II 

116TH CONGRESS 

2D SESSION S. 3872 

To amend the Federal Food, Drug, and Cosmetic Act to establish a timelimited 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 biological products, and for other purposes. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Promising Pathway 

5 Act’’. 

6 SEC. 2. FINDINGS. 

7 Congress finds as follows: 


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1 (1) The drugs and biological products intended 

2 to be reviewed under the pathway established under 

3 this Act are for the treatment and prevention of se4 rious diseases or conditions, especially those for 

5 which there are no available on-label meaningful or 

6 disease-modifying treatments, where speed to access 

7 is critical. 

8 (2) The approval pathway established under 

9 this Act is intended to allow drug and biological 

10 product applications to be more rapidly reviewed by 

11 the U.S. Food and Drug Administration (FDA), 

12 with the FDA reviewing various portions of new 

13 drug and biological product applications as they be14 come available. 

15 (3) The approval pathway established under 

16 this Act establishes a clear approval pathway that 

17 can be utilized by sponsors to receive rolling review 

18 of applications for drugs and biological products in19 tended to treat serious diseases, including drugs and 

20 biological products intended to treat COVID–19 that 

21 reduce the risk of death, severe disease, and progres22 sion of symptoms in those exposed to the virus. 

23 (4) The approval pathway established under 

24 this Act will enable sponsors to receive early, time25 limited, and provisional approval for drugs and bioVerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872

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1 logical products that have demonstrated substantial 

2 evidence of safety and relevant early evidence that 

3 establishes that the drug provides a positive thera4 peutic outcome. 

5 (5) The approval pathway established under 

6 this Act will allow for the use of real-world evidence 

7 and scientifically substantiated surrogates, other 

8 than those previously validated by the FDA, to pre9 dict the clinical benefits and ultimately support pro10 visional approval. 

11 (6) Drugs and biological products granted pro12 visional approval under the pathway established 

13 under this Act are limited to a 2-year approval pe14 riod, renewable every 2 years, for up to 6 years. Full 

15 approval can awarded at any time, for any drug or 

16 biological product provisionally approved under this 

17 pathway that establishes a 15 percent improvement 

18 in an important endpoint compared to standard 

19 therapies. 

20 (7) The approval pathway established under 

21 this Act prohibits denial of coverage for any drug or 

22 biological product provisionally approved under this 

23 approval pathway on account of it being experi24 mental. 


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1 (8) Informed consent is required for any pa2 tients using a drug or biological product approved 

3 under the provisional approval pathway established 

4 under this Act. Any patients using a drug or biologi5 cal product reviewed under this approval pathway 

6 must participate in an observational registry until 

7 those drugs or biological products receive full ap8 proval, with approval contingent on registry partici9 pation. 

10 (9) This Act requires that registries track ag11 gregated, de-identified data that will be readily avail12 able to approved researchers for public health re13 search purposes. 

14 (10) This Act creates, within the Office of the 

15 Commissioner at the FDA, the position of the Pa16 tient Advocate General to provide assistance to pa17 tients and their families utilizing drugs and biologi18 cal products. 

19 SEC. 3. PROVISIONAL APPROVAL OF NEW HUMAN DRUGS. 

20 (a) IN GENERAL.—Subchapter A of chapter V of the 

21 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351 

22 et seq.) is amended by adding at the end of the following: 


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1 ‘‘SEC. 524B. PROVISIONAL APPROVAL OF NEW HUMAN 

2 DRUGS. 

3 ‘‘(a) PRIORITY REVIEW AND EVALUATION OF APPLI4 CATIONS.— 

5 ‘‘(1) IN GENERAL.—The Secretary shall estab6 lish a priority review system to evaluate applications 

7 submitted under this pathway for provisional ap8 proval within 90 days of receipt of a completed ap9 plication. 

10 ‘‘(2) REVIEW OF APPLICATIONS DURING

11 EPIDEMICS AND PANDEMICS.—In the case of an epi12 demic or pandemic, including with respect to 

13 COVID–19, the Secretary shall accept and review 

14 various portions of an application submitted under 

15 the pathway under this section for provisional ap16 proval on a rolling basis, and the review of any part 

17 of an application so submitted shall be completed 

18 not later than 3 weeks after submission. 

19 ‘‘(3) OTHER DESIGNATIONS.—If a drug sub20 mitted for review under the pathway under this sec21 tion is eligible for a special designation by the Sec22 retary under this Act, including as a drug for a rare 

23 disease or condition under section 526, all benefits 

24 of such other designation shall be available for use 

25 under provisional approval, including any tax credits 

26 and waiving of fees under chapter VII. 


