107TH CONGRESS
2D SESSION
H. R. 4014
AN ACT
To amend the Federal Food, Drug, and Cosmetic
Act with respect to the development of products
for rare diseases.
107TH CONGRESS
2D SESSION
H. R. 4014
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 Representa-
tives 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 find-
ings:
(1) Rare diseases and disorders are those which
affect small patient populations, typically popu-
lations 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 dis-
orders were denied access to effective medicines be-
cause 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 treat-
ments. 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 Con-
gress for legislation to encourage the development of
orphan drugs.
(4) The Orphan Drug Act created financial in-
centives for the research and production of such or-
phan drugs. New Federal programs at the National
Institutes of Health and the Food and Drug Admin-
istration encouraged clinical research and commer-
cial product development for products that target
rare diseases. An Orphan Products Board was estab-
lished to promote the development of drugs and de-
vices 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 re-
search pipeline.
(6) Despite the tremendous success of the Or-
phan Drug Act, rare diseases and disorders deserve
greater emphasis in the national biomedical research
enterprise.
(7) The Food and Drug Administration sup-
ports 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 in-
crease 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 OR-
PHAN 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 al-
ready 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 Cos-
metic 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 certifi-
cation,’’.
Passed the House of Representatives October 1,
2002.
Attest:
Clerk.