Promising Pathway Act
This bill provides for expedited, provisional approval of drugs for serious or life-threatening diseases or conditions.
Specifically, the bill allows for provisional approval of drugs that are used to treat, prevent, or diagnose a serious or life-threatening disease or condition for which premature death is likely without early medical intervention. The Food and Drug Administration (FDA) must evaluate applications within 90 days of receipt. The FDA may approve applications if there is early evidence, including real-world and real-time evidence, of the drug's efficacy, as well as substantial evidence of the drug's safety. Provisional approval is valid for two years and may be renewed for up to eight years. The FDA may withdraw provisional approval in the event of serious adverse health effects.
Drug sponsors that receive provisional approval must require all patients receiving the drug to participate in an observational registry, through which the drug sponsor may collect and submit data relating to the drug until it is fully approved. Registries may be operated by the drug sponsor or another entity. The FDA must annually review these registries; sponsors of registries that fail to meet the bill's requirements are subject to civil penalties.
[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4408 Introduced in House (IH)]
<DOC>
118th CONGRESS
1st Session
H. R. 4408
To amend the Federal Food, Drug, and Cosmetic Act to establish a time-
limited provisional approval pathway, subject to specific obligations,
for certain drugs and biological products, and for other purposes.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
June 30, 2023
Mr. Gallagher (for himself, Mr. Quigley, Mr. Westerman, Mr. Swalwell,
and Mr. Fitzpatrick) 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 a time-
limited provisional 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''.
SEC. 2. PROVISIONAL APPROVAL OF NEW HUMAN DRUGS.
(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. PROVISIONAL APPROVAL OF NEW HUMAN DRUGS.
``(a) Priority Review and Evaluation of Applications.--
``(1) In general.--The Secretary shall establish a priority
review system to evaluate applications submitted under this
section for provisional approval within 90 days of receipt of a
completed application.
``(2) Other designations.--If a drug submitted for review
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 benefits of such
other designation shall be available for use under provisional
approval, including any tax credits and waiving of fees under
chapter VII.
``(b) Eligibility.--A drug may be eligible for provisional approval
under this section if the Secretary determines that the drug is
intended for the treatment, prevention, or medical diagnosis of a
serious or life-threatening disease or condition for which there is a
reasonable likelihood that premature death will occur without early
medical intervention for an individual contracting or being diagnosed
with such disease or condition.
``(c) Standard of Review for Provisional Approval.--
``(1) Requirements.--An application for provisional
approval under this section may be approved only if the
Secretary determines that--
``(A) there is substantial evidence of safety for
the drug, such that there is evidence consisting of
adequate and well-controlled investigations, including
clinical investigations, by experts qualified by
scientific training and experience to evaluate the
safety of the drug involved, on the basis of which it
could fairly and responsibly be concluded that the drug
will have the effect it purports or is represented to
have under the conditions of use prescribed,
recommended, or suggested in the labeling or proposed
labeling; and
``(B) there is relevant early evidence based on
adequate and well-controlled investigations, including
early-stage clinical investigations, to establish
that--
``(i) the drug provides a positive
therapeutic outcome; and
``(ii) the outcome of the drug is
consistent with or greater than currently
marketed on-label therapies, with equal or
fewer side effects, if there are currently
marketed on-label therapies.
``(2) Protocols.--The Secretary shall promulgate rules that
establish the appropriate protocols to enable rolling, real-
time, mid-trial submission while preserving the integrity of
the ongoing trial and without penalizing the sponsor for
seeking provisional approval under this section.
``(3) Real world evidence.--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,
to support an application for provisional approval under this
section, and to fulfill the follow-up requirements and support
applications for approval under section 505 or section 351 of
the Public Health Service Act, as applicable.
``(4) Use of scientifically substantiated surrogates.--
``(A) In general.--The sponsor of an application
for provisional approval under this section may use
scientifically substantiated surrogates to support such
application.
``(B) Definition.--In subparagraph (A), the term
`scientifically substantiated surrogates' means
surrogate endpoints to predict clinical benefit other
than such endpoints previously validated by the
Secretary, based on--
``(i) epidemiologic, therapeutic,
pathophysiologic, or other evidence; or
``(ii) an effect on a clinical endpoint
other than survival or irreversible morbidity
of interest.
``(d) Transparency and Patient Monitoring Requirements.--
``(1) Registries.--
``(A) In general.--The sponsor of a drug
provisionally approved under this section shall require
that all patients who use such drug participate in an
observational registry and consent to the sponsor's
collection, and submission to the registry, of data
related to the patient's use of such drug until such
drug receives approval under section 505 or section 351
of the Public Health Service Act, or the provisional
approval is rescinded.
