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receives provisional approval status is not brought to market within 180 days of the approval, the approval must be rescinded.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.
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 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; 
        or
            ``(2) a disease or condition that poses a threat of 
        epidemic or pandemic.
    ``(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>
Promising Pathway Act
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.
Promising Pathway Act
Rep. Gallagher, Mike