Orphan Drug Amendments of 1992

Orphan Drug Amendments of 1992 - Amends provisions of the Federal Food, Drug, and Cosmetic Act (FDCA) relating to designation of drugs for rare diseases or conditions to require that the number of people affected be determined three years after the request for designation as well as on the date of the request.

Increases the period during which the approval of an application, a certification, or a license for such a drug must be exclusive.

Creates additional exceptions from the protection of exclusive approval, certification, or license for drugs for rare diseases for drugs which qualified for protection because the disease or condition for which they are used affected less than a specified number of persons, but which have ceased to so qualify.

Provides for the termination of orphan drug status for drugs having cumulative sales over a certain amount, provided the costs of developing the drug in the United States are not over the same amount. Mandates a minimum two-year period after approval of an application, certification, or license before such a termination may be ordered. Sets forth transition rules requiring other minimum periods before such termination for drugs approved or awaiting approval at the time of enactment of this Act.

Amends the Public Health Service Act to replace provisions establishing the Orphan Products Board with provisions establishing the Office for Orphan Diseases and Conditions. Includes in the functions of the Office dealing with drugs, devices, and medical foods (currently, the Board deals with drugs and devices). Establishes an advisory committee within the Office.

Amends the FDCA to authorize appropriations for grants and contracts for development of drugs for rare diseases and conditions.