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1 ‘‘(b) ELIGIBILITY.—A drug may be eligible for provi2 sional approval under this section if the Secretary deter3 mines that the drug is intended for the treatment, preven4 tion, or medical diagnosis of— 

5 ‘‘(1) a serious or life-threatening disease or con6 dition for which there is a reasonable likelihood that 

7 premature death will occur without early medical 

8 intervention for an individual contracting or being 

9 diagnosed with such disease or condition; 

10 ‘‘(2) a disease or condition that poses a threat 

11 of epidemic or pandemic; or 

12 ‘‘(3) a disease or condition associated with mor13 bidity that has a substantial impact on day-to-day 

14 functioning. 

15 ‘‘(c) STANDARD OF REVIEW FOR APPROVAL.— 

16 ‘‘(1) REQUIREMENTS.—An application for pro17 visional approval under this section may be approved 

18 only if the Secretary determines that— 

19 ‘‘(A) there is substantial evidence of safety 

20 for the drug, such that there is evidence con21 sisting of adequate and well-controlled inves22 tigations, including clinical investigations, by 

23 experts qualified by scientific training and expe24 rience to evaluate the safety of the drug in25 volved, on the basis of which it could fairly and 


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1 responsibly be concluded that the drug will have 

2 the effect it purports or is represented to have 

3 under the conditions of use prescribed, rec4 ommended, or suggested in the labeling or pro5 posed labeling; and 

6 ‘‘(B) there is relevant early evidence based 

7 on adequate and well-controlled investigations, 

8 including early-stage clinical investigations, to 

9 establish that— 

10 ‘‘(i) the drug provides a positive 

11 therapeutic outcome; and 

12 ‘‘(ii) the outcome of the drug is con13 sistent with or greater than currently mar14 keted on-label therapies, with equal or 

15 fewer side effects, if there are currently 

16 marketed on-label therapies. 

17 ‘‘(2) PROTOCOLS.—The Secretary shall promul18 gate rules that establish the appropriate protocols 

19 for a sponsor of an application for provisional ap20 proval under this section and the Commissioner to 

21 follow to enable rolling, real-time, mid-trial submis22 sion while preserving the integrity of the ongoing 

23 trial and without penalizing the sponsor for making 

24 use of this pathway. 


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1 ‘‘(3) REAL WORLD EVIDENCE.—The Secretary 

2 shall allow the use of real world evidence (as defined 

3 in section 505F(b)), including real world data used 

4 to generate real world evidence, to support an appli5 cation for provisional approval under this section, 

6 and to fulfill the follow-up requirements and support 

7 applications for full approval as described under sec8 tion 505 or section 351 of the Public Health Service 

9 Act, as applicable. 

10 ‘‘(4) USE OF SCIENTIFICALLY-SUBSTANTIATED

11 SURROGATES.— 

12 ‘‘(A) IN GENERAL.—The sponsor of an ap13 plication for provisional approval under this sec14 tion may use scientifically-substantiated surro15 gates to support such application. 

16 ‘‘(B) DEFINITION.—In subparagraph (A), 

17 the term ‘scientifically-substantiated surrogates’ 

18 means surrogate endpoints to predict clinical 

19 benefit other than such endpoints previously 

20 validated by the Secretary, based on— 

21 ‘‘(i) epidemiologic, therapeutic, patho22 physiologic, or other evidence; or 

23 ‘‘(ii) an effect on a clinical endpoint 

24 other than survival or irreversible mor25 bidity of interest. 


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1 ‘‘(d) TRANSPARENCY AND PATIENT MONITORING

2 REQUIREMENTS.— 

3 ‘‘(1) REGISTRIES.— 

4 ‘‘(A) IN GENERAL.—The sponsor of a drug 

5 provisionally approved under this section shall 

6 require that all patients who use such drug par7 ticipate in an observational registry and consent 

8 to the sponsor’s collection, and submission to 

9 the registry, of data related to the patient’s use 

10 of such drug until such drug receives full ap11 proval under section 505 or section 351 of the 

12 Public Health Service Act, or the provisional 

13 approval is rescinded. 

14 ‘‘(B) REQUIREMENTS FOR REGISTRIES.— 

15 An observational registry described in subpara16 graph (A) may be run by a third party, such as 

17 a government, for profit, or nonprofit organiza18 tion, and shall track all patients who use the 

19 provisionally approved drug. 

