Promising Pathway Act

II 116TH CONGRESS 2D SESSION S. 3872 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 SENATE OF THE UNITED STATES JUNE 3, 2020 Mr. BRAUN (for himself, Ms. MURKOWSKI, and Ms. MCSALLY) introduced the following bill; which was read twice and referred to the Committee on Health, Education, Labor, and Pensions 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 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 2 •S 3872 IS (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 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 3 •S 3872 IS 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 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 4 •S 3872 IS (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 of the following: 22 VerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 5 •S 3872 IS ‘‘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 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 6 •S 3872 IS ‘‘(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; 9 ‘‘(2) a disease or condition that poses a threat 10 of epidemic or pandemic; or 11 ‘‘(3) a disease or condition associated with mor- 12 bidity that has a substantial impact on day-to-day 13 functioning. 14 ‘‘(c) STANDARD OF REVIEW FOR APPROVAL.— 15 ‘‘(1) REQUIREMENTS.—An application for pro- 16 visional approval under this section may be approved 17 only if the Secretary determines that— 18 ‘‘(A) there is substantial evidence of safety 19 for the drug, such that there is evidence con- 20 sisting of adequate and well-controlled inves- 21 tigations, including clinical investigations, by 22 experts qualified by scientific training and expe- 23 rience to evaluate the safety of the drug in- 24 volved, on the basis of which it could fairly and 25 VerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 7 •S 3872 IS responsibly be concluded that the drug will have 1 the effect it purports or is represented to have 2 under the conditions of use prescribed, rec- 3 ommended, or suggested in the labeling or pro- 4 posed labeling; and 5 ‘‘(B) there is relevant early evidence based 6 on adequate and well-controlled investigations, 7 including early-stage clinical investigations, to 8 establish that— 9 ‘‘(i) the drug provides a positive 10 therapeutic outcome; and 11 ‘‘(ii) the outcome of the drug is con- 12 sistent with or greater than currently mar- 13 keted on-label therapies, with equal or 14 fewer side effects, if there are currently 15 marketed on-label therapies. 16 ‘‘(2) PROTOCOLS.—The Secretary shall promul- 17 gate rules that establish the appropriate protocols 18 for a sponsor of an application for provisional ap- 19 proval under this section and the Commissioner to 20 follow to enable rolling, real-time, mid-trial submis- 21 sion while preserving the integrity of the ongoing 22 trial and without penalizing the sponsor for making 23 use of this pathway. 24 VerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 8 •S 3872 IS ‘‘(3) REAL WORLD EVIDENCE.—The Secretary 1 shall allow the use of real world evidence (as defined 2 in section 505F(b)), including real world data used 3 to generate real world evidence, to support an appli- 4 cation for provisional approval under this section, 5 and to fulfill the follow-up requirements and support 6 applications for full approval as described under sec- 7 tion 505 or section 351 of the Public Health Service 8 Act, as applicable. 9 ‘‘(4) USE OF SCIENTIFICALLY-SUBSTANTIATED 10 SURROGATES.— 11 ‘‘(A) IN GENERAL.—The sponsor of an ap- 12 plication for provisional approval under this sec- 13 tion may use scientifically-substantiated surro- 14 gates to support such application. 15 ‘‘(B) DEFINITION.—In subparagraph (A), 16 the term ‘scientifically-substantiated surrogates’ 17 means surrogate endpoints to predict clinical 18 benefit other than such endpoints previously 19 validated by the Secretary, based on— 20 ‘‘(i) epidemiologic, therapeutic, patho- 21 physiologic, or other evidence; or 22 ‘‘(ii) an effect on a clinical endpoint 23 other than survival or irreversible mor- 24 bidity of interest. 25 VerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 9 •S 3872 IS ‘‘(d) TRANSPARENCY AND PATIENT MONITORING 1 REQUIREMENTS.