[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4013 Enrolled Bill (ENR)]

        H.R.4013

                      One Hundred Seventh Congress

                                 of the

                        United States of America


                          AT THE SECOND SESSION

         Begun and held at the City of Washington on Wednesday,
          the twenty-third day of January, two thousand and two


                                 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 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.
            ``(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 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.''.

                               Speaker of the House of Representatives.

                            Vice President of the United States and    
                                               President of the Senate.