II 

113TH CONGRESS 

1ST SESSION S. 908 

To amend the Public Health Service Act to improve the diagnosis and 

treatment of hereditary hemorrhagic telangiectasia, and for other purposes. 

IN THE SENATE OF THE UNITED STATES 

MAY 8, 2013 

Mr. JOHNSON of South Dakota introduced the following bill; which was read 

twice and referred to the Committee on Health, Education, Labor, and 

Pensions 

A BILL 

To amend the Public Health Service Act to improve the 

diagnosis and treatment of hereditary hemorrhagic 

telangiectasia, and for other purposes. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Hereditary Hemor5 rhagic Telangiectasia Diagnosis and Treatment Act of 

6 2013’’. 

7 SEC. 2. FINDINGS. 

8 The Congress finds as follows: 


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1 (1) Hereditary hemorrhagic telangiectasia (re2 ferred to in this section as ‘‘HHT’’) is a largely 

3 undiagnosed or misdiagnosed vascular genetic bleed4 ing disorder resulting in artery-vein malformations 

5 (referred to in this section as ‘‘AVMs’’), which lead 

6 to preventable, catastrophic and disabling con7 sequences. HHT can cause sudden death at any age, 

8 unless detected and treated. Early detection, screen9 ing, and use of readily available treatment can pre10 vent premature deaths and long-term health com11 plications resulting from HHT. A person with HHT 

12 has the tendency to form blood vessels that lack the 

13 capillaries between an artery and vein. HHT often 

14 results in spontaneous hemorrhage or stroke from 

15 brain or lung AVMs. In addition to hemorrhagic 

16 stroke, embolic stroke and brain abscess occur in ap17 proximately 30 percent of individuals with HHT 

18 AVMs in the lung (due to lack of capillaries between 

19 the arterial and venous systems that normally filter 

20 out clots and bacteria). 

21 (2) One in 5,000 American children and adults 

22 suffer from HHT. 

23 (3) Studies have found an increase in morbidity 

24 and mortality rates for individuals who suffer from 

25 HHT. 


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1 (4) Due to the widespread lack of knowledge, 

2 accurate diagnosis, and appropriate intervention, 90 

3 percent of HHT-affected families are at risk for pre4 ventable life-threatening and disabling medical inci5 dents such as stroke. 

6 (5) Early detection, screening, and treatment 

7 can prevent premature deaths, spontaneous hemor8 rhage, hemorrhagic stroke, embolic stroke, brain ab9 scess, and other long-term health care complications 

10 resulting from HHT. 

11 (6) HHT is an important health condition with 

12 serious health consequences which are amenable to 

13 early identification and diagnosis with suitable tests, 

14 and acceptable and available treatments in estab15 lished treatment centers. 

16 (7) Timely identification and management of 

17 HHT cases is an important public health objective 

18 because it will save lives, prevent disability, and re19 duce direct and indirect health care costs expendi20 tures. A recent study has found use of a genetic 

21 testing model for HHT diagnosis saves $9,900,000 

22 if screening is limited to individuals within the fam23 ily groups that have the gene defect, leading to early 

24 intervention in individuals found to have treatable 

25 AVMs. 


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1 (8) Without a new program for early detection, 

2 screening, and treatment, 14,000 children and 

3 adults who suffer from HHT in the population today 

4 will suffer premature death and disability. 

5 SEC. 3. PURPOSE. 

6 The purpose of this Act is to create a federally led 

7 and financed initiative for early diagnosis and appropriate 

8 treatment of hereditary hemorrhagic telangiectasia that 

9 will result in the reduction of the suffering of families, 

10 prevent premature death and disability, and lower health 

11 care costs through proven treatment interventions. 

12 SEC. 4. NATIONAL INSTITUTES OF HEALTH. 

13 Part B of title IV of the Public Health Service Act 

14 (42 U.S.C. 284 et seq.) is amended by adding at the end 

15 the following: 

16 ‘‘SEC. 409K. HEREDITARY HEMORRHAGIC TELANGIECTASIA. 

17 ‘‘(a) HHT INITIATIVE.— 

18 ‘‘(1) ESTABLISHMENT.—The Secretary shall es19 tablish and implement an HHT initiative to assist in 

20 coordinating activities to improve early detection, 

21 screening, and treatment of people who suffer from 

22 HHT. Such initiative shall focus on— 

23 ‘‘(A) advancing research on the causes, di24 agnosis, and treatment of HHT, including 


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1 through the conduct or support of such re2 search; and 

3 ‘‘(B) increasing physician and public 

4 awareness of HHT. 

