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-
tives 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: 
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
logical products that have demonstrated substantial 
evidence of safety and relevant early evidence that 
establishes that the drug provides a positive thera-
peutic 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 pre-
dict the clinical benefits and ultimately support pro-
visional approval. 
(6) Drugs and biological products granted pro-
visional approval under the pathway established 
under this Act are limited to a 2-year approval pe-
riod, 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 experi-
mental. 
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
(8) Informed consent is required for any pa-
tients using a drug or biological product approved 
under the provisional approval pathway established 
under this Act. Any patients using a drug or biologi-
cal product reviewed under this approval pathway 
must participate in an observational registry until 
those drugs or biological products receive full ap-
proval, with approval contingent on registry partici-
pation. 
(9) This Act requires that registries track ag-
gregated, de-identified data that will be readily avail-
able to approved researchers for public health re-
search purposes. 
(10) This Act creates, within the Office of the 
Commissioner at the FDA, the position of the Pa-
tient Advocate General to provide assistance to pa-
tients and their families utilizing drugs and biologi-
cal 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: 
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
‘‘SEC. 524B. PROVISIONAL APPROVAL OF NEW HUMAN 
DRUGS. 
‘‘(a) PRIORITY REVIEW AND EVALUATION OF APPLI-
CATIONS.— 
‘‘(1) IN GENERAL.—The Secretary shall estab-
lish a priority review system to evaluate applications 
submitted under this pathway for provisional ap-
proval within 90 days of receipt of a completed ap-
plication. 
‘‘(2) 
REVIEW
OF
APPLICATIONS
DURING 
EPIDEMICS AND PANDEMICS.—In the case of an epi-
demic 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 ap-
proval 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 sub-
mitted for review under the pathway under this sec-
tion is eligible for a special designation by the Sec-
retary 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. 
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
‘‘(b) ELIGIBILITY.—A drug may be eligible for provi-
sional approval under this section if the Secretary deter-
mines that the drug is intended for the treatment, preven-
tion, or medical diagnosis of— 
‘‘(1) a serious or life-threatening disease or con-
dition 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 pro-
visional 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 con-
sisting of adequate and well-controlled inves-
tigations, including clinical investigations, by 
experts qualified by scientific training and expe-
rience to evaluate the safety of the drug in-
volved, 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, rec-
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
ommended, or suggested in the labeling or pro-
posed 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 con-
sistent with or greater than currently mar-
keted on-label therapies, with equal or 
fewer side effects, if there are currently 
marketed on-label therapies. 
‘‘(2) PROTOCOLS.—The Secretary shall promul-
gate rules that establish the appropriate protocols 
for a sponsor of an application for provisional ap-
proval under this section and the Commissioner to 
follow to enable rolling, real-time, mid-trial submis-
sion 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 appli-
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
cation for provisional approval under this section, 
and to fulfill the follow-up requirements and support 
applications for full approval as described under sec-
tion 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 ap-
plication for provisional approval under this sec-
tion may use scientifically substantiated surro-
gates 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, patho-
physiologic, or other evidence; or 
‘‘(ii) an effect on a clinical endpoint 
other than survival or irreversible mor-
bidity of interest. 
‘‘(d) TRANSPARENCY
AND PATIENT MONITORING 
REQUIREMENTS.— 
‘‘(1) REGISTRIES.— 
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
‘‘(A) IN GENERAL.—The sponsor of a drug 
provisionally approved under this section shall 
require that all patients who use such drug par-
ticipate 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 ap-
proval 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 subpara-
graph (A) may be run by a third party, such as 
a government, for profit, or non-profit organiza-
tion, 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, unre-
stricted (or transparent) access for such 
patients to their patient data and related 
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
information regarding their usage of the 
provisionally approved drug; and 
‘‘(ii) approved researchers and med-
ical 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 appli-
cable— 
‘‘(A) by the sponsor of the drug provision-
ally approved under this section that is the sub-
ject of the registry; 
‘‘(B) by a third party, such as a govern-
ment, 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 applica-
tion 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. 
