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
To amend the Public Health Service Act to improve the diagnosis and
treatment of hereditary hemorrhagic telangiectasia, and for other purposes.
Be it enacted by the Senate and House of Representatives of the United
States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the `Hereditary Hemorrhagic Telangiectasia Diagnosis
and Treatment Act of 2011'.
SEC. 2. FINDINGS.
The Congress finds as follows:
(1) Hereditary hemorrhagic telangiectasia (`HHT') is a largely undiagnosed
or misdiagnosed vascular genetic bleeding disorder that causes abnormalities
of the blood vessels. A person with HHT has the tendency to form blood vessels
that lack the capillaries between an artery and vein. HHT can cause spontaneous
hemorrhage or stroke when brain or lung arteriovenous malformations, which
are tangled blood vessels, rupture unexpectedly, in all age groups. In addition
to hemorrhagic stroke, embolic stroke and brain abscess occur in approximately
30% of persons with HHT caused by artery-vein malformations in the lung
(due to lack of capillaries between the arterial and venous systems which
normally filter out clots and bacteria), causing disability and sudden premature
(2) One in 5,000 American children and adults suffer from HHT.
(3) Studies have found an increase in morbidity and mortality rates for
individuals who suffer from HHT.
(4) Due to the widespread lack of knowledge, accurate diagnosis, and appropriate
intervention, 90 percent of HHT-affected families are at risk for preventable
life-threatening and disabling medical incidents such as stroke.
(5) Early detection, screening, and treatment can prevent premature deaths,
spontaneous hemorrhage, hemorrhagic stroke, embolic stroke, brain abscess,
and other long-term health care complications resulting from HHT.
(6) HHT is an important health condition with serious health consequences
which are amenable to early identification and diagnosis with suitable tests,
and acceptable and available treatments in established treatment centers.
(7) Timely identification and management of HHT cases is an important public
health objective because it will save lives, prevent disability, and reduce
direct and indirect health care costs expenditures.
(8) Without a new program for early detection, screening, and treatment,
14,000 children and adults who suffer from HHT in the population today will
suffer premature death and disability.
SEC. 3. PURPOSE.
The purpose of this Act is to create a federally led and financed initiative
for early diagnosis and appropriate treatment of hereditary hemorrhagic telangiectasia
that will result in the reduction of the suffering of families, prevent premature
death and disability, and lower health care costs through proven treatment
SEC. 4. NATIONAL INSTITUTES OF HEALTH.
Part B of title IV of the Public Health Service Act (42 U.S.C. 284 et seq.)
is amended by adding at the end the following:
`SEC. 409K. HEREDITARY HEMORRHAGIC TELANGIECTASIA.
`(1) ESTABLISHMENT- The Secretary shall establish and implement an HHT initiative
to assist in coordinating activities to improve early detection, screening,
and treatment of people who suffer from HHT. Such initiative shall focus
`(A) advancing research on the causes, diagnosis, and treatment of HHT,
including through the conduct or support of such research; and
`(B) increasing physician and public awareness of HHT.
`(2) CONSULTATION- In carrying out this subsection, the Secretary shall
consult with the Director of the National Institutes of Health and the Director
of the Centers for Disease Control and Prevention.
`(b) HHT Coordinating Committee-
`(1) ESTABLISHMENT- Not later than 60 days after the date of the enactment
of this section, the Secretary, in consultation with the Director of the
National Institutes of Health, shall establish a committee to be known as
the HHT Coordinating Committee.
`(A) IN GENERAL- The members of the Committee shall be appointed by the
Secretary, in consultation with the Director of the National Institutes
of Health, and shall consist of 12 individuals who are experts in HHT
or arteriovenous malformation (AVM) as follows:
`(i) Four representatives of HHT Treatment Centers of Excellence designated
under section 317U(c)(1).
`(ii) Four experts in vascular, molecular, or basic science.
`(iii) Four representatives of the National Institutes of Health.
`(B) CHAIR- The Secretary shall designate the Chair of the Committee from
among its members.
`(C) INTERIM MEMBERS- In place of the 4 members otherwise required to
be appointed under paragraph (2)(A)(i), the Secretary may appoint 4 experts
in vascular, molecular, or basic science to serve as members of the Committee
during the period preceding designation and establishment of HHT Treatment
Centers of Excellence under section 317U.
