[107th Congress Public Law 281]
[From the U.S. Government Printing Office]

[DOCID: f:publ281.107]

[[Page 116 STAT. 1992]]

Public Law 107-281
107th Congress

                                 An Act

 To amend the Federal Food, Drug, and Cosmetic Act with respect to the 
development of products for rare diseases. <<NOTE: Nov. 6, 2002 -  [H.R. 

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

SECTION 1. <<NOTE: 21 USC 301 note.>> SHORT TITLE.

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

SEC. 2. <<NOTE: 21 USC 360ee 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. 1993]]

        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.
            (7) The Food and Drug Administration supports small clinical 
        trials through Orphan Products Research Grants. Such grants 
        embody successful partnerships of government and industry, and 
        have led to the development of at least 23 drugs and four 
        medical devices for rare diseases and disorders. Yet the 
        appropriations in fiscal year 2001 for such grants were less 
        than in fiscal year 1995.

    (b) Purposes.--The purpose of this Act is to increase the national 
investment in the development of diagnostics and treatments for patients 
with rare diseases and disorders.


    Subsection (c) of section 5 of the Orphan Drug Act (21 U.S.C. 
360ee(c)) is amended to read as follows:
    ``(c) <<NOTE: Appropriation authorization.>> For grants and 
contracts under subsection (a), there are authorized to be appropriated 
such sums as already have been appropriated for fiscal year 2002, and 
$25,000,000 for each of the fiscal years 2003 through 2006.''.


    Section 527(a) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 360cc(a)) is amended in the matter following paragraph (2)--
            (1) by striking ``, of such certification,''; and
            (2) by striking ``, the issuance of the certification,''.

    Approved November 6, 2002.


HOUSE REPORTS: No. 107-702 (Comm. on Energy and Commerce).
            Oct. 1, considered and passed House.
            Oct. 17, considered and passed Senate.