I
103D CONGRESS
2D SESSION
H. R. 4160
To amend the Federal Food, Drug, and Cosmetic Act, the Public Health
Service Act, and the Orphan Drug Act to revise the provisions of such
Acts relating to orphan drugs.
IN THE HOUSE OF REPRESENTATIVES
MARCH 24, 1994
Mr. WAXMAN (for himself and Mr. STUDDS) introduced the following bill;
which was referred to the Committee on Energy and Commerce
A BILL
To amend the Federal Food, Drug, and Cosmetic Act, the
Public Health Service Act, and the Orphan Drug Act
to revise the provisions of such Acts relating to orphan
drugs.
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled,
SECTION 1. SHORT TITLE AND REFERENCE.
(a) SHORT TITLE.—This Act may be cited as the
‘‘Orphan Drug Act Amendments of 1994 ’’.
(b) REFERENCE.—Whenever in this Act (other than
sections 5 and 6) an amendment or repeal is expressed
in terms of an amendment to, or repeal of, a section or
other provision, the reference shall be considered to be
made to a section or other provision of the Federal Food,
Drug, and Cosmetic Act.
SEC. 2. PERIOD OF EXCLUSIVITY.
(a) INITIAL PERIOD.—Subsection (a) of section 527
(21 U.S.C. 360cc) is amended—
(1) by inserting ‘‘(1)’’ after ‘‘(a)’’,
(2) by redesignating paragraphs (1), (2), and
(3) as subparagraphs (A), (B), and (C), respectively,
(3) by striking ‘‘seven years’’ and inserting ‘‘4
years’’, and
(4) by striking ‘‘505(c)(2)’’ and inserting
‘‘505(c)(1)(B)’’.
(b) ADDITIONAL PERIOD.—Subsection (a) of such
section 527 (21 U.S.C. 360cc) (as amended by subsection
(a)) is amended by adding at the end the following:
‘‘(2) The holder of the approved application, certifi-
cation, or license of a drug to which the 4-year period of
exclusivity applies under paragraph (1) may, after the ex-
piration of 31⁄2 years of such period but not later than
90 days before the expiration of such period, apply to the
Secretary for a 3-year extension of such period. Such an
application shall contain such information as the Secretary
determines is necessary to evaluate such application.
‘‘(3) The Secretary shall approve an application sub-
mitted under paragraph (2) if the applicant—
‘‘(A) demonstrates that the drug has a limited
commercial potential as determined under regula-
tions of the Secretary, taking into account sales in-
formation respecting such drug and any other factor
identified by the Secretary in such regulations which
is relevant to the commercial potential of such drug,
and
‘‘(B) makes such demonstration on the basis of
the regulations of the Secretary referred to in sub-
paragraph (A) which were in effect—
‘‘(i) on the date—
‘‘(I) such drug received its designation
under section 526(a), or
‘‘(II) such applicant applied for an ex-
emption for such drug under section 505(i)
or 507(d),
whichever first occurs, or
‘‘(ii) if the date under clause (i) occurred
before the date such regulations were in effect,
or the date such regulations were in effect.’’.
(c) CONFORMING AMENDMENT.—Section 527(b) (21
U.S.C. 360cc(b)) is amended—
(1) by striking ‘‘during the seven-year period
beginning on the date of the application approval’’
and inserting ‘‘during the applicable period of exclu-
sivity under subsection (a)’’, and
(2) by striking ‘‘such seven year period’’ and in-
serting ‘‘the applicable period of exclusivity under
subsection (a)’’.
(d) EFFECTIVE DATE.—The amendments made by
subsections (a) and (b) shall not apply to a drug—
(1) for which an application under section 505
or 507 of the Federal Food, Drug, and Cosmetic Act
or section 351 of the Public Health Service Act was
submitted before March 1, 1994, or
(2) for which an exemption under section 505(i)
or 507(d) of the Federal Food, Drug, and Cosmetic
Act was in effect before March 1, 1994, for which
human clinical trials were actively being conducted
before such date, and for which an application for
designation under section 526 of such Act was sub-
mitted before the date of enactment of the Orphan
Drug Act Amendments of 1994.
The 7 year period of exclusivity provided by section 527(a)
of the Federal Food, Drug, and Cosmetic Act before the
date of the enactment of this Act shall, after such date,
apply to a drug described in paragraph (1) or (2).
(e) REGULATIONS.—The Secretary shall issue final
regulations to implement paragraphs (2) and (3) of sec-
tion 527(a) of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 360cc) (as amended by subsection (b)) not
later than 6 months after the date of the enactment of
this Act.
SEC. 3. DESIGNATIONS.
(a) IN GENERAL.—Section 526(a)(2) (21 U.S.C.
360bb(a)(2)) is amended to read as follows:
‘‘(2) For purposes of paragraph (1), the term ‘rare
disease or condition’ means any disease or condition
which—
‘‘(A) affects fewer than 200,000 persons in the
United States determined on the basis of—
‘‘(i) the facts and circumstances as of the
date the request for designation of the drug
under this subsection is made, and
‘‘(ii) projections as to the number of per-
sons who will be affected by the disease or con-
dition on a date which is 3 years from date
such request was made, or
‘‘(B) affects more than 200,000 persons in the
United States and for which there is no reasonable
expectation that the cost of developing and making
available in the United States a drug for such dis-
ease or condition will be recovered from sales in the
United States of such drug.’’.
(b) EXCLUSIVITY.—Section 527(b) (21 U.S.C.
360cc(b)) is amended—
(1) in paragraph (1), by striking ‘‘The’’ and in-
serting ‘‘the’’ and by striking ‘‘or’’ at the end of
such paragraph,
(2) by striking the period at the end of para-
graph (2) and inserting ‘‘; or’’, and
(3) by adding at the end the following:
‘‘(3) a drug has been designated under section
526 for a rare disease or condition described in sec-
tion 526(a)(2)(A) and if after such designation it is
determined that—
‘‘(A) such disease or condition affects more
than 200,000 persons in the United States, and
‘‘(B) such drug does not meet the require-
ment of section 526(a)(2)(B).’’.
SEC. 4. SIMULTANEOUS DEVELOPMENT.
(a) 
IN
GENERAL.—Section 
527(b) 
(21 
U.S.C.
360cc(b)), as amended by section 3(b), is amended by in-
serting ‘‘(1)’’ after ‘‘(b)’’, by redesignating paragraphs
(1), (2), and (3) as subparagraphs (A), (B), and (C), re-
spectively, by striking ‘‘for a person who is not’’ and by