PUBLIC LAW 107–84—DEC. 18, 2001 115 STAT. 823

Public Law 107–84

107th Congress

An Act

To amend the Public Health Service Act to provide for research with respect to

various forms of muscular dystrophy, including Duchenne, Becker, limb girdle,

congenital, facioscapulohumeral, myotonic, oculopharyngeal, distal, and EmeryDreifuss muscular dystrophies.

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 ‘‘Muscular Dystrophy Community

Assistance, Research and Education Amendments of 2001’’, or the

‘‘MD–CARE Act’’.

SEC. 2. FINDINGS.

Congress makes the following findings:

(1) Of the childhood muscular dystrophies, Duchenne Muscular Dystrophy (DMD) is the world’s most common and catastrophic form of genetic childhood disease, and is characterized

by a rapidly progressive muscle weakness that almost always

results in death, usually by 20 years of age.

(2) Duchenne muscular dystrophy is genetically inherited,

and mothers are the carriers in approximately 70 percent of

all cases.

(3) If a female is a carrier of the dystrophin gene, there

is a 50 percent chance per birth that her male offspring will

have Duchenne muscular dystrophy, and a 50 percent chance

per birth that her female offspring will be carriers.

(4) Duchenne is the most common lethal genetic disorder

of childhood worldwide, affecting approximately 1 in every 3,500

boys worldwide.

(5) Children with muscular dystrophy exhibit extreme

symptoms of weakness, delay in walking, waddling gait, difficulty in climbing stairs, and progressive mobility problems

often in combination with muscle hypertrophy.

(6) Other forms of muscular dystrophy affecting children

and adults include Becker, limb girdle, congenital,

facioscapulohumeral, myotonic, oculopharyngeal, distal, and

Emery-Dreifuss muscular dystrophies.

(7) Myotonic muscular dystrophy (also known as Steinert’s

disease and dystrophia myotonica) is the second most prominent

form of muscular dystrophy and the type most commonly found

in adults. Unlike any of the other muscular dystrophies, the

muscle weakness is accompanied by myotonia (delayed relaxation of muscles after contraction) and by a variety of abnormalities in addition to those of muscle.

42 USC 247b–18

note.

42 USC 201 note.

Muscular

Dystrophy

Community

Assistance,

Research and

Education

Amendments of

2001.

Dec. 18, 2001

[H.R. 717]

115 STAT. 824 PUBLIC LAW 107–84—DEC. 18, 2001

(8) Facioscapulohumeral muscular dystrophy (referred to

in this section as ‘‘FSHD’’) is a neuromuscular disorder that

is inherited genetically and has an estimated frequency of

1 in 20,000. FSHD, affecting between 15,000 to 40,000 persons,

causes a progressive and sever loss of skeletal muscle gradually

bringing weakness and reduced mobility. Many persons with

FSHD become severely physically disabled and spend many

decades in a wheelchair.

(9) FSHD is regarded as a novel genetic phenomenon

resulting from a crossover of subtelomeric DNA and may be

the only human disease caused by a deletion-mutation.

(10) Each of the muscular dystrophies, though distinct

in progressivity and severity of symptoms, have a devastating

impact on tens of thousands of children and adults throughout

the United States and worldwide and impose severe physical

and economic burdens on those affected.

(11) Muscular dystrophies have a significant impact on

quality of life—not only for the individual who experiences

its painful symptoms and resulting disability, but also for family

members and caregivers.

(12) Development of therapies for these disorders, while

realistic with recent advances in research, is likely to require

costly investments and infrastructure to support gene and other

therapies.

(13) There is a shortage of qualified researchers in the

field of neuromuscular research.

(14) Many family physicians and health care professionals

lack the knowledge and resources to detect and properly

diagnose the disease as early as possible, thus exacerbating

the progressiveness of symptoms in cases that go undetected

or misdiagnosed.

(15) There is a need for efficient mechanisms to translate

clinically relevant findings in muscular dystrophy research from

basic science to applied work.

(16) Educating the public and health care community

throughout the country about this devastating disease is of

paramount importance and is in every respect in the public

interest and to the benefit of all communities.

SEC. 3. EXPANSION, INTENSIFICATION, AND COORDINATION OF

ACTIVITIES OF NATIONAL INSTITUTES OF HEALTH WITH

RESPECT TO RESEARCH ON MUSCULAR DYSTROPHY.

Part A of title IV of the Public Health Service Act (42 U.S.C.

281 et seq.) is amended by adding at the end the following:

‘‘SEC. 404E. MUSCULAR DYSTROPHY; INITIATIVE THROUGH DIRECTOR

OF NATIONAL INSTITUTES OF HEALTH.

