us, also, as we talked about with some of 

what risk is tolerable in those situations. It is different. And there 

109 

are 7,000 different rare diseases. Each one of these is unique in its 

own regard. But there are some commonalities like that. 

Mr. ENGEL. Well, thank you. And thank you for your comments, 

and also thank you for your interest. 

And I want to thank the panel for a very interesting discussion. 

Thank you, Mr. Chairman. 

Mr. PITTS. The chair thanks the gentleman. 

Now recognizes the gentleman from Florida, Mr. Bilirakis, 5 minutes for questions. 

Mr. BILIRAKIS. Thank you, Mr. Chairman. I appreciate it. 

And I thank the panel for their testimony today. 

I know we have been talking about this and you have had an opportunity. I want to give you more of an opportunity to respond on 

this. 

On Wednesday, I asked one of the witnesses about his statement 

in including patients in the clinical trial process, but I want to 

make sure that you all have every opportunity to respond to this. 

If patients had a greater role in clinical trial design—and I know 

you have touched upon this—if trials measured qualitative data 

from patients like, ‘‘How do you feel?’’, ‘‘Is it less painful?’’, what 

have you; ‘‘How would things be different?’’ and ‘‘What would you 

like to see?’’ 

We will start with the—— 

Mr. BEALL. I would like to start. 

First of all, the patient-reported outcomes I think has been part 

of every clinical trial in cystic fibrosis for the last 10 or 15 years. 

Some of the tools are not the best at this point, but we are working 

to refine them. 

We have just spent as a Foundation a large effort to look at the 

patient-reported outcomes as a kind of specific validated tool for 

CF, and it is going to be submitted to the FDA and go through a 

validation process. In the past, we have used one that was generally for lung disease, but it may not be specific. 

So this is a science that is evolving. A decade ago or 15 years 

ago, 20 years ago, PROs were not really incorporated. 

So it is a science that is evolving, and it has to be evolved not 

only with the FDA, it has to be evolved with the sponsors, too, because they have got to be willing to incorporate those into the clinical-trial process. 

So I am encouraged by the process, but I will tell you the—just 

in this last trial we had where their lung function went up and the 

exacerbations went down, we didn’t have a statistically significant 

improvement in the patient-reported outcomes. 

Because when you are starting to treat the basic defect, you are 

treating the whole disease process and you are looking at extending 

lives. 

And the patients may not feel that from day to day, but over 

years, you may have a tremendous impact on those patients. So it 

is a tool that can be used, but it shouldn’t be used exclusively. 

Mr. BILIRAKIS. Thank you very much. 

Ms. Furlong, do you have a response? 

Ms. FURLONG. Sure. 

So I agree with Dr. Beall. And patient-reported outcomes are incredibly important, but I think this is where involving the patients 

110 

in the design and conduct of clinical trials is really going to be important. 

Because, for instance, how do we measure energy and endurance? How do we know that turning over in bed is important to patients as opposed to an outcome measure such as the 6-minute 

walk test? 

So I think things are important to patients that have a real effect on their lives. For instance, as you can imagine, if a boy can 

still text at the age of 18, that gives him independence. If a child 

can walk up a single step, they can enter buildings. If a child can 

roll over in bed, that makes the families’ quality of life overall, in 

general, much, much better. 

So I think the use of patient-reported outcomes and including the 

patient voice in the discussion about what the clinical trial looks 

like and what the measurements are, both primary and secondary, 

is going to be incredibly important. 

Mr. BILIRAKIS. Thank you so much. 

Mr. Pops, do you have a comment or—— 

Mr. POPS. I think these outcomes are so critical. In the world 

that we are developing drugs in, which is in psychiatry often, in 

schizophrenia, depression, addiction, the end point of the clinical 

trial—the hard end point is asking people essentially how they feel. 

And so how you feel is typically embodied in the set of validated 

scales, but those often don’t capture some of the most important 

parts of how they actually feel over time. 

A perfect example might be an opioid dependence or an alcohol 

dependence, where a critical question the patients ask us when 

they take our medicine is, ‘‘Is my craving going to go down? Am 

I going to crave this less? It may block the receptor and keep me 

from drinking, but is my craving going to change?’’ 

That was not a validated end point. That was something we 

couldn’t incorporate in the label, but it is essential to the patient’s 

perception of the disease. 

Mr. BILIRAKIS. Good point. 

Doctor? 

Dr. LICHTENFELD. About 4 weeks ago at ASCO, the oncology 

meeting, they showed a picture of a lady who was 96 years old who 

had received a phase 1 drug—that in itself is a fascinating point— 

whose cancer completely resolved. 

And on the bottom end of the before and after picture—on the 

after picture, you saw a trace of a little smile. And I noticed that 

smile and I tweeted it, actually. I took a picture and tweeted it and 

it got re-tweeted quite a bit. And then the lecturer said, ‘‘Yes. That 

really is a smile’’ in front of 2,000 people. 

What I am trying to say by that example is that is what we have 

to be able to measure and aggregate in a scientific way to show 

that the treatments make a difference. 

One example of one lady in an unusual situation, but something 

that I think all of us agree—I would echo the comments that were 

already made—is so critical to understanding, particularly in the 

oncology world, what we do and how we do it and the goal that we 

have to have of improving quality of life. 

Mr. BILIRAKIS. Thank you. 

Dr. Summar? 

111 

Dr. SUMMAR. Yes. I will just use another example, too. 

We had a new medication we were looking at. Most of the patients with rare diseases are on the medicines they take for life. So 

it is every day, day in and day out. And these care plans are often 

complex and they really affect the whole family. 