 
Calendar No. 591
102d CONGRESS
2d Session
S. 2060
[Report No. 102-358]
A BILL
To revise the orphan drug provisions of the Federal Food, Drug, and Cosmetic
Act, the Public Health Service Act, and the Orphan Drug Act, and for other
purposes.
August 4 (legislative day, JULY 23), 1992
Reported with an amendment
S 2060 RS
Calendar No. 591
102d CONGRESS
2d Session
S. 2060
[Report No. 102-358]
To revise the orphan drug provisions of the Federal Food, Drug, and Cosmetic
Act, the Public Health Service Act, and the Orphan Drug Act, and for other
purposes.
IN THE SENATE OF THE UNITED STATES
November 26 (legislative day, NOVEMBER 23), 1991
Mrs. KASSEBAUM (for herself, Mr. METZENBAUM, Mr. BINGAMAN, and Mr. INOUYE)
introduced the following bill; which was read twice and referred to the
Committee on Labor and Human Resources
August 4 (legislative day, JULY 23), 1992
Reported by Mr. KENNEDY, with an amendment
[Strike out all after the enacting clause and insert the part printed
in italic]
A BILL
To revise the orphan drug provisions of the Federal Food, Drug, and Cosmetic
Act, the Public Health Service Act, and the Orphan Drug Act, and for other
purposes.
  Be it enacted by the Senate and House of Representatives of the United
  States of America in Congress assembled,
 [Struck out->] SECTION 1. SHORT TITLE. [<-Struck out]
 [Struck out->]   This Act may be cited as the `Orphan Drug Amendments of
 1991'. [<-Struck out]
 [Struck out->] SEC. 2. DESIGNATIONS. [<-Struck out]
 [Struck out->]   (a) IN GENERAL- Section 526(a)(2) of the Federal Food,
 Drug, and Cosmetic Act (21 U.S.C. 360bb(a)(2)) is amended by adding before
 the period at the end the following: `, and on the basis of projections as
 to the number of persons who will be affected by the disease or condition
 3 years from such date'. [<-Struck out]
 [Struck out->]   (b) EXCLUSIVITY- Section 527(b) of such Act (21
 U.S.C. 360cc(b)) is amended-- [<-Struck out]
 [Struck out->]   (1) by striking `or' at the end of paragraph (1);
 [<-Struck out]
 [Struck out->]   (2) by striking the period at the end of paragraph (2)
 and inserting `;' and [<-Struck out]
 [Struck out->]   (3) by adding at the end the following new paragraphs:
 [<-Struck out]
 [Struck out->]   `(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 such disease or condition does not meet such description;
 or [<-Struck out]
 [Struck out->]   `(4) the Secretary has issued a termination notice under,
 and acted in accordance with, subsection (c).'. [<-Struck out]
 [Struck out->] SEC. 3. TERMINATION OF ORPHAN DRUG STATUS FOR DRUGS OF
 SIGNIFICANT COMMERCIAL VALUE. [<-Struck out]
 [Struck out->]   Section 527 of the Federal Food, Drug, and Cosmetic Act (21
 U.S.C. 360cc) is amended by adding at the end the following new subsection:
 [<-Struck out]
 [Struck out->]   `(c)(1) If the Secretary determines that the cumulative
 net sales of a drug which is designated under section 526 are more than
 $150,000,000 during the 7-year period described in subsection (a), the
 Secretary shall commence the review of any other application under section
 505, certification under section 507, or license under section 351 of the
 Public Health Service Act (42 U.S.C. 262), for such drug for such designation,
 if such review has not already commenced. [<-Struck out]
 [Struck out->]   `(2) If the Secretary determines that the cumulative
 net sales of a drug which is designated under section 526 are more than
 $200,000,000 during the 7-year period described in subsection (a), the
 Secretary shall issue a termination notice to the holder of the approved
 application of such designated drug. The notice shall state that the Secretary
 is authorized, no earlier than 90 days after the date of the notice, to
 approve other applications under section 505, issue other certifications
 under section 507, or issue other licenses under section 351 of the Public
 Health Service Act, for such drug for any such designation, unless such
 holder makes the showing described in paragraph (3). [<-Struck out]
 [Struck out->]   `(3) Not later than 30 days after the date of the termination
 notice, the holder described in paragraph (2) may submit evidence to the
 Secretary to demonstrate that-- [<-Struck out]
 [Struck out->]   `(A) the cumulative net sales of the drug are not more
 than $200,000,000; or [<-Struck out]
 [Struck out->]   `(B) the exceptionally high costs of developing the drug
 in the United States exceeded $200,000,000. [<-Struck out]
 [Struck out->]   `(4) Not later than 90 days after the date of the termination
 notice, the Secretary shall review any evidence submitted by the holder--
 [<-Struck out]
 [Struck out->]   `(A) in accordance with paragraph (3)(A), and determine
 whether cumulative net sales of the drug are more than $200,000,000, or
 [<-Struck out]
 [Struck out->]   `(B) in accordance with paragraph (3)(B), and determine
 whether the costs of developing the drug in the United States exceeded
 $200,000,000. [<-Struck out]
 [Struck out->] If the Secretary determines that the cumulative net sales of
 the drug are not more than $200,000,000, the Secretary shall, within such
 90 days, vacate the notice until such time as such sales are more than
 $200,000,000. If the Secretary determines that the costs of developing
 the drug in the United States exceeded $200,000,000 the Secretary shall,
 within such 90 days, determine the amount of costs incurred in developing
