Promising Pathway Act

I 117TH CONGRESS 1ST SESSION H. R. 3761 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 8, 2021 Mr. GALLAGHER (for himself, Mr. QUIGLEY, Mr. WESTERMAN, and Mr. SWALWELL) introduced the following bill; which was referred to the Com- mittee 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 bio- logical products, and for other purposes. Be it enacted by the Senate and House of Representa- 1 tives of the United States of America in Congress assembled, 2 SECTION 1. SHORT TITLE. 3 This Act may be cited as the ‘‘Promising Pathway 4 Act’’. 5 SEC. 2. FINDINGS. 6 Congress finds as follows: 7 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 2 •HR 3761 IH (1) The drugs and biological products intended 1 to be reviewed under the pathway established under 2 this Act are for the treatment and prevention of se- 3 rious diseases or conditions, especially those for 4 which there are no available on-label meaningful or 5 disease-modifying treatments, where speed to access 6 is critical. 7 (2) The approval pathway established under 8 this Act is intended to allow drug and biological 9 product applications to be more rapidly reviewed by 10 the U.S. Food and Drug Administration (FDA), 11 with the FDA reviewing various portions of new 12 drug and biological product applications as they be- 13 come available. 14 (3) The approval pathway established under 15 this Act establishes a clear approval pathway that 16 can be utilized by sponsors to receive rolling review 17 of applications for drugs and biological products in- 18 tended to treat serious diseases, including drugs and 19 biological products intended to treat COVID–19 that 20 reduce the risk of death, severe disease, and progres- 21 sion of symptoms in those exposed to the virus. 22 (4) The approval pathway established under 23 this Act will enable sponsors to receive early, time- 24 limited, and provisional approval for drugs and bio- 25 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 3 •HR 3761 IH logical products that have demonstrated substantial 1 evidence of safety and relevant early evidence that 2 establishes that the drug provides a positive thera- 3 peutic outcome. 4 (5) The approval pathway established under 5 this Act will allow for the use of real-world evidence 6 and scientifically substantiated surrogates, other 7 than those previously validated by the FDA, to pre- 8 dict the clinical benefits and ultimately support pro- 9 visional approval. 10 (6) Drugs and biological products granted pro- 11 visional approval under the pathway established 12 under this Act are limited to a 2-year approval pe- 13 riod, renewable every 2 years, for up to 6 years. Full 14 approval can awarded at any time, for any drug or 15 biological product provisionally approved under this 16 pathway that establishes a 15 percent improvement 17 in an important endpoint compared to standard 18 therapies. 19 (7) The approval pathway established under 20 this Act prohibits denial of coverage for any drug or 21 biological product provisionally approved under this 22 approval pathway on account of it being experi- 23 mental. 24 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 4 •HR 3761 IH (8) Informed consent is required for any pa- 1 tients using a drug or biological product approved 2 under the provisional approval pathway established 3 under this Act. Any patients using a drug or biologi- 4 cal product reviewed under this approval pathway 5 must participate in an observational registry until 6 those drugs or biological products receive full ap- 7 proval, with approval contingent on registry partici- 8 pation. 9 (9) This Act requires that registries track ag- 10 gregated, de-identified data that will be readily avail- 11 able to approved researchers for public health re- 12 search purposes. 13 (10) This Act creates, within the Office of the 14 Commissioner at the FDA, the position of the Pa- 15 tient Advocate General to provide assistance to pa- 16 tients and their families utilizing drugs and biologi- 17 cal products. 18 SEC. 3. PROVISIONAL APPROVAL OF NEW HUMAN DRUGS. 19 (a) IN GENERAL.—Subchapter A of chapter V of the 20 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351 21 et seq.) is amended by adding at the end the following: 22 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 5 •HR 3761 IH ‘‘SEC. 524B. PROVISIONAL APPROVAL OF NEW HUMAN 1 DRUGS. 2 ‘‘(a) PRIORITY REVIEW AND EVALUATION OF APPLI- 3 CATIONS.— 4 ‘‘(1) IN GENERAL.