3397 
† HR 2617 EAS2 
(C) in paragraph (3), by striking ‘‘or abbre-
viated application number’’ and inserting ‘‘, ab-
breviated application number, or biologics license 
application number’’; and 
(2) in subsection (b)— 
(A) in the matter preceding paragraph (1), 
by striking ‘‘The holder of an application ap-
proved under subsection (c) or (j)’’ and inserting 
‘‘The holder of an application approved under 
subsection (c) or (j) of section 505 of this Act or 
subsection (a) or (k) of section 351 of the Public 
Health Service Act’’; 
(B) in paragraph (1), by striking ‘‘estab-
lished name’’ and inserting ‘‘established name 
(or, in the case of a biological product, the prop-
er name)’’; and 
(C) in paragraph (2), by striking ‘‘or abbre-
viated application number’’ and inserting ‘‘, ab-
breviated application number, or biologics license 
application number’’. 
(b) ADDITIONAL ONE-TIME REPORT.—Subsection (c) 
of section 506I of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 356i) is amended to read as follows: 
‘‘(c) ADDITIONAL ONE-TIME REPORT.—Within 180 
days of the date of enactment of the Food and Drug Omni-
3398 
† HR 2617 EAS2 
bus Reform Act of 2022, all holders of applications ap-
proved under subsection (a) or (k) of section 351 of the Pub-
lic Health Service Act shall review the information in the 
list published under section 351(k)(9)(A) of the Public 
Health Service Act and shall submit a written notice to 
the Secretary— 
‘‘(1) stating that all of the application holder’s 
biological products in the list published under such 
section 351(k)(9)(A) that are not listed as discon-
tinued are available for sale; or 
‘‘(2) including the information required pursu-
ant to subsection (a) or (b), as applicable, for each of 
the application holder’s biological products that are 
in the list published under such section 351(k)(9)(A) 
and not listed as discontinued, but have been discon-
tinued from sale or never have been available for 
sale.’’. 
(c) PURPLE BOOK.—Section 506I of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 356i) is amended— 
(1) by striking subsection (d) and inserting the 
following: 
‘‘(d) FAILURE TO MEET REQUIREMENTS.—If a holder 
of an approved application fails to submit the information 
required under subsection (a), (b), or (c), the Secretary 
may—
3399 
† HR 2617 EAS2 
‘‘(1) move the application holder’s drugs from 
the active section of the list published under section 
505(j)(7)(A) to the discontinued section of the list, ex-
cept that the Secretary shall remove from the list in 
accordance with section 505(j)(7)(C) drugs the Sec-
retary determines have been withdrawn from sale for 
reasons of safety or effectiveness; and 
‘‘(2) identify the application holder’s biological 
products as discontinued in the list published under 
section 351(k)(9)(A) of the Public Health Service Act, 
except that the Secretary shall remove from the list in 
accordance with section 351(k)(9)(B) of such Act bio-
logical products for which the license has been revoked 
or suspended for reasons of safety, purity, or po-
tency.’’; and 
(2) in subsection (e)— 
(A) by inserting after the first sentence the 
following: ‘‘The Secretary shall update the list 
published under section 351(k)(9)(A) of the Pub-
lic Health Service Act based on information pro-
vided under subsections (a), (b), and (c) by iden-
tifying as discontinued biological products that 
are not available for sale, except that biological 
products for which the license has been revoked 
or suspended for safety, purity, or potency rea-
3400 
† HR 2617 EAS2 
sons shall be removed from the list in accordance 
with section 351(k)(9)(B) of the Public Health 
Service Act.’’; 
(B) by striking ‘‘monthly updates to the 
list’’ and inserting ‘‘monthly updates to the lists 
referred to in the preceding sentences’’; and 
(C) by striking ‘‘and shall update the list 
based on’’ and inserting ‘‘and shall update such 
lists based on’’. 
(d) TECHNICAL CORRECTIONS.—Section 506I(e) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356i(e)) 
is amended— 
(1) by striking ‘‘subsection 505(j)(7)(A)’’ and in-
serting ‘‘section 505(j)(7)(A)’’; and 
(2) by striking ‘‘subsection 505(j)(7)(C)’’ and in-
serting ‘‘section 505(j)(7)(C)’’. 
SEC. 3202. IMPROVING THE TREATMENT OF RARE DISEASES 
AND CONDITIONS. 