 the drug in the United States and vacate the notice until such time as the
 cumulative net sales are more than such amount. [<-Struck out]
 [Struck out->]   `(5) In determining the cumulative net sales of a drug,
 or determining the costs of developing a drug for purposes of paragraph
 (4), the Secretary shall consider-- [<-Struck out]
 [Struck out->]   `(A) evidence submitted in accordance with paragraph (3)(A)
 or paragraph (3)(B); [<-Struck out]
 [Struck out->]   `(B) data obtained through contracts with independent
 sources of comparative sales data; [<-Struck out]
 [Struck out->]   `(C) data submitted by interested parties; or [<-Struck out]
 [Struck out->]   `(D) other relevant data available to the
 Secretary.' [<-Struck out]
 [Struck out->]   `(6) Any person may submit to the Secretary a petition which
 contains evidence of the cumulative net sales of a drug and which requests
 the Secretary to determine whether a termination notice under paragraph
 (2) must be issued. [<-Struck out]
 [Struck out->]   `(7) As used in this subsection, the term `cumulative net
 sales' of a drug means total sales of the drug in the United States minus
 discounts, allowances, and returns.'. [<-Struck out]
 [Struck out->] SEC. 4. OFFICE FOR ORPHAN AND RARE DISEASES AND
 CONDITIONS. [<-Struck out]
 [Struck out->]   Section 227 of the Public Health Service Act (42 U.S.C. 236)
 is amended-- [<-Struck out]
 [Struck out->]   (1) by striking subsection (a) and inserting the following
 new subsection: [<-Struck out]
 [Struck out->]   `(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.'; [<-Struck out]
 [Struck out->]   (2) by striking `Board' each place the term appears and
 inserting `Office'; [<-Struck out]
 [Struck out->]   (3) by striking `drugs and devices' in subsection (b)
 and inserting `Drugs, devices, and medical foods'; [<-Struck out]
 [Struck out->]   (4) by inserting `of chapter V' after `subchapter B'
 in subsection (c)(1)(A); and [<-Struck out]
 [Struck out->]   (5) by adding at the end the following new subsection:
 [<-Struck out]
 [Struck out->]   `(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. [<-Struck out]
 [Struck out->]   `(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-- [<-Struck out]
 [Struck out->]   `(A) 5 representatives of organizations of persons with
 rare diseases or conditions; [<-Struck out]
 [Struck out->]   `(B) 3 research scientists; and [<-Struck out]
 [Struck out->]   `(C) 3 representatives of health-related companies. [<-Struck
 out]
 [Struck out->]   `(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. [<-Struck out]
 [Struck out->]   `(4) Any vacancy occurring in the membership of the
 advisory committee shall be filled in the same manner as the original
 appointment for the position being vacated. The vacancy shall not affect
 the power of the remaining members to execute the duties of the advisory
 committee. [<-Struck out]
 [Struck out->]   `(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. [<-Struck out]
 [Struck out->]   `(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.'. [<-Struck out]
 [Struck out->] SEC. 5. AUTHORIZATION FOR ORPHAN DRUG ACT. [<-Struck out]
 [Struck out->]   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 1992, $25,000,000 for fiscal year 1993, and
 $30,000,000 for fiscal year 1994.'. [<-Struck out]
SECTION 1. SHORT TITLE.
  This Act may be cited as the `Orphan Drug Amendments of 1992'.
SEC. 2. DESIGNATIONS.
  (a) IN GENERAL- Section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act
  (21 U.S.C. 360bb(a)(2)) is amended by adding before the period at the end
  the following: `, and on the basis of projections as to the number of persons
  who will be affected by the disease or condition 3 years from such date'.
  (b) PERIOD OF EXCLUSIVITY- The matter following paragraph (3) of the first
  sentence of section 527(a) of such Act (21 U.S.C. 360cc(a)) is amended by
  striking `seven' and inserting `9'.
  (c) EXCLUSIVITY- Section 527(b) of such Act  is amended--
  (1) by striking `or' at the end of paragraph (1);
  (2) by striking the period at the end of paragraph (2) and inserting `;' and
  (3) by adding at the end the following new paragraphs:
  `(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
  such disease or condition does not meet such description; or
  `(4) the Secretary has issued a termination notice under, and acted in
  accordance with, subsection (c).'.
SEC. 3. TERMINATION OF ORPHAN DRUG STATUS FOR DRUGS OF SIGNIFICANT COMMERCIAL
VALUE.
  (a) IN GENERAL- Section 527 of the Federal Food, Drug, and Cosmetic Act (21
  U.S.C. 360cc) is amended by adding at the end the following new subsection:
  `(c)(1) If the Secretary determines that the cumulative net sales of a drug
  that is designated under section 526 are more than $150,000,000 during the
  9-year period described in subsection (a), the Secretary shall commence
  the review of any other application under section 505, certification under
  section 507, or license under section 351 of the Public Health Service Act
  (42 U.S.C. 262), for such drug for such designation, if such review has
  not already commenced.
  `(2)(A) Except as provided in subparagraph (C), if the Secretary determines
  that the cumulative net sales of a drug that is designated under section 526
  are more than $200,000,000 during the 9-year period described in subsection