`(D) PUBLICATION OF NAMES- Not later than 30 days after the establishment
of the Committee, the Secretary shall publish the names of the Chair and
members of the Committee on the Website of the Department of Health and
`(E) TERMS- The members of the Committee shall each be appointed for a
3-year term and, at the end of each such term, may be reappointed.
`(F) VACANCIES- A vacancy on the Committee shall be filled by the Secretary
in the same manner in which the original appointment was made.
`(3) RESPONSIBILITIES- The Committee shall develop and coordinate implementation
of a plan to advance research and understanding of HHT by--
`(A) conducting or supporting basic, translational, and clinical research
on HHT across the relevant national research institutes, national centers,
and offices of the National Institutes of Health, including the National
Heart, Lung, and Blood Institute; the National Institute of Neurological
Disorders and Stroke; the National Institutes of Diabetes and Digestive
and Kidney Diseases; the Eunice Kennedy Shriver National Institute of
Child Health and Human Development; the National Cancer Institute; and
the Office of Rare Diseases; and
`(B) conducting evaluations and making recommendations to the Secretary,
the Director of the National Institutes of Health, and the Director of
the National Cancer Institute regarding the prioritization and award of
National Institutes of Health research grants relating to HHT, including
with respect to grants for--
`(i) expand understanding of HHT through basic, translational, and clinical
research on the cause, diagnosis, prevention, control, and treatment
`(ii) training programs on HHT for scientists and health professionals;
`(iii) HHT genetic testing research to improve the accuracy of genetic
`(c) Definitions- In this section:
`(1) The term `Committee' means the HHT Coordinating Committee established
under subsection (b).
`(2) The term `HHT' means hereditary hemorrhagic telangiectasia.'.
SEC. 5. CENTERS FOR DISEASE CONTROL AND PREVENTION.
Part B of title III of the Public Health Service Act is amended by inserting
after section 317T (42 U.S.C. 247b-22) the following:
`SEC. 317U. HEREDITARY HEMORRHAGIC TELANGIECTASIA.
`(a) In General- With respect to hereditary hemorrhagic telangiectasia (in
this section referred to as `HHT'), the Director of the Centers for Disease
Control and Prevention (in this section referred to as the `Director') shall
carry out the following activities:
`(1) The conduct of population screening described in subsection (c).
`(2) The identification and conduct of investigations to further develop
and support guidelines for diagnosis of, and intervention for, HHT, including
`(3) The development of a standardized survey and screening tool on family
`(4) The establishment, in collaboration with a voluntary health organization
representing HHT families, of an HHT resource center within the Centers
for Disease Control and Prevention to provide comprehensive education on,
and disseminate information about, HHT to health professionals, patients,
industry, and the public.
`(5) The conduct or support of public awareness programs in collaboration
with medical, genetic, and professional organizations to improve the education
of health professionals about HHT.
`(b) Collaborative Approaches- The Director shall carry out this section through
collaborative approaches within the National Center on Birth Defects and Developmental
Disabilities and the Division for Heart Disease and Stroke Prevention of the
Centers for Disease Control and Prevention.
