Orphan Drug Amendments of 1985
PL 99-91 (S.1147), enacted 08/15/1985

PUBLIC LAW 99-91—AUG. 15, 1985 
99 STAT. 387 
Public Law 99-91 
99th Congress 
An Act 
To amend the orphan drug provisions of the Federal Food, Drug, and Cosmetic Act 
Aug. 15, 1985 
and related laws. 
rg 1147] 
Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled. 
Orphan Drug 
Amendments of 
SECTION 1. SHORT TITLE. 
1985, 
This Act may be cited as the "Orphan Drug Amendments of 21 use 30i note. 
1985". 
SEC. 2. MARKET PROTECTION. 
Section 527 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360cc) is amended— 
(1) by striking out "and for which a United States Letter of 
Patent may not be issued" in subsection (a); 
(2) by striking out "and if a United States Letter of Patent 
may not be issued for the drug" in subsection (b); and 
(3) by striking out "UNPATENTED" in the title of the section. 
SEC. 3. ANTIBIOTIC DRUGS. 
(a) DESIGNATION.— 
(1) Section 525(a) of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 360aa(a)) is amended— 
(A) by striking out "or" at the end of paragraph (1), by 
redesignating paragraph (2) as paragraph (3), and by insert-
ing after paragraph (1) the following: 
"(2) if the drug is an antibiotic, it may be certified for such 
disease or condition under section 507, or ; 
(B) by striking out "before" in paragraph (3) (as so re-
designated); 
(C) by inserting after "505" in the last sentence a comma 
and the following: "certification of such drug for such 
disease or condition under section 507,"; and 
(D) by striking out "licensing under section 351 of the 
Public Health Service Act for such disease or condition" 
and inserting in lieu thereof "licensing of such drug for 
such disease or condition under section 351 of the Public 
Health Service Act". 
(2) Section 526(aXl) of such Act (21 U.S.C. 360bb(aXl)) is 
amended— 
i 
(A) by striking out "or" at the end of subparagraph (A) 
and by striking out subparagraph (B) and inserting in lieu 
thereof the following: 
"(B) if a certification for such drug is issued under section 507, 
or 
"(C) if a license for such drug is issued under section 351 of 
the Public Health Service Act,"; and 
(B) by striking out "the approval or license" and inserting 
in lieu thereof "the approval, certification, or license . 
99 STAT. 388 
PUBLIC LAW 99-91—AUG. 15, 1985 
(3) Section 527 of such Act (21 U.S.C. 360cc) is amended— 
(A) by striking out "or" at the end of paragraph (1) in 
subsection (a), by redesignating paragraph (2) as paragraph 
(3), and by inserting after paragraph (1) the following: 
"(2) issues a certification under section 507, or"; 
-" ., . 
(B) by inserting after "505" in the first sentence of subsec-
' ^ ' ''• 
tion (a) a comma and the following: "issue another certifi-
s
^
. 
A 
cation under section 507,"; 
(C) by inserting after "holder of such approved applica-
tion" in subsection (a) a comma and the following: "of such 
certification,"; 
" 
(D) by inserting after "approval of the approved applica-
tion" in subsection (a) a comma and the following: "the 
issuance of the certification,"; 
(E) by striking out "or a license" in subsection (b) and 
inserting in lieu thereof a comma and the following: "if a 
certification is issued under section 507 for such a drug, or 
if a license"; 
(F) by inserting after "application approval" in subsec-
tion (b) a comma and the following: "of the issuance of the 
certification under section 507,"; 
«
• 
(G) by striking out ", if the drug is a biological product," 
in subsection (b); 
(H) by inserting after "under section 505" in subsection 
(b) a comma and the following: "issue another certification 
under section 507,"; 
(I) by inserting after "holder of such approved applica-
tion" in subsection (b) a comma and the following: "of such 
certification,"; 
(J) by inserting after "application" in subsection (bXD a 
comma and the following: "of the certification,"; and 
(K) by inserting after "other applications" in subsection 
(bX2) a comma and the following: "issuance of other certifi-
cations,". 
42 use 236 note. SEC. 4. NATIONAL COMMISSION ON ORPHAN DISEASES. 
(a) EsTABUSHMENT.—There is established the National Commis-
sion on Orphan Diseases (hereinafter referred to as the "Commis-
sion"). 
