I 
117TH CONGRESS 
1ST SESSION H. R. 5416 
To amend the Federal Food, Drug, and Cosmetic Act to establish additional 
authorities of the Food and Drug Administration regarding the conduct 
of pediatric investigations of molecularly targeted drugs to treat cancer, 
and for other purposes. 
IN THE HOUSE OF REPRESENTATIVES 
SEPTEMBER 29, 2021 
Mr. BUTTERFIELD (for himself, Mr. MCCAUL, and Ms. WILD) 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 additional authorities of the Food and Drug 
Administration regarding the conduct of pediatric inves-
tigations of molecularly targeted drugs to treat cancer, 
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 ‘‘Give Kids a Chance 
Act’’. 
VerDate Sep 11 2014 
17:05 Oct 28, 2021
Jkt 029200
PO 00000
Frm 00001
Fmt 6652
Sfmt 6201
E:\BILLS\H5416.IH
H5416
pamtmann on DSKBC07HB2PROD with BILLS
SEC. 2. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDI-
TIONAL AUTHORITIES OF FOOD AND DRUG 
ADMINISTRATION 
REGARDING 
MOLECU-
LARLY TARGETED CANCER DRUGS. 
(a) IN GENERAL.— 
(1) AUTHORITY REGARDING INVESTIGATION OF 
COMBINATION
OF
ACTIVE
INGREDIENTS.—Section 
505B(a)(3) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355c(a)(3)) is amended— 
(A) by redesignating subparagraphs (B) 
and (C) as subparagraphs (C) and (D), respec-
tively; and 
(B) by striking subparagraph (A) and in-
serting the following: 
‘‘(A) IN GENERAL.—For purposes of para-
graph (1)(B), the investigation described in this 
paragraph is (as determined by the Secretary) 
a molecularly targeted pediatric cancer inves-
tigation of— 
‘‘(i) the drug or biological product for 
which the application referred to in such 
paragraph is submitted; or 
‘‘(ii) the active ingredient or ingredi-
ents of such drug or biological product in 
combination with— 
VerDate Sep 11 2014 
17:05 Oct 28, 2021
Jkt 029200
PO 00000
Frm 00002
Fmt 6652
Sfmt 6201
E:\BILLS\H5416.IH
H5416
pamtmann on DSKBC07HB2PROD with BILLS
‘‘(I) an active ingredient of a 
drug or biological product for which 
there is in effect an exemption for in-
vestigational use under section 505(i) 
that was submitted by the person sub-
mitting the application referred to in 
paragraph (1)(B); or 
‘‘(II) an active ingredient of a 
drug for which an approval under sec-
tion 505 is in effect, or an active in-
gredient of a biological product for 
which an approval under section 351 
of the Public Health Service Act is in 
effect. 
‘‘(B) ADDITIONAL REQUIREMENTS.— 
‘‘(i) DESIGN
OF
INVESTIGATION.—A 
molecularly targeted pediatric cancer inves-
tigation referred to in subparagraph (A) 
shall be designed to yield clinically mean-
ingful pediatric study data, gathered using 
appropriate formulations for each age 
group for which the study is required, re-
garding dosing, safety, and preliminary ef-
ficacy. 
VerDate Sep 11 2014 
17:05 Oct 28, 2021
Jkt 029200
PO 00000
Frm 00003
Fmt 6652
Sfmt 6201
E:\BILLS\H5416.IH
H5416
pamtmann on DSKBC07HB2PROD with BILLS
‘‘(ii) PURPOSE OF INVESTIGATION.— 
The purpose of a molecularly targeted pe-
diatric cancer investigation referred to in 
subparagraph (A) shall be— 
‘‘(I) in the case of such an inves-
tigation conducted with respect to a 
drug or biological product referred to 
in clause (i) of such subparagraph, to 
inform potential pediatric labeling of 
the drug or biological product in-
volved; and 
‘‘(II) in the case of such an in-
vestigation conducted with respect to 
a combination of active ingredients 
described to in clause (ii) of such sub-
paragraph, to assist in determining 
the relevance of its molecular target 
to the growth or progression of a pe-
diatric cancer. 
‘‘(iii) PRECLINICAL
DATA.—The re-
ports on an investigation required in para-
graph (1)(B) shall include the results of all 
preclinical studies on which the decision to 
conduct such investigation was based. 
VerDate Sep 11 2014 
17:05 Oct 28, 2021
Jkt 029200
PO 00000
Frm 00004
Fmt 6652
Sfmt 6201
E:\BILLS\H5416.IH
H5416
pamtmann on DSKBC07HB2PROD with BILLS
‘‘(iv) RULE
OF
CONSTRUCTION
RE-
GARDING
INACTIVE
INGREDIENTS.—With 
respect to a combination of active ingredi-
ents referred to in subparagraph (A)(ii), 
such subparagraph may not be construed 
as addressing the use of inactive ingredi-
ents with such combination. For purposes 
of such subparagraph, the Secretary may 
establish such requirements with respect to 
inactive ingredients as the Secretary deter-
mines to be appropriate.’’. 
(2) CLARIFYING
APPLICABILITY
OF
CERTAIN 
PROVISIONS.—Section 505B(a)(3) of the Federal 
Food, 
Drug, 
and 
Cosmetic 
Act 
(21 
U.S.C. 
355c(a)(3)), as amended by paragraph (1), is 
amended by adding at the end the following: 
‘‘(E) INTERNAL COMMITTEE REVIEW; LA-
BELING CHANGES; DISSEMINATION OF INFOR-
MATION; ADVERSE EVENTS; SCOPE OF AUTHOR-
ITY.—Subsections (f) through (j) shall apply 
with respect to investigations described in this 
paragraph to the same extent and in the same 
manner as such subsections apply with respect 
to the assessments required under paragraph 
(1)(A), except that subsection (g) does not 
VerDate Sep 11 2014 
17:05 Oct 28, 2021
Jkt 029200
PO 00000
Frm 00005
Fmt 6652
Sfmt 6201
E:\BILLS\H5416.IH
H5416
pamtmann on DSKBC07HB2PROD with BILLS
apply with respect to an investigation referred 
to in subparagraph (A)(ii).’’. 
(3) 
CONFORMING
AMENDMENTS.—Section 
505B(a) of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 355c(a)) is amended— 
(A) in paragraph (3)(C), as redesignated 
by paragraph (1)(A) of this subsection, by 
striking ‘‘investigations described in this para-
graph’’ and inserting ‘‘investigations referred to 
in subparagraph (A)(i)’’; 
(B) in paragraph (3)(D), as redesignated 
by paragraph (1)(A) of this subsection, by 
striking ‘‘the assessments under paragraph 
(2)(B)’’ and inserting ‘‘the assessments re-
quired under paragraph (1)(A)’’; and 
(C) in paragraph (5)(D), by inserting be-
fore the period at the end the following: ‘‘, ex-
cept this subparagraph is not applicable to a 
drug or biological product that is the subject of 
an investigation referred to in paragraph 
(3)(A)(ii)’’. 
(b) AUTHORITY REGARDING PRECLINICAL STUD-
IES.—Section 505B(a)(1) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355c(a)(1)), as amended by sub-
VerDate Sep 11 2014 
17:05 Oct 28, 2021
Jkt 029200
PO 00000
Frm 00006
Fmt 6652
Sfmt 6201
E:\BILLS\H5416.IH
H5416
pamtmann on DSKBC07HB2PROD with BILLS