[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[S. 4426 Introduced in Senate (IS)]
<DOC>
118th CONGRESS
2d Session
S. 4426
To amend the Federal Food, Drug, and Cosmetic Act to establish a time-
limited conditional approval pathway, subject to specific obligations,
for certain drugs and biological products, and for other purposes.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
May 23, 2024
Mr. Braun (for himself, Mrs. Gillibrand, Mr. Cramer, Mr. Manchin, Mr.
Schmitt, Mr. Padilla, Mr. Vance, Mr. Booker, Mr. Hawley, Mr. Welch, and
Ms. Murkowski) introduced the following bill; which was read twice and
referred to the Committee on Health, Education, Labor, and Pensions
_______________________________________________________________________
A BILL
To amend the Federal Food, Drug, and Cosmetic Act to establish a time-
limited conditional approval pathway, subject to specific obligations,
for certain drugs and biological products, 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 ``Promising Pathway Act 2.0''.
SEC. 2. CONDITIONAL APPROVAL OF NEW HUMAN DRUGS FOR INDIVIDUALS WITH
RARE, PROGRESSIVE, AND SERIOUS DISEASES.
(a) In General.--Subchapter A of chapter V of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 351 et seq.) is amended by adding at
the end of the following:
``SEC. 524C. CONDITIONAL APPROVAL OF HUMAN DRUGS FOR INDIVIDUALS WITH
RARE, PROGRESSIVE, AND SERIOUS DISEASES.
``(a) Conditional Approval; Priority Review; Other Designations.--
``(1) In general.--The sponsor of a drug may file with the
Secretary an application for conditional approval of an
eligible drug described in subsection (b). The Secretary shall
approve or deny such application in accordance with subsection
(c).
``(2) Priority review.--The Secretary shall give priority
review to an application for conditional approval of an
eligible drug described in subsection (b).
``(3) Other designations.--If a drug that is granted
conditional approval under this section is eligible for a
special designation by the Secretary under this Act, including
as a drug for a rare disease or condition under section 526,
all applicable benefits of such other designation shall be
available for use under such conditional approval, including
any tax credits and waiving of fees under chapter VII.
``(4) Other programs.--A sponsor of a drug seeking
conditional approval of such drug under this section may also
seek designation, exclusivity, or approval, as applicable, of
such drug under other applicable provisions of this Act or the
Public Health Service Act, subject to the requirements of such
provisions.
``(b) Eligibility.--
``(1) In general.--A drug may be eligible for conditional
approval under this section if such drug is intended to treat a
disease or condition that is--
``(A) rapidly progressive, terminal, and has
substantial unmet medical need, as determined by the
Secretary; or
``(B) a rare disease or condition (as defined in
section 526(a)(2)) that results in a substantially
shortened lifespan, substantial reduction in quality of
life, or other substantial adverse health effects, as
determined by the Secretary.
``(2) Exclusion from eligibility.--A drug that is intended
to treat or respond to a material threat identified by the
Secretary of Homeland Security under section 319F-
2(c)(2)(A)(ii) shall not be eligible for conditional approval
under this section.
``(c) Standard of Review for Conditional Approval.--
``(1) Requirements.--The Secretary shall only approve an
application for conditional approval of a drug under this
section if--
``(A) the Secretary determines that--
``(i)(I) evidence of safety for the drug
has been established by--
``(aa) the completion of a phase 1
clinical investigation of the drug (as
described in section 312.21 of title
21, Code of Federal Regulations (or
successor regulations)); or
``(bb) another demonstration of
safety, as determined appropriate by
the Secretary; and
``(II) evidence of effectiveness in
treating a given indication (which indication
is congruent with the eligibility requirements
of subsection (b)), as established by an
ongoing or completed phase 2 clinical
investigation of the drug (as described in
section 312.21 of title 21, Code of Federal
Regulations (or successor regulations)); or
``(ii) in the case of a drug that is
intended to treat a terminal pediatric rare
disease or condition (as defined in section
526(a)(2)) that does not predominately affect
adults--
``(I) evidence of safety for the
drug has been established in accordance
with clause (i)(I); and
``(II) the drug shows preliminary
evidence of clinical effectiveness
based upon studies in animal models;
and
``(B) the sponsor has provided a written
affirmation of the sponsor's intent to pursue under
section 505 of this Act or section 351 of the Public
Health Service Act approval of the drug, which
affirmation shall include a justification and a plan
for pursuing such approval.
