I 

108TH CONGRESS 

1ST SESSION H. R. 2857

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

Food and Drug Administration to require certain research into drugs 

used in pediatric patients. 

IN THE HOUSE OF REPRESENTATIVES 

JULY 24, 2003

Mr. GREENWOOD (for himself, Ms. ESHOO, and Ms. PRYCE of Ohio) introduced the following bill; which was referred to the Committee on Energy 

and Commerce 

A BILL

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

authorize the Food and Drug Administration to require 

certain research into drugs used in pediatric patients.

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled,
2

•HR 2857 IH 

1 SECTION 1. SHORT TITLE. 

2 This Act may be cited as the ‘‘Pediatric Research Eq3 uity Act of 2003’’. 

4 SEC. 2. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND 

5 BIOLOGICAL PRODUCTS. 

6 (a) IN GENERAL.—Subchapter A of chapter V of the 

7 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351 

8 et seq.) is amended by inserting after section 505A the 

9 following: 

10 ‘‘SEC. 505B. RESEARCH INTO PEDIATRIC USES FOR DRUGS 

11 AND BIOLOGICAL PRODUCTS. 

12 ‘‘(a) NEW DRUGS AND BIOLOGICAL PRODUCTS.—

13 ‘‘(1) IN GENERAL.—A person that submits an 

14 application (or supplement to an application)—

15 ‘‘(A) under section 505 for a new active in16 gredient, new indication, new dosage form, new 

17 dosing regimen, or new route of administration; 

18 or 

19 ‘‘(B) under section 351 of the Public 

20 Health Service Act (42 U.S.C. 262) for a new 

21 active ingredient, new indication, new dosage 

22 form, new dosing regimen, or new route of ad23 ministration; 

24 shall submit with the application the assessments de25 scribed in paragraph (2). 

26 ‘‘(2) ASSESSMENTS.—
3

•HR 2857 IH 

1 ‘‘(A) IN GENERAL.—The assessments re2 ferred to in paragraph (1) shall contain data, 

3 gathered using appropriate formulations for 

4 each age group for which the assessment is re5 quired, that are adequate—

6 ‘‘(i) to assess the safety and effective7 ness of the drug or the biological product 

8 for the claimed indications in all relevant 

9 pediatric subpopulations; and 

10 ‘‘(ii) to support dosing and adminis11 tration for each pediatric subpopulation for 

12 which the drug or the biological product is 

13 safe and effective. 

14 ‘‘(B) SIMILAR COURSE OF DISEASE OR

15 SIMILAR EFFECT OF DRUG OR BIOLOGICAL

16 PRODUCT.—

17 ‘‘(i) IN GENERAL.—If the course of 

18 the disease and the effects of the drug are 

19 sufficiently similar in adults and pediatric 

20 patients, the Secretary may conclude that 

21 pediatric effectiveness can be extrapolated 

22 from adequate and well-controlled studies 

23 in adults, usually supplemented with other 

24 information obtained in pediatric patients, 

25 such as pharmacokinetic studies. 
4

•HR 2857 IH 

1 ‘‘(ii) EXTRAPOLATION BETWEEN AGE

2 GROUPS.—A study may not be needed in 

3 each pediatric age group if data from 1 

4 age group can be extrapolated to another 

5 age group. 

6 ‘‘(3) DEFERRAL.—On the initiative of the Sec7 retary or at the request of the applicant, the Sec8 retary may defer submission of some or all assess9 ments required under paragraph (1) until a specified 

10 date after approval of the drug or issuance of the li11 cense for a biological product if—

12 ‘‘(A) the Secretary finds that—

13 ‘‘(i) the drug or biological product is 

14 ready for approval for use in adults before 

15 pediatric studies are complete; 

16 ‘‘(ii) pediatric studies should be de17 layed until additional safety or effective18 ness data have been collected; or 

19 ‘‘(iii) there is another appropriate rea20 son for deferral; and 

21 ‘‘(B) the applicant submits to the Sec22 retary—

23 ‘‘(i) certification of the grounds for 

24 deferring the assessments; 
5

•HR 2857 IH 

1 ‘‘(ii) a description of the planned or 

2 ongoing studies; and 

3 ‘‘(iii) evidence that the studies are 

4 being conducted or will be conducted with 

5 due diligence and at the earliest possible 

6 time. 