``(B) Requirements for registries.--An
observational registry described in subparagraph (A)
may be run by a third party, such as a government, for
profit, or nonprofit organization, and shall track all
patients who use the provisionally approved drug.
``(C) Accessibility.--An observational registry
described in subparagraph (A) shall be easily
accessible for--
``(i) all patients who are participating in
any registry related to a provisionally
approved drug that allows for easy,
unrestricted (or transparent) access for such
patients to their patient data and related
information regarding their usage of the
provisionally approved drug; and
``(ii) approved researchers and medical
professionals who may access data maintained in
the registry, which access shall be for public
health research and only in a de-identified,
aggregated manner.
``(2) Funding.--An observational registry under this
subsection shall be maintained, as applicable--
``(A) by the sponsor of the drug provisionally
approved under this section that is the subject of the
registry; or
``(B) by a third party, such as a government, for
profit, or nonprofit organization.
``(3) Sponsor requirements.--
``(A) In general.--For any drug application
provisionally approved under this section, the
Secretary shall notify the sponsor of the exact data
such sponsor is required to submit to an observational
registry.
``(B) Annual review of the registry; penalties.--
The Secretary shall conduct an annual review of
observational registries established under this
subsection. If, at such an annual review, fewer than 90
percent of patients are participating in an
observational registry with respect to a drug approved
under this section, the Secretary shall issue to the
sponsor of such drug a civil monetary penalty of not
more than $100,000. If a violation of this section is
not corrected within the 30-day period following
notification, the sponsor shall, in addition to any
penalty under this subparagraph be subject to a civil
monetary penalty of not more than $10,000 for each day
of the violation after such period until the violation
is corrected. If application patient participation in
an observational registry is not at or above 90 percent
within 6 months of issuance of such penalty, the
provisional approval shall be withdrawn.
``(4) Annual report to congress.--The Secretary shall
submit an annual report to Congress on all drugs granted
provisional approval under this section. Such report shall
include--
``(A) the number of patients treated with each such
drug, and the number of patients tracked in an
observational registry with respect to each such drug;
``(B) a discussion of the minimum amount of data
required in the registries, including patient
treatments and uses, length of use, side effects
encountered, relevant biomarkers or scientifically
substantiated surrogates, scan results, cause of death
and how long the patient lived, and adverse drug
effects;
``(C) a list of all such drugs for which an
application for approval under section 505 of this Act
or section 351 of the Public Health Service Act, or an
application for an extension of provisional approval
under this section, has been submitted; and
``(D) a list of all applications denied provisional
approval under this section, together with an
explanation for the decisions to deny each such
application.
``(e) Withdrawal of Provisional Approval.--
``(1) In general.--The Secretary shall withdraw provisional
approval under this section if there are a significant numbers
of patients who experience serious adverse effects, compared to
the other currently marketed on-label therapies that are
available for the applicable disease or condition.
``(2) Effect of withdrawal.--If a provisional approval is
withdrawn under this subsection, the sponsor may not make the
drug available to any new patients, but may be allowed to
continue to make such drug available to patients who started
taking the drug prior to the date of withdrawal, for as long a
period as dictated by patient need, as determined by the
Secretary.
``(f) Transparency.--Any scientific, medical, academic, or health
care journal publishing an article explaining, releasing, conveying, or
announcing research findings which were funded by the Department of
Health and Human Services shall be prohibited from publishing such
research unless--
``(1) such article conveying research findings is made
publicly available on the journal's internet website without a
paywall or charge not later than 3 months after the date on
which such article was first provided to subscribers of such
journal (or first made available for purchase); and
``(2) the article's author or researcher or author's
institution (or, in the case of multiple authors, researchers,
or institutions, all such authors, researchers, or
institutions) received less than 30 percent of funding for such
research from the Department of Health and Human Services
throughout the period of time the research was conducted.
``(g) Informed Consent.--Prior to receiving a drug provisionally
approved under this section, the sponsor of the drug shall receive from
each patient, or the patient's representative, informed consent,
through a signed informed consent form, acknowledging that such patient
understands that the drug did not undergo the usual process for
approval of a drug by the Food and Drug Administration, and that such
patient is willing to accept the risks involved in taking such drug.
``(h) Postmarket Controls and Labeling.--
``(1) FDA annual review of registry data.--The Secretary
shall annually review the data made available through the
observational registries under subsection (d) and make a
determination regarding whether the side effect profile of any
drug provisionally approved under this section does not support
the benefit provided, or the data shows the benefit is less
than the benefits offered through other, fully approved drugs.
``(2) Labeling.--The sponsor of the provisionally approved
drug shall ensure that all labeling and promotional materials
for the drug bear the statement `provisionally approved by the
FDA pending a full demonstration of effectiveness under
application number ______' (specifying the application number
assigned by the Secretary in place of the blank). All
promotional, educational and marketing materials for
provisionally approved products shall be reviewed and approved
by the Secretary before such materials are distributed.