`(c) Population Screening- In carrying out population screening under subsection
(a)(1), the Director shall--
`(1) designate and provide funding for a sufficient number of HHT Treatment
Centers of Excellence to improve patient access to information, treatment,
and care by HHT experts;
`(2) conduct surveillance by means of a regional population study, supplemented
by sentinel health care provider or center surveillance, and by administrative
database analyses as useful, to accurately identify--
`(A) the prevalence of HHT; and
`(B) the prevalence of hemorrhagic and embolic stroke, and brain abscess,
resulting from HHT;
`(3) include HHT screening questions in the Behavioral Risk Factor Surveillance
System survey conducted by the Centers for Disease Control and Prevention
in order to screen a broader population and more accurately determine the
prevalence of HHT;
`(4) provide data collected under paragraph (2)(B) to the Paul Coverdell
National Acute Stroke Registry to facilitate--
`(A) analyses of the natural history of hemorrhagic and embolic stroke
in HHT; and
`(B) development of screening and artery-vein malformation treatment guidelines
specific to prevention of complications from HHT;
`(5) develop and implement programs, targeted for physicians and health
care professional groups likely to be accessed by families with HHT, to
increase HHT diagnosis and treatment rates through the--
`(A) establishment of a partnership with HHT Treatment Centers of Excellence
designated under paragraph (1) through the creation of an international
database of patients assessed at such HHT Treatment Centers of Excellence
(including with respect to phenotype information, genotype information,
transfusion dependence, and radiological findings);
`(B) integration of such database with--
`(i) the universal data collection system used by the Centers for monitoring
hemophilia with the blood disorders; and
`(ii) the Paul Coverdell National Acute Stroke Registry; and
`(C) inclusion of other medical providers who treat HHT patients; and
`(6) use existing administrative databases on non-HHT Treatment Center of
`(A) to learn about the natural history of HHT and the efficacy of various
treatment modalities; and
`(B) to better inform and develop screening and treatment guidelines associated
with improvement in health care outcomes, and research priorities relevant
`(d) Eligibility for Designation as HHT Treatment Center of Excellence- In
carrying out subsection (c)(1), the Director may designate as an HHT Treatment
Center of Excellence only academic health centers demonstrating each of the
`(1) The academic health center possesses a team of medical experts capable
of providing comprehensive evaluation, treatment, and education to individuals
with known or suspected HHT and their health care providers.
`(2) The academic health center has sufficient personnel with knowledge
about HHT, or formal collaboration with one or more partnering organizations
for personnel or resources, to be able to--
`(A) respond in a coordinated, multidisciplinary way to patient inquiries;
`(B) coordinate evaluation, treatment, and education of patients and their
families in a timely manner.
`(3) The academic health center has the following personnel, facilities,
and patient volume:
`(A) A medical director with--
`(i) specialized knowledge of the main organ manifestations of HHT;
`(ii) the ability to coordinate the multidisciplinary diagnosis and
treatment of patients referred to the center.
`(B) Administrative staff with--
`(i) sufficient knowledge to respond to patient inquiries and coordinate
patient care in a timely fashion; and
`(ii) adequate financial support to allow the staff to commit at least
25 to 50 of their time on the job to HHT.
`(C) An otolaryngologist with experience and expertise in the treatment
of recurrent epistaxis in HHT patients.
`(D) An interventional radiologist with experience and expertise in the
treatment of pulmonary arteriovenous malformations (AVM).
`(E) A genetic counselor or geneticist with the expertise to provide HHT-specific
genetic counseling to patients and families.
`(F) On-site facilities to screen for all major organ manifestations of
`(G) A patient volume of at least 25 new HHT patients per year.
`(H) Established mechanisms to coordinate surveillance and outreach with
HHT patient advocacy organizations.'.
SEC. 6. ADDITIONAL HEALTH AND HUMAN SERVICES ACTIVITIES.
With respect to hereditary hemorrhagic telangiectasia (in this section referred
to as `HHT'), the Secretary of Health and Human Services, acting through the
Administrator of the Centers for Medicare & Medicaid Services, shall award
grants on a competitive basis--
(1) for an analysis by grantees of the Medicare Provider Analysis and Review
(MEDPAR) file to develop preliminary estimates on the total costs to the
Medicare program under title XVIII of the Scial Security Act for items,
services, and treatments for HHT furnished to individuals with HHT who are
entitled to benefits under part A of title XVIII of the Social Security
Act or enrolled under part B of such title; and
(2) to make recommendations regarding an enhanced data collection protocol
to permit a more precise determination of the total costs described in paragraph
SEC. 7. AUTHORIZATION OF APPROPRIATIONS.
(a) In General- To carry out section 409K of the Public Health Service Act
as added by section 4 of this Act, section 317U of the Public Health Service
Act as added by section 5 of this Act, and section 6 of this Act, there is
authorized to be appropriated $5,000,000 for each of fiscal years 2012 through
(b) Resource Center- Of the amount authorized to be appropriated under subsection
(a) for each of fiscal years 2012 through 2016, $1,000,000 shall be for carrying
out section 317U(a)(4) of the Public Health Service Act, as added by section
5 of this Act.
(c) Offset- There is authorized to be appropriated to the Department of Health
and Human Services for salaries and expenses of the Department for each of
fiscal years 2012 through 2016 the amount that is $5,000,000 less than the
amount appropriated for such salaries and expenses for fiscal year 2011.