S 1167 112th
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
June 9, 2011
JOHNSON of South Dakota (for himself and Mr. BINGAMAN) introduced the following
bill; which was read twice and referred to the Committee on Health, Education,
Labor, and Pensions
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.
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 percent of individuals with HHT caused by artery-vein malformations in the
lung (due to lack of capillaries between the arterial and venous systems which
prevent or normally filter out clots and bacteria), causing disability and sudden
(2) One in 5,000 American children and
adults suffer from HHT.
(3) Studies have found an increase
in morbidity and mortality rate 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.
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.
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
SEC. 3. 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 interventions.
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
`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 on--
research on the causes, diagnosis, and treatment of HHT, including through the
conduct or support of such research; and
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
`(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
`(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 Human Services.
`(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 of HHT;
`(ii) training programs on HHT for scientists and health professionals; and
`(iii) HHT genetic testing research to improve the accuracy of genetic testing.
`(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.'.
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
`(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 cost-benefit
`(3) The development of a standardized survey
and screening tool on family history.
`(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
`(c) Population Screening- In carrying out population
screening under subsection (a)(1), the Director shall--
designate and provide funding for a sufficient number of HHT Treatment Centers
of Excellence to improve patient access to information, treatment, and care by
`(2) conduct surveillance through a regional
population study, supplemented by sentinel health care provider or center surveillance,
and 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
`(4) disseminate data collected under paragraph
(2)(B) to the Paul Coverdell National Acute Stroke Registry, to be utilized for
analyses of natural history of hemorrhagic and embolic stroke in HHT, and to develop
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
`(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 the universal data collection system used
by the Centers for monitoring hemophilia with the blood disorders and 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 Excellence
patients to learn about the natural history of HHT, the efficacy of various treatment
modalities, and 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 following:
`(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
`(2) The academic health center has sufficient
personnel with knowledge about HHT, or formal collaboration with partnering organizations
for personnel or resources, to be able to--
respond in a coordinated, multidisciplinary way to patient inquiries; and
`(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--
knowledge of the main organ manifestations of HHT; and
`(ii) the ability to coordinate the multidisciplinary diagnosis and treatment
of patients referred to the center.
Administrative staff with--
knowledge to respond to patient inquiries and coordinate patient care in a timely
`(ii) adequate financial
support to allow the staff to commit at least 25 to 50 percent 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 HHT.
`(G) A patient volume of at least 25 new HHT patients per year.
`(H) Established mechanisms to coordinate surveillance and outreach with HHT patient
SEC. 6. ADDITIONAL HEALTH AND
HUMAN SERVICES ACTIVITIES.
With respect to hereditary hemorrhagic
telangiectasia (in this sec 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 Social 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;
(2) to make recommendations regarding an enhanced
data collection protocol to permit a more precise determination of the total costs
described in paragraph (1).
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 2016.
(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.