Union Calendar No. 410

103D CONGRESS

2D SESSION H. R. 4865

[Report No. 103–746]

A BILL

To amend the Federal Food, Drug, and Cosmetic

Acts relating to orphan drugs.phan Drug Act to revise the provisions of suchAct, the Public Health Service Act, and the OrSEPTEMBER 26, 1994

Reported with an amendment, committed to the Commitordered to be printed tee of the Whole House on the State of the Union, and
IB

Union Calendar No. 410

103D CONGRESS

2D SESSION H. R. 4865

[Report No. 103–746]

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

AUGUST 1, 1994

Mr. WAXMAN (for himself and Mr. STUDDS) introduced the following bill;

which was referred to the Committee on Energy and Commerce

SEPTEMBER 26, 1994

Reported with an amendment, committed to the Committee of the Whole

House on the State of the Union, and ordered to be printed

[Strike out all after the enacting clause and insert the part printed in italic]

[For text of introduced bill, see copy of bill as introduced on August 1, 1994]

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.

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled,
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1 SECTION 1. SHORT TITLE AND REFERENCE.

2 (a) SHORT TITLE.—This Act may be cited as the

3 ‘‘Orphan Drug Act Amendments of 1994’’.

4 (b) REFERENCE.—Whenever in this Act (other than

5 sections 5 and 6) an amendment or repeal is expressed in

6 terms of an amendment to, or repeal of, a section or other

7 provision, the reference shall be considered to be made to

8 a section or other provision of the Federal Food, Drug, and

9 Cosmetic Act.

10 SEC. 2. PERIOD OF EXCLUSIVITY.

11 (a) INITIAL PERIOD.—Subsection (a) of section 527

12 (21 U.S.C. 360cc) is amended—

13 (1) by inserting ‘‘(1)’’ after ‘‘(a)’’,

14 (2) by redesignating paragraphs (1), (2), and (3)

15 as subparagraphs (A), (B), and (C), respectively,

16 (3) by striking ‘‘seven years’’ and inserting ‘‘4

17 years’’, and

18 (4) by striking ‘‘505(c)(2)’’ and inserting

19 ‘‘505(c)(1)(B)’’.

20 (b) ADDITIONAL PERIOD.—Subsection (a) of such sec21 tion 527 (21 U.S.C. 360cc) (as amended by subsection (a))

22 is amended by adding at the end the following:

23 ‘‘(2) The holder of the approved application, certifi24 cation, or license of a drug to which the 4-year period of

25 exclusivity applies under paragraph (1) may, after the expiration of 31 26 ⁄2 years of such period but not later than 90
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1 days before the expiration of such period, apply to the Sec2 retary for a 3-year extension of such period. Such an appli3 cation shall contain such information as the Secretary de4 termines is necessary to evaluate such application.

5 ‘‘(3) The Secretary shall approve an application sub6 mitted under paragraph (2) if the applicant—

7 ‘‘(A) demonstrates that the drug has a limited

8 commercial potential, as determined under regula9 tions of the Secretary, on the basis of—

10 ‘‘(i) total sales revenue for such drug during

11 the 4-year period of exclusivity under paragraph

12 (1), or

13 ‘‘(ii) factors other than total sales revenue

14 identified by the Secretary, and

15 ‘‘(B) makes such demonstration on the basis of

16 the regulations of the Secretary referred to in sub17 paragraph (A) which were in effect—

18 ‘‘(i) on the date—

19 ‘‘(I) such drug received its designation

20 under section 526(a)(1), or

21 ‘‘(II) such applicant applied for an ex22 emption for such drug under section 505(i)

23 or clause (3) of section 507(d),

24 whichever first occurs, or
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1 ‘‘(ii) if the date under clause (i) occurred

2 before the date such regulations were in effect, on

3 the date such regulations were in effect.’’.

4 (c) CONFORMING AMENDMENTS.—Section 527(b) (21

5 U.S.C. 360cc(b)) is amended—

6 (1) by striking ‘‘during the seven-year period’’

7 and inserting ‘‘during the applicable period of exclu8 sivity under subsection (a)’’,

9 (2) in paragraph (2), by striking ‘‘such seven

10 year period’’ and inserting ‘‘the applicable period of

11 exclusivity under subsection (a)’’,

12 (3) by striking ‘‘507,,’’ and inserting ‘‘507,’’, and

13 (4) in paragraph (1), by striking ‘‘The Sec14 retary’’ and inserting ‘‘the Secretary’’.

