Rare Diseases Orphan Product Development Act of 2002
PL 107-281 (H.R.4014), enacted 11/06/2002

116 STAT. 1992 
PUBLIC LAW 107-281—NOV. 6, 2002 
Public Law 107-281 
107th Congress 
An Act 
Nov. 6, 2002 
To amend the Federal Food, Drug, and Cosmetic Act with respect to the development 
[H R 4014] 
^^ products for rare diseases. 
Be it enacted by the Senate and House of Representatives 
of 
Rare Diseases 
the United States of America in Congress assembled, 
Orphan Product 
Development Act 
SECTION 1. SHORT TITLE. 
2i^USC 301 note 
'^^^^ ^^^ "^^^ ^^ ^^^^^ ^^ ^^^ "Rare Diseases Orphan Product 
• Development Act of 2002". 
21 u s e 360ee 
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 
PUBLIC LAW 107-281—NOV. 6, 2002 
116 STAT. 1993 
United States and more than 800 additional drugs are in the 
research pipehne. 
(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 Appropriation 
authorized to be appropriated such sums as already have been authorization. 
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.