7 ‘‘(4) WAIVERS.—

8 ‘‘(A) FULL WAIVER.—On the initiative of 

9 the Secretary or at the request of an applicant, 

10 the Secretary shall grant a full waiver, as ap11 propriate, of the requirement to submit assess12 ments for a drug or biological product under 

13 this subsection if the applicant certifies and the 

14 Secretary finds that—

15 ‘‘(i) necessary studies are impossible 

16 or highly impracticable (because, for exam17 ple, the number of patients is so small or 

18 the patients are geographically dispersed); 

19 ‘‘(ii) there is evidence strongly sug20 gesting that the drug or biological product 

21 would be ineffective or unsafe in all pedi22 atric age groups; or 

23 ‘‘(iii) the drug or biological product—
6

•HR 2857 IH 

1 ‘‘(I) does not represent a mean2 ingful therapeutic benefit over existing 

3 therapies for pediatric patients; and 

4 ‘‘(II) is not likely to be used in a 

5 substantial number of pediatric pa6 tients.

7 ‘‘(B) PARTIAL WAIVER.—On the initiative 

8 of the Secretary or at the request of an appli9 cant, the Secretary shall grant a partial waiver, 

10 as appropriate, of the requirement to submit as11 sessments for a drug or biological product 

12 under this subsection with respect to a specific 

13 pediatric age group if the applicant certifies 

14 and the Secretary finds that—

15 ‘‘(i) necessary studies are impossible 

16 or highly impracticable (because, for exam17 ple, the number of patients in that age 

18 group is so small or patients in that age 

19 group are geographically dispersed); 

20 ‘‘(ii) there is evidence strongly sug21 gesting that the drug or biological product 

22 would be ineffective or unsafe in that age 

23 group; 

24 ‘‘(iii) the drug or biological product—
7

•HR 2857 IH 

1 ‘‘(I) does not represent a mean2 ingful therapeutic benefit over existing 

3 therapies for pediatric patients in that 

4 age group; and 

5 ‘‘(II) is not likely to be used by 

6 a substantial number of pediatric pa7 tients in that age group; or 

8 ‘‘(iv) the applicant can demonstrate 

9 that reasonable attempts to produce a pe10 diatric formulation necessary for that age 

11 group have failed. 

12 ‘‘(C) PEDIATRIC FORMULATION NOT POS13 SIBLE.—If a waiver is granted on the ground 

14 that it is not possible to develop a pediatric for15 mulation, the waiver shall cover only the pedi16 atric groups requiring that formulation. 

17 ‘‘(D) LABELING REQUIREMENT.—If the 

18 Secretary grants a full or partial waiver because 

19 there is evidence that a drug or biological prod20 uct would be ineffective or unsafe in pediatric 

21 populations, the information shall be included 

22 in the labeling for the drug or biological prod23 uct. 

24 ‘‘(b) MARKETED DRUGS AND BIOLOGICAL PROD25 UCTS.—
8

•HR 2857 IH 

1 ‘‘(1) IN GENERAL.—After providing notice in 

2 the form of a letter and an opportunity for written 

3 response and a meeting, which may include an advi4 sory committee meeting, the Secretary may (by 

5 order in the form of a letter) require the holder of 

6 an approved application for a drug under section 

7 505 or the holder of a license for a biological prod8 uct under section 351 of the Public Health Service 

9 Act (42 U.S.C. 262) to submit by a specified date 

10 the assessments described in subsection (a)(2) if the 

11 Secretary finds that—

12 ‘‘(A)(i) the drug or biological product is 

13 used for a substantial number of pediatric pa14 tients for the labeled indications; and 

15 ‘‘(ii) the absence of adequate labeling could 

16 pose significant risks to pediatric patients; or 

17 ‘‘(B)(i) there is reason to believe that the 

18 drug or biological product would represent a 

19 meaningful therapeutic benefit over existing 

20 therapies for pediatric patients for 1 or more of 

21 the claimed indications; and 

22 ‘‘(ii) the absence of adequate labeling could 

23 pose significant risks to pediatric patients. 

