[Congressional Bills 103th Congress]
[From the U.S. Government Publishing Office]
[S. 1981 Reported in Senate (RS)]
Calendar No. 609
103d CONGRESS
2d Session
S. 1981
[Report No. 103-366]
_______________________________________________________________________
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, and for other purposes.
_______________________________________________________________________
September 14 (legislative day, September 12), 1994
Reported without amendment
Calendar No. 609
103d CONGRESS
2d Session
S. 1981
[Report No. 103-366]
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, and for other purposes.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
March 24 (legislative day, February 22), 1994
Mrs. Kassebaum (for herself, Mr. Metzenbaum, Mr. Kennedy, and Mr.
Simon) introduced the following bill; which was read twice and referred
to the Committee on Labor and Human Resources
September 14 (legislative day, September 12), 1994
Reported by Mr. Kennedy, without amendment
_______________________________________________________________________
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, and for other purposes.
Be it enacted by the Senate and House of Representatives 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 (21 U.S.C. 201 et seq.).
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 section 527 (21 U.S.C.
360cc) (as amended by subsection (a)) is amended by adding at the end
the following new paragraphs:
``(2) The holder of the approved application, certification, or
license of a drug to which the 4-year period of exclusivity applies
under paragraph (1) may, after the expiration of 3\1/2\ 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 submitted under
paragraph (2) if the applicant--
``(A) demonstrates that the drug has a limited commercial
potential as determined under regulations of the Secretary,
taking into account sales information respecting such drug and
any other factor identified by the Secretary in such
regulations that 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 subparagraph (A)
that were in effect--
``(i) on the date--
``(I) such drug received its designation
under section 526(a), or
``(II) such applicant applied for an
exemption 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, on 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 exclusivity under subsection (a)'';
and
(2) by striking ``such seven year period'' and inserting
``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
submitted 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 section 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 that--
``(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 persons who
will be affected by the disease or condition 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 disease 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 ``or'' at the end of such
paragraph;
(2) by striking the period at the end of paragraph (2) and
inserting ``; or'', and
(3) by adding at the end the following new paragraph:
``(3) a drug has been designated under section 526 for a
rare disease or condition described in section 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 requirement 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--
(1) inserting ``(1)'' after ``(b)'';
(2) by redesignating paragraphs (1), (2), and (3) as
subparagraphs (A), (B), and (C), respectively;
(3) by striking ``for a person who is not'' and inserting
``for an applicant who is not''; and
(4) by adding at the end the following new subparagraphs:
``(D) the Secretary finds, after providing the holder, such
applicant, and any other interested person an opportunity to
present their views, that the drugs of the holder and such
applicant were developed 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 developed simultaneously only if--
``(A) the applicant requested that its drug be designated
under section 526 not 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 studies necessary for
approval, certification, or licensing, not more than 12 months
after the holder submitted its application, including such
reports, for such action.
``(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 application for designation under section 526 was
submitted 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 condition'' after
``(a)''; and
(2) by striking ``shall be made available to the public''
and inserting ``shall be promptly published in the Federal
Register and otherwise made available to the public in a manner
designed 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) in subsection (a), to read as follows:
``(a) There is established in the Department of Health and Human
Services an Office for Orphan Diseases and Conditions. Such Office
shall be established at a level within the Department with sufficient
authority to assure full implementation of the functions and
responsibilities established by this section.'';
(2) by striking ``Board'' each place the term appears and
inserting ``Office'';
(3) in subsection (b), by striking ``drugs and devices''
and inserting ``drugs, devices, and medical foods'';
(4) in subsection (c)(1)(A), by inserting ``of chapter V''
after ``subchapter B'';
(5) by adding at the end the following new subsection:
``(f)(1) There is established in the Office an advisory committee
to advise the Office in carrying out the functions 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 persons with
rare diseases or conditions;
``(B) 3 research scientists; and
``(C) 3 representatives of health-related companies.
``(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 agencies under subchapter 1 of chapter 57
of title 5, United States Code, for each day the member or liaison is
engaged 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 voluntary services provided by a
member of the advisory committee or a liaison to the advisory
committee.''; and
(6) by amending the section heading to read as follows:
``office for orphan diseases and conditions''.
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.''.