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
‘‘(B) ANNUAL REVIEW OF THE REGISTRY; 
PENALTIES.—The Secretary shall conduct an 
annual review of observational registries estab-
lished under this subsection. If, at such an an-
nual 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 no-
tification, 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 appli-
cation patient participation in an observational 
registry is not at or above 90 percent within 6 
months of issuance of such penalty, the provi-
sional 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— 
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
‘‘(A) the number of patients treated with 
each such drug, and the number of patients 
tracked in an observational registry with re-
spect to each such drug; 
‘‘(B) a discussion of the minimum amount 
of data required in the registries, including pa-
tient treatments and uses, length of use, side 
effects encountered, relevant biomarkers or sci-
entifically substantiated surrogates, scan re-
sults, 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 provi-
sional 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 with-
draw provisional approval under this section if there 
are a significant numbers of patients who experience 
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00012
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS
serious adverse effects, compared to the other cur-
rently marketed on-label therapies that are available 
for the applicable disease or condition. 
‘‘(2) EFFECT
OF
WITHDRAWAL.—If a provi-
sional 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 de-
termined by the Secretary. 
‘‘(f) TRANSPARENCY.—Any scientific, medical, aca-
demic, or health care journal publishing an article explain-
ing, 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 au-
thor’s institution (or, in the case of multiple authors, 
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00013
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS
researchers, or institutions, all such authors, re-
searchers, or institutions) received less than 30 per-
cent of funding for such research from the Depart-
ment 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 in-
formed consent form, acknowledging that such patient un-
derstands that the drug did not undergo the usual process 
for full approval of a drug by the Food and Drug Adminis-
tration, 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 re-
garding 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. 
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00014
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS
‘‘(2) LABELING.—The sponsor of the provision-
ally approved drug shall ensure that all labeling and 
promotional materials for the drug bear the state-
ment ‘provisionally approved by the FDA pending a 
full demonstration of effectiveness under application 
number llllll’ (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
AP-
PROVAL.—If the Secretary determines that the side 
effect profile of any drug included in such observa-
tional registries does not support the benefit pro-
vided 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 re-
scind such provisional approval. 
‘‘(i) DURATION
OF PROVISIONAL APPROVAL; RE-
QUIREMENT 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 
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00015
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS
for up to 3 subsequent 2-year periods by the Sec-
retary. Provisional approval status with respect to a 
drug shall not exceed a total of 6 years from the ini-
tial date the sponsor was awarded provisional ap-
proval 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 other-
wise made available pursuant to a grant of provisional ap-
proval under this section is unsafe or ineffective, no liabil-
ity in a cause of action shall lie against a sponsor or manu-
facturer, 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 provi-
sional 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. 
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00016
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS
‘‘(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 sub-
section (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 estab-
lishes a 15 percent improvement in an impor-
tant endpoint, including surrogate endpoints 
not validated by the Food and Drug Adminis-
tration, compared to a standard drug. 
‘‘(3) REAL-TIME EPIDEMIC AND PANDEMIC VAC-
CINE APPROVAL.— 
‘‘(A) IN GENERAL.—In the case of a vac-
cine developed in response to an epidemic or 
pandemic, including COVID–19, the Secretary 
shall share data information regarding the ap-
proval of the vaccine with the Advisory Com-
mittee on Immunization Practices of the Cen-
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00017
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS
ters 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 Com-
mittee on Immunization Practices of the Cen-
ters 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 Of-
fice 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 sec-
tion 351 of the Public Health Service Act. Such assistance 
shall include providing bi-informational communication 
about maintaining patient health, delivery of proper in-
formed consent, participating in clinical investigations, 
completing required documentation in order to participate 
in the applicable programs, and providing other informa-
tion.’’. 
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00018
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS
(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 indi-
vidual 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 sec-
tion 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 in-
surance issuer to cover any specific drug provisionally ap-
proved under such section 524B.’’. 
VerDate Sep 11 2014 
22:12 Jul 13, 2021
Jkt 019200
PO 00000
Frm 00019
Fmt 6652
Sfmt 6201
E:\BILLS\H3761.IH
H3761
pbinns on DSKJLVW7X2PROD with BILLS