I 

115TH CONGRESS 

1ST SESSION H. R. 1231 

To amend the Federal Food, Drug, and Cosmetic Act to establish a program 

to increase the development of new drugs to treat pediatric cancers, 

and for other purposes. 

IN THE HOUSE OF REPRESENTATIVES 

FEBRUARY 27, 2017 

Mr. MCCAUL (for himself, Mr. BUTTERFIELD, Mr. DUFFY, and Ms. CLARKE

of New York) 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 

establish a program to increase the development of new 

drugs to treat pediatric cancers, and for other purposes. 

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 ‘‘Research to Accelerate 

5 Cures and Equity for Children Act’’ or the ‘‘RACE for 

6 Children Act’’. 


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1 SEC. 2. DRUG DEVELOPMENT FOR PEDIATRIC CANCER. 

2 (a) MOLECULAR TARGETS REGARDING CANCER

3 DRUGS.—Section 505B of the Federal Food, Drug, and 

4 Cosmetic Act (21 U.S.C. 355c) is amended— 

5 (1) in subsection (a)(2)(A)(i) by striking ‘‘prod6 uct for the claimed indications in all relevant pedi7 atric subpopulations; and’’ and inserting ‘‘product in 

8 all relevant pediatric subpopulations— 

9 ‘‘(I) for the claimed indications; 

10 or 

11 ‘‘(II) for a pediatric cancer indi12 cation, if the drug is intended for the 

13 treatment of an adult cancer and is 

14 directed at a molecular target consid15 ered to be germane to the growth and 

16 progression of such pediatric cancer; 

17 and’’; 

18 (2) in subsection (b)(1)— 

19 (A) by amending subparagraph (A)(i) to 

20 read as follows: 

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

22 used for a substantial number of pediatric pa23 tients— 

24 ‘‘(I) for the labeled indications; or 

25 ‘‘(II) for a pediatric cancer indication, 

26 if the drug is intended for the treatment of 


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1 an adult cancer and is directed at a molec2 ular target considered to be germane to 

3 the growth and progression of such pedi4 atric cancer; and’’; and 

5 (B) by amending subparagraph (B) to read 

6 as follows: 

7 ‘‘(B) there is reason to believe that the 

8 drug or biological product would represent a 

9 meaningful therapeutic benefit over existing 

10 therapies for pediatric patients— 

11 ‘‘(i) for one or more of the claimed in12 dications; or 

13 ‘‘(ii) for a pediatric cancer indication, 

14 if the drug is intended for the treatment of 

15 an adult cancer and is directed at a molec16 ular target considered to be germane to 

17 the growth and progression of such pedi18 atric cancer; or’’; and 

19 (3) by amending paragraph (2) of subsection 

20 (c) to read as follows: 

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

22 of products, is for an indication, or is directed at a 

23 specific molecular target in an adult cancer and such 

24 molecular target is germane to the growth or proVerDate Sep 11 2014 23:48 Mar 01, 2017 Jkt 069200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H1231.IH H1231

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1 gression of cancer in a pediatric cancer, for which 

2 there is need for additional options.’’. 

3 (b) EARLY MEETING ON PEDIATRIC STUDY PLAN.— 

4 (1) IN GENERAL.—Clause (i) of section 

5 505B(e)(2)(C) of the Federal Food, Drug, and Cos6 metic Act (21 U.S.C. 355c(e)(2)(C)) is amended to 

7 read as follows: 

8 ‘‘(i) shall meet with the applicant— 

9 ‘‘(I) if requested by the applicant 

10 with respect to a drug that is in11 tended to treat a serious or life12 threatening disease or condition, to 

13 discuss preparation of the initial pedi14 atric study plan, not later than the 

15 end-of-Phase 1 meeting (as such term 

16 is used in section 312.47(b) of title 

17 21, Code of Federal Regulations, or 

18 successor regulations) or within 30 

19 days of receipt of such request, which20 ever is later; 

21 ‘‘(II) to discuss the initial pedi22 atric study plan as soon as prac23 ticable, but not later than 90 calendar 

24 days after the receipt of such plan 

25 under subparagraph (A); and 


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1 ‘‘(III) to discuss any scientific or 

2 operational challenges that may be the 

3 basis of a deferral under subsection 

4 (a)(3) or a full or partial waiver under 

5 subsection (a)(4);’’. 

