Give Kids a Chance Act
This bill authorizes the Food and Drug Administration (FDA) to take various actions regarding pediatric cancer treatments, such as requiring pediatric cancer trials involving a combination of drugs when an applicant seeks market approval for a new drug (or biological product).
For an application for market approval for a new adult cancer treatment drug, if the FDA determines that the drug in question is substantially relevant to the growth or progression of a pediatric cancer, the FDA may require a pediatric cancer investigation into that drug's active ingredient in combination with another active ingredient that has received market approval or an exemption for investigational use. Currently, the FDA may only require a pediatric cancer trial for the drug in the application.
Furthermore, when the FDA receives an application for an investigational use exemption for an adult cancer treatment drug, the FDA may require the applicant to conduct up to two preclinical studies to determine the relevance of the drug to pediatric cancer.
[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 5416 Introduced in House (IH)]
<DOC>
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 investigations of molecularly targeted drugs
to treat cancer, and for other purposes.
Be it enacted by the Senate and House of Representatives 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''.
SEC. 2. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDITIONAL AUTHORITIES
OF FOOD AND DRUG ADMINISTRATION REGARDING MOLECULARLY
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 Cosmetic Act (21 U.S.C. 355c(a)(3)) is amended--
(A) by redesignating subparagraphs (B) and (C) as
subparagraphs (C) and (D), respectively; and
(B) by striking subparagraph (A) and inserting the
following:
``(A) In general.--For purposes of paragraph
(1)(B), the investigation described in this paragraph
is (as determined by the Secretary) a molecularly
targeted pediatric cancer investigation of--
``(i) the drug or biological product for
which the application referred to in such
paragraph is submitted; or
``(ii) the active ingredient or ingredients
of such drug or biological product in
combination with--
``(I) an active ingredient of a
drug or biological product for which
there is in effect an exemption for
investigational use under section
505(i) that was submitted by the person
submitting the application referred to
in paragraph (1)(B); or
``(II) an active ingredient of a
drug for which an approval under
section 505 is in effect, or an active
ingredient 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
investigation referred to in subparagraph (A)
shall be designed to yield clinically
meaningful pediatric study data, gathered using
appropriate formulations for each age group for
which the study is required, regarding dosing,
safety, and preliminary efficacy.
``(ii) Purpose of investigation.--The
purpose of a molecularly targeted pediatric
cancer investigation referred to in
subparagraph (A) shall be--
``(I) in the case of such an
investigation 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
involved; and
``(II) in the case of such an
investigation conducted with respect to
a combination of active ingredients
described to in clause (ii) of such
subparagraph, to assist in determining
the relevance of its molecular target
to the growth or progression of a
pediatric cancer.
``(iii) Preclinical data.--The reports on
an investigation required in paragraph (1)(B)
shall include the results of all preclinical
studies on which the decision to conduct such
investigation was based.
``(iv) Rule of construction regarding
inactive ingredients.--With respect to a
combination of active ingredients referred to
in subparagraph (A)(ii), such subparagraph may
not be construed as addressing the use of
inactive ingredients with such combination. For
purposes of such subparagraph, the Secretary
may establish such requirements with respect to
inactive ingredients as the Secretary
determines 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; labeling changes;
dissemination of information; adverse events; scope of
authority.--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).''.
(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 paragraph'' 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 required under paragraph (1)(A)'';
and
(C) in paragraph (5)(D), by inserting before the
period at the end the following: ``, except 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 Studies.--Section 505B(a)(1) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(1)), as
amended by subsection (b)(1), is further amended by adding at the end
the following:
``(D) Preclinical studies generally.--
``(i) In general.--With respect to an
application for an exemption for
investigational use under section 505(i) for a
drug or biological product that is intended for
the treatment of an adult cancer, the Secretary
may require, as a condition of permitting the
exemption to go into effect, that the sponsor
involved enter into an agreement with the
Secretary to conduct not more than two
preclinical studies of the drug or biological
product in order to assist in determining the
relevance of its molecular target to the growth
or progression of a pediatric cancer.
``(ii) Timeframe for preclinical studies.--
With respect to the drug or biological product
involved, an agreement under clause (i) for a
preclinical study shall specify the date by
which an initial plan for the study will be
submitted to the Secretary. The results of the
preclinical study shall be submitted to the
Secretary in accordance with a timeframe to
which the Secretary and the sponsor involved
have agreed.''.
(c) Applicability.--The amendments made by this section apply with
respect to any application under section 505(i) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355(i)), any application under
section 505 of such Act (21 U.S.C. 355), and any application under
section 351(a) of the Public Health Service Act (42 U.S.C. 262), that
is submitted on or after the expiration of the 3-year period beginning
on the date of the enactment of this Act.
<all>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
Referred to the Subcommittee on Health.
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