5 ‘‘(2) CONSULTATION.—In carrying out this sub6 section, the Secretary shall consult with the Director 

7 of the National Institutes of Health and the Director 

8 of the Centers for Disease Control and Prevention. 

9 ‘‘(b) HHT COORDINATING COMMITTEE.— 

10 ‘‘(1) ESTABLISHMENT.—Not later than 60 days 

11 after the date of the enactment of this section, the 

12 Secretary, in consultation with the Director of the 

13 National Institutes of Health, shall establish a com14 mittee to be known as the HHT Coordinating Com15 mittee. 

16 ‘‘(2) MEMBERSHIP.— 

17 ‘‘(A) IN GENERAL.—The members of the 

18 Committee shall be appointed by the Secretary, 

19 in consultation with the Director of the Na20 tional Institutes of Health, and shall consist of 

21 12 individuals who are experts in HHT or 

22 arteriovenous malformation (AVM) as follows: 

23 ‘‘(i) Four representatives of HHT 

24 Treatment Centers of Excellence des25 ignated under section 317U(c)(1). 


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1 ‘‘(ii) Four experts in vascular, molec2 ular, or basic science. 

3 ‘‘(iii) Four representatives of the Na4 tional Institutes of Health. 

5 ‘‘(B) CHAIR.—The Secretary shall des6 ignate the Chair of the Committee from among 

7 its members. 

8 ‘‘(C) INTERIM MEMBERS.—In place of the 

9 4 members otherwise required to be appointed 

10 under subparagraph (A)(i), the Secretary may 

11 appoint 4 experts in vascular, molecular, or 

12 basic science to serve as members of the Com13 mittee during the period preceding designation 

14 and establishment of HHT Treatment Centers 

15 of Excellence under section 317U. 

16 ‘‘(D) PUBLICATION OF NAMES.—Not later 

17 than 30 days after the establishment of the 

18 Committee, the Secretary shall publish the 

19 names of the Chair and members of the Com20 mittee on the Website of the Department of 

21 Health and Human Services. 

22 ‘‘(E) TERMS.—The members of the Com23 mittee shall each be appointed for a 3-year term 

24 and, at the end of each such term, may be re25 appointed. 


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1 ‘‘(F) VACANCIES.—A vacancy on the Com2 mittee shall be filled by the Secretary in the 

3 same manner in which the original appointment 

4 was made. 

5 ‘‘(3) RESPONSIBILITIES.—The Committee shall 

6 develop and coordinate implementation of a plan to 

7 advance research and understanding of HHT by— 

8 ‘‘(A) conducting or supporting basic, 

9 translational, and clinical research on HHT 

10 across the relevant national research institutes, 

11 national centers, and offices of the National In12 stitutes of Health, including the National 

13 Heart, Lung, and Blood Institute; the National 

14 Institute of Neurological Disorders and Stroke; 

15 the National Institutes of Diabetes and Diges16 tive and Kidney Diseases; the Eunice Kennedy 

17 Shriver National Institute of Child Health and 

18 Human Development; the National Cancer In19 stitute; and the Office of Rare Diseases; and 

20 ‘‘(B) conducting evaluations and making 

21 recommendations to the Secretary, the Director 

22 of the National Institutes of Health, and the 

23 Director of the National Cancer Institute re24 garding the prioritization and award of Na25 tional Institutes of Health research grants reVerDate Mar 15 2010 03:08 May 10, 2013 Jkt 029200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\S908.IS S908

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1 lating to HHT, including with respect to grants 

2 for— 

3 ‘‘(i) expand understanding of HHT 

4 through basic, translational, and clinical 

5 research on the cause, diagnosis, preven6 tion, control, and treatment of HHT; 

7 ‘‘(ii) training programs on HHT for 

8 scientists and health professionals; and 

9 ‘‘(iii) HHT genetic testing research to 

10 improve the accuracy of genetic testing. 

11 ‘‘(c) DEFINITIONS.—In this section: 

12 ‘‘(1) The term ‘Committee’ means the HHT 

13 Coordinating Committee established under sub14 section (b). 

15 ‘‘(2) The term ‘HHT’ means hereditary hemor16 rhagic telangiectasia.’’. 

17 SEC. 5. CENTERS FOR DISEASE CONTROL AND PREVEN18 TION. 

19 Part B of title III of the Public Health Service Act 

20 is amended by inserting after section 317T (42 U.S.C. 