6 (2) CONFORMING CHANGES.—Section 505B(e) 

7 of the Federal Food, Drug, and Cosmetic Act (21 

8 U.S.C. 355c(e)) is amended— 

9 (A) in the heading of paragraph (2), by 

10 striking ‘‘MEETING’’ and inserting ‘‘MEETINGS’’; 

11 (B) in the heading of paragraph (2)(C), by 

12 striking ‘‘MEETING’’ and inserting ‘‘MEET13 INGS’’; 

14 (C) in clauses (ii) and (iii) of paragraph 

15 (2)(C), by striking ‘‘no meeting’’ each place it 

16 appears and inserting ‘‘no meeting under clause 

17 (i)(II)’’; and 

18 (D) in paragraph (3) by striking ‘‘meeting 

19 under paragraph (2)(C)(i)’’ and inserting 

20 ‘‘meeting under paragraph (2)(C)(i)(II)’’. 

21 (c) ORPHAN DRUGS.—Section 505B(k) of the Fed22 eral Food, Drug, and Cosmetic Act (21 U.S.C. 355c(k)) 

23 is amended by inserting ‘‘except in the case of a drug or 

24 biological product that is intended for the treatment of 

25 an adult cancer and is directed at a molecular target conVerDate Sep 11 2014 23:48 Mar 01, 2017 Jkt 069200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H1231.IH H1231

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1 sidered to be germane to the growth and progression of 

2 a pediatric cancer,’’ after ‘‘regulation,’’. 

3 (d) GUIDANCE.—Not later than 1 year after the date 

4 of enactment of this Act, the Secretary of Health and 

5 Human Services, acting through the Commissioner of 

6 Food and Drugs, shall issue guidance on the implementa7 tion of the amendments to section 505B of the Federal 

8 Food, Drug, and Cosmetic Act (21 U.S.C. 355c) made by 

9 this section, including— 

10 (1) study designs; 

11 (2) molecular targets considered to be germane 

12 to the growth and progression present in one or 

13 more cancers in pediatric populations that may be 

14 appropriate for assessment under such section 505B, 

15 as so amended; and 

16 (3) considerations for implementation of such 

17 section 505B, as so amended, and waivers of the re18 quirements of such section 505B with regard to mo19 lecular targets for which several drugs may be under 

20 investigation. 

21 (e) APPLICABILITY.—This section and the amend22 ments made by this section apply with respect to applica23 tions for a drug submitted under section 505 of the Fed24 eral Food, Drug, and Cosmetic Act (21 U.S.C. 355) or 

25 section 351 of the Public Health Service Act (42 U.S.C. 


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1 262) on or after the date that is 18 months after the date 

2 of enactment of this Act. 

3 (f) REPORT TO CONGRESS.—Section 508(b) of the 

4 FDA Safety and Innovation Act (21 U.S.C. 355c–1(b)) 

5 is amended— 

6 (1) in paragraph (10), by striking ‘‘; and’’ and 

7 inserting ‘‘;’’; and 

8 (2) by striking paragraph (11) and inserting 

9 the following: 

10 ‘‘(11) an assessment of the impact of the 

11 amendments to such section 505B made by the 

12 RACE for Children Act on pediatric labeling of 

13 drugs and pediatric labeling of molecularly targeted 

14 drugs for the treatment of cancer; 

15 ‘‘(12) an assessment of the efforts of the Sec16 retary to implement the plan developed under sec17 tion 505C–1 of the Federal Food, Drug, and Cos18 metic Act, regarding earlier submission of pediatric 

19 studies under sections 505A and 505B, including— 

20 ‘‘(A) the average length of time after the 

21 approval of an application under section 

22 505(b)(1) of the Federal Food, Drug, and Cos23 metic Act (21 U.S.C. 355(b)(1)) before studies 

24 conducted pursuant to such sections 505A or 


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1 505B are completed, submitted, and incor2 porated into labeling; 