(b) DUTY.—The Commission shall assess the activities of the Na-
tional Institutes of Health, the Alcohol, Drug Abuse, and Mental 
Health Administration, the Food and Drug Administration, other 
public agencies, and private entities in connection with— 
Research and 
(1) basic research conducted on rare diseases; 
development. 
(2) the use in research on rare diseases of knowledge developed 
in other research; 
(3) applied and clinical research on the prevention, diagnosis, 
and treatment of rare diseases; and 
(4) the dissemination to the public, health care professionals, 
researchers, and drug and medical device manufacturers of 
knowledge developed in research on rare diseases and other 
diseases which can be used in the prevention, diagnosis, and 
treatment of rare diseases. 
(c) REVIEW REQUIREMENTS.—In assessing the activities of the Na-
tional Institutes of Health, the Alcohol, Drug Abuse, and Mental 
Health Administration, and the Food and Drug Administration in 
connection with research on rare diseases, the Commission shall 
review— 
(1) the appropriateness of the priorities currently placed on 
research on rare diseases; 
Grants. 
(2) the relative effectiveness of grants and contracts when 
Contracts. 
used to fund research on rare diseases; 
PUBLIC LAW 99-91—AUG. 15, 1985 
99 STAT. 389 
(3) the appropriateness of specific requirements applicable to 
Research and 
applications for funds for research on rare diseases taking into 
development, 
consideration the reasonable capacity of applicants to meet such 
requirements; 
(4) the adequacy of the scientific basis for such research, 
including the adequacy of the research facilities and research 
resources used in such research and the appropriateness of the 
j 
scientific training of the personnel engaged in such research; 
(5) the effectiveness of activities undertaken to encourage 
such research; 
(6) the organization of the peer review process applicable to 
applications for funds for such research to determine if the 
organization of the peer review process could be revised to 
improve the effectiveness of the review provided to proposals for 
research on rare diseases; 
(7) the effectiveness of the coordination between the national 
research institutes of the National Institutes of Health, the 
institutes of the Alcohol, Drug Abuse, and Mental Health 
Administration, the Food and Drug Administration, and private 
entities in supporting such research; and 
(8) the effectiveness of activities undertaken to assure that 
knowledge developed in research on nonrare diseases is, when 
appropriate, used in research on rare diseases. 
(d) COMPOSITION.—The Commission shall be composed of twenty 
members appointed by the Secretary of Health and Human Services 
as follows: 
(1) Ten members shall be appointed from individuals who are 
not officers or employees of the Government and who by virtue 
of their training or experience in research on rare diseases or in 
the treatment of rare diseases are qualified to serve on the 
Commission. 
(2) Five members shall be appointed from individuals who are 
not officers or employees of the Government and who have a 
rare disease or are employed to represent or are members of an 
organization concerned about rare disease. 
(3) Four nonvoting members shall be appointed from— 
i 
(A) the directors of the national research institutes of the 
National Institutes of Health; or 
(B) the directors of the institutes of the Alcohol, Drug 
Abuse, and Mental Health Administration, 
which the Secretary determines are involved with rare diseases. 
(4) One nonvoting member shall be appointed from officers or 
employees of the Food and Drug Administration who the Sec-
retary determines are involved with rare diseases. 
A vacancy in the Commission shall be filled in the manner in which 
the original appointment was made. If any member of the Commis-
sion who was appointed to the Commission as a director of a 
national research institute or an institute of the Alcohol, Drug 
Abuse, and Mental Health Administration or as an officer or em-
ployee of the Food and Drug Administration leaves that office or 
position, or if any member of the Commission who was appointed 
from persons who are not officers or employees of the Government 
becomes an officer or employee of the Government, such member 
may continue as a member of the Commission for not longer than 
the ninety-day period beginning on the date such member leaves 
that office or position or becomes such an officer or employee, as the 
case may be. 
99 STAT. 390 
PUBLIC LAW 99-91—AUG. 15, 1985 
5 use 5332. 
5 use 5101 et 
seq., 5331. 
Government 
organization and 
employees. 
(e) TERM.—Members shall be appointed for the life of the Commis-
sion. 
(f) COMPENSATION.— 
(1) Except as provided in paragraph (2), members of the 
Commission shall each be entitled to receive compensation at a 
rate not to exceed the daily equivalent of the annual rate of 
basic pay in effect for grade GS-18 of the General Schedule for 
each day (including traveltime) during which they are engaged 
in the actual performance of duties as members of the Commis-
sion. 
