I 

112TH CONGRESS 

1ST SESSION H. R. 2123 

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

treatment of hereditary hemorrhagic telangiectasia, and for other purposes. 

IN THE HOUSE OF REPRESENTATIVES 

JUNE 3, 2011 

Mr. GALLEGLY (for himself, Mr. WU, and Mr. HIMES) introduced the following bill; which was referred to the Committee on Energy and Commerce, and in addition to the Committee on Ways and Means, for a period to be subsequently determined by the Speaker, in each case for consideration of such provisions as fall within the jurisdiction of the committee concerned 

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 2011’’. 

7 SEC. 2. FINDINGS. 

8 The Congress finds as follows: 


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1 (1) Hereditary hemorrhagic telangiectasia 

2 (‘‘HHT’’) is a largely undiagnosed or misdiagnosed 

3 vascular genetic bleeding disorder that causes abnor4 malities of the blood vessels. A person with HHT 

5 has the tendency to form blood vessels that lack the 

6 capillaries between an artery and vein. HHT can 

7 cause spontaneous hemorrhage or stroke when brain 

8 or lung arteriovenous malformations, which are tan9 gled blood vessels, rupture unexpectedly, in all age 

10 groups. In addition to hemorrhagic stroke, embolic 

11 stroke and brain abscess occur in approximately 

12 30% of persons with HHT caused by artery-vein 

13 malformations in the lung (due to lack of capillaries 

14 between the arterial and venous systems which nor15 mally filter out clots and bacteria), causing disability 

16 and sudden premature death. 

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

18 suffer from HHT. 

19 (3) Studies have found an increase in morbidity 

20 and mortality rates for individuals who suffer from 

21 HHT. 

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

23 accurate diagnosis, and appropriate intervention, 90 

24 percent of HHT-affected families are at risk for preVerDate Mar 15 2010 22:17 Jun 09, 2011 Jkt 099200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H2123.IH H2123

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1 ventable life-threatening and disabling medical inci2 dents such as stroke. 

3 (5) Early detection, screening, and treatment 

4 can prevent premature deaths, spontaneous hemor5 rhage, hemorrhagic stroke, embolic stroke, brain ab6 scess, and other long-term health care complications 

7 resulting from HHT. 

8 (6) HHT is an important health condition with 

9 serious health consequences which are amenable to 

10 early identification and diagnosis with suitable tests, 

11 and acceptable and available treatments in estab12 lished treatment centers. 

13 (7) Timely identification and management of 

14 HHT cases is an important public health objective 

15 because it will save lives, prevent disability, and re16 duce direct and indirect health care costs expendi17 tures. 

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

19 screening, and treatment, 14,000 children and 

20 adults who suffer from HHT in the population today 

21 will suffer premature death and disability. 

22 SEC. 3. PURPOSE. 

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

24 and financed initiative for early diagnosis and appropriate 

25 treatment of hereditary hemorrhagic telangiectasia that 


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

2 prevent premature death and disability, and lower health 

3 care costs through proven treatment interventions. 

4 SEC. 4. NATIONAL INSTITUTES OF HEALTH. 

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

6 (42 U.S.C. 284 et seq.) is amended by adding at the end 

7 the following: 

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

9 ‘‘(a) HHT INITIATIVE.— 

10 ‘‘(1) ESTABLISHMENT.—The Secretary shall es11 tablish and implement an HHT initiative to assist in 

12 coordinating activities to improve early detection, 

13 screening, and treatment of people who suffer from 

14 HHT. Such initiative shall focus on— 

15 ‘‘(A) advancing research on the causes, di16 agnosis, and treatment of HHT, including 

17 through the conduct or support of such re18 search; and 

19 ‘‘(B) increasing physician and public 

20 awareness of HHT. 

21 ‘‘(2) CONSULTATION.—In carrying out this sub22 section, the Secretary shall consult with the Director 

23 of the National Institutes of Health and the Director 

24 of the Centers for Disease Control and Prevention. 

25 ‘‘(b) HHT COORDINATING COMMITTEE.— 


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

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

3 Secretary, in consultation with the Director of the 

4 National Institutes of Health, shall establish a com5 mittee to be known as the HHT Coordinating Com6 mittee. 

7 ‘‘(2) MEMBERSHIP.— 

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

9 Committee shall be appointed by the Secretary, 

10 in consultation with the Director of the Na11 tional Institutes of Health, and shall consist of 

12 12 individuals who are experts in HHT or 

13 arteriovenous malformation (AVM) as follows: 

14 ‘‘(i) Four representatives of HHT 

15 Treatment Centers of Excellence des16 ignated under section 317U(c)(1). 

17 ‘‘(ii) Four experts in vascular, molec18 ular, or basic science. 

19 ‘‘(iii) Four representatives of the Na20 tional Institutes of Health. 

21 ‘‘(B) CHAIR.—The Secretary shall des22 ignate the Chair of the Committee from among 

23 its members. 