``(2) Rolling, real-time review.--
``(A) In general.--If the Secretary determines,
after preliminary evaluation of data submitted by the
sponsor, that a drug may meet the standard for
conditional approval, the sponsor may submit portions
of an application for conditional approval of a drug
under this section for evaluation by the Secretary
before the sponsor submits a complete application,
which submission shall include--
``(i) a schedule for submission of
information necessary to make the application
complete; and
``(ii) a payment of any fee that may be
required under section 736.
``(B) Review.--The Secretary--
``(i) shall evaluate each application
submitted under subparagraph (A) to assess
whether such application is complete or ready
to be filed; and
``(ii) may commence review of portions of
such application for approval.
``(3) Use of real-world evidence.--
``(A) In general.--The Secretary shall allow the
use of real world evidence (as defined in section
505F(b)), including real world data used to generate
real world evidence, and of external sources of data,
including prospective or retrospective natural history
data, to support an application for conditional
approval under this section.
``(B) Data integrity requirements.--In using
evidence described in subparagraph (A) to support an
application for conditional approval under this
section, the sponsor shall consider the guidance of the
Food and Drug Administration entitled `Data Standards
for Drug and Biological Product Submissions Containing
Real-World Data' and dated December 2023 (or successor
guidance).
``(d) FDA Authority To Withdraw Conditional Approval.--
``(1) In general.--The Secretary may withdraw the
conditional approval of a drug under this section if--
``(A) after adequate review of appropriate safety
data, including data from an observational registry
established under subsection (g), the Secretary
determines that such data no longer supports
conditional approval;
``(B) the Secretary determines that the application
for conditional approval submitted under subsection
(a)(1) contained an untrue statement of material fact;
or
``(C) the Secretary determines that the drug is no
longer eligible under subsection (b).
``(2) FDA examination authority.--
``(A) In general.--For purposes determining whether
to withdraw the conditional approval of a drug under
paragraph (1), the Secretary may--
``(i) review any available clinical data
made available through clinical trials or an
observational registry under subsection (g),
applicable to such drug; and
``(ii) determine whether the sponsor of
such drug is in violation of a requirement
established under paragraph (3) or (4) of
section 505(o) or section 505-1 with respect to
the drug.
``(B) Transparency.--
``(i) In general.--The Secretary may
require drug sponsors and observational
registries under subsection (g) to submit the
data described in subparagraph (A) for the
purposes of the review under that subparagraph.
``(ii) Fines.--The Secretary may levy fines
on sponsors and observational registries that
do not comply with a request for data under
clause (i) within such reasonable timeframe as
is established by the Secretary.
``(3) Effect of withdrawal.--
``(A) Availability to new patients.--
``(i) In general.--If a conditional
approval is withdrawn under this subsection,
the sponsor may not make the drug available to
any new patients, but may continue to make such
drug available to patients who started taking
the drug prior to the date of withdrawal.
``(ii) Effect.--Nothing in this
subparagraph shall be construed to require--
``(I) a patient to continue taking
a conditionally approved drug if such
patient decides to stop taking such
drug; or
``(II) the sponsor to ensure such
drug continues to be manufactured after
the date of withdrawal.
``(B) Civil monetary penalty.--Any sponsor who
makes available to new patients a drug for which
conditional approval has been withdrawn under this
subsection shall be subject to such civil monetary
penalty as is determined by the Secretary.
``(4) Withdrawal notice.--Upon determining to withdraw the
conditional approval of a drug under paragraph (1), the
Secretary shall submit written notice to the sponsor of such
drug and such withdrawal shall be effective on the date that is
14 days after the date of such submission of notice.