24 ‘‘(2) WAIVERS.—
9

•HR 2857 IH 

1 ‘‘(A) FULL WAIVER.—At the request of an 

2 applicant, the Secretary shall grant a full waiv3 er, as appropriate, of the requirement to submit 

4 assessments under this subsection if the appli5 cant certifies and the Secretary finds that—

6 ‘‘(i) necessary studies are impossible 

7 or highly impracticable (because, for exam8 ple, the number of patients in that age 

9 group is so small or patients in that age 

10 group are geographically dispersed); or 

11 ‘‘(ii) there is evidence strongly sug12 gesting that the drug or biological product 

13 would be ineffective or unsafe in all pedi14 atric age groups. 

15 ‘‘(B) PARTIAL WAIVER.—At the request of 

16 an applicant, the Secretary shall grant a partial 

17 waiver, as appropriate, of the requirement to 

18 submit assessments under this subsection with 

19 respect to a specific pediatric age group if the 

20 applicant certifies and the Secretary finds 

21 that—

22 ‘‘(i) necessary studies are impossible 

23 or highly impracticable (because, for exam24 ple, the number of patients in that age 
10

•HR 2857 IH 

1 group is so small or patients in that age 

2 group are geographically dispersed); 

3 ‘‘(ii) there is evidence strongly sug4 gesting that the drug or biological product 

5 would be ineffective or unsafe in that age 

6 group; 

7 ‘‘(iii)(I) the drug or biological prod8 uct—

9 ‘‘(aa) does not represent a mean10 ingful therapeutic benefit over existing 

11 therapies for pediatric patients in that 

12 age group; and 

13 ‘‘(bb) is not likely to be used in 

14 a substantial number of pediatric pa15 tients in that age group; and 

16 ‘‘(II) the absence of adequate labeling 

17 could not pose significant risks to pediatric 

18 patients; or 

19 ‘‘(iv) the applicant can demonstrate 

20 that reasonable attempts to produce a pe21 diatric formulation necessary for that age 

22 group have failed. 

23 ‘‘(C) PEDIATRIC FORMULATION NOT POS24 SIBLE.—If a waiver is granted on the ground 

25 that it is not possible to develop a pediatric for-
11

•HR 2857 IH 

1 mulation, the waiver shall cover only the pedi2 atric groups requiring that formulation. 

3 ‘‘(D) LABELING REQUIREMENT.—If the 

4 Secretary grants a full or partial waiver because 

5 there is evidence that a drug or biological prod6 uct would be ineffective or unsafe in pediatric 

7 populations, the information shall be included

8 in the labeling for the drug or biological prod9 uct. 

10 ‘‘(3) RELATIONSHIP TO OTHER PEDIATRIC PRO11 VISIONS.—

12 ‘‘(A) NO ASSESSMENT WITHOUT WRITTEN

13 REQUEST.—No assessment may be required 

14 under paragraph (1) for a drug subject to an 

15 approved application under section 505 un16 less—

17 ‘‘(i) the Secretary has issued a written 

18 request for a related pediatric study under 

19 section 505A(c) of this Act or section 409I 

20 of the Public Health Service Act (42 

21 U.S.C. 284m); 

22 ‘‘(ii)(I) if the request was made under 

23 section 505A(c)—
12

•HR 2857 IH 

1 ‘‘(aa) the recipient of the written 

2 request does not agree to the request; 

3 or 

4 ‘‘(bb) the Secretary does not re5 ceive a response as specified under 

6 section 505A(d)(4)(A); or 

7 ‘‘(II) if the request was made under 

8 section 409I of the Public Health Service 

9 Act (42 U.S.C. 284m)—

10 ‘‘(aa) the recipient of the written 

11 request does not agree to the request; 

12 or 

13 ‘‘(bb) the Secretary does not re14 ceive a response as specified under 

15 section 409I(c)(2) of that Act; and 

16 ‘‘(iii)(I) the Secretary certifies under 

17 subparagraph (B) that there are insuffi18 cient funds under sections 409I and 499 of 

19 the Public Health Service Act (42 U.S.C. 

