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. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Research to Accelerate 
Cures and Equity for Children Act’’ or the ‘‘RACE for 
Children Act’’. 
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SEC. 2. DRUG DEVELOPMENT FOR PEDIATRIC CANCER. 
(a) MOLECULAR
TARGETS
REGARDING
CANCER 
DRUGS.—Section 505B of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355c) is amended— 
(1) in subsection (a)(2)(A)(i) by striking ‘‘prod-
uct for the claimed indications in all relevant pedi-
atric subpopulations; and’’ and inserting ‘‘product in 
all relevant pediatric subpopulations— 
‘‘(I) for the claimed indications; 
or 
‘‘(II) for a pediatric cancer indi-
cation, if the drug is intended for the 
treatment of an adult cancer and is 
directed at a molecular target consid-
ered to be germane to the growth and 
progression of such pediatric cancer; 
and’’; 
(2) in subsection (b)(1)— 
(A) by amending subparagraph (A)(i) to 
read as follows: 
‘‘(A)(i) the drug or biological product is 
used for a substantial number of pediatric pa-
tients— 
‘‘(I) for the labeled indications; or 
‘‘(II) for a pediatric cancer indication, 
if the drug is intended for the treatment of 
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an adult cancer and is directed at a molec-
ular target considered to be germane to 
the growth and progression of such pedi-
atric cancer; and’’; and 
(B) by amending subparagraph (B) to read 
as follows: 
‘‘(B) there is reason to believe that the 
drug or biological product would represent a 
meaningful therapeutic benefit over existing 
therapies for pediatric patients— 
‘‘(i) for one or more of the claimed in-
dications; or 
‘‘(ii) for a pediatric cancer indication, 
if the drug is intended for the treatment of 
an adult cancer and is directed at a molec-
ular target considered to be germane to 
the growth and progression of such pedi-
atric cancer; or’’; and 
(3) by amending paragraph (2) of subsection 
(c) to read as follows: 
‘‘(2) the drug or biological product is in a class 
of products, is for an indication, or is directed at a 
specific molecular target in an adult cancer and such 
molecular target is germane to the growth or pro-
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gression of cancer in a pediatric cancer, for which 
there is need for additional options.’’. 
(b) EARLY MEETING ON PEDIATRIC STUDY PLAN.— 
(1) 
IN
GENERAL.—Clause 
(i) 
of 
section 
505B(e)(2)(C) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355c(e)(2)(C)) is amended to 
read as follows: 
‘‘(i) shall meet with the applicant— 
‘‘(I) if requested by the applicant 
with respect to a drug that is in-
tended to treat a serious or life- 
threatening disease or condition, to 
discuss preparation of the initial pedi-
atric study plan, not later than the 
end-of-Phase 1 meeting (as such term 
is used in section 312.47(b) of title 
21, Code of Federal Regulations, or 
successor regulations) or within 30 
days of receipt of such request, which-
ever is later; 
‘‘(II) to discuss the initial pedi-
atric study plan as soon as prac-
ticable, but not later than 90 calendar 
days after the receipt of such plan 
under subparagraph (A); and 
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‘‘(III) to discuss any scientific or 
operational challenges that may be the 
basis of a deferral under subsection 
(a)(3) or a full or partial waiver under 
subsection (a)(4);’’. 
(2) CONFORMING CHANGES.—Section 505B(e) 
of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355c(e)) is amended— 
(A) in the heading of paragraph (2), by 
striking ‘‘MEETING’’ and inserting ‘‘MEETINGS’’; 
(B) in the heading of paragraph (2)(C), by 
striking ‘‘MEETING’’ and inserting ‘‘MEET-
INGS’’; 
(C) in clauses (ii) and (iii) of paragraph 
(2)(C), by striking ‘‘no meeting’’ each place it 
appears and inserting ‘‘no meeting under clause 
(i)(II)’’; and 
(D) in paragraph (3) by striking ‘‘meeting 
under paragraph (2)(C)(i)’’ and inserting 
‘‘meeting under paragraph (2)(C)(i)(II)’’. 
(c) ORPHAN DRUGS.—Section 505B(k) of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 355c(k)) 
is amended by inserting ‘‘except in the case of a drug or 
biological product that is intended for the treatment of 
an adult cancer and is directed at a molecular target con-
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sidered to be germane to the growth and progression of 
a pediatric cancer,’’ after ‘‘regulation,’’. 
(d) GUIDANCE.—Not later than 1 year after the date 
of enactment of this Act, the Secretary of Health and 
Human Services, acting through the Commissioner of 
Food and Drugs, shall issue guidance on the implementa-
tion of the amendments to section 505B of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 355c) made by 
this section, including— 
(1) study designs; 
(2) molecular targets considered to be germane 
to the growth and progression present in one or 
more cancers in pediatric populations that may be 
appropriate for assessment under such section 505B, 
as so amended; and 
(3) considerations for implementation of such 
section 505B, as so amended, and waivers of the re-
quirements of such section 505B with regard to mo-
lecular targets for which several drugs may be under 
investigation. 
