II

107TH CONGRESS

1ST SESSION S. 1301

To amend the Federal Food, Drug, and Cosmetic Act to improve the safety

and efficacy of pharmaceuticals for children.

IN THE SENATE OF THE UNITED STATES

AUGUST 1, 2001

Mr. BOND introduced the following bill; which was read twice and referred to

the Committee on Health, Education, Labor, and Pensions

A BILL

To amend the Federal Food, Drug, and Cosmetic Act to

improve the safety and efficacy of pharmaceuticals for

children.

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled,

3 SECTION 1. SHORT TITLE.

4 This Act may be cited as the ‘‘Better Medicine for

5 Children Act’’.

6 SEC. 2. SECOND PERIOD OF EXCLUSIVITY FOR ALREADY7 MARKETED DRUGS.

8 (a) IN GENERAL.—Section 505A of the Federal

9 Food, Drug, and Cosmetic Act (21 U.S.C. 355a) (as

10 amended by section 11) is amended—
2

•S 1301 IS

1 (1) by inserting after subsection (c) the fol2 lowing:

3 ‘‘(d) SECOND PERIOD OF MARKET EXCLUSIVITY FOR

4 ALREADY-MARKETED DRUGS.—

5 ‘‘(1) IN GENERAL.—If—

6 ‘‘(A) the Secretary determines that infor7 mation relating to the use of an approved drug

8 in an additional pediatric population may

9 produce health benefits in that population and

10 makes a second written request to the holder of

11 an approved application under section

12 505(b)(1) for pediatric studies (which shall

13 specify a timeframe for completing the studies);

14 ‘‘(B) the holder has received a patent ex15 tension under paragraph (1) or (2) of sub16 section (b) or paragraph (1) or (2) of sub17 section (c);

18 ‘‘(C) the holder agrees to the request and

19 the studies are completed within the specified

20 timeframe; and

21 ‘‘(D) reports on the studies are submitted

22 in accordance with subsection (e)(2) or accepted

23 in accordance with subsection (e)(3);
3

•S 1301 IS

1 the applicable extension period referred to in sub2 section (b) or (c) shall be extended by an additional

3 3 months.

4 ‘‘(2) ONE EXTENSION ONLY.—Not more than 1

5 patent extension may be granted under paragraph

6 (1) for any single approved drug.’’; and

7 (2) in subsection (a), by striking ‘‘As used in

8 this section, the term’’ and inserting the following:

9 ‘‘In this section:

10 ‘‘(1) ADDITIONAL PEDIATRIC POPULATION.—

11 The term ‘additional pediatric population’ means a

12 pediatric subpopulation for which information was

13 not requested by the Secretary in a written

14 request—

15 ‘‘(A) under subsection (b) or (c), because

16 the approved drug in question was not used in

17 that pediatric subpopulation at the time when

18 the request was made; or

19 ‘‘(B) under subsection (c), because obtain20 ing such information would have been impos21 sible or inadvisable because of a need to pursue

22 pediatric studies in a sequential manner for sci23 entific, medical, or ethical reasons.

24 ‘‘(2) PEDIATRIC STUDIES; STUDIES.—The

25 term’’.
4

•S 1301 IS

1 SEC. 3. INFRASTRUCTURE FOR PEDIATRIC PHARMA2 COLOGICAL RESEARCH.

3 (a) INVESTMENT IN TOMORROW’S PEDIATRIC RE4 SEARCHERS.—Section 452G of the Public Health Service

5 Act (42 U.S.C. 285g–10) is amended—

6 (1) by redesignating subsection (b) as sub7 section (c); and

8 (2) by inserting after subsection (a), the fol9 lowing:

10 ‘‘(b) PRIORITY.—In awarding grants under sub11 section (a), the Secretary shall give priority to pediatric

12 specialty areas in which there is significant need (includ13 ing pediatric pharmacology, as appropriate).’’.