(2) Members of the Commission who are full-time officers or 
employees of the Government shall receive no additional pay by 
reason of their service on the Commission. 
(g) CHAIRMAN.—The Chairman of the Commission shall be des-
ignated by the members of the Commission. 
(h) STAFF.—Subject to such rules as may be prescribed by the 
Commission, the Commission may appoint and fix the pay of such 
personnel as it determines are necessary to enable the Commission 
to carry out its functions. Personnel shall be appointed subject to 
the provisions of title 5, United States Code, governing appoint-
ments in the competitive service, and shall be paid in accordance 
with the provisions of chapter 51 and subchapter III of chapter 53 of 
such title relating to classification and General Schedule pay rates. 
(i) EXPERTS AND CONSULTANTS.—Subject to such rules as may be 
prescribed by the Commission, the Commission may procure tem-
porary and intermittent services under section 3109(b) of title 5 of 
the United States Code, but at rates for individuals not to exceed the 
daily equivalent of the basic pay payable for grade GS-15 of the 
General Schedule. 
(j) DETAIL OF PERSONNEL.—Upon request of the Commission, the 
head of any Federal agency is authorized to detail, on a reimburs-
able basis, any of the personnel of such agency to the Commission to 
assist the Commission in carrying out its functions. 
(k) ADMINISTRATIVE SUPPORT SERVICES.—The Administrator of 
General Services shall provide to the Commission on a reimbursable 
basis such administrative support services as the Commission may 
request. 
(1) GENERAL AUTHORITY.—The Commission may, for the purpose of 
carrying out this section, hold such hearings, sit and act at such 
times and places, take such testimony, and receive such evidence, as 
the Commission considers appropriate. 
(m) INFORMATION.—The Commission may secure directly from any 
department or agency of the United States information necessary to 
enable it to carry out this section. Upon request of the Chairman, 
the head of such department or agency shall furnish such informa-
tion to the Commission. 
(n) REPORT.—The Commission shall transmit to the Secretary and 
to each House of the Congress a report not later than September 30, 
1987, on the activities of the Commission. The report shall contain a 
detailed statement of the findings and conclusions of the Commis-
sion, together with its recommendations for— 
(1) a long range plan for the use of public and private re-
sources to improve research into rare diseases and to assist in 
the prevention, diagnosis, and treatment of rare diseases; and 
(2) such legislation or administrative actions as it considers 
appropriate. 
PUBLIC LAW 99-91—AUG. 15, 1985 
99 STAT. 391 
(o) TERMINATION.—The Commission shall terminate 90 days after 
the date of the submittal of its report under subsection (n). 
(p) FUNDS.—The Director of the National Institutes of Health 
shall make available $1,000,000 to the Commission from appropria-
tions for fiscal year 1986 for the National Institutes of Health. 
SEC. 5. FINANCIAL ASSISTANCE. 
(a) QuAUFiED TESTING.—Section 5 of the Orphan Drug Act (21 
U.S.C. 360ee) is amended— 
(1) in subsection (a) by striking out "clinical"; and 
(2) by amending subsection (b)(1) to read as follows: 
"(1) The term 'qualified testing' means— 
"(A) human clinical testing— 
"(i) which is carried out under an exemption for a 
drug for a rare disease or condition under section 505(i) 
of the Federal Food, Drug, and Cosmetic Act (or 
regulations issued under such section); and 
"(ii) which occurs after the date such drug is des-
ignated under section 526 of such Act and before the 
date on which an application with respect to such drug 
is submitted under section 505(b) or 507 of such Act or 
under section 351 of the Public Health Service Act; and 
"(B) preclinical testing involving a drug for a rare disease 
or condition which occurs after the date such drug is des-
ignated under section 526 of such Act and before the date 
on which an application with respect to such drug is 
submitted under section 505(b) or 507 of such Act or under 
section 351 of the Public Health Service Act.". 
(b) AUTHORIZATION.—Subsection (c) of such section 5 is amended to 
read as follows: 
"(c) For grants and contracts under subsection (a) there are 
authorized to be appropriated $4,000,000 for fiscal year 1986, 
$4,000,000 for fiscal year 1987, and $4,000,000 for fiscal year 1988.". 
SEC. 6. TECHNICAL CORRECTIONS. 