20 284m, 290b) to conduct the study; or 

21 ‘‘(II) the Secretary publishes in the 

22 Federal Register a certification that cer23 tifies that—

24 ‘‘(aa) no contract or grant has 

25 been awarded under section 409I or 
13

•HR 2857 IH 

1 499 of the Public Health Service Act 

2 (42 U.S.C. 284m, 290b); and 

3 ‘‘(bb) not less than 270 days 

4 have passed since the date of a certifi5 cation under subparagraph (B) that 

6 there are sufficient funds to conduct 

7 the study. 

8 ‘‘(B) NO AGREEMENT TO REQUEST.—Not 

9 later than 60 days after determining that no 

10 holder will agree to the written request (includ11 ing a determination that the Secretary has not 

12 received a response specified under section 

13 505A(d) of this Act or section 409I of the Pub14 lic Health Service Act (42 U.S.C. 284m), the 

15 Secretary shall certify whether the Secretary 

16 has sufficient funds to conduct the study under 

17 section 409I or 499 of the Public Health Serv18 ice Act (42 U.S.C. 284m, 290b), taking into ac19 count the prioritization under section 409I. 

20 ‘‘(c) MEANINGFUL THERAPEUTIC BENEFIT.—For 

21 the purposes of paragraph (4)(A)(iii)(I) and (4)(B)(iii)(I) 

22 of subsection (a) and paragraphs (1)(B)(i) and 

23 (2)(B)(iii)(I)(aa) of subsection (b), a drug or biological 

24 product shall be considered to represent a meaningful 
14

•HR 2857 IH 

1 therapeutic benefit over existing therapies if the Secretary 

2 estimates that—

3 ‘‘(1) if approved, the drug or biological product 

4 would represent a significant improvement in the 

5 treatment, diagnosis, or prevention of a disease, 

6 compared with marketed products adequately labeled 

7 for that use in the relevant pediatric population; or 

8 ‘‘(2) the drug or biological product is in a class 

9 of products or for an indication for which there is 

10 a need for additional options. 

11 ‘‘(d) SUBMISSION OF ASSESSMENTS.—If a person 

12 fails to submit an assessment described in subsection 

13 (a)(2), or a request for approval of a pediatric formulation 

14 described in subsection (a) or (b), in accordance with ap15 plicable provisions of subsections (a) and (b)—

16 ‘‘(1) the drug or biological product that is the 

17 subject of the assessment or request may be consid18 ered misbranded solely because of that failure and 

19 subject to relevant enforcement action (except that 

20 the drug or biological product shall not be subject to 

21 action under section 303); but 

22 ‘‘(2) the failure to submit the assessment or re23 quest shall not be the basis for a proceeding—

24 ‘‘(A) to withdraw approval for a drug 

25 under section 505(e); or 
15

•HR 2857 IH 

1 ‘‘(B) to revoke the license for a biological 

2 product under section 351 of the Public Health 

3 Service Act (42 U.S.C. 262). 

4 ‘‘(e) MEETINGS.—Before and during the investiga5 tional process for a new drug or biological product, the 

6 Secretary shall meet at appropriate times with the sponsor 

7 of the new drug or biological product to discuss—

8 ‘‘(1) information that the sponsor submits on 

9 plans and timelines for pediatric studies; or 

10 ‘‘(2) any planned request by the sponsor for 

11 waiver or deferral of pediatric studies.