‘‘(a) EXPANSION, INTENSIFICATION, AND COORDINATION OF

ACTIVITIES.—

‘‘(1) IN GENERAL.—The Director of NIH, in coordination

with the Directors of the National Institute of Neurological

Disorders and Stroke, the National Institute of Arthritis and

Muscoskeletal and Skin Diseases, the National Institute of

Child Health and Human Development, and the other national

research institutes as appropriate, shall expand and intensify

programs of such Institutes with respect to research and related

activities concerning various forms of muscular dystrophy,

including Duchenne, myotonic, facioscapulohumeral muscular

42 USC 283g.

PUBLIC LAW 107–84—DEC. 18, 2001 115 STAT. 825

dystrophy (referred to in this section as ‘FSHD’) and other

forms of muscular dystrophy.

‘‘(2) COORDINATION.—The Directors referred to in paragraph (1) shall jointly coordinate the programs referred to

in such paragraph and consult with the Muscular Dystrophy

Interagency Coordinating Committee established under section

6 of the MD–CARE Act.

‘‘(3) ALLOCATIONS BY DIRECTOR OF NIH.—The Director of

NIH shall allocate the amounts appropriated to carry out this

section for each fiscal year among the national research

institutes referred to in paragraph (1).

‘‘(b) CENTERS OF EXCELLENCE.—

‘‘(1) IN GENERAL.—The Director of NIH shall award grants

and contracts under subsection (a)(1) to public or nonprofit

private entities to pay all or part of the cost of planning,

establishing, improving, and providing basic operating support

for centers of excellence regarding research on various forms

of muscular dystrophy.

‘‘(2) RESEARCH.—Each center under paragraph (1) shall

supplement but not replace the establishment of a comprehensive research portfolio in all the muscular dystrophies. As a

whole, the centers shall conduct basic and clinical research

in all forms of muscular dystrophy including early detection,

diagnosis, prevention, and treatment, including the fields of

muscle biology, genetics, noninvasive imaging, genetics,

pharmacological and other therapies.

‘‘(3) COORDINATION OF CENTERS; REPORTS.—The Director

of NIH—

‘‘(A) shall, as appropriate, provide for the coordination

of information among centers under paragraph (1) and

ensure regular communication between such centers; and

‘‘(B) shall require the periodic preparation of reports

on the activities of the centers and the submission of the

reports to the Director.

‘‘(4) ORGANIZATION OF CENTERS.—Each center under paragraph (1) shall use the facilities of a single institution, or

be formed from a consortium of cooperating institutions,

meeting such requirements as may be prescribed by the

Director of NIH.

‘‘(5) DURATION OF SUPPORT.—Support for a center established under paragraph (1) may be provided under this section

for a period of not to exceed 5 years. Such period may be

extended for 1 or more additional periods not exceeding 5

years if the operations of such center have been reviewed

by an appropriate technical and scientific peer review group

established by the Director of NIH and if such group has

recommended to the Director that such period should be

extended.

‘‘(c) FACILITATION OF RESEARCH.—The Director of NIH shall

provide for a program under subsection (a)(1) under which samples

of tissues and genetic materials that are of use in research on

muscular dystrophy are donated, collected, preserved, and made

available for such research. The program shall be carried out in

accordance with accepted scientific and medical standards for the

donation, collection, and preservation of such samples.

‘‘(d) COORDINATING COMMITTEE.—

Grants.

Contracts.

115 STAT. 826 PUBLIC LAW 107–84—DEC. 18, 2001

‘‘(1) IN GENERAL.—The Secretary shall establish the Muscular Dystrophy Coordinating Committee (referred to in this

section as the ‘Coordinating Committee’) to coordinate activities

across the National Institutes and with other Federal health

programs and activities relating to the various forms of muscular dystrophy.

‘‘(2) COMPOSITION.—The Coordinating Committee shall consist of not more than 15 members to be appointed by the

Secretary, of which— ‘‘(A) 2⁄3 of such members shall represent governmental

agencies, including the directors or their designees of each

of the national research institutes involved in research

with respect to muscular dystrophy and representatives

of all other Federal departments and agencies whose programs involve health functions or responsibilities relevant

to such diseases, including the Centers for Disease Control

and Prevention, the Health Resources and Services

Administration and the Food and Drug Administration and

representatives of other governmental agencies that serve

children with muscular dystrophy, such as the Department

of Education; and

‘‘(B) 1⁄3 of such members shall be public members,

including a broad cross section of persons affected with

muscular dystrophies including parents or legal guardians,

affected individuals, researchers, and clinicians.

Members appointed under subparagraph (B) shall serve for

a term of 3 years, and may serve for an unlimited number

of terms if reappointed.

