[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4014 Enrolled Bill (ENR)]

        H.R.4014

                      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 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,''.

                               Speaker of the House of Representatives.

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