12 ‘‘(f) SCOPE OF AUTHORITY.—Nothing in this section 

13 provides to the Secretary any authority to require a pedi14 atric assessment of any drug or biological product, or any 

15 assessment regarding other populations or uses of a drug 

16 or biological product, other than the pediatric assessments 

17 described in this section. 

18 ‘‘(g) ORPHAN DRUGS.—Unless the Secretary requires 

19 otherwise by regulation, this section does not apply to any 

20 drug for an indication for which orphan designation has 

21 been granted under section 526. 

22 ‘‘(h) INTEGRATION WITH OTHER PEDIATRIC STUD23 IES.—The authority under this section shall remain in ef24 fect so long as an application subject to this section may 
16

•HR 2857 IH 

1 be accepted for filing by the Secretary on or before the 

2 date specified in section 505A(n).’’.

3 (b) CONFORMING AMENDMENTS.—

4 (1) Section 505(b)(1) of the Federal Food, 

5 Drug, and Cosmetic Act (21 U.S.C. 355(b)(1)) is 

6 amended in the second sentence—

7 (A) by striking ‘‘and (F)’’ and inserting 

8 ‘‘(F)’’; and 

9 (B) by striking the period at the end and 

10 inserting ‘‘, and (G) any assessments required 

11 under section 505B.’’.

12 (2) Section 505A(h) of the Federal Food, Drug, 

13 and Cosmetic Act (21 U.S.C. 355a(h)) is amended—

14 (A) in the subsection heading, by striking 

15 ‘‘REGULATIONS’’ and inserting ‘‘PEDIATRIC RE16 SEARCH REQUIREMENTS’’; and 

17 (B) by striking ‘‘pursuant to regulations 

18 promulgated by the Secretary’’ and inserting 

19 ‘‘by a provision of law (including a regulation) 

20 other than this section’’. 

21 (3) Section 351(a)(2) of the Public Health 

22 Service Act (42 U.S.C. 262(a)(2)) is amended—

23 (A) by redesignating subparagraph (B) as 

24 subparagraph (C); and
17

•HR 2857 IH 

1 (B) by inserting after subparagraph (A) 

2 the following: 

3 ‘‘(B) PEDIATRIC STUDIES.—A person that 

4 submits an application for a license under this 

5 paragraph shall submit to the Secretary as part 

6 of the application any assessments required 

7 under section 505B of the Federal Food, Drug, 

8 and Cosmetic Act.’’. 

9 SEC. 3. TECHNICAL AND CONFORMING AMENDMENTS. 

10 (a) ABBREVIATED NEW DRUG APPLICATION.—Sec11 tion 505A of the Federal Food, Drug, and Cosmetic Act 

12 (21 U.S.C. 355a) is amended in subparagraphs (A) and 

13 (B) of subsection (b)(2) and subparagraphs (A) and (B) 

14 of subsection (c)(2) by striking ‘‘505(j)(4)(B)’’ and insert15 ing ‘‘505(j)(5)(B)’’. 

16 (b) PEDIATRIC ADVISORY COMMITTEE.—

17 (1) Section 505A(i)(2) of the Federal Food, 

18 Drug, and Cosmetic Act (21 U.S.C. 355a(i)(2)) is 

19 amended by striking ‘‘Advisory Subcommittee of the 

20 Anti-Infective Drugs’’ each place it appears. 

21 (2) Section 14 of the Best Pharmaceuticals for 

22 Children Act (42 U.S.C. 284m note; Public Law 

23 107–109) is amended—

24 (A) in the section heading, by striking 

25 ‘‘PHARMACOLOGY’’; 
18

•HR 2857 IH 

1 (B) in subsection (a), by striking ‘‘(42 

2 U.S.C. 217a),’’ and inserting (42 U.S.C. 217a) 

3 or other appropriate authority,’’; 

4 (C) in subsection (b)—

5 (i) in paragraph (1), by striking ‘‘and 

6 in consultation with the Director of the 

7 National Institutes of Health’’; and 

8 (ii) in paragraph (2), by striking ‘‘and 

9 505A’’ and inserting ‘‘505A, and 505B’’; 

10 and 

11 (D) by striking ‘‘pharmacology’’ each place 

12 it appears and inserting ‘‘therapeutics’’. 