‘‘(3) CHAIR.—

‘‘(A) IN GENERAL.—With respect to muscular dystrophy,

the Chair of the Coordinating Committee shall serve as

the principal advisor to the Secretary, the Assistant Secretary for Health, and the Director of NIH, and shall provide advice to the Director of the Centers for Disease

Control and Prevention, the Commissioner of Food and

Drugs, and to the heads of other relevant agencies. The

Coordinating Committee shall select the Chair for a term

not to exceed 2 years.

‘‘(B) APPOINTMENT.—The Chair of the Committee shall

be appointed by and be directly responsible to the Secretary.

‘‘(4) ADMINISTRATIVE SUPPORT; TERMS OF SERVICE; OTHER

PROVISIONS.—The following shall apply with respect to the

Coordinating Committee:

‘‘(A) The Coordinating Committee shall receive necessary and appropriate administrative support from the

Department of Health and Human Services.

‘‘(B) The Coordinating Committee shall meet as appropriate as determined by the Secretary, in consultation with

the chair.

‘‘(e) PLAN FOR HHS ACTIVITIES.— ‘‘(1) IN GENERAL.—Not later than 1 year after the date

of enactment of this section, the Coordinating Committee shall

develop a plan for conducting and supporting research and

education on muscular dystrophy through the national research

institutes and shall periodically review and revise the plan.

The plan shall—

Deadline.

Applicability.

Establishment.

PUBLIC LAW 107–84—DEC. 18, 2001 115 STAT. 827

‘‘(A) provide for a broad range of research and education activities relating to biomedical, epidemiological,

psychosocial, and rehabilitative issues, including studies

of the impact of such diseases in rural and underserved

communities;

‘‘(B) identify priorities among the programs and activities of the National Institutes of Health regarding such

diseases; and

‘‘(C) reflect input from a broad range of scientists,

patients, and advocacy groups.

‘‘(2) CERTAIN ELEMENTS OF PLAN.—The plan under paragraph (1) shall, with respect to each form of muscular dystrophy, provide for the following as appropriate: ‘‘(A) Research to determine the reasons underlying the

incidence and prevalence of various forms of muscular dystrophy. ‘‘(B) Basic research concerning the etiology and genetic

links of the disease and potential causes of mutations. ‘‘(C) The development of improved screening techniques. ‘‘(D) Basic and clinical research for the development

and evaluation of new treatments, including new biological

agents. ‘‘(E) Information and education programs for health

care professionals and the public. ‘‘(f) REPORTS TO CONGRESS.—The Coordinating Committee shall

biennially submit to the Committee on Energy and Commerce of

the House of Representatives, and the Committee on Health, Education, Labor, and Pensions of the Senate, a report that describes

the research, education, and other activities on muscular dystrophy

being conducted or supported through the Department of Health

and Human Services. Each such report shall include the following:

‘‘(1) The plan under subsection (e)(1) (or revisions to the

plan, as the case may be).

‘‘(2) Provisions specifying the amounts expended by the

Department of Health and Human Services with respect to

various forms of muscular dystrophy, including Duchenne,

myotonic, FSHD and other forms of muscular dystrophy.

‘‘(3) Provisions identifying particular projects or types of

projects that should in the future be considered by the national

research institutes or other entities in the field of research

on all muscular dystrophies.

‘‘(g) PUBLIC INPUT.—The Secretary shall, under subsection

(a)(1), provide for a means through which the public can obtain

information on the existing and planned programs and activities

of the Department of Health and Human Services with respect

to various forms of muscular dystrophy and through which the

Secretary can receive comments from the public regarding such

programs and activities.

‘‘(h) AUTHORIZATION OF APPROPRIATIONS.—For the purpose of

carrying out this section, there are authorized to be appropriated

such sums as may be necessary for each of fiscal years 2002 through

2006. The authorization of appropriations established in the preceding sentence is in addition to any other authorization of appropriations that is available for conducting or supporting through

the National Institutes of Health research and other activities with

respect to muscular dystrophy.’’.

115 STAT. 828 PUBLIC LAW 107–84—DEC. 18, 2001

SEC. 4. DEVELOPMENT AND EXPANSION OF ACTIVITIES OF CENTERS

FOR DISEASE CONTROL AND PREVENTION WITH

RESPECT TO EPIDEMIOLOGICAL RESEARCH ON MUSCULAR DYSTROPHY.

Part B of title III of the Public Health Service Act (42 U.S.C.

243 et seq.) is amended by inserting after section 317P the following:

‘‘SEC. 317Q. SURVEILLANCE AND RESEARCH REGARDING MUSCULAR

DYSTROPHY.

‘‘(a) IN GENERAL.—The Secretary, acting through the Director

of the Centers for Disease Control and Prevention, may award

grants and cooperative agreements to public or nonprofit private

entities (including health departments of States and political subdivisions of States, and including universities and other educational

entities) for the collection, analysis, and reporting of data on

Duchenne and other forms of muscular dystrophy. In making such

awards, the Secretary may provide direct technical assistance in

lieu of cash.