20 ‘‘(C) ACCESSIBILITY.—An observational 

21 registry described in subparagraph (A) shall be 

22 easily accessible for— 

23 ‘‘(i) all patients who are participating 

24 in any registry related to a provisionally 

25 approved drug that allows for easy, unreVerDate Sep 11 2014 21:59 Jun 20, 2020 Jkt 099200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\S3872.IS S3872

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1 stricted (or transparent) access for such 

2 patients to their patient data and related 

3 information regarding their usage of the 

4 provisionally approved drug; and 

5 ‘‘(ii) approved researchers and med6 ical professionals who may access data 

7 maintained in the registry, which access 

8 shall be for public health research and only 

9 in a de-identified, aggregated manner. 

10 ‘‘(2) FUNDING.—An observational registry 

11 under this subsection shall be maintained, as appli12 cable— 

13 ‘‘(A) by the sponsor of the drug provision14 ally approved under this section that is the sub15 ject of the registry; 

16 ‘‘(B) by a third party, such as a govern17 ment, for profit, or nonprofit organization; or 

18 ‘‘(C) the Federal Government, in the case 

19 of any drug so approved that is intended to 

20 treat a disease or condition associated with an 

21 epidemic or pandemic. 

22 ‘‘(3) SPONSOR REQUIREMENTS.— 

23 ‘‘(A) IN GENERAL.—For any drug applica24 tion provisionally approved under this section, 

25 the Secretary shall notify the sponsor of the 


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1 exact data such sponsor is required to submit 

2 to an observational registry. 

3 ‘‘(B) ANNUAL REVIEW OF THE REGISTRY; 

4 PENALTIES.—The Secretary shall conduct an 

5 annual review of observational registries estab6 lished under this subsection. If, at such an an7 nual review, less than 90 percent of patients are 

8 participating in an observational registry with 

9 respect to a drug approved under this section, 

10 the Secretary shall issue to the sponsor of such 

11 drug a civil monetary penalty of not more than 

12 $100,000. If a violation of this section is not 

13 corrected within the 30-day period following no14 tification, the sponsor shall, in addition to any 

15 penalty under this subparagraph be subject to 

16 a civil monetary penalty of not more than 

17 $10,000 for each day of the violation after such 

18 period until the violation is corrected. If appli19 cation patient participation in an observational 

20 registry is not at or above 90 percent within 6 

21 months of issuance of such penalty, the provi22 sional approval shall be withdrawn. 

23 ‘‘(4) ANNUAL REPORT TO CONGRESS.—The 

24 Secretary shall submit an annual report to Congress 


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1 on all drugs granted provisional approval under this 

2 section. Such report shall include— 

3 ‘‘(A) the number of patients treated with 

4 each such drug, and the number of patients 

5 tracked in an observational registry with re6 spect to each such drug; 

7 ‘‘(B) a discussion of the minimum amount 

8 of data required in the registries, including pa9 tient treatments and uses, length of use, side 

10 effects encountered, relevant biomarkers or sci11 entifically substantiated surrogates, scan re12 sults, cause of death and how long the patient 

13 lived, and adverse drug effects; 

14 ‘‘(C) a list of all such drugs for which an 

15 application for full approval under section 505 

16 of this Act or section 351 of the Public Health 

17 Service Act, or an application for an extension 

18 of provisional approval under this section, has 

19 been submitted; and 

20 ‘‘(D) a list of all applications denied provi21 sional approval under this section, together with 

22 an explanation for the decisions to deny each 

23 such application. 

24 ‘‘(e) WITHDRAWAL OF PROVISIONAL APPROVAL.— 


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1 ‘‘(1) IN GENERAL.—The Secretary shall with2 draw provisional approval under this section if there 

3 are a significant numbers of patients who experience 

4 serious adverse effects, compared to the other cur5 rently marketed on-label therapies that are available 

6 for the applicable disease or condition. 

7 ‘‘(2) EFFECT OF WITHDRAWAL.—If a provi8 sional approval is withdrawn under this subsection, 

9 the sponsor may not make the drug available to any 

10 new patients, but may be allowed to continue to 

11 make such drug available to patients who started 

12 taking the drug prior to the date of withdrawal, for 

13 as long a period as dictated by patient need, as de14 termined by the Secretary. 