14 (b) PEDIATRIC RESEARCH LOAN REPAYMENT PRO15 GRAM.—The second section 487F of the Public Health

16 Service Act, relating to the pediatric research loan repay17 ment program, (42 U.S.C. 288–6) is amended—

18 (1) by redesignating that section as section

19 487G;

20 (2) by redesignating subsection (c) as sub21 section (d); and

22 (3) by inserting after subsection (b) the fol23 lowing:

24 ‘‘(c) PRIORITY.—In entering into loan repayment

25 contracts under subsection (a)(1), the Secretary shall give

26 priority to pediatric specialty areas in which there is sig-
5

•S 1301 IS

1 nificant need (including pediatric pharmacology, as appro2 priate).’’.

3 (c) SENSE OF THE SENATE.—It is the sense of the

4 Senate that the National Institutes of Health should con5 sider the formation of a Pediatric/Developmental Pharma6 cology Scientific Review Group or Special Emphasis Panel

7 to evaluate Federal grant programs relating to pediatric

8 pharmacology.

9 SEC. 4. PEDIATRIC STUDIES OF ALREADY-MARKETED

10 DRUGS.

11 Section 505A of the Federal Food, Drug, and Cos12 metic Act (21 U.S.C. 355a) is amended—

13 (1) by striking subsection (b); and

14 (2) in subsection (c)—

15 (A) by inserting after ‘‘the Secretary’’ the

16 following: ‘‘determines that information relating

17 to the use of an approved drug in the pediatric

18 population may produce health benefits in that

19 population and’’; and

20 (B) by striking ‘‘concerning a drug identi21 fied in the list described in subsection (b)’’.

22 SEC. 5. RESEARCH FUND FOR THE STUDY OF OFF-PATENT

23 DRUGS.

24 Part B of title IV of the Public Health Service Act

25 (42 U.S.C. 284 et seq.) is amended—
6

•S 1301 IS

1 (1) by redesignating the second section 409C,

2 relating to clinical research (42 U.S.C. 284k), as

3 section 409G;

4 (2) by redesignating the second section 409D,

5 relating to enhancement awards (42 U.S.C. 284l), as

6 section 409H; and

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

8 ‘‘SEC. 409I. PROGRAM FOR PEDIATRIC STUDIES OF OFF9 PATENT DRUGS.

10 ‘‘(a) LIST OF OFF-PATENT DRUGS FOR WHICH PE11 DIATRIC STUDIES ARE NEEDED.—

12 ‘‘(1) IN GENERAL.—Not later than 1 year after

13 the date of enactment of this section, the Secretary,

14 acting through the Director of the National Insti15 tutes of Health and in consultation with the Com16 missioner of Food and Drugs and experts in pedi17 atric research (including United States Pharma18 copoeia), shall develop, and publish a list of priority

19 approved drugs for which—

20 ‘‘(A) there is no patent or market exclu21 sivity protection; and

22 ‘‘(B) additional studies are needed to as23 sess the safety and effectiveness of the use of

24 the drug in the pediatric population.
7

•S 1301 IS

1 ‘‘(2) CONSIDERATION OF AVAILABLE INFORMA2 TION.—In developing the list under paragraph (1),

3 the Secretary shall consider, for each drug on the

4 list—

5 ‘‘(A) the availability of information con6 cerning the safe and effective use of the drug

7 in the pediatric population;

8 ‘‘(B) whether additional information is

9 needed; and

10 ‘‘(C) whether new pediatric studies con11 cerning the drug may produce health benefits in

12 the pediatric population.

13 ‘‘(b) CONTRACTS FOR PEDIATRIC STUDIES.—

14 ‘‘(1) IN GENERAL.—The Secretary shall award

15 contracts to entities that have the expertise to con16 duct pediatric clinical trials (including qualified uni17 versities, hospitals, laboratories, contract research

18 organizations, federally funded programs such as pe19 diatric pharmacology research units, other public or

20 private institutions, or individuals) to enable the en21 tities to conduct pediatric studies concerning 1 or

22 more drugs identified in the list described in sub23 section (a).

24 ‘‘(2) REQUIREMENTS.—In awarding contracts

25 under paragraph (1), the Secretary shall, to the ex-
8

•S 1301 IS

1 tent practicable award grants for a significant num2 ber of approved drugs on the list.

