IB 
Union Calendar No. 435
107TH CONGRESS 
2D SESSION 
H. R. 4014
[Report No. 107–702] 
To amend the Federal Food, Drug, and Cosmetic Act with respect to the 
development of products for rare diseases. 
IN THE HOUSE OF REPRESENTATIVES 
MARCH 20, 2002
Mr. FOLEY (for himself, Mr. WAXMAN, Mr. SHIMKUS, Mr. BROWN of Ohio, 
Mrs. ROUKEMA, Mr. RUSH, Mr. KING, Mr. GREENWOOD, and Mr. DIN-
GELL) introduced the following bill; which was referred to the Committee 
on Energy and Commerce 
OCTOBER 1, 2002
Additional sponsors: Mr. MCGOVERN, Mr. HORN, Mr. SMITH of New Jersey, 
Ms. MCKINNEY, Mrs. MORELLA, Mr. WYNN, Mr. GREEN of Texas, Mr. 
TOWNS, Mr. FRANK, Mr. LYNCH, Mr. PALLONE, Ms. RIVERS, Mr. 
GEORGE
MILLER
of California, Mrs. CAPPS, Ms. DEGETTE, Mr. 
WELDON of Florida, Mr. HOEFFEL, Mr. LANTOS, Ms. DELAURO, Mr. 
STUPAK, Mrs. MINK of Hawaii, Mrs. KELLY, Ms. SLAUGHTER, Mr. 
MCDERMOTT, Ms. WOOLSEY, Mr. PLATTS, Ms. VELA´ZQUEZ, Mr. KIND, 
Mrs. JOHNSON of Connecticut, Mr. BONIOR, Mr. WEXLER, Ms. NORTON, 
Mr. LUCAS of Oklahoma, Mr. ISAKSON, Mrs. LOWEY, Ms. SCHAKOWSKY, 
Mr. KENNEDY of Rhode Island, Mr. COOKSEY, Mr. PRICE of North Caro-
lina, Mr. WOLF, Mr. DEUTSCH, Ms. CARSON of Indiana, Mr. SANDERS, 
Mr. WATT of North Carolina, Mr. ENGEL, Mr. JENKINS, Mr. BALDACCI, 
Ms. HARMAN, Mr. SAWYER, and Mr. TOM DAVIS of Virginia 
OCTOBER 1, 2002
Committed to the Committee of the Whole House on the State of the Union 
and ordered to be printed
A BILL 
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,’’.
Union Calendar No. 435
107TH CONGRESS 
2D SESSION 
H. R. 4014
[Report No. 107–702] 
A BILL 
To amend the Federal Food, Drug, and Cosmetic 
Act with respect to the development of products 
for rare diseases. 
OCTOBER 1, 2002
Committed to the Committee of the Whole House on the 
State of the Union and ordered to be printed