‘‘(b) NATIONAL MUSCULAR DYSTROPHY EPIDEMIOLOGY PROGRAM.—The Secretary, acting through the Director of the Centers

for Disease Control and Prevention, may award grants to public

or nonprofit private entities (including health departments of States

and political subdivisions of States, and including universities and

other educational entities) for the purpose of carrying out

epidemiological activities regarding Duchenne and other forms of

muscular dystrophies, including collecting and analyzing information on the number, incidence, correlates, and symptoms of cases.

In carrying out the preceding sentence, the Secretary shall provide

for a national surveillance program. In making awards under this

subsection, the Secretary may provide direct technical assistance

in lieu of cash.

‘‘(c) COORDINATION WITH CENTERS OF EXCELLENCE.—The Secretary shall ensure that epidemiological information under subsections (a) and (b) is made available to centers of excellence supported under section 404E(b) by the Director of the National

Institutes of Health.

‘‘(d) AUTHORIZATION OF APPROPRIATIONS.—There are authorized

to be appropriated such sums as may be necessary to carry out

this section.’’.

SEC. 5. INFORMATION AND EDUCATION.

(a) IN GENERAL.—The Secretary of Health and Human Services

(referred to in this Act as the ‘‘Secretary’’) shall establish and

implement a program to provide information and education on

muscular dystrophy to health professionals and the general public,

including information and education on advances in the diagnosis

and treatment of muscular dystrophy and training and continuing

education through programs for scientists, physicians, medical students, and other health professionals who provide care for patients

with muscular dystrophy.

(b) STIPENDS.—The Secretary may use amounts made available

under this section provides stipends for health professionals who

are enrolled in training programs under this section.

(c) AUTHORIZATION OF APPROPRIATIONS.—There are authorized

to be appropriated such sums as may be necessary to carry out

this section.

42 USC 247b–19.

42 USC 247b–18.

PUBLIC LAW 107–84—DEC. 18, 2001 115 STAT. 829

SEC. 6. REPORT TO CONGRESS.

Not later than January 1, 2003, and each January 1 thereafter,

the Secretary shall prepare and submit to the appropriate committees of Congress, a report concerning the implementation of this

Act and the amendments made by this Act.

SEC. 7. STUDY ON THE USE OF CENTERS OF EXCELLENCE AT THE

NATIONAL INSTITUTES OF HEALTH.

(a) REVIEW.—Not later than 60 days after the date of enactment

of this Act, the Secretary of Health and Human Services shall

enter into a contract with the Institute of Medicine for the purpose

of conducting a study and making recommendations on the impact

of, need for, and other issues associated with Centers of Excellence

at the National Institutes of Health.

(b) AREAS OF REVIEW.—In conducting the study under subsection (a), the Institute of Medicine shall at a minimum consider

the following:

(1) The current areas of research incorporating Centers

of Excellence (which shall include a description of such areas)

and the relationship of this form of funding mechanism to

other forms of funding for research grants, including investigator initiated research, contracts and other types of research

support awards.

(2) The distinctive aspects of Centers of Excellence,

including the additional knowledge that may be expected to

be gained through Centers of Excellence as compared to other

forms of grant or contract mechanisms.

(3) The costs associated with establishing and maintaining

Centers of Excellence, and the record of scholarship and

training resulting from such Centers. The research and training

contributions of Centers should be assessed on their own merits

and in comparison with other forms of research support.

(4) Specific areas of research in which Centers of Excellence

may be useful, needed, or underused, as well as areas of

research in which Centers of Excellence may not be helpful.

(5) Criteria that may be applied in determining when Centers of Excellence are an appropriate and cost-effective research

investment and conditions that should be present in order

to consider the establishment of Centers of Excellence.

(6) Alternative research models that may accomplish

results similar to or greater than Centers of Excellence.

(c) REPORT.—Not later than 1 year after the date on which

the contract is entered into under subsection (a), the Institute

of Medicine shall complete the study under such subsection and

submit a report to the Secretary of Health and Human Services

Deadline.

Deadline.

Contracts.

42 USC 281 note.

Deadline.

42 USC 247b–18

note.

115 STAT. 830 PUBLIC LAW 107–84—DEC. 18, 2001

LEGISLATIVE HISTORY—H.R. 717:

HOUSE REPORTS: No. 107–195 (Comm. on Energy and Commerce).

CONGRESSIONAL RECORD, Vol. 147 (2001):

Sept. 24, considered and passed House.

Nov. 15, considered and passed Senate, amended.

Nov. 29, House concurred in Senate amendment.

Æ

and the appropriate committees of Congress that contains the

results of such study.

Approved December 18, 2001.