3 ‘‘(c) PROCESS FOR CONTRACTS AND LABELING

4 CHANGES.—

5 ‘‘(1) WRITTEN REQUEST TO HOLDERS OF AP6 PROVED APPLICATIONS FOR OFF-PATENT DRUGS.—

7 ‘‘(A) IN GENERAL.—The Commissioner of

8 Food and Drugs, in consultation with the Di9 rector of National Institutes of Health, may

10 issue a written request for pediatric studies

11 concerning a drug identified in the list de12 scribed in subsection (a) to all holders of an ap13 proved application for the drug under section

14 505 of the Federal Food, Drug, and Cosmetic

15 Act. Such a request shall be made in accord16 ance with section 505A of the Federal Food,

17 Drug, and Cosmetic Act.

18 ‘‘(B) PUBLICATION OF REQUEST.—If the

19 Commissioner of Food and Drugs does not re20 ceive a response to a written request issued

21 under subparagraph (A) within 30 days of the

22 date on which a request was issued, the Sec23 retary, acting through the Director of National

24 Institutes of Health, shall publish a request for
9

•S 1301 IS

1 contract proposals to conduct the pediatric

2 studies described in the written request.

3 ‘‘(2) CONTRACTS.—A contract under this sec4 tion may be awarded only if a proposal for the con5 tract is submitted to the Secretary in such form and

6 manner, and containing such agreements, assur7 ances, and information as the Secretary determines

8 to be necessary to carry out this section.

9 ‘‘(3) REPORTING OF STUDIES.—

10 ‘‘(A) Upon completion of a pediatric study

11 in accordance with a contract awarded under

12 this section, a report concerning the study shall

13 be submitted to the Director of National Insti14 tutes of Health and the Commissioner of Food

15 and Drugs. The report shall include all data

16 generated in connection with the study.

17 ‘‘(B) AVAILABILITY OF REPORTS.—Each

18 report submitted under subparagraph (A) shall

19 be considered to be in the public domain, and

20 shall be assigned a docket number by the Com21 missioner of Food and Drugs. An interested

22 person may submit written comments con23 cerning such pediatric studies to the Commis24 sioner of Food and Drugs, and the written com-
10

•S 1301 IS

1 ments shall become part of the docket file with

2 respect to each drug.

3 ‘‘(C) ACTION BY COMMISSIONER.—The

4 Commissioner of Food and Drugs shall take ap5 propriate action in response to the reports sub6 mitted under subparagraph (A) in accordance

7 with paragraph (4).

8 ‘‘(4) REQUEST FOR LABELING CHANGES.—Dur9 ing the 180-day period after the date on which a re10 port is submitted under paragraph (3)(A), the Com11 missioner of Food and Drugs shall—

12 ‘‘(A) review the report and such other data

13 as are available concerning the safe and effec14 tive use in the pediatric population of the drug

15 studied;

16 ‘‘(B) negotiate with the holders of ap17 proved applications for the drug studied for any

18 labeling changes that the Commissioner of Food

19 and Drugs determines to be appropriate and re20 quests the holders to make; and

21 ‘‘(C)(i) place in the public docket file a

22 copy of the report and of any requested labeling

23 changes; and
11

•S 1301 IS

1 ‘‘(ii) publish in the Federal Register a

2 summary of the report and a copy of any re3 quested labeling changes.

4 ‘‘(5) DISPUTE RESOLUTION.—If, not later than

5 the end of the 180-day period specified in paragraph

6 (4), the holder of an approved application for the

7 drug involved does not agree to any labeling change

8 requested by the Commissioner of Food and Drugs

9 under that paragraph—

10 ‘‘(A) the Commissioner of Food and Drugs

11 shall immediately refer the request to the Pedi12 atric Advisory Subcommittee of the Anti-Infec13 tive Drugs Advisory Committee; and

14 ‘‘(B) not later than 60 days after receiving

15 the referral, the Subcommittee shall—

16 ‘‘(i) review the available information

17 on the safe and effective use of the drug

18 in the pediatric population, including study

19 reports submitted under this section; and

20 ‘‘(ii) make a recommendation to the

21 Commissioner of Food and Drugs as to ap22 propriate labeling changes, if any.

23 ‘‘(6) FDA DETERMINATION.—Not later than 30

24 days after receiving a recommendation from the

25 Subcommittee under paragraph (5)B(ii) with respect
12

•S 1301 IS

1 to a drug, the Commissioner of Food and Drugs

2 shall consider the recommendation and, if appro3 priate, make a request to the holders of approved

4 applications for the drug to make any labeling

5 change that the Commissioner of Food and Drugs

6 determines to be appropriate.