—The Secretary shall estab- 5 lish a priority review system to evaluate applications 6 submitted under this pathway for provisional ap- 7 proval within 90 days of receipt of a completed ap- 8 plication. 9 ‘‘(2) REVIEW OF APPLICATIONS DURING 10 EPIDEMICS AND PANDEMICS.—In the case of an epi- 11 demic or pandemic, including with respect to 12 COVID–19, the Secretary shall accept and review 13 various portions of an application submitted under 14 the pathway under this section for provisional ap- 15 proval on a rolling basis, and the review of any part 16 of an application so submitted shall be completed 17 not later than 3 weeks after submission. 18 ‘‘(3) OTHER DESIGNATIONS.—If a drug sub- 19 mitted for review under the pathway under this sec- 20 tion is eligible for a special designation by the Sec- 21 retary under this Act, including as a drug for a rare 22 disease or condition under section 526, all benefits 23 of such other designation shall be available for use 24 under provisional approval, including any tax credits 25 and waiving of fees under chapter VII. 26 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 6 •HR 3761 IH ‘‘(b) ELIGIBILITY.—A drug may be eligible for provi- 1 sional approval under this section if the Secretary deter- 2 mines that the drug is intended for the treatment, preven- 3 tion, or medical diagnosis of— 4 ‘‘(1) a serious or life-threatening disease or con- 5 dition for which there is a reasonable likelihood that 6 premature death will occur without early medical 7 intervention for an individual contracting or being 8 diagnosed with such disease or condition; or 9 ‘‘(2) a disease or condition that poses a threat 10 of epidemic or pandemic. 11 ‘‘(c) STANDARD OF REVIEW FOR APPROVAL.— 12 ‘‘(1) REQUIREMENTS.—An application for pro- 13 visional approval under this section may be approved 14 only if the Secretary determines that— 15 ‘‘(A) there is substantial evidence of safety 16 for the drug, such that there is evidence con- 17 sisting of adequate and well-controlled inves- 18 tigations, including clinical investigations, by 19 experts qualified by scientific training and expe- 20 rience to evaluate the safety of the drug in- 21 volved, on the basis of which it could fairly and 22 responsibly be concluded that the drug will have 23 the effect it purports or is represented to have 24 under the conditions of use prescribed, rec- 25 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 7 •HR 3761 IH ommended, or suggested in the labeling or pro- 1 posed labeling; and 2 ‘‘(B) there is relevant early evidence based 3 on adequate and well-controlled investigations, 4 including early-stage clinical investigations, to 5 establish that— 6 ‘‘(i) the drug provides a positive 7 therapeutic outcome; and 8 ‘‘(ii) the outcome of the drug is con- 9 sistent with or greater than currently mar- 10 keted on-label therapies, with equal or 11 fewer side effects, if there are currently 12 marketed on-label therapies. 13 ‘‘(2) PROTOCOLS.—The Secretary shall promul- 14 gate rules that establish the appropriate protocols 15 for a sponsor of an application for provisional ap- 16 proval under this section and the Commissioner to 17 follow to enable rolling, real-time, mid-trial submis- 18 sion while preserving the integrity of the ongoing 19 trial and without penalizing the sponsor for making 20 use of this pathway. 21 ‘‘(3) REAL WORLD EVIDENCE.—The Secretary 22 shall allow the use of real world evidence (as defined 23 in section 505F(b)), including real world data used 24 to generate real world evidence, to support an appli- 25 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 8 •HR 3761 IH cation for provisional approval under this section, 1 and to fulfill the follow-up requirements and support 2 applications for full approval as described under sec- 3 tion 505 or section 351 of the Public Health Service 4 Act, as applicable. 5 ‘‘(4) USE OF SCIENTIFICALLY SUBSTANTIATED 6 SURROGATES.— 7 ‘‘(A) IN GENERAL.—The sponsor of an ap- 8 plication for provisional approval under this sec- 9 tion may use scientifically substantiated surro- 10 gates to support such application. 11 ‘‘(B) DEFINITION.—In subparagraph (A), 12 the term ‘scientifically substantiated surrogates’ 13 means surrogate endpoints to predict clinical 14 benefit other than such endpoints previously 15 validated by the Secretary, based on— 16 ‘‘(i) epidemiologic, therapeutic, patho- 17 physiologic, or other evidence; or 18 ‘‘(ii) an effect on a clinical endpoint 19 other than survival or irreversible mor- 20 bidity of interest. 