(a) REPORT ON ORPHAN DRUG PROGRAM.— 
(1) IN GENERAL.—Not later than September 30, 
2026, the Secretary shall submit to the Committee on 
Energy and Commerce of the House of Representa-
tives and the Committee on Health, Education, 
Labor, and Pensions of the Senate a report summa-
rizing the activities of the Food and Drug Adminis-
3401 
† HR 2617 EAS2 
tration, with respect to the period of fiscal years 2023 
through fiscal year 2025, related to designating drugs 
under section 526 of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 360bb) for a rare disease or 
condition and approving such drugs under section 
505 of such Act (21 U.S.C. 355) or licensing such 
drugs under section 351 of the Public Health Service 
Act (42 U.S.C. 262), including— 
(A) the number of applications for such 
drugs under section 505 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355) or sec-
tion 351 of the Public Health Service Act (42 
U.S.C. 262) received by the Food and Drug Ad-
ministration, the number of such applications 
accepted and rejected for filing, and the numbers 
of such applications pending, approved, and for 
which a complete response letter has been issued 
by the Food and Drug Administration; 
(B) the number of applications for which 
the sponsor requested written recommendations 
pursuant to section 525 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360aa) and 
the number of such applications for which the 
sponsor received such written recommendations;
3402 
† HR 2617 EAS2 
(C) a description of trends in drug approv-
als for rare diseases and conditions across review 
divisions at the Food and Drug Administration; 
(D) the extent to which the Food and Drug 
Administration is consulting with external ex-
perts pursuant to section 569(a)(2) of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb–8(a)(2)) on topics pertaining to drugs 
for a rare disease or condition, including how 
and when any such consultation is occurring; 
(E) the number of applications for which 
the Secretary allowed the sponsor to rely upon 
data and information pursuant to section 529A 
of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 360ff–1); and 
(F) a description of the Food and Drug Ad-
ministration’s efforts to promote best practices in 
the development of novel treatments for rare dis-
eases or conditions, including— 
(i) reviewer training on policies, meth-
ods, and tools related to rare diseases and 
conditions; and 
(ii) new regulatory science and coordi-
nated support for patient and stakeholder 
engagement.
3403 
† HR 2617 EAS2 
(2) PUBLIC AVAILABILITY.—The Secretary shall 
make the report under paragraph (1) available to the 
public, including by posting the report on the website 
of the Food and Drug Administration. 
(3) INFORMATION DISCLOSURE.—Nothing in this 
subsection shall be construed to authorize the disclo-
sure of information that is prohibited from disclosure 
under section 301(j) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 331(j)) or section 1905 of 
title 18, United States Code, or subject to withholding 
under paragraph (4) of section 552(b) of title 5, 
United States Code (commonly referred to as the 
‘‘Freedom of Information Act’’). 
(b) GUIDANCE.—Not later than 9 months after the date 
of enactment of this Act, the Secretary shall publish final 
guidance related to the draft guidance titled, ‘‘Rare Dis-
eases: Common Issues in Drug Development’’, issued on 
February 1, 2019. 
(c) STUDY ON EUROPEAN UNION SAFETY AND EFFI-
CACY REVIEWS OF DRUGS FOR RARE DISEASES AND CON-
DITIONS.— 
(1) IN GENERAL.—The Secretary shall enter into 
a contract with the National Academies of Sciences, 
Engineering, and Medicine (referred to in this section 
as the ‘‘National Academies’’) to conduct a study on
3404 
† HR 2617 EAS2 
processes for evaluating the safety and efficacy of 
drugs for rare diseases or conditions in the United 
States and the European Union, including— 
(A) flexibilities, authorities, or mechanisms 
available to regulators in the United States and 
the European Union specific to rare diseases or 
conditions; 
(B) the consideration and use of supple-
mental data submitted during review processes 
in the United States and the European Union, 
including data associated with open label exten-
sion studies and expanded access programs spe-
cific to rare diseases or conditions; 
(C) an assessment of collaborative efforts be-
tween United States and European Union regu-
lators related to— 
(i) 
product 
development 
programs 
under review; 
(ii) policies under development and 
those recently issued; and 
(iii) scientific information related to 
product development or regulation; and 
(D) recommendations for how Congress can 
support collaborative efforts described in sub-
paragraph (C).