``(3) Rescission of provisional approval.--If the Secretary
determines that the side effect profile of any drug included in
such observational registries does not support the benefit
provided by such drug, or that the data shows that the benefit
is less than the benefits offered through other drugs approved
under section 505 of this Act or section 351 of the Public
Health Service Act, the Secretary shall rescind such
provisional approval.
``(i) Duration of Provisional Approval; Requirement To Bring Drug
to Market.--
``(1) Duration; renewals.--The provisional approval for a
drug under this section is effective for a 2-year period. The
sponsor may request renewal for provisional approval status for
up to 3 subsequent 2-year periods. Provisional approval status
with respect to a drug shall not exceed a total of 8 years from
the initial date the sponsor was awarded provisional approval
status.
``(2) Marketing requirement.--If any drug that receives
provisional approval under this section is not brought to
market within 180 days of the provisional approval, such
provisional approval shall be rescinded.
``(j) Limitation on Liability.--With respect to any claim under
State law alleging that a drug sold or otherwise made available
pursuant to a grant of provisional approval under this section is
unsafe or ineffective, no liability in a cause of action shall lie
against a sponsor or manufacturer, unless the relevant conduct
constitutes reckless or willful misconduct, gross negligence, or an
intentional tort under any applicable State law.
``(k) Right To Petition an Advisory Committee for Approval.--
``(1) In general.--The sponsor of a drug granted
provisional approval pursuant to this section may request, at
any time after provisional approval is granted under this
section, a meeting with the appropriate advisory committee (or
advisory committees) to present safety and efficacy data for
the purposes of receiving a recommendation from such an
advisory committee for approval under section 505 of this Act
or section 351 of the Public Health Service Act of the
provisionally approved drug. Such a requested meeting shall be
granted not later than 90 days after a request is made. Nothing
in this paragraph shall be construed to alter the processes and
timeframes for recommendation for approval by such an advisory
committee of the provisionally approved drug or for approval of
the provisionally approved drug under section 505 of this Act
or section 351 of the Public Health Service Act.
``(2) Waiver of adequate and well-controlled study
requirements.--
``(A) In general.--In considering whether to
recommend a drug that was provisionally approved under
this section for approval under section 505, the
Director of the Center for Drug Evaluation and Research
shall consider the option to waive requirements for
adequate and well-controlled studies in accordance with
the process described in section 314.126(c) of title
21, Code of Federal Regulations (or successor
regulations).
``(B) Biological products.--In considering whether
to recommend a biological product that was
provisionally approved under this section for licensure
under section 351 of the Public Health Service Act, the
Director of the Center for Biologics Evaluation and
Research may, and shall consider the option to, waive
requirements, as applicable, for adequate and well-
controlled studies for such biological product in
accordance with the process described in section
314.126(c) of title 21, Code of Federal Regulations (or
successor regulations).''.
(b) Conforming Amendment.--Section 505(a) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355(a)) is amended by inserting ``,
or there is in effect a provisional approval under section 524C with
respect to such drug'' before the period.
(c) Reimbursement.--
(1) Private health insurers.--Section 2719A of the Public
Health Service Act (42 U.S.C. 300gg-19a) is amended by adding
at the end the following:
``(f) Treatment of Certain Drugs.--A group health plan or health
insurance issuer of group or individual health insurance coverage shall
not deny coverage of any drug provisionally approved under section 524C
of the Federal Food, Drug, and Cosmetic Act on the basis of such drug
being experimental. In determining coverage under the applicable plan
or coverage, a group health plan or health insurance issuer shall treat
a drug provisionally approved under such section in the same manner as
such plan or coverage would treat a drug approved under section 505 of
the Federal Food, Drug, and Cosmetic Act or section 351 of this Act.
Nothing in this subsection shall be construed to require a group health
plan or health insurance issuer to cover any specific drug
provisionally approved under such section 524C.''.
(2) Federal health care programs.--The requirement under
subsection (f) of section 2719A of the Public Health Service
Act (as added by paragraph (1)) shall apply with respect to
coverage determinations under a Federal health care program (as
defined in section 1128B(f) of the Social Security Act (42
U.S.C. 1320a-7b(f))) in the same manner such requirement
applies under such subsection (f).
(3) Conforming amendment.--Section 1927(k)(2)(A)(i) of the
Social Security Act (42 U.S.C. 1396r-8(k)(2)(A)(i)) is
amended--
(A) by striking ``or which'' and inserting ``,
which''; and
(B) by inserting ``, or which is provisionally
approved under section 524C of such Act'' before the
semicolon.
<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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