15 ‘‘(f) TRANSPARENCY.—Any scientific, medical, aca16 demic, or health care journal publishing an article explain17 ing, releasing, conveying or announcing research findings 

18 which were funded by the Department of Health and 

19 Human Services shall be prohibited from publishing such 

20 research unless— 

21 ‘‘(1) such article conveying research findings is 

22 made publicly available on the journal’s internet 

23 website without a paywall or charge not later than 

24 3 months after the date on which such article was 


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1 first provided to subscribers of such journal (or first 

2 made available for purchase); and 

3 ‘‘(2) the article’s author or researcher or au4 thor’s institution (or, in the case of multiple authors, 

5 researchers, or institutions, all such authors, re6 searchers, or institutions) received less than 30 per7 cent of funding for such research from the Depart8 ment of Health and Human Services throughout the 

9 period of time the research was conducted. 

10 ‘‘(g) INFORMED CONSENT.—Prior to receiving a drug 

11 provisionally approved under this section, the sponsor of 

12 the drug shall receive from each patient, or the patient’s 

13 representative, informed consent, through a signed in14 formed consent form, acknowledging that such patient un15 derstands that the drug did not undergo the usual process 

16 for full approval of a drug by the Food and Drug Adminis17 tration, and that such patient is willing to accept the risks 

18 involved in taking such drug. 

19 ‘‘(h) POSTMARKET CONTROLS AND LABELING.— 

20 ‘‘(1) FDA ANNUAL REVIEW OF REGISTRY

21 DATA.—The Secretary shall annually review the data 

22 made available through the observational registries 

23 under subsection (d) and make a determination re24 garding whether the side effect profile of any drug 

25 approved under this pathway does not support the 


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1 benefit provided, or the data shows the benefit is 

2 less than the benefits offered through other, fully 

3 approved drugs. 

4 ‘‘(2) LABELING.—The sponsor of the provision5 ally approved drug shall ensure that all labeling and 

6 promotional materials for the drug bear the state7 ment ‘provisionally approved by the FDA pending a 

8 full demonstration of effectiveness under application 

9 number llllll’ (specifying the application 

10 number assigned by the Secretary in place of the 

11 blank). All promotional, educational and marketing 

12 materials for provisionally approved products shall 

13 be reviewed and approved by the Secretary before 

14 such materials are distributed. 

15 ‘‘(3) RESCISSION OF PROVISIONAL AP16 PROVAL.—If the Secretary determines that the side 

17 effect profile of any drug included in such observa18 tional registries does not support the benefit pro19 vided by such drug, or that the data shows that the 

20 benefit is less than the benefits offered through 

21 other, fully approved drugs, the Secretary shall re22 scind such provisional approval. 

23 ‘‘(i) DURATION OF PROVISIONAL APPROVAL; RE24 QUIREMENT TO BRING DRUG TO MARKET.— 


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1 ‘‘(1) DURATION; RENEWALS.—The period of 

2 provisional approval for a drug approved under this 

3 section is effective for a 2-year period. The sponsor 

4 may request renewal for provisional approval status 

5 for up to 3 subsequent 2-year periods by the Sec6 retary. Provisional approval status with respect to a 

7 drug shall not exceed a total of 6 years from the ini8 tial date the sponsor was awarded provisional ap9 proval status. 

10 ‘‘(2) MARKETING REQUIREMENT.—If any drug 

11 that receives provisional approval status under this 

12 section is not brought to market within 180 days of 

13 the approval, such approval shall be rescinded. 

14 ‘‘(j) LIMITATION ON LIABILITY.—With respect to any 

15 claim under State law alleging that a drug sold or other16 wise made available pursuant to a grant of provisional ap17 proval under this section is unsafe or ineffective, no liabil18 ity in a cause of action shall lie against a sponsor or manu19 facturer, unless the relevant conduct constitutes reckless 

20 or willful misconduct, gross negligence, or an intentional 

21 tort under any applicable State law. 

22 ‘‘(k) APPLYING FOR FULL APPROVAL.— 

23 ‘‘(1) IN GENERAL.—Except as provided under 

24 paragraph (2), the sponsor of a drug granted provi25 sional approval pursuant to this section may, at any 


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1 point, submit an application for full approval of such 

2 drug under section 505 of this Act or section 351 

3 of the Public Health Service Act, as applicable. 