  (a), the Secretary shall issue a termination notice to the holder of the
  approved application under section 505, certification under section 507,
  or license under section 351 of the Public Health Service Act, of such
  designated drug.
  `(B) The notice shall state that the Secretary is authorized, not earlier
  than 90 days after the date of the notice, to approve other applications
  under section 505, issue other certifications under section 507, or issue
  other licenses under section 351 of the Public Health Service Act, for
  such drug for any such designation, unless such holder makes the showing
  described in paragraph (3).
  `(C) The Secretary shall not issue such a termination notice until a period
  of 2 years has expired after the date on which the Secretary approves such
  an application, or issues such a certification or license, for such drug.
  `(3) Not later than 30 days after the date of the termination notice, the
  holder described in paragraph (2)(A) may submit evidence to the Secretary
  to demonstrate that--
  `(A) the cumulative net sales of the drug are not more than $200,000,000; or
  `(B) the exceptionally high costs of developing the drug in the United
  States exceeded $200,000,000.
  `(4)(A) Not later than 90 days after the date of the termination notice,
  the Secretary shall review any evidence submitted by the holder--
  `(i) in accordance with paragraph (3)(A), and determine whether the
  cumulative net sales of the drug are more than $200,000,000; or
  `(ii) in accordance with paragraph (3)(B), and determine whether the costs
  of developing the drug in the United States exceeded $200,000,000.
  `(B)(i) If the Secretary determines that the cumulative net sales of the drug
  are not more than $200,000,000, the Secretary shall, within such 90 days,
  vacate the notice until such time as such sales are more than $200,000,000.
  `(ii) If the Secretary determines that the costs of developing the drug in
  the United States exceeded $200,000,000, the Secretary shall, within such
  90 days, determine the amount of costs incurred in developing the drug in
  the United States and vacate the notice until such time as the cumulative
  net sales are more than such amount.
  `(5) In determining the cumulative net sales of a drug, or determining the
  costs of developing a drug for purposes of paragraph (4), the Secretary
  shall consider--
  `(A) evidence submitted in accordance with paragraph (3)(A) or paragraph
  (3)(B);
  `(B) data obtained through contracts with independent sources of comparative
  sales data;
  `(C) data submitted by interested parties; or
  `(D) other relevant data available to the Secretary.'
  `(6) Any person may submit to the Secretary a petition that contains evidence
  of the cumulative net sales of a drug and that requests the Secretary to
  determine whether a termination notice under paragraph (2) must be issued.
  `(7) As used in this subsection, the term `cumulative net sales' of a
  drug means total sales of the drug in the United States minus discounts,
  allowances, and returns.'.
  (b) TRANSITION RULES-
  (1) APPROVED DRUGS- Notwithstanding section 527(c)(2)(C) of the Federal Food,
  Drug, and Cosmetic Act, with respect to a drug for which the Secretary
  approved an application under section 505 of the Federal Food, Drug,
  and Cosmetic Act, issued a certification under section 507 of such Act
  or issued a license under section 351 of the Public Health Service Act,
  before the date of enactment of the Orphan Drug Amendments of 1992, the
  Secretary shall not issue a termination notice under section 527(c)(2)(A)
  of the Federal Food, Drug, and Cosmetic Act until a period of 5 years has
  expired after the date on which the Secretary approved such an application,
  or issued such a certification or license, for such drug.
  (2) DRUGS AWAITING APPROVAL- Notwithstanding section 527(c)(2)(C) of the
  Federal Food, Drug, and Cosmetic Act, with respect to a drug that has
  been designated as a drug for a rare disease or condition under section
  526 before such date of enactment, and for which the Secretary approves
  such an application or issues such a certification or license after such
  date of enactment and before the date that is 2 years after such date
  of enactment, the Secretary shall not issue a termination notice under
  section 527(c)(2)(A) of the Federal Food, Drug, and Cosmetic Act until 4
  years after such date of enactment.
SEC. 4. OFFICE FOR ORPHAN AND RARE DISEASES AND CONDITIONS.
  Section 227 of the Public Health Service Act (42 U.S.C. 236) is amended--
  (1) by striking subsection (a) and inserting the following new subsection:
  `(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) by striking `drugs and devices' in subsection (b) and inserting `drugs,
  devices, and medical foods';
  (4) by inserting `of chapter V' after `subchapter B' in subsection
  (c)(1)(A); and
  (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) Any vacancy occurring in the membership of the advisory committee
  shall be filled in the same manner as the original appointment for the
  position being vacated. The vacancy 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.'.
SEC. 5. 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 1992, $25,000,000 for fiscal year 1993, and $30,000,000
  for fiscal year 1994.'.
S 2060 RS----2