15 (d) EFFECTIVE DATE.—The amendments made by

16 subsections (a) and (b) shall not apply to a drug—

17 (1) for which an application under section 505

18 or 507 of the Federal Food, Drug, and Cosmetic Act

19 or section 351 of the Public Health Service Act was

20 submitted or approved before March 1, 1994, or

21 (2) for which an exemption under section 505(i)

22 or clause (3) of section 507(d) of the Federal Food,

23 Drug, and Cosmetic Act was in effect before March 1,

24 1994, for which human clinical trials were actively

25 being conducted before such date, and for which an
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1 application for designation under section 526 of such

2 Act was submitted before the date of enactment of the

3 Orphan Drug Act Amendments of 1994.

4 The 7-year period of exclusivity provided by section 527(a)

5 of the Federal Food, Drug, and Cosmetic Act before the date

6 of the enactment of this Act shall, after such date, apply

7 to a drug described in paragraph (1) or (2).

8 (e) REGULATIONS.—The Secretary shall issue final

9 regulations to implement paragraphs (2) and (3) of section

10 527(a) of the Federal Food, Drug, and Cosmetic Act (21

11 U.S.C. 360cc) (as added by subsection (b)) not later than

12 6 months after the date of the enactment of this Act.

13 SEC. 3. DESIGNATIONS.

14 (a) IN GENERAL.—Section 526(a)(2) (21 U.S.C.

15 360bb(a)(2)) is amended to read as follows:

16 ‘‘(2) For purposes of paragraph (1), the term ‘rare dis17 ease or condition’ means any disease or condition which—

18 ‘‘(A) affects fewer than 200,000 persons in the

19 United States determined on the basis of—

20 ‘‘(i) the facts and circumstances as of the

21 date the request for designation of the drug

22 under this subsection is made, and

23 ‘‘(ii) projections as to the number of persons

24 who will be affected by the disease or condition
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1 on a date which is 3 years from date such re2 quest was made, or

3 ‘‘(B) affects more than 200,000 persons in the

4 United States and for which there is no reasonable ex5 pectation that the cost of developing and making

6 available in the United States a drug for such disease

7 or condition will be recovered from sales in the Unit8 ed States of such drug.’’.

9 (b) EXCLUSIVITY.—Section 527(b) (21 U.S.C.

10 360cc(b)) is amended—

11 (1) by striking ‘‘or’’ at the end of paragraph (1),

12 (2) by striking the period at the end of para13 graph (2) and inserting a semicolon, and

14 (3) by adding at the end the following:

15 ‘‘(3) such drug was designated under section

16 526(a)(1) for a rare disease or condition described in

17 paragraph (2) of section 526(a) and, after such des18 ignation, it is determined that such disease or condi19 tion affects more than 200,000 persons in the United

20 States; or’’.

21 SEC. 4. SIMULTANEOUS DEVELOPMENT.

22 (a) IN GENERAL.—Section 527(b) (21 U.S.C.

23 360cc(b)), as amended by section 3(b), is amended by in24 serting ‘‘(1)’’ after ‘‘(b)’’, by redesignating paragraphs (1),

25 (2), and (3) as subparagraphs (A), (B), and (C), respec-
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1 tively, by striking ‘‘for a person who is not’’ and by insert2 ing ‘‘for an applicant who is not’’, and by adding at the

3 end the following:

4 ‘‘(D) the Secretary finds, after providing the

5 holder, such applicant, and any other interested per6 son an opportunity to present their views, that the

7 drugs of the holder and such applicant were developed

8 simultaneously.

9 The Secretary shall make a decision on a request for a find10 ing under subparagraph (D) not later than 60 days after

11 the filing of the request.

12 ‘‘(2) For purposes of paragraph (1)(D), drugs of a

13 holder and an applicant shall be considered to be developed

14 simultaneously only if—

15 ‘‘(A) the applicant requested that its drug be des16 ignated under section 526 no later than 6 months

17 after publication of the designation under section

18 526(c) of the holder’s drug,

19 ‘‘(B) the applicant initiated the human clinical

20 trials that the applicant relied on in its application

21 for such approval, certification, or license not more

22 than 12 months after the date the holder initiated the

23 human clinical trials that the holder relied on in its

24 application for such approval, certification, or

25 license, and
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1 ‘‘(C) the applicant submitted such application,

2 including the reports of the clinical and animal stud3 ies necessary for approval, certification, or license,

4 not more than 12 months after the holder submitted

5 its application, including such reports, for such

6 action.

