I 
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 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’’. 
VerDate Sep 11 2014 
03:53 Aug 02, 2023
Jkt 039200
PO 00000
Frm 00001
Fmt 6652
Sfmt 6201
E:\BILLS\H4408.IH
H4408
pbinns on DSKJLVW7X2PROD with $$_JOB
SEC. 2. PROVISIONAL APPROVAL OF NEW HUMAN DRUGS. 
(a) IN GENERAL.—Subchapter A of chapter V of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351 
et seq.) is amended by adding at the end of the following: 
‘‘SEC. 524C. PROVISIONAL APPROVAL OF NEW HUMAN 
DRUGS. 
‘‘(a) PRIORITY REVIEW AND EVALUATION OF APPLI-
CATIONS.— 
‘‘(1) IN GENERAL.—The Secretary shall estab-
lish a priority review system to evaluate applications 
submitted under this section for provisional approval 
within 90 days of receipt of a completed application. 
‘‘(2) OTHER
DESIGNATIONS.—If a drug sub-
mitted for review under this section is eligible for a 
special designation by the Secretary under this Act, 
including as a drug for a rare disease or condition 
under section 526, all benefits of such other designa-
tion shall be available for use under provisional ap-
proval, including any tax credits and waiving of fees 
under chapter VII. 
‘‘(b) ELIGIBILITY.—A drug may be eligible for provi-
sional approval under this section if the Secretary deter-
mines that the drug is intended for the treatment, preven-
tion, or medical diagnosis of a serious or life-threatening 
disease or condition for which there is a reasonable likeli-
hood that premature death will occur without early med-
VerDate Sep 11 2014 
03:53 Aug 02, 2023
Jkt 039200
PO 00000
Frm 00002
Fmt 6652
Sfmt 6201
E:\BILLS\H4408.IH
H4408
pbinns on DSKJLVW7X2PROD with $$_JOB
ical intervention for an individual contracting or being di-
agnosed with such disease or condition. 
‘‘(c) STANDARD OF REVIEW FOR PROVISIONAL AP-
PROVAL.— 
‘‘(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-
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 
VerDate Sep 11 2014 
03:53 Aug 02, 2023
Jkt 039200
PO 00000
Frm 00003
Fmt 6652
Sfmt 6201
E:\BILLS\H4408.IH
H4408
pbinns on DSKJLVW7X2PROD with $$_JOB
‘‘(ii) the outcome of the drug is con-
sistent with or greater than currently mar-
keted on-label therapies, with equal or 
fewer side effects, if there are currently 
marketed on-label therapies. 
‘‘(2) PROTOCOLS.—The Secretary shall promul-
gate rules that establish the appropriate protocols to 
enable rolling, real-time, mid-trial submission while 
preserving the integrity of the ongoing trial and 
without penalizing the sponsor for seeking provi-
sional approval under this section. 
‘‘(3) REAL WORLD EVIDENCE.—The Secretary 
shall allow the use of real world evidence (as defined 
in section 505F(b)), including real world data used 
to generate real world evidence, to support an appli-
cation for provisional approval under this section, 
and to fulfill the follow-up requirements and support 
applications for approval under section 505 or sec-
tion 351 of the Public Health Service Act, as appli-
cable. 
‘‘(4) USE OF SCIENTIFICALLY SUBSTANTIATED 
SURROGATES.— 
‘‘(A) IN GENERAL.—The sponsor of an ap-
plication for provisional approval under this sec-
VerDate Sep 11 2014 
03:53 Aug 02, 2023
Jkt 039200
PO 00000
Frm 00004
Fmt 6652
Sfmt 6201
E:\BILLS\H4408.IH
H4408
pbinns on DSKJLVW7X2PROD with $$_JOB
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.— 
‘‘(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 approval 
under section 505 or section 351 of the Public 
Health Service Act, or the provisional approval 
is rescinded. 
VerDate Sep 11 2014 
03:53 Aug 02, 2023
Jkt 039200
PO 00000
Frm 00005
Fmt 6652
Sfmt 6201
E:\BILLS\H4408.IH
H4408
pbinns on DSKJLVW7X2PROD with $$_JOB
‘‘(B) REQUIREMENTS FOR REGISTRIES.— 
An observational registry described in subpara-
graph (A) may be run by a third party, such as 
a government, for profit, or nonprofit organiza-
tion, and shall track all patients who use the 
provisionally approved drug. 
‘‘(C) ACCESSIBILITY.—An observational 
registry described in subparagraph (A) shall be 
easily accessible for— 
‘‘(i) all patients who are participating 
in any registry related to a provisionally 
approved drug that allows for easy, unre-
stricted (or transparent) access for such 
patients to their patient data and related 
information regarding their usage of the 
provisionally approved drug; and 
‘‘(ii) approved researchers and med-
ical professionals who may access data 
maintained in the registry, which access 
shall be for public health research and only 
in a de-identified, aggregated manner. 
‘‘(2) 
FUNDING.—An 
observational 
registry 
under this subsection shall be maintained, as appli-
cable— 
VerDate Sep 11 2014 
03:53 Aug 02, 2023
Jkt 039200
PO 00000
Frm 00006
Fmt 6652
Sfmt 6201
E:\BILLS\H4408.IH
H4408
pbinns on DSKJLVW7X2PROD with $$_JOB