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<PRE>[DOCID: f:h4014ih.txt]






107th CONGRESS
  2d Session
                                H. R. 4014

 To amend the Federal Food, Drug, and Cosmetic Act with respect to the 
               development of products for rare diseases.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                             March 20, 2002

  Mr. Foley (for himself, Mr. Waxman, Mr. Shimkus, Mr. Brown of Ohio, 
   Mrs. Roukema, Mr. Rush, Mr. King, Mr. Greenwood, and Mr. Dingell) 
 introduced the following bill; which was referred to the Committee on 
                          Energy and Commerce

_______________________________________________________________________

                                 A BILL


 
 To amend the Federal Food, Drug, and Cosmetic Act with respect to the 
               development of products for rare diseases.

    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 Orphan Product 
Development 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.
            (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.

SEC. 3. FOOD AND DRUG ADMINISTRATION; GRANTS AND CONTRACTS FOR THE 
              DEVELOPMENT OF ORPHAN DRUGS.

    Subsection (c) of section 5 of the Orphan Drug Act (21 U.S.C. 
360ee(c)) is amended to read as follows:
    ``(c) 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.''.

SEC. 4. TECHNICAL AMENDMENT.

    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,''.
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