``(5) Appeals.--Not later than 180 days after the date of
enactment of the Promising Pathway Act 2.0, the Secretary, by
rule, shall establish a process by which a sponsor of a drug
for which conditional approval was withdrawn under paragraph
(1) may appeal such withdrawal.
``(6) Automatic withdrawal.--
``(A) In general.--If the sponsor of a drug that
receives conditional approval under this section does
not submit an application for renewal of such
conditional approval under subsection (f)(2) by the
deadline under that subsection, such conditional
approval shall automatically be withdrawn in accordance
with paragraph (3) on the date on which such
conditional approval expires.
``(B) Marketing requirement.--If any drug that
receives conditional approval under this section is not
brought to market within 1 year of the date on which
the conditional approval is granted, such conditional
approval, along with any benefits described in
subsection (a)(3), shall automatically be withdrawn in
accordance with paragraph (3) on such date.
``(C) No right to appeal; effect of automatic
withdrawal.--
``(i) In general.--A sponsor shall not have
the right to appeal an automatic withdrawal
under this paragraph.
``(ii) Effect.--The Secretary shall have no
means or power to prevent an automatic
withdrawal under this paragraph from occurring.
``(e) Labeling; Review of Materials.--
``(1) In general.--Sponsors may not make available to
patients a drug conditionally approved under this section,
unless--
``(A) all labeling and advertising of such drug
contains the statement `conditionally approved for a
limited population' in a prominent manner and adjacent
to, and not more prominent than--
``(i) the proprietary name of such drug, if
any; or
``(ii) if there is no proprietary name, the
established name of such drug, if any, as
defined in section 502(e)(3), or, in the case
of a drug that is a biological product, the
proper name, as defined by regulation; and
``(B) the prescribing information for the drug
required by section 201.57 of title 21, Code of Federal
Regulations (or any successor regulation) includes the
following statement: `This drug is conditionally
approved for use in a limited and specific population.
This drug has not received full approval by the Food
and Drug Administration. Conditional approval of this
drug may be withdrawn at short notice.'.
``(2) Submission.--Not later than 45 days before such
materials are distributed, all promotional, educational, and
marketing materials for such drug shall be submitted to the
Secretary for review.
``(3) Public list.--The Secretary shall maintain a list of
all drugs conditionally approved under this section on a
publicly accessible website. Such website shall briefly
describe what each conditionally approved drugs is and list the
1 or more diseases or conditions for which the drug is
indicated.
``(f) Renewal of Conditional Approval; Requirement To Bring Drug to
Market.--
``(1) Duration; renewals.--The conditional approval for a
drug under this section is effective for a 2-year period. The
sponsor may request renewal of such conditional approval for up
to 3 subsequent 2-year periods. Conditional approval with
respect to a drug shall not exceed a total of 8 years from the
initial date the drug was granted conditional approval.
``(2) Applications for renewal of conditional approval.--
``(A) In general.--Except as provided in
subparagraph (C), the sponsor of a drug seeking a
renewal of conditional approval for such drug under
this subsection shall submit to the Secretary, not
later than 180 days before the date on which such
conditional approval expires, an application that
contains the applicable information described in
paragraph (3) in a standardized format determined by
the Secretary.
``(B) Process for granting renewals.--Not later
than 180 days after the date of enactment of the
Promising Pathway Act 2.0, the Secretary, by rule,
shall establish the process for granting a renewal
under this subsection.
``(C) Exemption for small population diseases.--
``(i) In general.--The Secretary shall
exempt from the requirements of subparagraph
(A) and paragraph (3) an application for a
renewal of conditional approval for a drug
under this subsection if the Secretary
determines that the population affected by the
disease or condition that the drug is intended
to treat does not support additional
preliminary evidence of effectiveness (as
defined in paragraph (3)(D)).
``(ii) Application for exemption.--Sponsors
may submit an application for exemption under
this subparagraph not later than 180 days
before the date on which the conditional
approval expires.
``(iii) Application process.--Not later
than 180 days after the date of enactment of
the Promising Pathway Act 2.0, the Secretary
shall establish a sta