Text: H.R.4014 — 107th Congress (2001-2002)All Information (Except Text)
Public Law No: 107-281 (11/06/2002)
[107th Congress Public Law 281]
[From the U.S. Government Printing Office]
[[Page 116 STAT. 1992]]
Public Law 107-281
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
(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) <<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.''.
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,''.
Approved November 6, 2002.
LEGISLATIVE HISTORY--H.R. 4014:
HOUSE REPORTS: No. 107-702 (Comm. on Energy and Commerce).
CONGRESSIONAL RECORD, Vol. 148 (2002):
Oct. 1, considered and passed House.
Oct. 17, considered and passed Senate.