3405 
† HR 2617 EAS2 
(2) CONSULTATION.—The contract under para-
graph (1) shall provide for consultation with relevant 
stakeholders, including— 
(A) representatives from the Food and Drug 
Administration and the European Medicines 
Agency; 
(B) patients with rare diseases or condi-
tions; and 
(C) patient groups that— 
(i) represent patients with rare dis-
eases or conditions; and 
(ii) have international patient out-
reach. 
(3) REPORT.—The contract under paragraph (1) 
shall provide for, not later than 2 years after the date 
of entering into such contract— 
(A) the completion of the study under para-
graph (1); and 
(B) the submission of a report on the results 
of such study to the Committee on Energy and 
Commerce of the House of Representatives and 
the Committee on Health, Education, Labor, and 
Pensions of the Senate. 
(4) PUBLIC AVAILABILITY.—The contract under 
paragraph (1) shall provide for the National Acad-
3406 
† HR 2617 EAS2 
emies to make the report under paragraph (3) avail-
able to the public, including by posting the report on 
the website of the National Academies. 
(d) PUBLIC MEETING.— 
(1) IN GENERAL.—Not later than December 31, 
2023, the Secretary, acting through the Commissioner 
of Food and Drugs, shall convene one or more public 
meetings to solicit input from stakeholders regarding 
the approaches described in paragraph (2). 
(2) APPROACHES.—The public meeting or meet-
ings under paragraph (1) shall address approaches to 
increasing and improving engagement with rare dis-
ease or condition patients, groups representing such 
patients, rare disease or condition experts, and ex-
perts on small population studies, in order to improve 
the understanding with respect to rare diseases or 
conditions of— 
(A) patient burden; 
(B) treatment options; and 
(C) side effects of treatments, including un-
derstanding the risks of side effects relative to the 
health status of the patient and the progression 
of the disease or condition. 
(3) PUBLIC DOCKET.—The Secretary shall estab-
lish a public docket to receive written comments re-
3407 
† HR 2617 EAS2 
lated to the approaches addressed during each public 
meeting under paragraph (1). Such public docket 
shall remain open for 60 days following the date of 
each such public meeting. 
(4) REPORTS.—Not later than 180 days after 
each public meeting under paragraph (1), the Com-
missioner of Food and Drugs shall develop and pub-
lish on the website of the Food and Drug Administra-
tion a report on— 
(A) the approaches discussed at the public 
meeting; and 
(B) any related recommendations. 
(e) CONSULTATION ON THE SCIENCE OF SMALL POPU-
LATION STUDIES.—Section 569(b) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360bbb–8(b)) is amend-
ed— 
(1) in paragraph (6), by striking ‘‘; and’’ and 
inserting a semicolon; 
(2) in paragraph (7), by striking the period and 
inserting ‘‘; and’’; and 
(3) by adding at the end the following: 
‘‘(8) the science of small population studies.’’. 
(f) GAO REPORT.— 
(1) IN
GENERAL.—Not later than 18 months 
after the date of enactment of this Act, the Comp-
3408 
† HR 2617 EAS2 
troller General of the United States shall submit to 
the Committee on Health, Education, Labor, and 
Pensions of the Senate and the Committee on Energy 
and Commerce of the House of Representatives, a re-
port assessing the policies, practices, and programs of 
the Food and Drug Administration with respect to 
the review of applications for approval of drugs under 
section 505 of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 355) and licensing of biological prod-
ucts under section 351 of the Public Health Service 
Act (42 U.S.C. 262) intended to treat rare diseases 
and conditions. 
