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. 
‘‘(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 for 
which the application referred to in 
paragraph (1)(B) is submitted; 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. 
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‘‘(iii) LIMITATION REGARDING INVES-
TIGATION OF NOVEL COMBINATION.—For 
purposes of paragraph (1)(B), a novel 
combination is a combination of two or 
more active ingredients for which an appli-
cation under section 505 of this Act or sec-
tion 351 of the Public Health Service Act 
for such combination has not previously 
been approved. A pediatric investigation 
under this paragraph of such novel com-
bination is required only if each of the ac-
tive ingredients in the combination has 
been approved under such section 505 or 
such section 351 to treat an adult cancer. 
‘‘(iv) PRECLINICAL DATA.—The Sec-
retary may require that reports on an in-
vestigation required pursuant to paragraph 
(1)(B) shall include the results of all pre-
clinical studies on which the decision to 
conduct such investigation was based. 
‘‘(v) 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 
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as addressing the use of inactive ingredi-
ents with such combination.’’. 
(3) 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 (2), is fur-
ther 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 
apply with respect to an investigation referred 
to in subparagraph (A)(ii) of this paragraph.’’. 
(4) 
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 (2)(A) of this subsection, by 
striking ‘‘investigations described in this para-
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graph’’ and inserting ‘‘investigations referred to 
in subparagraph (A)(i)’’; 
(B) in paragraph (3)(D), as redesignated 
by paragraph (2)(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 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-
section (a)(1), is further amended by adding at the end 
the following: 
‘‘(C) 
PRECLINICAL
STUDIES
GEN-
ERALLY.— 
‘‘(i) IN GENERAL.—With respect to an 
application for an exemption for investiga-
tional use under section 505(i) for a drug 
or biological product that is intended for 
the treatment of an adult cancer, the Sec-
retary may require, as a condition of per-
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