116 STAT. 1992 PUBLIC LAW 107–281—NOV. 6, 2002

Public Law 107–281

107th Congress

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 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

21 USC 360ee

note.

21 USC 301 note.

Rare Diseases

Orphan Product

Development Act

of 2002.

Nov. 6, 2002

[H.R. 4014]

PUBLIC LAW 107–281—NOV. 6, 2002 116 STAT. 1993

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.

Æ

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 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) 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.

Appropriation

authorization.