(e) APPLICABILITY.—This section and the amend-
ments made by this section apply with respect to applica-
tions for a drug submitted under section 505 of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 355) or 
section 351 of the Public Health Service Act (42 U.S.C. 
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262) on or after the date that is 18 months after the date 
of enactment of this Act. 
(f) REPORT TO CONGRESS.—Section 508(b) of the 
FDA Safety and Innovation Act (21 U.S.C. 355c–1(b)) 
is amended— 
(1) in paragraph (10), by striking ‘‘; and’’ and 
inserting ‘‘;’’; and 
(2) by striking paragraph (11) and inserting 
the following: 
‘‘(11) an assessment of the impact of the 
amendments to such section 505B made by the 
RACE for Children Act on pediatric labeling of 
drugs and pediatric labeling of molecularly targeted 
drugs for the treatment of cancer; 
‘‘(12) an assessment of the efforts of the Sec-
retary to implement the plan developed under sec-
tion 505C–1 of the Federal Food, Drug, and Cos-
metic Act, regarding earlier submission of pediatric 
studies under sections 505A and 505B, including— 
‘‘(A) the average length of time after the 
approval of an application under section 
505(b)(1) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355(b)(1)) before studies 
conducted pursuant to such sections 505A or 
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505B are completed, submitted, and incor-
porated into labeling; 
‘‘(B) the average length of time after the 
receipt of a proposed pediatric study request be-
fore the Secretary responds to such request; 
‘‘(C) the average length of time after the 
submission of a proposed pediatric study re-
quest before the Secretary issues a written re-
quest for such studies; 
‘‘(D) the number of written requests issued 
for each investigational new drug prior to the 
submission of an application under section 
505(b)(1) of the Federal Food, Drug, and Cos-
metic Act; and 
‘‘(E) the average number, and range of 
numbers, of amendments to written requests 
issued; 
‘‘(13) a list of sponsors of applications or hold-
ers of approved applications who received exclusivity 
under such section 505A after receiving a letter 
issued under such section 505B(d)(1) and before the 
studies referred to in such letter were completed and 
submitted; and 
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‘‘(14) a list of assessments required under sub-
sections (a)(2)(A)(i)(II) and (b)(1)(B)(ii) of section 
505B.’’. 
(g) RULE OF CONSTRUCTION.—Nothing in this sec-
tion, including the amendments made by this section, shall 
limit the authority of the Secretary of Health and Human 
Services to issue written requests under section 505A of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a). 
SEC. 3. IMPROVING THE TIMELINESS OF PEDIATRIC STUD-
IES. 
(a) INFORMING INTERNAL REVIEW COMMITTEE.— 
Section 505A(f) of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 355a(f)) is amended by adding at the end 
the following: 
‘‘(7) INFORMING
INTERNAL
REVIEW
COM-
MITTEE.—The Secretary shall provide to the com-
mittee referred to in paragraph (1) any response 
issued to an applicant or holder with respect to a 
proposed pediatric study request.’’. 
(b) ACTION ON SUBMISSIONS.—Section 505A(d) of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a(d)) is amended— 
(1) by redesignating paragraphs (3) through 
(5) as paragraphs (4) through (6), respectively; and 
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(2) by inserting after paragraph (2) the fol-
lowing: 
‘‘(3) ACTION ON SUBMISSIONS.—The Secretary 
shall review and act upon a submission of a pro-
posed pediatric study request or a sponsor’s pro-
posed amendment to a written request for pediatric 
studies within 120 days of the submission.’’. 
(c) STUDY.—The Secretary of Health and Human 
Services, acting through the internal review committee es-
tablished under section 505C of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 355d) shall, not later than 
one year after the date of enactment of this Act, develop 
and implement a plan to achieve, when appropriate, earlier 
submission of pediatric studies under section 505A of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a). 
Such plan shall include recommendations to achieve— 
(1) earlier discussion of proposed pediatric 
study requests and written requests with sponsors, 
and if appropriate, at the meeting required under 
section 505B(e)(2)(C) of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 355c(e)(2)(C)), as 
amended by section 2; 
(2) earlier issuance of written requests for a pe-
diatric study under such section 505A, including for 
investigational new drugs prior to the submission of 
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an application under section 505(b)(1) of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 
355(b)(1)); and 
(3) shorter timelines, when appropriate, for the 
completion of studies pursuant to a written request 
under such section 505A. 
SEC. 4. NEONATOLOGY EXPERTISE. 
Section 6(d) of the Best Pharmaceuticals for Chil-
dren Act (21 U.S.C. 393a(d)) is amended by striking ‘‘For 
the 5-year period beginning on the date of enactment of 
this subsection, at’’ and inserting ‘‘At’’. 
Æ 
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