21 247b–22) the following: 

22 ‘‘SEC. 317U. HEREDITARY HEMORRHAGIC 

23 TELANGIECTASIA. 

24 ‘‘(a) IN GENERAL.—With respect to hereditary hem25 orrhagic telangiectasia (in this section referred to as 


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1 ‘HHT’), the Director of the Centers for Disease Control 

2 and Prevention (in this section referred to as the ‘Direc3 tor’) shall carry out the following activities: 

4 ‘‘(1) The conduct of population screening de5 scribed in subsection (c). 

6 ‘‘(2) The identification and conduct of inves7 tigations to further develop and support guidelines 

8 for diagnosis of, and intervention for, HHT, includ9 ing cost-benefit studies. 

10 ‘‘(3) The development of a standardized survey 

11 and screening tool on family history. 

12 ‘‘(4) The establishment, in collaboration with a 

13 voluntary health organization representing HHT 

14 families, of an HHT resource center within the Cen15 ters for Disease Control and Prevention to provide 

16 comprehensive education on, and disseminate infor17 mation about, HHT to health professionals, pa18 tients, industry, and the public. 

19 ‘‘(5) The conduct or support of public aware20 ness programs in collaboration with medical, genetic, 

21 and professional organizations to improve the edu22 cation of health professionals about HHT. 

23 ‘‘(b) COLLABORATIVE APPROACHES.—The Director 

24 shall carry out this section through collaborative ap25 proaches within the National Center on Birth Defects and 


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1 Developmental Disabilities and the Division for Heart Dis2 ease and Stroke Prevention of the Centers for Disease 

3 Control and Prevention with respect to clotting and bleed4 ing disorders. 

5 ‘‘(c) POPULATION SCREENING.—In carrying out pop6 ulation screening under subsection (a)(1), the Director 

7 shall— 

8 ‘‘(1) designate and provide funding for a suffi9 cient number of HHT Treatment Centers of Excel10 lence to improve patient access to information, treat11 ment, and care by HHT experts; 

12 ‘‘(2) conduct surveillance through a population 

13 study, supplemented by sentinel health care provider 

14 or center surveillance, and by administrative data15 base analyses, as useful, to accurately identify— 

16 ‘‘(A) the prevalence of HHT; and 

17 ‘‘(B) the prevalence of hemorrhagic and 

18 embolic stroke and brain abscess, resulting 

19 from HHT; 

20 ‘‘(3) include HHT screening questions in the 

21 Behavioral Risk Factor Surveillance System survey 

22 conducted by the Centers for Disease Control and 

23 Prevention in order to screen a broader population 

24 and more accurately determine the prevalence of 

25 HHT; 


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1 ‘‘(4) provide data collected under paragraph 

2 (2)(B) to the Paul Coverdell National Acute Stroke 

3 Registry, to facilitate— 

4 ‘‘(A) analyses of the natural history of 

5 hemorrhagic and embolic stroke in HHT; and 

6 ‘‘(B) the development of screening and ar7 tery-vein malformation treatment guidelines 

8 specific to prevention of complications from 

9 HHT; 

10 ‘‘(5) develop and implement programs, targeted 

11 for physicians and health care professional groups 

12 likely to be accessed by families with HHT, to in13 crease HHT diagnosis and treatment rates through 

14 the— 

15 ‘‘(A) establishment of a partnership with 

16 HHT Treatment Centers of Excellence des17 ignated under paragraph (1) through the cre18 ation of a database of patients assessed at such 

19 HHT Treatment Centers of Excellence (includ20 ing with respect to phenotype information, gen21 otype information, transfusion dependence, and 

22 radiological findings); 

23 ‘‘(B) integration of such database with— 


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1 ‘‘(i) the universal data collection sys2 tem used by the Centers for monitoring he3 mophilia with the blood disorders; and 

4 ‘‘(ii) the Paul Coverdell National 

5 Acute Stroke Registry; and 

6 ‘‘(C) inclusion of other medical providers 

7 who treat HHT patients; and 

8 ‘‘(6) use existing administrative databases on 

9 non-HHT Treatment Center of Excellence pa10 tients— 

11 ‘‘(A) to learn about the natural history of 

12 HHT and the efficacy of various treatment mo13 dalities; and 

14 ‘‘(B) to better inform and develop screen15 ing and treatment guidelines associated with 

16 improvement in health care outcomes, and re17 search priorities relevant to HHT. 