7 ‘‘(7) FAILURE TO AGREE.—If a holder of an

8 approved application for a drug, within 30 days

9 after receiving a request to make a labeling change

10 under paragraph (6), does not agree to make a re11 quested labeling change, the Commissioner may

12 deem the drug to be misbranded under the Federal

13 Food, Drug, and Cosmetic Act.

14 ‘‘(d) AUTHORIZATION OF APPROPRIATIONS.—

15 ‘‘(1) IN GENERAL.—There are authorized to be

16 appropriated to carry out this section—

17 ‘‘(A) $200,000,000 for fiscal year 2002;

18 and

19 ‘‘(B) such sums as are necessary for each

20 of the 5 succeeding fiscal years.

21 ‘‘(2) AVAILABILITY.—Any amount appropriated

22 under paragraph (1) shall remain available to carry

23 out this section until expended.’’.
13

•S 1301 IS

1 SEC. 6. TIMELY LABELING CHANGES FOR DRUGS GRANTED

2 EXCLUSIVITY; DRUG FEES.

3 (a) ELIMINATION OF USER FEE WAIVER FOR PEDI4 ATRIC SUPPLEMENTS.—Section 736(a)(1) of the Federal

5 Food, Drug, and Cosmetic Act (21 U.S.C. 379h(A)(1))

6 is amended—

7 (1) by striking subparagraph (F); and

8 (2) by redesignating subparagraph (G) as sub9 paragraph (F).

10 (b) LABELING CHANGES.—Section 505A of the Fed11 eral Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is

12 amended by adding at the end the following:

13 ‘‘(l) LABELING SUPPLEMENTS.—

14 ‘‘(1) PRIORITY STATUS FOR PEDIATRIC SUP15 PLEMENTS.—Any supplement to a human drug ap16 plication submitted under this section—

17 ‘‘(A) shall be considered to be a priority

18 supplement; and

19 ‘‘(B) shall be subject to the performance

20 goals established by the Commissioner for pri21 ority drugs.

22 ‘‘(2) DISPUTE RESOLUTION.—If the Commis23 sioner determines that a supplemental application

24 submitted under this section is approvable and that

25 the only open issue for final action on the supple26 ment is the reaching of an agreement between the
14

•S 1301 IS

1 sponsor of the application and the Commissioner on

2 appropriate changes to the labeling for the drug that

3 is the subject of the application—

4 ‘‘(A) not later than 180 days after the date

5 of submission of the supplemental application—

6 ‘‘(i) the Commissioner shall request

7 that the sponsor of the application make

8 any labeling change that the Commissioner

9 determines to be appropriate; and

10 ‘‘(ii) if the sponsor of the application

11 does not agree to make a labeling change

12 requested by the Commissioner by that

13 date, the Commissioner shall immediately

14 refer the matter to the Pediatric Advisory

15 Subcommittee of the Anti-Infective Drugs

16 Advisory Committee;

17 ‘‘(B) not later than 60 days after receiving

18 the referral, the Pediatric Advisory Sub19 committee of the Anti-Infective Drugs Advisory

20 Committee shall—

21 ‘‘(i) review the pediatric study reports;

22 and

23 ‘‘(ii) make a recommendation to the

24 Commissioner concerning appropriate la25 beling changes, if any;
15

•S 1301 IS

1 ‘‘(C) the Commissioner shall consider the

2 recommendations of the Pediatric Advisory

3 Subcommittee of the Anti-Infective Drugs Advi4 sory Committee and, if appropriate, not later

5 than 30 days after receiving the recommenda6 tion, make a request to the sponsor of the ap7 plication to make any labeling change that the

8 Commissioner determines to be appropriate;

9 and

10 ‘‘(D) if the sponsor of the application,

11 within 30 days after receiving a request under

12 subparagraph (D), does not agree to make a la13 beling change requested by the Commissioner,

14 the Commissioner may deem the drug that is

15 the subject of the application to be mis16 branded.’’.

