[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[S. 4446 Introduced in Senate (IS)]
<DOC>
117th CONGRESS
2d Session
S. 4446
To modernize the process of accelerated approval of a drug for a
serious or life-threatening disease or condition.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
June 22, 2022
Ms. Collins (for herself and Mr. Kaine) introduced the following bill;
which was read twice and referred to the Committee on Health,
Education, Labor, and Pensions
_______________________________________________________________________
A BILL
To modernize the process of accelerated approval of a drug for a
serious or life-threatening disease or condition.
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 ``Modernizing the Accelerated Approval
Pathway Act of 2022''.
SEC. 2. MODERNIZING ACCELERATED APPROVAL.
(a) In General.--Section 506(c) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 356(c)) is amended--
(1) in paragraph (2)--
(A) by redesignating subparagraphs (A) and (B) as
clauses (i) and (ii), respectively, and adjusting the
margins accordingly;
(B) by striking ``Approval of a product'' and
inserting the following:
``(A) In general.--Approval of a product'';
(C) in clause (i) of such subparagraph (A), as so
redesignated, by striking ``appropriate postapproval
studies'' and inserting ``an appropriate postapproval
study or studies (which may be augmented or supported
by real world evidence)''; and
(D) by adding at the end the following:
``(B) Studies not required.--If the Secretary does
not require that the sponsor of a product approved
under accelerated approval conduct a postapproval study
under this paragraph, the Secretary shall publish on
the website of the Food and Drug Administration the
rationale for why such study is not appropriate or
necessary.
``(C) Postapproval study conditions.--Not later
than the time of approval of a product under
accelerated approval, the Secretary shall specify the
conditions for a postapproval study or studies required
to be conducted under this paragraph with respect to
such product, which may include enrollment targets, the
study protocol, and milestones, including the target
date of study completion.
``(D) Studies begun before approval.--The Secretary
may require such study or studies to be underway prior
to approval.''; and
(2) in paragraph (3)--
(A) by redesignating subparagraphs (A) through (D)
as clauses (i) through (iv), respectively, and
adjusting the margins accordingly;
(B) by striking ``The Secretary may'' and inserting
the following:
``(A) In general.--The Secretary may'';
(C) in clause (i) of such subparagraph (A), as so
redesignated, by striking ``drug with due diligence''
and inserting ``product with due diligence, including
with respect to conditions specified by the Secretary
under paragraph (2)(C)'';
(D) in clause (iii) of such subparagraph (A), as so
redesignated, by inserting ``shown to be'' after
``product is not''; and
(E) by adding at the end the following:
``(B) Expedited procedures described.--Expedited
procedures described in this subparagraph shall consist
of, prior to the withdrawal of accelerated approval--
``(i) providing the sponsor with--
``(I) due notice;
``(II) an explanation for the
proposed withdrawal;
``(III) an opportunity for a
meeting with the Commissioner or the
Commissioner's designee; and
``(IV) an opportunity for written
appeal to--
``(aa) the Commissioner; or
``(bb) a designee of the
Commissioner who has not
participated in the proposal
withdrawal of approval (other
than a meeting pursuant to
subclause (III)) and is not
subordinate of an individual
(other than the Commissioner)
who participated in such
proposed withdrawal;
``(ii) providing an opportunity for public
comment on the proposing to withdrawal
approval;
``(iii) the publication of a summary of the
public comments received, and the Secretary's
response to such comments, on the website of
the Food and Drug Administration; and
``(iv) convening and consulting an advisory
committee on issues related to the proposed
withdrawal, if requested by the sponsor and if
no such advisory committee has previously
advised the Secretary on such issues with
respect to the withdrawal of the product prior
to the sponsor's request.''.
(b) Reports of Postmarketing Studies.--Section 506B(a) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356b(a)) is amended--
(1) by redesignating paragraph (2) as paragraph (3); and
(2) by inserting after paragraph (1) the following:
``(2) Accelerated approval.--Notwithstanding paragraph (1),
a sponsor of a drug approved under accelerated approval shall
submit to the Secretary a report of the progress of any study
required under section 506(c), including progress toward
enrollment targets, milestones, and other information as
required by the Secretary, not later than 180 days after the
approval of such drug and not less frequently than every 180
days thereafter, until the study is completed or terminated.
The Secretary shall promptly publish on the website of the Food
and Drug Administration the information so reported, in an
easily searchable format.''.
(c) Enforcement.--Section 301 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 331), is amended by adding at the end the
following:
``(fff) The failure of a sponsor of a product approved under
accelerated approval pursuant to section 506(c)--
``(1) to conduct with due diligence any postapproval study
required under section 506(c) with respect to such product; or
``(2) to submit timely reports with respect to such product
in accordance with section 506B(a)(2).''.
