```

[107th Congress Public Law 280]
[From the U.S. Government Printing Office]

<DOC>
[DOCID: f:publ280.107]

[[Page 1987]]

                        RARE DISEASES ACT OF 2002

[[Page 116 STAT. 1988]]

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. <<NOTE: Nov. 6, 2002 -  [H.R. 4013]>> 

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress <<NOTE: Rare Diseases Act of 
2002.>> assembled,

SECTION 1. <<NOTE: 42 USC 201 note.>> SHORT TITLE.

    This Act may be cited as the ``Rare Diseases Act of 2002''.

SEC. 2. <<NOTE: 42 USC 283h note.>> 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

[[Page 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. <<NOTE: 42 USC 283h.>> (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.

[[Page 116 STAT. 1990]]

                    ``(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) <<NOTE: Reports.>> 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) <<NOTE: Reports.>> 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. <<NOTE: 42 USC 283i.>> (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

[[Page 116 STAT. 1991]]

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 Agreements 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.

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.

                                  <all>
```