(2) CONTENT
OF
REPORT.—The report under 
paragraph (1) shall— 
(A) describe the activities of the Food and 
Drug Administration dedicated to the develop-
ment and review of drugs and biological prod-
ucts intended to treat rare diseases and condi-
tions under section 505 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355) and 
section 351 of the Public Health Service Act (42 
U.S.C. 262); 
(B) describe challenges with developing and 
obtaining approval or licensure of drugs and bio-
logical products intended to treat rare diseases
3409 
† HR 2617 EAS2 
and conditions, such as challenges related to de-
signing and conducting clinical trials, clinical 
trial subject recruitment and enrollment, study 
endpoints, and ensuring data quality, assessing 
the benefit-risk profile of drugs and biological 
products intended to treat rare diseases and con-
ditions, and meeting requirements for approval 
or licensure; 
(C) assess the effectiveness of policies and 
practices of the Food and Drug Administration 
related to the review of applications for drugs 
and biological products intended to treat rare 
diseases and conditions, including— 
(i) initiatives to support the develop-
ment and review of drugs and biological 
products intended to treat rare diseases and 
conditions, including initiatives related to 
regulatory science, clinical trial design, sta-
tistical analysis, and other relevant topics; 
(ii) consideration of relevant patient- 
focused drug development data and infor-
mation, including patient experience data 
and the views of patients, pursuant to sec-
tion 569C of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 360bbb–8c);
3410 
† HR 2617 EAS2 
(iii) training and other efforts to en-
sure the expertise of personnel of the Food 
and Drug Administration regarding the re-
view of applications for drugs and biologi-
cal products intended to treat rare diseases 
and conditions; and 
(iv) consultations and engagement with 
stakeholders and external experts pursuant 
to section 569 of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 360bbb–8); 
(D) assess the extent to which the Food and 
Drug Administration is applying the policies 
and practices described in subparagraph (C) 
consistently across review divisions, and the fac-
tors that influence the extent to which such ap-
plication is consistent; and 
(E) include recommendations to address 
challenges and deficiencies identified, including 
recommendations to improve the effectiveness, 
consistency, and coordination of policies, prac-
tices, and programs of the Food and Drug Ad-
ministration related to the review of applications 
for drugs and biological products intended to 
treat rare diseases and conditions.
3411 
† HR 2617 EAS2 
(g) DEFINITION.—In this section, the terms ‘‘rare dis-
ease or condition’’, ‘‘rare diseases or conditions’’, and ‘‘rare 
diseases and conditions’’ have the meaning given the term 
‘‘rare disease or condition’’ in section 526(a)(2) of the Fed-
eral 
Food, 
Drug, 
and 
Cosmetic 
Act 
(21 
U.S.C. 
360bbb(a)(2)). 
SEC. 3203. EMERGING TECHNOLOGY PROGRAM. 
Chapter V of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 201 et seq.) is amended by inserting after 
section 566 of such Act (21 U.S.C. 360bbb–5) the following: 
‘‘SEC. 566A. EMERGING TECHNOLOGY PROGRAM. 
‘‘(a) PROGRAM ESTABLISHMENT.— 
‘‘(1) IN GENERAL.—The Secretary shall establish 
a program to support the adoption of, and improve 
the development of, innovative approaches to drug de-
sign and manufacturing. 
‘‘(2) ACTIONS.—In carrying out the program 
under paragraph (1), the Secretary may— 
‘‘(A) facilitate and increase communication 
between public and private entities, consortia, 
and individuals with respect to innovative drug 
product design and manufacturing; 
‘‘(B) solicit information regarding, and 
conduct or support research on, innovative ap-
3412 
† HR 2617 EAS2 
proaches to drug product design and manufac-
turing; 
‘‘(C) convene meetings with representatives 
of industry, academia, other Federal agencies, 
international agencies, and other interested per-
sons, as appropriate; 
‘‘(D) convene working groups to support 
drug product design and manufacturing research 
and development; 
‘‘(E) support education and training for 
regulatory staff and scientists related to innova-
tive approaches to drug product design and 
manufacturing; 
‘‘(F) advance regulatory science related to 
the development and review of innovative ap-
proaches to drug product design and manufac-
turing; 
‘‘(G) convene or participate in working 
groups to support the harmonization of inter-
national regulatory requirements related to inno-
vative approaches to drug product design and 
manufacturing; and 
‘‘(H) award grants or contracts to carry out 
or support the program under paragraph (1).
3413 
† HR 2617 EAS2 
‘‘(3) GRANTS AND CONTRACTS.—To seek a grant 
or contract under this section, an entity shall submit 
an application— 
‘‘(A) in such form and manner as the Sec-
retary may require; and 
‘‘(B) containing such information as the 
Secretary may require, including a description 
of— 
‘‘(i) how the entity will conduct the ac-
tivities to be supported through the grant or 
contract; and 
‘‘(ii) how such activities will further 
research and development related to, or 
adoption of, innovative approaches to drug 
product design and manufacturing. 
