Promising Pathway Act
This bill establishes a provisional approval pathway for medicines intended for serious or life-threatening diseases, including illnesses posing a threat of epidemic or pandemic. The period of the provisional approval is for two years and is potentially renewable.
The Food and Drug Administration (FDA) must establish a priority review system to evaluate completed provisional approval applications within 90 days of receipt. A provisional approval application may be approved if the FDA determines that (1) there is substantial evidence of safety for the drug; and (2) there is relevant early evidence of efficacy, based on adequate and well-controlled investigations.
During the COVID-19 (i.e., coronavirus disease 2019) pandemic, or another epidemic or pandemic, the FDA must accept and review various portions of a provisional approval application on a rolling basis.
The manufacturer of a provisionally approved drug must require patients to participate in an observational registry. A manufacturer that fails to comply with registry requirements is subject to civil penalties.
A provisionally approved drug must be labeled as such.
If a drug that receives provisional approval status is not brought to market within 180 days of the approval, the approval must be rescinded.
The bill also limits the liability of a manufacturer of a provisionally approved drug with respect to any claim under state law alleging that the drug is unsafe or ineffective.
Private health insurers and federal health care programs shall not deny coverage of a provisionally approved drug on the basis of it being experimental.
[Congressional Bills 116th Congress]
[From the U.S. Government Publishing Office]
[H.R. 7269 Introduced in House (IH)]
<DOC>
116th CONGRESS
2d Session
H. R. 7269
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 18, 2020
Mr. Quigley (for himself, Mr. Gallagher, Mr. Swalwell of California,
and Mr. Westerman) 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. FINDINGS.
Congress finds as follows:
(1) The drugs and biological products intended to be
reviewed under the pathway established under this Act are for
the treatment and prevention of serious diseases or conditions,
especially those for which there are no available on-label
meaningful or disease-modifying treatments, where speed to
access is critical.
(2) The approval pathway established under this Act is
intended to allow drug and biological product applications to
be more rapidly reviewed by the U.S. Food and Drug
Administration (FDA), with the FDA reviewing various portions
of new drug and biological product applications as they become
available.
(3) The approval pathway established under this Act
establishes a clear approval pathway that can be utilized by
sponsors to receive rolling review of applications for drugs
and biological products intended to treat serious diseases,
including drugs and biological products intended to treat
COVID-19 that reduce the risk of death, severe disease, and
progression of symptoms in those exposed to the virus.
(4) The approval pathway established under this Act will
enable sponsors to receive early, time-limited, and provisional
approval for drugs and biological products that have
demonstrated substantial evidence of safety and relevant early
evidence that establishes that the drug provides a positive
therapeutic outcome.
(5) The approval pathway established under this Act will
allow for the use of real-world evidence and scientifically
substantiated surrogates, other than those previously validated
by the FDA, to predict the clinical benefits and ultimately
support provisional approval.
(6) Drugs and biological products granted provisional
approval under the pathway established under this Act are
limited to a 2-year approval period, renewable every 2 years,
for up to 6 years. Full approval can awarded at any time, for
any drug or biological product provisionally approved under
this pathway that establishes a 15-percent improvement in an
important endpoint compared to standard therapies.
(7) The approval pathway established under this Act
prohibits denial of coverage for any drug or biological product
provisionally approved under this approval pathway on account
of it being experimental.
(8) Informed consent is required for any patients using a
drug or biological product approved under the provisional
approval pathway established under this Act. Any patients using
a drug or biological product reviewed under this approval
pathway must participate in an observational registry until
those drugs or biological products receive full approval, with
approval contingent on registry participation.
(9) This Act requires that registries track aggregated, de-
identified data that will be readily available to approved
researchers for public health research purposes.
(10) This Act creates, within the Office of the
Commissioner at the FDA, the position of the Patient Advocate
General to provide assistance to patients and their families
utilizing drugs and biological products.
SEC. 3. 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. 524B. 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
pathway for provisional approval within 90 days of receipt of a
completed application.