21 ‘‘(d) TRANSPARENCY AND PATIENT MONITORING 22 REQUIREMENTS.— 23 ‘‘(1) REGISTRIES.— 24 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 9 •HR 3761 IH ‘‘(A) IN GENERAL.—The sponsor of a drug 1 provisionally approved under this section shall 2 require that all patients who use such drug par- 3 ticipate in an observational registry and consent 4 to the sponsor’s collection, and submission to 5 the registry, of data related to the patient’s use 6 of such drug until such drug receives full ap- 7 proval under section 505 or section 351 of the 8 Public Health Service Act, or the provisional 9 approval is rescinded. 10 ‘‘(B) REQUIREMENTS FOR REGISTRIES.— 11 An observational registry described in subpara- 12 graph (A) may be run by a third party, such as 13 a government, for profit, or non-profit organiza- 14 tion, and shall track all patients who use the 15 provisionally approved drug. 16 ‘‘(C) ACCESSIBILITY.—An observational 17 registry described in subparagraph (A) shall be 18 easily accessible for— 19 ‘‘(i) all patients who are participating 20 in any registry related to a provisionally 21 approved drug that allows for easy, unre- 22 stricted (or transparent) access for such 23 patients to their patient data and related 24 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 10 •HR 3761 IH information regarding their usage of the 1 provisionally approved drug; and 2 ‘‘(ii) approved researchers and med- 3 ical professionals who may access data 4 maintained in the registry, which access 5 shall be for public health research and only 6 in a de-identified, aggregated manner. 7 ‘‘(2) FUNDING.—An observational registry 8 under this subsection shall be maintained, as appli- 9 cable— 10 ‘‘(A) by the sponsor of the drug provision- 11 ally approved under this section that is the sub- 12 ject of the registry; 13 ‘‘(B) by a third party, such as a govern- 14 ment, for profit, or nonprofit organization; or 15 ‘‘(C) the Federal Government, in the case 16 of any drug so approved that is intended to 17 treat a disease or condition associated with an 18 epidemic or pandemic. 19 ‘‘(3) SPONSOR REQUIREMENTS.— 20 ‘‘(A) IN GENERAL.—For any drug applica- 21 tion provisionally approved under this section, 22 the Secretary shall notify the sponsor of the 23 exact data such sponsor is required to submit 24 to an observational registry. 25 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 11 •HR 3761 IH ‘‘(B) ANNUAL REVIEW OF THE REGISTRY; 1 PENALTIES.—The Secretary shall conduct an 2 annual review of observational registries estab- 3 lished under this subsection. If, at such an an- 4 nual review, less than 90 percent of patients are 5 participating in an observational registry with 6 respect to a drug approved under this section, 7 the Secretary shall issue to the sponsor of such 8 drug a civil monetary penalty of not more than 9 $100,000. If a violation of this section is not 10 corrected within the 30-day period following no- 11 tification, the sponsor shall, in addition to any 12 penalty under this subparagraph be subject to 13 a civil monetary penalty of not more than 14 $10,000 for each day of the violation after such 15 period until the violation is corrected. If appli- 16 cation patient participation in an observational 17 registry is not at or above 90 percent within 6 18 months of issuance of such penalty, the provi- 19 sional approval shall be withdrawn. 20 ‘‘(4) ANNUAL REPORT TO CONGRESS.—The 21 Secretary shall submit an annual report to Congress 22 on all drugs granted provisional approval under this 23 section. Such report shall include— 24 VerDate Sep 11 2014 22:12 Jul 13, 2021 Jkt 019200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\H3761.IH H3761 pbinns on DSKJLVW7X2PROD with BILLS 12 •HR 3761 IH ‘‘(A) the number of patients treated with 1 each such drug, and the number of patients 2 tracked in an observational registry with re- 3 spect to each such drug; 4 ‘‘(B) a discussion of the minimum amount 5 of data required in the registries, including pa- 6 tient treatments and uses, length of use, side 7 effects encountered, relevant biomarkers or sci- 8 entifically substantiated surrogates, scan re- 9 sults, cause of death and how long the patient 10 lived, and adverse drug effects; 11 ‘‘(C) a list of all such drugs for which an 12 application for full approval under section 505 13 of this Act or section 351 of the Public Health 14 Service Act, or an application for an extension 15 of provisi [Text truncated for display. Full text available on Congress.gov.]