‘‘(b) GUIDANCE.—The Secretary shall— 
‘‘(1) issue or update guidance to help facilitate 
the adoption of, and advance the development of, in-
novative approaches to drug product design and man-
ufacturing; and 
‘‘(2) include in such guidance descriptions of— 
‘‘(A) any regulatory requirements related to 
the development or review of technologies related 
to innovative approaches to drug product design 
and manufacturing, including updates and im-
3414 
† HR 2617 EAS2 
provements to such technologies after product ap-
proval; and 
‘‘(B) data that can be used to demonstrate 
the identity, safety, purity, and potency of drugs 
manufactured using such technologies. 
‘‘(c) REPORT TO CONGRESS.—Not later than 4 years 
after the date of enactment of this section, the Secretary 
shall submit to the Committee on Energy and Commerce 
of the House of Representatives and the Committee on 
Health, Education, Labor, and Pensions of the Senate a 
report containing— 
‘‘(1) an annual accounting of the allocation of 
funds made available to carry out this section; 
‘‘(2) a description of how Food and Drug Ad-
ministration staff were utilized to carry out this sec-
tion and, as applicable, any challenges or limitations 
related to staffing; 
‘‘(3) the number of public meetings held or par-
ticipated in by the Food and Drug Administration 
pursuant to this section, including meetings convened 
as part of a working group described in subparagraph 
(D) or (G) of subsection (a)(2), and the topics of each 
such meeting; and 
‘‘(4) the number of drug products approved or li-
censed, after the date of enactment of this section,
3415 
† HR 2617 EAS2 
using an innovative approach to drug product design 
and manufacturing.’’. 
SEC. 3204. NATIONAL CENTERS OF EXCELLENCE IN AD-
VANCED 
AND 
CONTINUOUS 
PHARMA-
CEUTICAL MANUFACTURING. 
(a) IN GENERAL.—Section 3016 of the 21st Century 
Cures Act (21 U.S.C. 399h) is amended to read as follows: 
‘‘SEC. 3016. NATIONAL CENTERS OF EXCELLENCE IN AD-
VANCED 
AND 
CONTINUOUS 
PHARMA-
CEUTICAL MANUFACTURING. 
‘‘(a) IN GENERAL.—The Secretary of Health and 
Human Services, acting through the Commissioner of Food 
and Drugs— 
‘‘(1) may, to support the advancement, develop-
ment, and implementation of advanced and contin-
uous pharmaceutical manufacturing— 
‘‘(A) solicit requests for designation as Na-
tional Centers of Excellence in Advanced and 
Continuous Pharmaceutical Manufacturing (in 
this section referred to as a ‘National Center of 
Excellence’); 
‘‘(B) beginning not later than one year after 
the date of enactment of the Food and Drug Om-
nibus Reform Act of 2022, designate as National 
Centers of Excellence institutions of higher edu-
3416 
† HR 2617 EAS2 
cation or consortia of institutions of higher edu-
cation that— 
‘‘(i) request such designation; and 
‘‘(ii) meet the eligibility criteria speci-
fied in subsection (c); and 
‘‘(C) award grants to such institutions or 
consortia of institutions; and 
‘‘(2) shall so designate not more than 5 institu-
tions of higher education or consortia of such institu-
tions. 
‘‘(b) REQUEST FOR DESIGNATION.—A request for des-
ignation under subsection (a) shall be made to the Secretary 
at such time, in such manner, and containing such infor-
mation as the Secretary may require. 