7 ‘‘(3) Paragraph (1)(D) does not apply to a drug—

8 ‘‘(A) for which an application under section 505

9 or 507 or section 351 of the Public Health Service Act

10 was submitted or approved before March 1, 1994, or

11 ‘‘(B) for which an exemption under section

12 505(i) or clause (3) of section 507(d) was in effect be13 fore March 1, 1994, for which human clinical trials

14 were actively being conducted before such date, and

15 for which an application for designation under sec16 tion 526 was submitted before the date of enactment

17 of the Orphan Drug Act Amendments of 1994.’’.

18 (b) PUBLICATION.—Section 526(c) (21 U.S.C.

19 360bb(c)) is amended—

20 (1) by inserting ‘‘for a rare disease or condition’’

21 after ‘‘(a)’’, and

22 (2) by striking out ‘‘shall be made available to

23 the public’’ and inserting in lieu thereof ‘‘shall be

24 promptly published in the Federal Register and other25 wise made available to the public in a manner de-
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1 signed to notify persons who have such disease or con2 dition’’.

3 SEC. 5. OFFICE FOR ORPHAN DISEASES AND CONDITIONS.

4 Section 227 of the Public Health Service Act (42

5 U.S.C. 236) is amended—

6 (1) by amending subsection (a) to read as

7 follows:

8 ‘‘(a) There is established in the Department of Health

9 and Human Services an Office for Orphan Diseases and

10 Conditions. Such Office shall be established at a level within

11 the Department with sufficient authority to assure full im12 plementation of the functions and responsibilities estab13 lished by this section.’’,

14 (2) by striking ‘‘Board’’ each place the term ap15 pears and inserting ‘‘Office’’,

16 (3) by striking ‘‘drugs and devices’’ in subsection

17 (b) and inserting ‘‘drugs, devices, and medical foods’’,

18 (4) by inserting ‘‘of chapter V’’ after ‘‘subchapter

19 B’’ in subsection (c)(1)(A),

20 (5) by adding at the end the following new sub21 section:

22 ‘‘(f)(1) There is established in the Office an advisory

23 committee to advise the Office in carrying out the functions

24 of the Office under this section.
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1 ‘‘(2) The advisory committee shall be comprised of 11

2 members appointed by the Secretary, in consultation with

3 the Office and the Commissioner of Food and Drugs, from

4 persons knowledgeable about rare diseases and conditions,

5 including—

6 ‘‘(A) 5 representatives of organizations of persons

7 with rare diseases or conditions;

8 ‘‘(B) 3 research scientists; and

9 ‘‘(C) 3 representatives of health-related compa10 nies.

11 ‘‘(3) The Secretary shall also appoint, as liaisons to

12 the advisory committee, individuals from the Food and

13 Drug Administration, the National Institutes of Health,

14 and other appropriate Federal agencies.

15 ‘‘(4) Vacancies occurring in the membership of the ad16 visory committee shall be filled in the same manner as the

17 original appointment for the position being vacated. Vacan18 cies shall not affect the power of the remaining members

19 to execute the duties of the advisory committee.

20 ‘‘(5) Members of the advisory committee, and liaisons

21 to the advisory committee, shall not be compensated, but

22 shall receive travel expenses, including per diem in lieu of

23 subsistence, at rates authorized for employees of agencies

24 under subchapter 1 of chapter 57 of title 5, United States

25 Code, for each day the member or liaison is engaged in the
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1 performance of duties away from the home or regular place

2 of business of the member or liaison.

3 ‘‘(6) Notwithstanding section 1342 of title 31, United

4 States Code, the advisory committee may accept the vol5 untary services provided by a member of the advisory com6 mittee or a liaison to the advisory committee.’’, and

7 (6) by amending the section heading to read as

8 follows: ‘‘OFFICE FOR ORPHAN DISEASES AND CONDI9 TIONS’’.

10 SEC. 6. AUTHORIZATION FOR ORPHAN DRUG ACT.

11 Section 5(c) of the Orphan Drug Act (21 U.S.C.

12 360ee(c)) is amended by striking ‘‘$10,000,000’’ and all

13 that follows and inserting ‘‘$20,000,000 for fiscal year

14 1995, $25,000,000 for fiscal year 1996, and $30,000,000 for

15 fiscal year 1997.’’.