(a) PuBuc LAW 98-619.—The paragraph following the heading 
"EDUCATION FOR THE HANDICAPPED" under title III of the Depart-
ments of Labor, Health and Human Services, and Education and 
Related Agencies Appropriation Act, 1985 (Public Law 98-619) is 
amended— 
(1) by inserting after "shall" the first time it appears a comma 
and the following: "except for part D of such Act,", and 
(2) by adding at the end thereof the following: "The amounts 
available for such part D shall be available for obligation on 
October 1, 1984, and shall remain available until September 30, 
1985.". 
Qj) PUBLIC LAW 98-527.—Section 122(b)(4)(C) of the Developmental 
Disabilities Assistance and Bill of Rights Act (42 U.S.C. 6022(b)(4)(C)) 
is amended to read as follows: 
"(C) Notwithstanding subparagraph (E)(i), upon application of a 
State, which under section 133(b)(4)(C) of this Act (as in effect on 
October 18, 1984) was permitted to make expenditures for services 
without regard to the requirements of section 133(b)(4)(B) of this Act 
(as so in effect) the Secretary, pursuant to regulations which the 
Secretary shall prescribe, may permit a portion of the funds which, 
pursuant to subparagraph (E)(i), must otherwise be expended under 
21 u s e 355. 
21 use 360bb. 
21 use 357. 
42 u s e 262. 
21 use 360ee. 
98 Stat. 3324. 
98 Stat. 2670. 
42 use 6063. 
99 STAT. 392 
PUBLIC LAW 99-91—AUG. 15, 1985 
42 u s e 6063. 
98 Stat. 2679. 
42 u s e 6041, 
6061, 6081. 
42 u s e 295g-l. 
Contracts. 
Schools and 
colleges. 
42 u s e 295g-l 
note. 
21 u s e 360aa 
note. 
the State plan of such State for service activities in the priority 
services, to be expended in fiscal years 1985, 1986, and 1987 for the 
additional services for which expenditure was permitted under sec-
tion 133(b)(4)(C) (as so in effect) if the Secretary determines that— 
"(i) such additional services are not priority services; 
"(ii) such additional services are not services for which funds 
are otherwise available under part C, D, or E; and 
"(lii) the expenditures of such State on service activities in the 
priority services has reasonably met the need for those services 
in such State in comparison to the extent to which the need for 
such'additional services has been met in such State.". 
SEC. 7. AREA HEALTH EDUCATION CENTERS. 
Section 781(a)(2) of the Public Health Service Act (42 U.S.C. 295g-
7(a)(2)) is amended by redesignating subparagraphs (A), (B), and (C) 
as clauses (i), (ii), and (iii), respectively, and by striking out all that 
precedes clause (i) (as so redesignated) and inserting in lieu thereof 
the following: 
**(2)(A) The Secretary shall enter into contracts with schools of 
medicine and osteopathy— 
"(i) Vhich have previously received Federal financial assist-
ance for an area health education center program under section 
802 of the Health Professionals Educational Assistance Act of 
1976 in fiscal year 1979 or under paragraph (1), or 
"(ii) which are receiving assistance under paragraph (1), 
to carry out projects described in subparagraph (B) through area 
health education centers for which Federal financial assistance was 
provided under paragraph (1) and which are no longer eligible to 
receive such assistance. 
"(B) Projects for which assistance may be provided under subpara-
graph (A) are—". 
SEC. 8. EFFECTIVE DATE. 
(a) GENERAL RULE.—Except as provided in subsection (b), this Act 
and the amendments made by this Act shall take effect October 1, 
1985. 
(b) EXCEPTION.—The amendments made by sections 2, 3, and 6(a) 
shall take effect on the date of the enactment of this Act. The 
amendment made by section 6(b) shall take effect October 19, 1984. 
The amendments made by section 7 shall take effect October 1,1984 
and shall cease to be in effect after September 30,1985. 
Approved August 15, 1985. 
LEGISLATIVE HISTORY—S. 1147 (H.R. 2290): 
HOUSE REPORT No. 99-153 accompanying H.R. 2290 (Comm. on Energy and 
Commerce). 
CONGRESSIONAL RECORD, Vol. 131 (1985): 
May 23, considered and passed Senate. 
June 17,18, H.R. 2290 considered and passed House; S. 1147, amended, passed 
in lieu. 
July 25, Senate concurred in House amendment with eimendments. 
July 31, House concurred in Senate amendments.