3 ‘‘(B) the average length of time after the 

4 receipt of a proposed pediatric study request be5 fore the Secretary responds to such request; 

6 ‘‘(C) the average length of time after the 

7 submission of a proposed pediatric study re8 quest before the Secretary issues a written re9 quest for such studies; 

10 ‘‘(D) the number of written requests issued 

11 for each investigational new drug prior to the 

12 submission of an application under section 

13 505(b)(1) of the Federal Food, Drug, and Cos14 metic Act; and 

15 ‘‘(E) the average number, and range of 

16 numbers, of amendments to written requests 

17 issued; 

18 ‘‘(13) a list of sponsors of applications or hold19 ers of approved applications who received exclusivity 

20 under such section 505A after receiving a letter 

21 issued under such section 505B(d)(1) and before the 

22 studies referred to in such letter were completed and 

23 submitted; and 


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1 ‘‘(14) a list of assessments required under sub2 sections (a)(2)(A)(i)(II) and (b)(1)(B)(ii) of section 

3 505B.’’. 

4 (g) RULE OF CONSTRUCTION.—Nothing in this sec5 tion, including the amendments made by this section, shall 

6 limit the authority of the Secretary of Health and Human 

7 Services to issue written requests under section 505A of 

8 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

9 355a). 

10 SEC. 3. IMPROVING THE TIMELINESS OF PEDIATRIC STUD11 IES. 

12 (a) INFORMING INTERNAL REVIEW COMMITTEE.— 

13 Section 505A(f) of the Federal Food, Drug, and Cosmetic 

14 Act (21 U.S.C. 355a(f)) is amended by adding at the end 

15 the following: 

16 ‘‘(7) INFORMING INTERNAL REVIEW COM17 MITTEE.—The Secretary shall provide to the com18 mittee referred to in paragraph (1) any response 

19 issued to an applicant or holder with respect to a 

20 proposed pediatric study request.’’. 

21 (b) ACTION ON SUBMISSIONS.—Section 505A(d) of 

22 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

23 355a(d)) is amended— 

24 (1) by redesignating paragraphs (3) through 

25 (5) as paragraphs (4) through (6), respectively; and 


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1 (2) by inserting after paragraph (2) the fol2 lowing: 

3 ‘‘(3) ACTION ON SUBMISSIONS.—The Secretary 

4 shall review and act upon a submission of a pro5 posed pediatric study request or a sponsor’s pro6 posed amendment to a written request for pediatric 

7 studies within 120 days of the submission.’’. 

8 (c) STUDY.—The Secretary of Health and Human 

9 Services, acting through the internal review committee es10 tablished under section 505C of the Federal Food, Drug, 

11 and Cosmetic Act (21 U.S.C. 355d) shall, not later than 

12 one year after the date of enactment of this Act, develop 

13 and implement a plan to achieve, when appropriate, earlier 

14 submission of pediatric studies under section 505A of the 

15 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a). 

16 Such plan shall include recommendations to achieve— 

17 (1) earlier discussion of proposed pediatric 

18 study requests and written requests with sponsors, 

19 and if appropriate, at the meeting required under 

20 section 505B(e)(2)(C) of the Federal Food, Drug, 

21 and Cosmetic Act (21 U.S.C. 355c(e)(2)(C)), as 

22 amended by section 2; 

23 (2) earlier issuance of written requests for a pe24 diatric study under such section 505A, including for 

25 investigational new drugs prior to the submission of 


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1 an application under section 505(b)(1) of the Fed2 eral Food, Drug, and Cosmetic Act (21 U.S.C. 

3 355(b)(1)); and 

4 (3) shorter timelines, when appropriate, for the 

5 completion of studies pursuant to a written request 

6 under such section 505A. 

7 SEC. 4. NEONATOLOGY EXPERTISE. 

8 Section 6(d) of the Best Pharmaceuticals for Chil9 dren Act (21 U.S.C. 393a(d)) is amended by striking ‘‘For 

10 the 5-year period beginning on the date of enactment of 

11 this subsection, at’’ and inserting ‘‘At’’. 

Æ 


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