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

25 4 members otherwise required to be appointed 


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1 under paragraph (2)(A)(i), the Secretary may 

2 appoint 4 experts in vascular, molecular, or 

3 basic science to serve as members of the Com4 mittee during the period preceding designation 

5 and establishment of HHT Treatment Centers 

6 of Excellence under section 317U. 

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

8 than 30 days after the establishment of the 

9 Committee, the Secretary shall publish the 

10 names of the Chair and members of the Com11 mittee on the Website of the Department of 

12 Health and Human Services. 

13 ‘‘(E) TERMS.—The members of the Com14 mittee shall each be appointed for a 3-year term 

15 and, at the end of each such term, may be re16 appointed. 

17 ‘‘(F) VACANCIES.—A vacancy on the Com18 mittee shall be filled by the Secretary in the 

19 same manner in which the original appointment 

20 was made. 

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

22 develop and coordinate implementation of a plan to 

23 advance research and understanding of HHT by— 

24 ‘‘(A) conducting or supporting basic, 

25 translational, and clinical research on HHT 


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1 across the relevant national research institutes, 

2 national centers, and offices of the National In3 stitutes of Health, including the National 

4 Heart, Lung, and Blood Institute; the National 

5 Institute of Neurological Disorders and Stroke; 

6 the National Institutes of Diabetes and Diges7 tive and Kidney Diseases; the Eunice Kennedy 

8 Shriver National Institute of Child Health and 

9 Human Development; the National Cancer In10 stitute; and the Office of Rare Diseases; and 

11 ‘‘(B) conducting evaluations and making 

12 recommendations to the Secretary, the Director 

13 of the National Institutes of Health, and the 

14 Director of the National Cancer Institute re15 garding the prioritization and award of Na16 tional Institutes of Health research grants re17 lating to HHT, including with respect to grants 

18 for— 

19 ‘‘(i) expand understanding of HHT 

20 through basic, translational, and clinical 

21 research on the cause, diagnosis, preven22 tion, control, and treatment of HHT; 

23 ‘‘(ii) training programs on HHT for 

24 scientists and health professionals; and 


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1 ‘‘(iii) HHT genetic testing research to 

2 improve the accuracy of genetic testing. 

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

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

5 Coordinating Committee established under sub6 section (b). 

7 ‘‘(2) The term ‘HHT’ means hereditary hemor8 rhagic telangiectasia.’’. 

9 SEC. 5. CENTERS FOR DISEASE CONTROL AND PREVEN10 TION. 

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

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

13 247b–22) the following: 

14 ‘‘SEC. 317U. HEREDITARY HEMORRHAGIC 

15 TELANGIECTASIA. 

16 ‘‘(a) IN GENERAL.—With respect to hereditary hem17 orrhagic telangiectasia (in this section referred to as 

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

19 and Prevention (in this section referred to as the ‘Direc20 tor’) shall carry out the following activities: 

21 ‘‘(1) The conduct of population screening de22 scribed in subsection (c). 

23 ‘‘(2) The identification and conduct of inves24 tigations to further develop and support guidelines 


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1 for diagnosis of, and intervention for, HHT, includ2 ing cost-benefit studies. 

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

4 and screening tool on family history. 

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

6 voluntary health organization representing HHT 

7 families, of an HHT resource center within the Cen8 ters for Disease Control and Prevention to provide 

9 comprehensive education on, and disseminate infor10 mation about, HHT to health professionals, pa11 tients, industry, and the public. 

12 ‘‘(5) The conduct or support of public aware13 ness programs in collaboration with medical, genetic, 

14 and professional organizations to improve the edu15 cation of health professionals about HHT. 

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

17 shall carry out this section through collaborative ap18 proaches within the National Center on Birth Defects and 

19 Developmental Disabilities and the Division for Heart Dis20 ease and Stroke Prevention of the Centers for Disease 

21 Control and Prevention. 

22 ‘‘(c) POPULATION SCREENING.—In carrying out pop23 ulation screening under subsection (a)(1), the Director 

24 shall— 


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1 ‘‘(1) designate and provide funding for a suffi2 cient number of HHT Treatment Centers of Excel3 lence to improve patient access to information, treat4 ment, and care by HHT experts; 

5 ‘‘(2) conduct surveillance by means of a re6 gional population study, supplemented by sentinel 

7 health care provider or center surveillance, and by 

8 administrative database analyses as useful, to accu9 rately identify— 

10 ‘‘(A) the prevalence of HHT; and 

11 ‘‘(B) the prevalence of hemorrhagic and 

12 embolic stroke, and brain abscess, resulting 

13 from HHT; 

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

15 Behavioral Risk Factor Surveillance System survey 

16 conducted by the Centers for Disease Control and 

17 Prevention in order to screen a broader population 

18 and more accurately determine the prevalence of 

19 HHT; 

20 ‘‘(4) provide data collected under paragraph 

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

22 Registry to facilitate— 

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

24 hemorrhagic and embolic stroke in HHT; and 


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1 ‘‘(B) development of screening and artery2 vein malformation treatment guidelines specific 