So the new drug looked like it was promising from the standpoint of maybe a little bit better efficacy, a little bit better control, 

but it was five times a day instead of two or three times a day compared to the old one. 

And the families were like, ‘‘Why would we add three more times 

a day of dosing for the small effect?’’ And no one had really bothered to ask them that before we started. 

So I think there is all of these things that really getting the patient input early on is going to make a difference. 

Mr. BILIRAKIS. Thank you so much. Appreciate it. 

Mr. Chairman, can I ask one more question? 

Mr. PITTS. You may proceed. 

Mr. BILIRAKIS. OK. Thank you so much. Appreciate it. 

Dr. Beall, the CF Foundation’s venture philanthropy model has 

produced incredible results. Congratulations. Your foundation 

found your breakthrough drug when it helped translate some of the 

early research through the valley of death, and now you have the 

Kalydeco. 

How are you able to establish this program? And how can other 

groups adopt this similar model? 

Mr. BEALL. Well, it is a willingness to take risks. That is what 

you have to do in drug discovery. And we were frustrated by the 

fact that companies were not getting involved in the orphan diseases. 

So the whole concept here was to say, ‘‘Take some of the risk out 

of biotech companies or pharmaceutical companies to get engaged 

in CF research.’’ And, as I said, we spent $42 million initially to 

start a high-throughput screening that led to Kalydeco. 

I think what is the most important and gratifying thing for the 

Cystic Fibrosis Foundation—and I know Ms. Furlong was in my office a number of years ago—and what we are seeing is so many 

other organizations are feeling the same impatience that our foundation felt 14 years ago in adopting this. 

One of the first times I talked about venture philanthropy at the 

bio meetings, we had 10 people in the audience. And now it is really becoming really inherent in what many voluntary health organizations are doing. 

In fact, FasterCures has been an organization that has been central to making some of that happen. There are law firms that specialize in it. 

So we love to share our ideas. We share our ideas all the time. 

And it has been very gratifying to our community that we happen 

to be fortunate enough to be able to start it because we had the 

resources. Bill and Melinda Gates gave us $20 million to start our 

program. We had other dollars to really make that initial investment. 

Mr. BILIRAKIS. Can you tell us how you successfully established 

the CF registry? 

112 

Mr. BEALL. As I say, it goes back a long time. But Dr. Zerhouni 

was here several years ago when he was the head of the NIH, and 

he says one thing about the CF community, it is a community with 

a culture of research. 

And every patient who goes to one of our care centers is asked, 

‘‘Do you want to participate in a patient registry?’’ And I think it 

is 99.5 percent of the patients that say, ‘‘Yes, I do’’ and then signs 

the informed consent. 

So it is all part of the culture. It is part of the culture the organization creates. It is the physicians and it is the relationships and 

the recognition that it is an important part of having a disease because we can’t cure this disease without their involvement. 

Mr. BILIRAKIS. Very good. Thank you very much. Appreciate it. 

I yield back, Mr. Chairman. 

Mr. PITTS. The chair thanks the gentleman. 

Dr. Summar, I didn’t get to you in my round. 

On Wednesday, we heard an idea thrown out there that there 

are vast amounts of data available that are not being utilized. And 

we all know what an organ donor is. The idea was that we have 

data donors. 

Now, how would this play—and you mentioned the IRB system, 

the risk enterprise. What is your reaction to that? 

Dr. SUMMAR. This is something we talk about when we are having coffee a lot. 

There are data sets all over the place. In fact, most of them end 

up usually lost when someone’s computer gets recycled. We had a 

physician lose 15 years of data because his Excel spreadsheet didn’t 

update. 

I think a way—find a way that balances, obviously, people’s desire for confidentiality versus the irreplaceable and oftentimes 

irreproducible amounts of data that are out there. We really do 

need to find that balance. 

My reaction to that would be I would love to find a way forward 

with that. That one is going to—you can see a lot of sides to that 

question. But I definitely think it is worth looking at. 

And I think what we find is a lot of patients are like, ‘‘Yes. I will 

put it out there. I am fine with that.’’ There will be a small core 

that won’t. You can take a count for that. But I think most folks, 

if you ask them, saying, ‘‘Would you feel OK if your data is out 

there so everybody can take a look at it?’’ would be fine. 

You see people opening up their genomes, who had their genomes 

sequenced, saying, ‘‘I will publicly post it along with my medical 

health history.’’ A lot of folks want to help. 

Mr. PITTS. Thank you. Thank you. 

Mr. BURGESS. I did that. We did that. I mean, that is a real thing 

that is happening right now. And, yes, privacy is something we all 

value, but it also is a voluntary relinquishing of a portion of that 

for the greater good. 

I think that is something we ought to not encourage—well, not 

encourage, but we certainly shouldn’t stand in the way if that is 

an activity that—— 

Dr. SUMMAR. Right. 

113 

Mr. BURGESS. And unfortunately, I can’t say that we don’t always respect that, that we shouldn’t stand in the way. But enough 

about that. 

Mr. PITTS. All right. The chair thanks the gentleman. 

That concludes the questions of the Members who are here. Another exciting, informative, very important hearing. Thank you so 

much for coming. 

Members will have followup questions, and we will send those to 

you. We ask that you please respond promptly. I remind Members 

they have 10 business days to submit questions for the record, and 

that means Members should submit their questions by the close of 

business on Friday, July 25th. 

I have a UC request, a statement for the record, from the National Health Council. Without objection, that will be inserted into 

the record. 

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

Mr. PITTS. Without objection, the subcommittee is adjourned. 

[Whereupon, at 12:19 p.m., the subcommittee was adjourned.] 

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