4 ‘‘(2) EFFECT OF RECESSION ON APPROVAL AND

5 AUTOMATIC APPROVAL.— 

6 ‘‘(A) IN GENERAL.—The sponsor of a drug 

7 granted provisional approval pursuant to this 

8 section that has been rescinded under sub9 section (h)(3), may submit an application for 

10 full approval of such drug under section 505 of 

11 this Act or section 351 of the Public Health 

12 Service Act at any time. 

13 ‘‘(B) AUTOMATIC APPROVAL.—Such full 

14 approval may be awarded at any time for any 

15 drug granted provisional approval pursuant to 

16 this section if the sponsor of the drug estab17 lishes a 15 percent improvement in an impor18 tant endpoint, including surrogate endpoints 

19 not validated by the Food and Drug Adminis20 tration, compared to a standard drug. 

21 ‘‘(3) REAL-TIME EPIDEMIC AND PANDEMIC VAC22 CINE APPROVAL.— 

23 ‘‘(A) IN GENERAL.—In the case of a vac24 cine developed in response to an epidemic or 

25 pandemic, including COVID–19, the Secretary 


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1 shall share data information regarding the ap2 proval of the vaccine with the Advisory Com3 mittee on Immunization Practices of the Cen4 ters for Disease Control and Prevention as the 

5 review nears completion. 

6 ‘‘(B) EVALUATION.—Any vaccine that has 

7 been approved by the Secretary for an epidemic 

8 or pandemic-related disease, including COVID– 

9 19, shall be evaluated by the Advisory Com10 mittee on Immunization Practices of the Cen11 ters for Disease Control and Prevention not 

12 later than 1 week after the date of submission 

13 to the Advisory Committee by the Secretary of 

14 the vaccine. 

15 ‘‘(l) PATIENT ADVOCATE GENERAL.—Not later than 

16 6 months after the date of enactment of the Promising 

17 Pathway Act, the Secretary shall establish within the Of18 fice of the Commissioner, the position of Patient Advocate 

19 General, who shall provide assistance to patients and their 

20 families who use drugs under evaluation in this pathway 

21 or drugs reviewed or approved under section 505 or sec22 tion 351 of the Public Health Service Act. Such assistance 

23 shall include providing bi-informational communication 

24 about maintaining patient health, delivery of proper in25 formed consent, participating in clinical investigations, 


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1 completing required documentation in order to participate 

2 in the applicable programs, and providing other informa3 tion.’’. 

4 (b) CONFORMING AMENDMENT.—Section 505(a) of 

5 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

6 355(a)) is amended by inserting ‘‘, or there is in effect 

7 a provisional approval under section 524B with respect to 

8 such drug’’ before the period. 

9 (c) REIMBURSEMENT.— 

10 (1) PRIVATE HEALTH INSURERS.—Section 

11 2719A of the Public Health Service Act (42 U.S.C. 

12 300gg–19a) is amended by adding at the end the 

13 following: 

14 ‘‘(e) TREATMENT OF CERTAIN DRUGS.—A group 

15 health plan or health insurance issuer of group or indi16 vidual health insurance coverage shall not deny coverage 

17 of any drug provisionally approved under section 524B of 

18 the Federal Food, Drug, and Cosmetic Act on the basis 

19 of such drug being experimental. In determining coverage 

20 under the applicable plan or coverage, a group health plan 

21 or health insurance issuer shall treat a drug provisionally 

22 approved under such section in the same manner as such 

23 plan or coverage would treat a drug approved under sec24 tion 505 of the Federal Food, Drug, and Cosmetic Act 

25 or section 351 of this Act. Nothing in this subsection shall 


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1 be construed to require a group health plan or health in2 surance issuer to cover any specific drug provisionally ap3 proved under such section 524B.’’. 

4 (2) FEDERAL HEALTH CARE PROGRAMS.—The 

5 requirement under subsection (e) of section 2719A 

6 of the Public Health Service Act (as added by para7 graph (1)) shall apply with respect to coverage de8 terminations under a Federal health care program 

9 (as defined in section 1128B(f) of the Social Secu10 rity Act (42 U.S.C. 1320a–7b(f))) in the same man11 ner such requirement applies under such subsection 

12 (e). 

13 (3) CONFORMING AMENDMENT.—Section 

14 1927(k)(2)(A)(i) of the Social Security Act (42 

15 U.S.C. 1396r–8(k)(2)(A)(i)) is amended— 

16 (A) by striking ‘‘or which’’ and inserting ‘‘, 

17 which’’; and 

18 (B) by inserting ‘‘, or which is provision19 ally approved under section 524B of such Act’’ 

20 before the semicolon. 

Æ 


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