PUBLIC LAW 107–280—NOV. 6, 2002

RARE DISEASES ACT OF 2002

116 STAT. 1988 PUBLIC LAW 107–280—NOV. 6, 2002

Public Law 107–280

107th Congress

An Act

To amend the Public Health Service Act to establish an Office of Rare Diseases

at the National Institutes of Health, 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 ‘‘Rare Diseases Act of 2002’’.

SEC. 2. FINDINGS AND PURPOSES.

(a) FINDINGS.—Congress makes the following findings:

(1) Rare diseases and disorders are those which affect

small patient populations, typically populations smaller than

200,000 individuals in the United States. Such diseases and

conditions include Huntington’s disease, amyotrophic lateral

sclerosis (Lou Gehrig’s disease), Tourette syndrome, Crohn’s

disease, cystic fibrosis, cystinosis, and Duchenne muscular dystrophy.

(2) For many years, the 25,000,000 Americans suffering

from the over 6,000 rare diseases and disorders were denied

access to effective medicines because prescription drug manufacturers could rarely make a profit from marketing drugs

for such small groups of patients. The prescription drug

industry did not adequately fund research into such treatments.

Despite the urgent health need for these medicines, they came

to be known as ‘‘orphan drugs’’ because no companies would

commercialize them.

(3) During the 1970s, an organization called the National

Organization for Rare Disorders (NORD) was founded to provide services and to lobby on behalf of patients with rare

diseases and disorders. NORD was instrumental in pressing

Congress for legislation to encourage the development of orphan

drugs.

(4) The Orphan Drug Act created financial incentives for

the research and production of such orphan drugs. New Federal

programs at the National Institutes of Health and the Food

and Drug Administration encouraged clinical research and

commercial product development for products that target rare

diseases. An Orphan Products Board was established to promote the development of drugs and devices for rare diseases

or disorders.

(5) Before 1983, some 38 orphan drugs had been developed.

Since the enactment of the Orphan Drug Act, more than 220

new orphan drugs have been approved and marketed in the

42 USC 283h

note.

42 USC 201 note.

Rare Diseases

Act of 2002.

Nov. 6, 2002

[H.R. 4013]

PUBLIC LAW 107–280—NOV. 6, 2002 116 STAT. 1989

United States and more than 800 additional drugs are in the

research pipeline.

(6) Despite the tremendous success of the Orphan Drug

Act, rare diseases and disorders deserve greater emphasis in

the national biomedical research enterprise. The Office of Rare

Diseases at the National Institutes of Health was created in

1993, but lacks a statutory authorization.

(7) The National Institutes of Health has received a

substantial increase in research funding from Congress for

the purpose of expanding the national investment of the United

States in behavioral and biomedical research.

(8) Notwithstanding such increases, funding for rare diseases and disorders at the National Institutes of Health has

not increased appreciably.

(9) To redress this oversight, the Department of Health

and Human Services has proposed the establishment of a network of regional centers of excellence for research on rare

diseases.

(b) PURPOSES.—The purposes of this Act are to—

(1) amend the Public Health Service Act to establish an

Office of Rare Diseases at the National Institutes of Health;

and

(2) increase the national investment in the development

of diagnostics and treatments for patients with rare diseases

and disorders.

SEC. 3. NIH OFFICE OF RARE DISEASES AT NATIONAL INSTITUTES

OF HEALTH.

Title IV of the Public Health Service Act (42 U.S.C. 281 et

seq.), as amended by Public Law 107–84, is amended by inserting

after section 404E the following:

‘‘OFFICE OF RARE DISEASES

‘‘SEC. 404F. (a) ESTABLISHMENT.—There is established within

the Office of the Director of NIH an office to be known as the

Office of Rare Diseases (in this section referred to as the ‘Office’),

which shall be headed by a Director (in this section referred to

as the ‘Director’), appointed by the Director of NIH.

‘‘(b) DUTIES.—

‘‘(1) IN GENERAL.—The Director of the Office shall carry

out the following:

‘‘(A) The Director shall recommend an agenda for conducting and supporting research on rare diseases through

the national research institutes and centers. The agenda

shall provide for a broad range of research and education

activities, including scientific workshops and symposia to

identify research opportunities for rare diseases.

‘‘(B) The Director shall, with respect to rare diseases,

promote coordination and cooperation among the national

research institutes and centers and entities whose research

is supported by such institutes.

‘‘(C) The Director, in collaboration with the directors

of the other relevant institutes and centers of the National

Institutes of Health, may enter into cooperative agreements

with and make grants for regional centers of excellence

on rare diseases in accordance with section 404G.

