Orphan Drug Amendments of 1988
PL 100-290 (H.R.3459), enacted 04/18/1988

102 STAT. 90 
PUBLIC LAW 100-290—APR. 18, 1988 
Public Law 100-290 
100th Congress 
An Act 
Apr. 18, 1988 
To amend the Federal Food, Drug, and Cosmetic Act to revise the provisions 
[H.R. 3459] 
respecting orphan drugs, and for other purposes. 
Be it enacted by the Senate and House of Representatives of the 
Orphan Drug 
United States of America in Congress assembled, 
Amendments of 
1988. 
SECTION 1. SHORT TITLE. 
21 use 301 note. 
'^^^^ ^^^ 
^^V 
^^ cited as the "Orphan Drug Amendments of 
19oo . 
SEC. 2. DESIGNATION AS AN ORPHAN DRUG. 
(a) REQUEST.—Section 526(a)(1) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 360bb(a)(l)) is amended by adding after the 
first sentence the following: "A request for designation of a drug 
shall be made before the submission of an application under section 
505(b) for the drug, the submission of an application for certification 
of the drug under section 507, or the submission of an application for 
licensing of the drug under section 351 of the Public Health Service 
Act". 
(b) DISCONTINUANCE.—Section 526 of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 360bb) is amended by redesignating subsec-
tions (b) and (c) as subsections (c) and (d), respectively, and by adding 
after subsection (a) the following: 
"(b) A designation of a drug under subsection (a) shall be subject 
to the condition that— 
"(1) if an application was approved for the drug under section 
505(b), a certificate was issued for the drug under section 507, or 
a license was issued for the drug under section 351 of the Public 
Health Service Act, the manufacturer of the drug will notify the 
Secretary of any discontinuance of the production of the drug at 
least one year before discontinuance, and 
"(2) if an application has not been approved for the drug 
under section 505(b), a certificate has not been issued for the 
drug under section 507, or a license has not been issued for the 
drug under section 351 of the Public Health Service Act and if 
preclinical investigations or investigations under section 505(i) 
are being conducted with the drug, the manufacturer or sponsor 
of the drug will notify the Secretary of any decision to dis-
continue active pursuit of approval of an application under 
section 505(b), approval of an application for certification under 
section 507, or approval of a license under section 351 of the 
Public Health Service Act.". 
SEC. 3. FINANCIAL ASSISTANCE. 
(a) MEDICAL DEVICES.—Section 5 of the Orphan Drug Act (21 
U.S.C. 360ee) is amended— 
(1) in subsection (a), by inserting "(1)" after "assist in" and by 
inserting before the period a comma and "(2) defraying the costs 
PUBLIC LAW 100-290—APR. 18, 1988 
102 STAT. 91 
of developing medical devices for rare diseases or conditions", 
and 
(2) in subsection (b)(2)— 
(A) by inserting "(1) in the case of a drug," after "means" 
in the first sentence and by adding before the period in that 
sentence a comma and "(2) in the case of a medical device, 
any disease or condition that occurs so infrequently in the 
United States that there is no reasonable expectation that a 
medical device for such disease or condition will be devel-
oped without assistance under subsection (a)", and 
(B) by striking out "under this subsection" in the last 
sentence and inserting in lieu thereof "under section 526 of 
the Federal Food, Drug, and Cosmetic Act". 
(b) MEDICAL FOODS.—Section 5 of the Orphan Drug Act (21 U.S.C. 
360ee) is amended— 
(1) in subsection (a) (as amended by subsection (a)), by insert-
ing before the period a comma and "and (3) defraying the costs 
of developing medical foods for rare diseases or conditions", 
(2) in subsection (b)(2) (as amended by subsection (a)), by 
inserting before the period at the end of the first sentence a 
comma and "and (3) in the case of a medical food, any disease or 
condition that occurs so infrequently in the United States that 
there is no reasonable expectation that a medical food for such 
disease or condition will be developed without assistance under 
subsection (a)", and 
(3) by adding at the end of subsection (b) the following: 
"(3) The term 'medical food' means a food which is formulated 
to be consumed or administered enterally under the supervision 
of a physician and which is intended for the specific dietary 
management of a disease or condition for which distinctive 
nutritional requirements, based on recognized scientific prin-
ciples, are established by medical evaluation.". 
(c) AUTHORIZATION.—Section 5(c) 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 $10,000,000 for fiscal year 1988, 
$12,000,000 for fiscal year 1989, $14,000,000 for fiscal year 1990.". 
(d) STUDY.—The Secretary of Health and Human Services shall 
conduct a study to determine whether the application of subchapter 
B of chapter V of the Federal Food, Drug, and Cosmetic Act (relating 
to drugs for rare diseases and conditions) and section 28 of the 
Internal Revenue Code of 1986 (relating to tax credit) to medical 
devices or medical foods for rare diseases or conditions or to both is 
needed to encourage the development of such devices and foods. The 
Secretary shall report the results of the study to the Committee on 
Energy and Commerce of the House of Representatives and the 
Committee on Labor and Human Resources of the Senate not later 
than one year after the date of the enactment of this Act. For 
purposes of this section, the term "rare diseases or conditions" has 
the meaning prescribed by section 5 of the Orphan Drug Act (21 
U.S.C. 360ee). 
21 u s e 360aa 
note. 
Reports. 
102 STAT. 92 
PUBLIC LAW 100-290—APR. 18, 1988 
SKC. 4. NATIONAL COMMISSION ON ORPHAN DISEASES. 
Section 4(n) of the Orphan Drug Amendments of 1985 (42 U.S.C. 
236 note) is amended by striking out "September 30, 1987" and 
inserting in lieu thereof "February 1, 1989". 
Approved April 18, 1988. 
LEGISLATIVE HISTORY—H.R. 3459: 
-HOUSE REPORTS: No. 100-473 (Comm. on Energy and Commerce). 
CONGRESSIONAL RECORD, Vol. 134 (1988): 
Mar. 22, considered and passed House. 
Mar. 31, considered and passed Senate.