(d) Guidance.--
(1) In general.--The Secretary of Health and Human Services
shall issue guidance describing--
(A) how sponsor questions related to the
identification of novel surrogate or intermediate
clinical endpoints may be addressed in early-stage
development meetings with the Food and Drug
Administration;
(B) the use of novel clinical trial designs that
may be used to conduct appropriate post-approval
studies as may be required under section 506(c)(2)(A)
of the Federal Food, Drug, and Cosmetic Act, as amended
by subsection (a);
(C) the expedited procedures described in section
506(c)(3)(B) of the Federal Food, Drug, and Cosmetic
Act; and
(D) considerations related to the use of surrogate
or intermediate clinical endpoints that may support the
accelerated approval of an application under
506(c)(1)(A), including considerations in evaluating
the evidence related to any such endpoints.
(2) Final guidance.--The Secretary shall issue--
(A) draft guidance under paragraph (1) not later
than 18 months after the date of enactment of this Act;
and
(B) final guidance not later than 1 year after the
close of the public comment period on such draft
guidance.
(e) Rare Disease Endpoint Advancement Pilot.--
(1) In general.--The Secretary of Health and Human Services
shall establish a pilot program under which the Secretary will
establish procedures to provide increased interaction with
sponsors of rare disease drug development programs for purposes
of advancing the development of efficacy endpoints, including
surrogate and intermediate endpoints, for drugs intended to
treat rare diseases, including through--
(A) determining eligibility of participants for
such program; and
(B) developing and implementing a process for
applying to, and participating in, such a program.
(2) Public workshops.--The Secretary shall conduct up to 3
public workshops, which shall be completed not later than
September 30, 2026, to discuss topics relevant to the
development of endpoints for rare diseases, which may include
discussions about--
(A) novel endpoints developed through the pilot
program established under this subsection; and
(B) as appropriate, the use of real world evidence
and real work data to support the validation of
efficacy endpoints, including surrogate and
intermediate endpoints, for rare diseases.
(3) Report.--Not later than September 30, 2027, the
Secretary shall submit to the Committee on Energy and Commerce
of the House of Representatives and the Committee on Health,
Education, Labor, and Pensions of the Senate a report
describing the outcomes of the pilot program established under
this subsection.
(4) Guidance.--Not later than September 30, 2027, the
Secretary shall issue guidance describing best practices and
strategies for development of efficacy endpoints, including
surrogate and intermediate endpoints, for rare diseases.
(5) Sunset.--The Secretary may not accept any new
application or request to participate in the program
established by this subsection on or after October 1, 2027.
(f) Accelerated Approval Council.--
(1) General.--Not later than 180 days after the date of
enactment of this Act, the Secretary of Health and Human
Services shall establish an intra-agency coordinating council
within the Food and Drug Administration to ensure the
consistent and appropriate use of accelerated approval across
the Food and Drug Administration, pursuant to section 506(c) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356(c)).
(2) Membership.--The members of the Council shall consist
of the following senior officials, or a designee of such
official, from the Food and Drug Administration and relevant
Centers:
(A) The Director of the Center for Drug Evaluation
and Research.
(B) The Director of the Center for Biologics
Evaluation and Research.
(C) The Director of the Oncology Center of
Excellence.
(D) The Director of the Office of New Drugs.
(E) The Director of the Office of Orphan Products
Development.
(F) The Director of the Office of Tissues and
Advanced Therapies.
(G) The Director of the Office of Medical Policy.
(H) At least 3 directors of review division
overseeing products approved under accelerated
approval, including at least one director of a review
division within the Office of Neuroscience.
(3) Duties of the council.--
(A) Meetings.--The Council shall convene not fewer
than 3 times per calendar year to discuss issues
related to accelerated approval, including any relevant
cross-disciplinary approaches related to product review
with respect to accelerated approval.
(B) Policy development.--The Council shall directly
engage with product review teams to support the
consistent and appropriate use of accelerated approval
across the Food and Drug Administration. Such
activities may include--
(i) developing guidance for Food and Drug
Administration staff and best practices for,
and across, product review teams, including
with respect to communication between sponsors
and the Food and Drug Administration and the
review of products under accelerated approval;
(ii) providing training for product review
teams; and
(iii) advising review divisions on product-
specific development, review, and withdrawal of
products under accelerated approval.
(4) Publication of a report.--Not later than 1 year after
the date of enactment of this Act, and annually thereafter, the
council shall publish on the public website of the Food and
Drug Administration a report on the activities of the council.
(g) Rule of Construction.--Nothing in this section (including the
amendments made by this section) shall be construed to affect products
approved pursuant to 506(c) of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 356(c)) prior to the date of enactment of this Act.
<all>