— 2 ‘‘(1) REGISTRIES.— 3 ‘‘(A) IN GENERAL.—The sponsor of a drug 4 provisionally approved under this section shall 5 require that all patients who use such drug par- 6 ticipate in an observational registry and consent 7 to the sponsor’s collection, and submission to 8 the registry, of data related to the patient’s use 9 of such drug until such drug receives full ap- 10 proval under section 505 or section 351 of the 11 Public Health Service Act, or the provisional 12 approval is rescinded. 13 ‘‘(B) REQUIREMENTS FOR REGISTRIES.— 14 An observational registry described in subpara- 15 graph (A) may be run by a third party, such as 16 a government, for profit, or nonprofit organiza- 17 tion, and shall track all patients who use the 18 provisionally approved drug. 19 ‘‘(C) ACCESSIBILITY.—An observational 20 registry described in subparagraph (A) shall be 21 easily accessible for— 22 ‘‘(i) all patients who are participating 23 in any registry related to a provisionally 24 approved drug that allows for easy, unre- 25 VerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 10 •S 3872 IS stricted (or transparent) access for such 1 patients to their patient data and related 2 information regarding their usage of the 3 provisionally approved drug; and 4 ‘‘(ii) approved researchers and med- 5 ical professionals who may access data 6 maintained in the registry, which access 7 shall be for public health research and only 8 in a de-identified, aggregated manner. 9 ‘‘(2) FUNDING.—An observational registry 10 under this subsection shall be maintained, as appli- 11 cable— 12 ‘‘(A) by the sponsor of the drug provision- 13 ally approved under this section that is the sub- 14 ject of the registry; 15 ‘‘(B) by a third party, such as a govern- 16 ment, for profit, or nonprofit organization; or 17 ‘‘(C) the Federal Government, in the case 18 of any drug so approved that is intended to 19 treat a disease or condition associated with an 20 epidemic or pandemic. 21 ‘‘(3) SPONSOR REQUIREMENTS.— 22 ‘‘(A) IN GENERAL.—For any drug applica- 23 tion provisionally approved under this section, 24 the Secretary shall notify the sponsor of the 25 VerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 11 •S 3872 IS exact data such sponsor is required to submit 1 to an observational registry. 2 ‘‘(B) ANNUAL REVIEW OF THE REGISTRY; 3 PENALTIES.—The Secretary shall conduct an 4 annual review of observational registries estab- 5 lished under this subsection. If, at such an an- 6 nual review, less than 90 percent of patients are 7 participating in an observational registry with 8 respect to a drug approved under this section, 9 the Secretary shall issue to the sponsor of such 10 drug a civil monetary penalty of not more than 11 $100,000. If a violation of this section is not 12 corrected within the 30-day period following no- 13 tification, the sponsor shall, in addition to any 14 penalty under this subparagraph be subject to 15 a civil monetary penalty of not more than 16 $10,000 for each day of the violation after such 17 period until the violation is corrected. If appli- 18 cation patient participation in an observational 19 registry is not at or above 90 percent within 6 20 months of issuance of such penalty, the provi- 21 sional approval shall be withdrawn. 22 ‘‘(4) ANNUAL REPORT TO CONGRESS.—The 23 Secretary shall submit an annual report to Congress 24 VerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872 pamtmann on DSKBC07HB2PROD with BILLS 12 •S 3872 IS on all drugs granted provisional approval under this 1 section. Such report shall include— 2 ‘‘(A) the number of patients treated with 3 each such drug, and the number of patients 4 tracked in an observational registry with re- 5 spect to each such drug; 6 ‘‘(B) a discussion of the minimum amount 7 of data required in the registries, including pa- 8 tient treatments and uses, length of use, side 9 effects encountered, relevant biomarkers or sci- 10 entifically substantiated surrogates, scan re- 11 sults, cause of death and how long the patient 12 lived, and adverse drug effects; 13 ‘‘(C) a list of all such drugs for which an 14 application [Text truncated for display. Full text available on Congress.gov.]