13 (3) Section 15(a)(2)(A) of the Best Pharma14 ceuticals for Children Act (115 Stat. 1419) is 

15 amended by striking ‘‘Pharmacology’’. 

16 (4) Section 16(1)(C) of the Best Pharma17 ceuticals for Children Act (21 U.S.C. 355a note; 

18 Public Law 107–109) is amended by striking ‘‘Advi19 sory Subcommittee of the Anti-Infective Drugs’’. 

20 (5) Section 17(b)(1) of the Best Pharma21 ceuticals for Children Act (21 U.S.C. 355b(b)(1)) is 

22 amended in the second sentence by striking ‘‘Advi23 sory Subcommittee of the Anti-Infective Drugs’’. 

24 (6) Paragraphs (8), (9), and (11) of section 

25 409I(c) of the Public Health Service Act (42 U.S.C. 
19

•HR 2857 IH 

1 284m(c)) are amended by striking ‘‘Advisory Sub2 committee of the Anti-Infective Drugs’’ each place it 

3 appears. 

4 SEC. 4. EFFECTIVE DATE. 

5 (a) IN GENERAL.—Subject to subsection (b), this Act 

6 and the amendments made by this Act take effect on the 

7 date of enactment of this Act. 

8 (b) APPLICABILITY TO NEW DRUGS AND BIOLOGICAL

9 PRODUCTS.—

10 (1) IN GENERAL.—Subsection (a) of section 

11 505B of the Federal Food, Drug, and Cosmetic Act 

12 (as added by section 2) shall apply to an application 

13 described in paragraph (1) of that subsection sub14 mitted to the Secretary of Health and Human Serv15 ices on or after April 1, 1999. 

16 (2) WAIVERS AND DEFERRALS.—

17 (A) WAIVER OR DEFERRAL GRANTED.—If, 

18 with respect to an application submitted to the 

19 Secretary of Health and Human Services be20 tween April 1, 1999, and the date of enactment 

21 of this Act, a waiver or deferral of pediatric as22 sessments was granted under regulations of the 

23 Secretary then in effect, the waiver or deferral 

24 shall be a waiver or deferral under subsection 

25 (a) of section 505B of the Federal Food, Drug, 
20

•HR 2857 IH 

1 and Cosmetic Act, except that any date speci2 fied in such a deferral shall be extended by the 

3 number of days that is equal to the number of 

4 days between October 17, 2002, and the date of 

5 enactment of this Act. 

6 (B) WAIVER OR DEFERRAL NOT GRANT7 ED.—If, with respect to an application sub8 mitted to the Secretary of Health and Human 

9 Services between April 1, 1999, and the date of 

10 enactment of this Act, neither a waiver nor de11 ferral of pediatric assessments was granted 

12 under regulations of the Secretary then in ef13 fect, the person that submitted the application 

14 shall be required to submit assessments under 

15 subsection (a)(2) of section 505B of the Fed16 eral Food, Drug, and Cosmetic Act on the date 

17 that is the later of—

18 (i) the date that is 1 year after the 

19 date of enactment of this Act; or 

20 (ii) such date as the Secretary may 

21 specify under subsection (a)(3) of that sec22 tion; 

23 unless the Secretary grants a waiver under sub24 section (a)(4) of that section. 
21

•HR 2857 IH 

1 (c) NO LIMITATION OF AUTHORITY.—Neither the 

2 lack of guidance or regulations to implement this Act or 

3 the amendments made by this Act nor the pendency of 

4 the process for issuing guidance or regulations shall limit 

5 the authority of the Secretary of Health and Human Serv6 ices under, or defer any requirement under, this Act or 

7 those amendments.

Æ