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 dys-
trophy.
(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 manu-
facturers 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 pro-
vide 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 pro-
mote 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
H. R. 4014—2
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 clin-
ical 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 treat-
ments 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.