17 SEC. 7. OFFICE OF PEDIATRIC THERAPEUTICS.

18 (a) ESTABLISHMENT.—The Secretary of Health and

19 Human Services shall establish an Office of Pediatric

20 Therapeutics within the Office of the Commissioner of

21 Food and Drugs.

22 (b) DUTIES.—The Office of Pediatric Therapeutics

23 shall be responsible for oversight and coordination of all

24 activities of the Food and Drug Administration that may

25 have any effect on a pediatric population or the practice
16

•S 1301 IS

1 of pediatrics or may in any other way involve pediatric

2 issues.

3 (c) STAFF.—The staff of the Office of Pediatric

4 Therapeutics shall include—

5 (1) 1 or more individuals with expertise con6 cerning ethical issues presented by the conduct of

7 clinical research in the pediatric population; and

8 (2) 1 or more individuals with expertise in pedi9 atrics who shall consult with all components of the

10 Food and Drug Administration concerning activities

11 described in subsection (b).

12 SEC. 8. NEONATES.

13 Section 505A(g) of the Federal Food, Drug, and Cos14 metic Act (21 U.S.C. 355a(g)) is amended by inserting

15 ‘‘(including neonates in appropriate cases)’’ after ‘‘pedi16 atric age groups’’.

17 SEC. 9. SUNSET.

18 Section 505A of the Federal Food, Drug, and Cos19 metic Act (21 U.S.C. 355a) is amended by striking sub20 section (j) and inserting the following:

21 ‘‘(j) SUNSET.—A drug may not receive any 6-month

22 period under subsection (a) or (c) unless—

23 ‘‘(1) on or before October 1, 2007, the Sec24 retary makes a written request for pediatric studies

25 of the drug;
17

•S 1301 IS

1 ‘‘(2) on or before October 1, 2007, an applica2 tion for the drug is submitted under section

3 505(b)(1); and

4 ‘‘(3) all requirements of this section are met.’’.

5 SEC. 10. DISSEMINATION OF PEDIATRIC INFORMATION.

6 Section 505A of the Federal Food, Drug, and Cos7 metic Act (21 U.S.C 355a) (as amended by section 4(b))

8 is amended by adding at the end the following:

9 ‘‘(m) DISSEMINATION OF PEDIATRIC INFORMA10 TION.—

11 ‘‘(1) IN GENERAL.—Not later than 180 days

12 after the date of submission of a supplemental appli13 cation under this section, the Commissioner shall

14 make available to the public a summary of the med15 ical and clinical pharmacology reviews of pediatric

16 studies conducted for the supplement, including by

17 publication in the Federal Register.

18 ‘‘(2) EFFECT OF SUBSECTION.—Nothing in this

19 subsection alters or amends in any way section 552

20 of title 5 or section 1905 of title 18, United States

21 Code.’’.

22 SEC. 11. TECHNICAL AND CONFORMING AMENDMENTS.

23 Section 505A of the Federal Food, Drug, and Cos24 metic Act (21 U.S.C. 355a) (as amended by sections 2(1),

25 4(b), 7, and 8) is amended—
18

•S 1301 IS

1 (1) by redesignating subsections (a), (d), (e),

2 (f), (g), (j), (l), and (m) as subsections (b), (e), (f),

3 (g), (a), (m), (k), and (l), respectively;

4 (2) by moving the subsections so as to appear

5 in alphabetical order;

6 (3) in subsections (b) and (c)—

7 (A) by striking ‘‘subsection (d)(2)’’ and in8 serting ‘‘subsection (e)(2)’’; and

9 (B) by striking ‘‘subsection (d)(3)’’ and in10 serting ‘‘subsection (e)(3)’’;

11 (4) in subsection (c)(2), by striking ‘‘subsection

12 (d)’’ each place it appears and inserting ‘‘subsection

13 (e)’’;

14 (5) in paragraphs (1), (2), and (3) of sub15 section (e) and subsections (f), (h) (as redesignated

16 by paragraph (1)), and (m) (as redesignated by

17 paragraph (1)), by striking ‘‘subsection (a) or (c)’’

18 and inserting ‘‘subsection (b) or (c)’’; and

19 (6) in subsection (i), by striking ‘‘(a) or (b)’’

20 and inserting ‘‘(a), (c), or (d)’’.

Æ