``(2) Review of applications during epidemics and
pandemics.--In the case of an epidemic or pandemic, including
with respect to COVID-19, the Secretary shall accept and review
various portions of an application submitted under the pathway
under this section for provisional approval on a rolling basis,
and the review of any part of an application so submitted shall
be completed not later than 3 weeks after submission.
``(3) Other designations.--If a drug submitted for review
under the pathway 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--
``(1) 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;
``(2) a disease or condition that poses a threat of
epidemic or pandemic; or
``(3) a disease or condition associated with morbidity that
has a substantial impact on day-to-day functioning.
``(c) Standard of Review for 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 for a sponsor of an
application for provisional approval under this section and the
Commissioner to follow to enable rolling, real-time, mid-trial
submission while preserving the integrity of the ongoing trial
and without penalizing the sponsor for making use of this
pathway.
``(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 full approval as described 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 full 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 non-profit 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;
``(B) by a third party, such as a government, for
profit, or nonprofit organization; or
``(C) the Federal Government, in the case of any
drug so approved that is intended to treat a disease or
condition associated with an epidemic or pandemic.
``(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, less 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 full 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 full
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 approved under this pathway 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, fully approved
drugs, the Secretary shall rescind such provisional approval.
``(i) Duration of Provisional Approval; Requirement To Bring Drug
to Market.--
``(1) Duration; renewals.--The period of provisional
approval for a drug approved 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 by the Secretary. Provisional approval status with
respect to a drug shall not exceed a total of 6 years from the
initial date the sponsor was awarded provisional approval
status.
``(2) Marketing requirement.--If any drug that receives
provisional approval status under this section is not brought
to market within 180 days of the approval, such 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) Applying for Full Approval.--
``(1) In general.--Except as provided under paragraph (2),
the sponsor of a drug granted provisional approval pursuant to
this section may, at any point, submit an application for full
approval of such drug under section 505 of this Act or section
351 of the Public Health Service Act, as applicable.
``(2) Effect of recession on approval and automatic
approval.--
``(A) In general.--The sponsor of a drug granted
provisional approval pursuant to this section that has
been rescinded under subsection (h)(3), may submit an
application for full approval of such drug under
section 505 of this Act or section 351 of the Public
Health Service Act at any time.
``(B) Automatic approval.--Such full approval may
be awarded at any time for any drug granted provisional
approval pursuant to this section if the sponsor of the
drug establishes a 15 percent improvement in an
important endpoint, including surrogate endpoints not
validated by the Food and Drug Administration, compared
to a standard drug.
``(3) Real-time epidemic and pandemic vaccine approval.--
``(A) In general.--In the case of a vaccine
developed in response to an epidemic or pandemic,
including COVID-19, the Secretary shall share data
information regarding the approval of the vaccine with
the Advisory Committee on Immunization Practices of the
Centers for Disease Control and Prevention as the
review nears completion.
``(B) Evaluation.--Any vaccine that has been
approved by the Secretary for an epidemic or pandemic-
related disease, including COVID-19, shall be evaluated
by the Advisory Committee on Immunization Practices of
the Centers for Disease Control and Prevention not
later than 1 week after the date of submission to the
Advisory Committee by the Secretary of the vaccine.
``(l) Patient Advocate General.--Not later than 6 months after the
date of enactment of the Promising Pathway Act, the Secretary shall
establish within the Office of the Commissioner, the position of
Patient Advocate General, who shall provide assistance to patients and
their families who use drugs under evaluation in this pathway or drugs
reviewed or approved under section 505 or section 351 of the Public
Health Service Act. Such assistance shall include providing bi-
informational communication about maintaining patient health, delivery
of proper informed consent, participating in clinical investigations,
completing required documentation in order to participate in the
applicable programs, and providing other information.''.
(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 524B 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:
``(e) 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 524B
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 524B.''.
(2) Federal health care programs.--The requirement under
subsection (e) 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 (e).
(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 524B of such Act'' before the
semicolon.
<all>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
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