3 to prevention of complications from HHT; 

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

5 for physicians and health care professional groups 

6 likely to be accessed by families with HHT, to in7 crease HHT diagnosis and treatment rates through 

8 the— 

9 ‘‘(A) establishment of a partnership with 

10 HHT Treatment Centers of Excellence des11 ignated under paragraph (1) through the cre12 ation of an international database of patients 

13 assessed at such HHT Treatment Centers of 

14 Excellence (including with respect to phenotype 

15 information, genotype information, transfusion 

16 dependence, and radiological findings); 

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

18 ‘‘(i) the universal data collection sys19 tem used by the Centers for monitoring he20 mophilia with the blood disorders; and 

21 ‘‘(ii) the Paul Coverdell National 

22 Acute Stroke Registry; and 

23 ‘‘(C) inclusion of other medical providers 

24 who treat HHT patients; and 


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1 ‘‘(6) use existing administrative databases on 

2 non-HHT Treatment Center of Excellence pa3 tients— 

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

5 HHT and the efficacy of various treatment mo6 dalities; and 

7 ‘‘(B) to better inform and develop screen8 ing and treatment guidelines associated with 

9 improvement in health care outcomes, and re10 search priorities relevant to HHT. 

11 ‘‘(d) ELIGIBILITY FOR DESIGNATION AS HHT 

12 TREATMENT CENTER OF EXCELLENCE.—In carrying out 

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

14 Treatment Center of Excellence only academic health cen15 ters demonstrating each of the following: 

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

17 team of medical experts capable of providing com18 prehensive evaluation, treatment, and education to 

19 individuals with known or suspected HHT and their 

20 health care providers. 

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

22 personnel with knowledge about HHT, or formal col23 laboration with one or more partnering organizations 

24 for personnel or resources, to be able to— 


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1 ‘‘(A) respond in a coordinated, multidisci2 plinary way to patient inquiries; and 

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

4 education of patients and their families in a 

5 timely manner. 

6 ‘‘(3) The academic health center has the fol7 lowing personnel, facilities, and patient volume: 

8 ‘‘(A) A medical director with— 

9 ‘‘(i) specialized knowledge of the main 

10 organ manifestations of HHT; and 

11 ‘‘(ii) the ability to coordinate the mul12 tidisciplinary diagnosis and treatment of 

13 patients referred to the center. 

14 ‘‘(B) Administrative staff with— 

15 ‘‘(i) sufficient knowledge to respond to 

16 patient inquiries and coordinate patient 

17 care in a timely fashion; and 

18 ‘‘(ii) adequate financial support to 

19 allow the staff to commit at least 25 to 50 

20 of their time on the job to HHT. 

21 ‘‘(C) An otolaryngologist with experience 

22 and expertise in the treatment of recurrent epi23 staxis in HHT patients. 


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1 ‘‘(D) An interventional radiologist with ex2 perience and expertise in the treatment of pul3 monary arteriovenous malformations (AVM). 

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

5 the expertise to provide HHT-specific genetic 

6 counseling to patients and families. 

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

8 major organ manifestations of HHT. 

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

10 HHT patients per year. 

11 ‘‘(H) Established mechanisms to coordi12 nate surveillance and outreach with HHT pa13 tient advocacy organizations.’’. 

14 SEC. 6. ADDITIONAL HEALTH AND HUMAN SERVICES AC15 TIVITIES. 

16 With respect to hereditary hemorrhagic telangiectasia 

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

18 Health and Human Services, acting through the Adminis19 trator of the Centers for Medicare & Medicaid Services, 

20 shall award grants on a competitive basis— 

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

22 Provider Analysis and Review (MEDPAR) file to de23 velop preliminary estimates on the total costs to the 

24 Medicare program under title XVIII of the Social 

25 Security Act for items, services, and treatments for 


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1 HHT furnished to individuals with HHT who are 

2 entitled to benefits under part A of title XVIII of 

3 the Social Security Act or enrolled under part B of 

4 such title; and 

5 (2) to make recommendations regarding an en6 hanced data collection protocol to permit a more 

7 precise determination of the total costs described in 

8 paragraph (1). 

9 SEC. 7. AUTHORIZATION OF APPROPRIATIONS. 

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

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

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

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

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

15 of fiscal years 2012 through 2016. 

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

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

18 years 2012 through 2016, $1,000,000 shall be for car19 rying out section 317U(a)(4) of the Public Health Service 

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

21 (c) OFFSET.—There is authorized to be appropriated 

22 to the Department of Health and Human Services for sal23 aries and expenses of the Department for each of fiscal 

24 years 2012 through 2016 the amount that is $5,000,000 


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1 less than the amount appropriated for such salaries and 

2 expenses for fiscal year 2011. 

Æ 


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