18 ‘‘(d) ELIGIBILITY FOR DESIGNATION AS HHT 

19 TREATMENT CENTER OF EXCELLENCE.—In carrying out 

20 subsection (c)(1), the Director may designate as an HHT 

21 Treatment Center of Excellence an academic health center 

22 demonstrating each of the following: 

23 ‘‘(1) The academic health center possesses a 

24 team of medical experts capable of providing com25 prehensive evaluation, treatment, and education to 


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1 individuals with known or suspected HHT and their 

2 health care providers. 

3 ‘‘(2) The academic health center has sufficient 

4 personnel with knowledge about HHT, or formal col5 laboration with one or more partnering organizations 

6 for personnel or resources, to be able to— 

7 ‘‘(A) respond in a coordinated, multidisci8 plinary way to patient inquiries; and 

9 ‘‘(B) coordinate evaluation, treatment, and 

10 education of patients and their families in a 

11 timely manner. 

12 ‘‘(3) The academic health center has the fol13 lowing personnel, facilities, and patient volume: 

14 ‘‘(A) A medical director with— 

15 ‘‘(i) specialized knowledge of the main 

16 organ manifestations of HHT; and 

17 ‘‘(ii) the ability to coordinate the mul18 tidisciplinary diagnosis and treatment of 

19 patients referred to the center. 

20 ‘‘(B) Administrative staff with— 

21 ‘‘(i) sufficient knowledge to respond to 

22 patient inquiries and coordinate patient 

23 care in a timely fashion; and 


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1 ‘‘(ii) adequate financial support to 

2 allow the staff to commit at least 25 to 50 

3 percent of their time on the job to HHT. 

4 ‘‘(C) An otolaryngologist with experience 

5 and expertise in the treatment of recurrent epi6 staxis in HHT patients. 

7 ‘‘(D) An interventional radiologist with ex8 perience and expertise in the treatment of pul9 monary arteriovenous malformations (AVM). 

10 ‘‘(E) A genetic counselor or geneticist with 

11 the expertise to provide HHT-specific genetic 

12 counseling to patients and families. 

13 ‘‘(F) On-site facilities to screen for all 

14 major organ manifestations of HHT. 

15 ‘‘(G) A patient volume of at least 25 new 

16 HHT patients per year. 

17 ‘‘(H) Established mechanisms to coordi18 nate surveillance and outreach with HHT pa19 tient advocacy organizations.’’. 

20 SEC. 6. ADDITIONAL HEALTH AND HUMAN SERVICES AC21 TIVITIES. 

22 With respect to hereditary hemorrhagic telangiectasia 

23 (referred to in this section as ‘‘HHT’’), the Secretary of 

24 Health and Human Services, acting through the AdminisVerDate Mar 15 2010 03:08 May 10, 2013 Jkt 029200 PO 00000 Frm 00014 Fmt 6652 Sfmt 6201 E:\BILLS\S908.IS S908

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1 trator of the Centers for Medicare & Medicaid Services, 

2 shall award grants on a competitive basis— 

3 (1) for an analysis by grantees of the Medicare 

4 Provider Analysis and Review (MEDPAR) file to de5 velop preliminary estimates from the Medicare pro6 gram under title XVIII of the Social Security Act 

7 for— 

8 (A) preventable costs of annual health care 

9 expenditures associated with untreated HHT 

10 furnished to individuals with HHT, including 

11 items, services, and treatments; and 

12 (B) socioeconomic costs associated with 

13 preventable medical events among individuals 

14 with HHT who are entitled to benefits under 

15 part A of title XVIII of the Social Security Act 

16 or enrolled under part B of such title, such as 

17 disability expenditures; and 

18 (2) to make recommendations regarding an en19 hanced data collection protocol to permit a more 

20 precise determination of the total costs described in 

21 paragraph (1). 

22 SEC. 7. AUTHORIZATION OF APPROPRIATIONS. 

23 (a) IN GENERAL.—To carry out section 409K of the 

24 Public Health Service Act as added by section 4 of this 

25 Act, section 317U of the Public Health Service Act as 


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1 added by section 5 of this Act, and section 6 of this Act, 

2 there is authorized to be appropriated $5,000,000 for each 

3 of fiscal years 2014 through 2018. 

4 (b) RESOURCE CENTER.—Of the amount authorized 

5 to be appropriated under subsection (a) for each of fiscal 

6 years 2014 through 2018, $1,000,000 shall be for car7 rying out section 317U(a)(4) of the Public Health Service 

8 Act, as added by section 5 of this Act. 

Æ 


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