(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
inserting ‘‘for an applicant who is not’’, and by adding
at the end the following:
‘‘(D) the Secretary finds, after providing the
holder, such applicant, and any other interested per-
son an opportunity to present their views, that the
drugs of the holder and such applicant were devel-
oped simultaneously.
The Secretary shall make a decision on a request for a
finding under subparagraph (D) not later than 60 days
after the filing of the request.
‘‘(2) For purposes of paragraph (1)(D), drugs of a
holder and an applicant shall be considered to be devel-
oped simultaneously only if—
‘‘(A) the applicant requested that its drug be
designated under section 526 no later than 6 months
after publication of the designation under section
526(c) of the holder’s drug,
‘‘(B) the applicant initiated the human clinical
trials that the applicant relied on in its application
for such approval, certification, or license not more
than 12 months after the date the holder initiated
the human clinical trials that the holder relied on in
its application for such approval, certification, or
license, and
‘‘(C) the applicant submitted such application,
including the reports of the clinical and animal stud-
ies necessary for approval, certification, or licensing,
not more than 12 months after the holder submitted
its application, including such reports, for such ac-
tion.
‘‘(3) Paragraph (1)(D) does not apply to a drug—
‘‘(A) for which an application under section 505
or 507 or section 351 of the Public Health Service
Act was submitted before March 1, 1994, or
‘‘(B) for which an exemption under section
505(i) or 507(d) was in effect before March 1, 1994,
for which human clinical trials were actively being
conducted before such date, and for which an appli-
cation for designation under section 526 was submit-
ted before the date of enactment of the Orphan
Drug Act Amendments of 1994.’’.
(b) PUBLICATION.—Section 526(c) (21 U.S.C.
360bb(c) is amended—
(1) by inserting ‘‘for a rare disease or condi-
tion’’ after ‘‘(a)’’, and
(2) by striking out ‘‘shall be made available to
the public’’ and inserting in lieu thereof ‘‘shall be
promptly published in the Federal Register and oth-
erwise made available to the public in a manner de-
signed to notify persons who have such disease or
condition’’.
SEC. 5. OFFICE FOR ORPHAN DISEASES AND CONDITIONS.
Section 227 of the Public Health Service Act (42
U.S.C. 236) is amended—
(1) by amending subsection (a) to read as
follows:
‘‘(a) There is established in the Department of
Health and Human Services an Office for Orphan Dis-
eases and Conditions. Such Office shall be established at
a level within the Department with sufficient authority to
assure full implementation of the functions and respon-
sibilities established by this section.’’,
(2) by striking ‘‘Board’’ each place the term ap-
pears and inserting ‘‘Office’’,
(3) by striking ‘‘drugs and devices’’ in sub-
section (b) and inserting ‘‘drugs, devices, and medi-
cal foods’’,
(4) by inserting ‘‘of chapter V’’ after ‘‘sub-
chapter B’’ in subsection (c)(1)(A),
(5) by adding at the end the following new sub-
section:
‘‘(f)(1) There is established in the Office an advisory
committee to advise the Office in carrying out the func-
tions of the Office under this section.
‘‘(2) The advisory committee shall be comprised of
11 members appointed by the Secretary, in consultation
with the Office and the Commissioner of the Food and
Drug Administration, from persons knowledgeable about
rare diseases and conditions, including—
‘‘(A) 5 representatives of organizations of per-
sons with rare diseases or conditions;
‘‘(B) 3 research scientists; and
‘‘(C) 3 representatives of health-related compa-
nies.
‘‘(3) The Secretary shall also appoint, as liaisons to
the advisory committee, individuals from the Food and
Drug Administration, the National Institutes of Health,
and other appropriate Federal agencies.
‘‘(4) Vacancies occurring in the membership of the
advisory committee shall be filled in the same manner as
the original appointment for the position being vacated.
Vacancies shall not affect the power of the remaining
members to execute the duties of the advisory committee.
‘‘(5) Members of the advisory committee, and liaisons
to the advisory committee, shall not be compensated, but
shall receive travel expenses, including per diem in lieu
of subsistence, at rates authorized for employees of agen-
cies under subchapter 1 of chapter 57 of title 5, United
States Code, for each day the member or liaison is en-
gaged in the performance of duties away from the home
or regular place of business of the member or liaison.
‘‘(6) Notwithstanding section 1342 of title 31, United
States Code, the advisory committee may accept the vol-
untary services provided by a member of the advisory com-
mittee or a liaison to the advisory committee.’’, and
(6) by amending the section heading to read as
follows: ‘‘OFFICE FOR ORPHAN DISEASES AND CON-
DITIONS’’.
SEC. 6. AUTHORIZATION FOR ORPHAN DRUG ACT.
Section 5(c) of the Orphan Drug Act (21 U.S.C.
360ee(c)) is amended by striking ‘‘$10,000,000’’ and all
that follows and inserting ‘‘$20,000,000 for fiscal year
1995, $25,000,000 for fiscal year 1996, and $30,000,000
for fiscal year 1997.’’.
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HR 4865 IH——2