[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4408 Introduced in House (IH)]

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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.
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