42 USC 283h.

116 STAT. 1990 PUBLIC LAW 107–280—NOV. 6, 2002

‘‘(D) The Director shall promote the sufficient allocation

of the resources of the National Institutes of Health for

conducting and supporting research on rare diseases. ‘‘(E) The Director shall promote and encourage the

establishment of a centralized clearinghouse for rare and

genetic disease information that will provide understandable information about these diseases to the public, medical

professionals, patients and families. ‘‘(F) The Director shall biennially prepare a report

that describes the research and education activities on

rare diseases being conducted or supported through the

national research institutes and centers, and that identifies

particular projects or types of projects that should in the

future be conducted or supported by the national research

institutes and centers or other entities in the field of

research on rare diseases. ‘‘(G) The Director shall prepare the NIH Director’s

annual report to Congress on rare disease research conducted by or supported through the national research

institutes and centers.

‘‘(2) PRINCIPAL ADVISOR REGARDING ORPHAN DISEASES.— With respect to rare diseases, the Director shall serve as the

principal advisor to the Director of NIH and shall provide

advice to other relevant agencies. The Director shall provide

liaison with national and international patient, health and

scientific organizations concerned with rare diseases.

‘‘(c) DEFINITION.—For purposes of this section, the term ‘rare

disease’ means any disease or condition that affects less than

200,000 persons in the United States.

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

carrying out this section, there are authorized to be appropriated

such sums as already have been appropriated for fiscal year 2002,

and $4,000,000 for each of the fiscal years 2003 through 2006.’’.

SEC. 4. RARE DISEASE REGIONAL CENTERS OF EXCELLENCE.

Title IV of the Public Health Service Act (42 U.S.C. 281 et

seq.), as amended by section 3, is further amended by inserting

after section 404F the following:

‘‘RARE DISEASE REGIONAL CENTERS OF EXCELLENCE

‘‘SEC. 404G. (a) COOPERATIVE AGREEMENTS AND GRANTS.—

‘‘(1) IN GENERAL.—The Director of the Office of Rare Diseases (in this section referred to as the ‘Director’), in collaboration with the directors of the other relevant institutes and

centers of the National Institutes of Health, may enter into

cooperative agreements with and make grants to public or

private nonprofit entities to pay all or part of the cost of

planning, establishing, or strengthening, and providing basic

operating support for regional centers of excellence for clinical

research into, training in, and demonstration of diagnostic,

prevention, control, and treatment methods for rare diseases.

‘‘(2) POLICIES.—A cooperative agreement or grant under

paragraph (1) shall be entered into in accordance with policies

established by the Director of NIH.

‘‘(b) COORDINATION WITH OTHER INSTITUTES.—The Director

shall coordinate the activities under this section with similar activities conducted by other national research institutes, centers and

42 USC 283i.

Reports.

Reports.

PUBLIC LAW 107–280—NOV. 6, 2002 116 STAT. 1991

LEGISLATIVE HISTORY—H.R. 4013 (S. 1379):

HOUSE REPORTS: No. 107–543 (Comm. on Energy and Commerce).

SENATE REPORTS: No. 107–129 accompanyng S. 1379 (Comm. on Health, Education, Labor, and Pensions).

CONGRESSIONAL RECORD, Vol. 148 (2002):

Oct. 1, considered and passed House.

Oct. 17, considered and passed Senate.

Æ

agencies of the National Institutes of Health and by the Food

and Drug Administration to the extent that such institutes, centers

and agencies have responsibilities that are related to rare diseases. ‘‘(c) USES FOR FEDERAL PAYMENTS UNDER COOPERATIVE AGREE- MENTS OR GRANTS.—Federal payments made under a cooperative

agreement or grant under subsection (a) may be used for— ‘‘(1) staffing, administrative, and other basic operating

costs, including such patient care costs as are required for

research; ‘‘(2) clinical training, including training for allied health

professionals, continuing education for health professionals and

allied health professions personnel, and information programs

for the public with respect to rare diseases; and ‘‘(3) clinical research and demonstration programs. ‘‘(d) PERIOD OF SUPPORT; ADDITIONAL PERIODS.—Support of a

center under subsection (a) may be for a period of not to exceed

5 years. Such period may be extended by the Director for additional

periods of not more than 5 years if the operations of such center

have been reviewed by an appropriate technical and scientific peer

review group established by the Director and if such group has

recommended to the Director that such period should be extended. ‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—For the purpose of

carrying out this section, there are authorized to be appropriated

such sums as already have been appropriated for fiscal year 2002,

and $20,000,000 for each of the fiscal years 2003 through 2006.’’.

Approved November 6, 2002.