‘‘(c) ELIGIBILITY CRITERIA FOR DESIGNATION.—To be 
eligible to receive a designation under this section, an insti-
tution of higher education or consortium of institutions of 
higher education shall include in its request for designation 
a description of the institution’s or consortium’s— 
‘‘(1) physical capacity and technical capabilities 
to conduct advanced research on, and to develop and 
implement, advanced and continuous pharmaceutical 
manufacturing; 
‘‘(2) collaboration or partnerships with other in-
stitutions of higher education, nonprofit organiza-
3417 
† HR 2617 EAS2 
tions, and large and small pharmaceutical manufac-
turers, including generic and nonprescription manu-
facturers, contract manufacturers, and other relevant 
entities; 
‘‘(3) proven capacity to design, develop, imple-
ment, and demonstrate new, highly effective tech-
nologies for use in advanced and continuous pharma-
ceutical manufacturing; 
‘‘(4) proven ability to facilitate training of a 
qualified workforce for advanced research on, and de-
velopment and implementation of, advanced and con-
tinuous pharmaceutical manufacturing; and 
‘‘(5)(A) experience in participating in and lead-
ing advanced and continuous pharmaceutical manu-
facturing technology partnerships with other institu-
tions of higher education, nonprofit organizations, 
and large and small pharmaceutical manufacturers, 
including generic and nonprescription manufacturers, 
contract manufacturers, and other relevant entities 
to— 
‘‘(i) support the implementation of ad-
vanced or continuous pharmaceutical manufac-
turing for companies manufacturing or seeking 
to manufacture in the United States;
3418 
† HR 2617 EAS2 
‘‘(ii) support Federal agencies with tech-
nical assistance and workforce training, which 
may include regulatory and quality metric guid-
ance as applicable, and hands-on training, for 
advanced and continuous pharmaceutical manu-
facturing; 
‘‘(iii) organize and conduct advanced re-
search and development activities, with respect to 
advanced or continuous pharmaceutical manu-
facturing, needed to develop new and more effec-
tive technology, and to develop and support tech-
nological leadership; 
‘‘(iv) develop best practices for designing, 
developing, and implementing advanced and 
continuous pharmaceutical manufacturing proc-
esses; and 
‘‘(v) identify and assess workforce needs for 
advanced and continuous pharmaceutical manu-
facturing, and address such workforce needs, 
which may include the development and imple-
menting of training programs; or 
‘‘(B) a plan, to be implemented within 2 years, 
to establish partnerships described in subparagraph 
(A).
3419 
† HR 2617 EAS2 
‘‘(d) TERMINATION OF DESIGNATION.—The Secretary 
may terminate the designation of any National Center of 
Excellence designated under this section if the Secretary de-
termines such National Center of Excellence no longer meets 
the criteria specified in subsection (c). Not later than 90 
days before the effective date of such a termination, the Sec-
retary shall provide written notice to the National Center 
of Excellence, including the rationale for such termination. 
‘‘(e) CONDITIONS FOR DESIGNATION.—As a condition 
of designation as a National Center of Excellence under this 
section, the Secretary shall require that an institution of 
higher education or consortium of institutions of higher 
education enter into an agreement with the Secretary under 
which the institution or consortium agrees— 
‘‘(1) to collaborate directly with the Food and 
Drug Administration to publish the reports required 
by subsection (g); 
‘‘(2) to share data with the Food and Drug Ad-
ministration regarding best practices and research 
generated through the funding under subsection (f); 
‘‘(3) to develop, along with industry partners 
(which may include large and small pharmaceutical 
manufacturers, including generic and nonprescription 
manufacturers, and contract research organizations 
or contract manufacturers that carry out drug devel-
3420 
† HR 2617 EAS2 
opment and manufacturing activities) and another 
institution or consortium designated under this sec-
tion, if any, a strategic plan for developing an ad-
vanced and continuous pharmaceutical manufac-
turing workforce; 
‘‘(4) to develop, along with industry partners 
and other institutions or consortia of such institu-
tions designated under this section, a strategic plan 
for strengthening existing, and developing new, part-
nerships with other institutions of higher education or 
consortia thereof, or nonprofit organizations; and 
‘‘(5) to provide an annual report to the Food 
and Drug Administration regarding the designee’s ac-
tivities under this section, including a description of 
how the designee continues to meet and make progress 
on the criteria specified in subsection (c). 
‘‘(f) FUNDING.— 
‘‘(1) IN GENERAL.—The Secretary shall award 
funding, through grants, contracts, or cooperative 
agreements, to the entities designated as National 
Centers of Excellence under this section for the pur-
poses of supporting the advanced research on, and de-
velopment and implementation of, advanced and con-
tinuous pharmaceutical manufacturing, and recom-
3421 
† HR 2617 EAS2 
mending improvements to advanced and continuous 
pharmaceutical manufacturing, including— 
‘‘(A) expanding capacity for advanced re-
search on, and development of, advanced and 
continuous pharmaceutical manufacturing; and 
‘‘(B) implementing advanced research ca-
pacity and capabilities in advanced and contin-
uous pharmaceutical manufacturing suitable for 
accelerating the development of drug products 
needed to respond to public health threats, miti-
gate or prevent drug shortages, address drug 
quality issues and supply chain disruptions, and 
other circumstances with respect to which the 
Secretary may determine the rapid development 
of new products or new manufacturing processes 
may be appropriate. 
‘‘(2) CONSISTENCY
WITH
FDA
MISSION.—As a 
condition on receipt of funding under this subsection, 
a National Center of Excellence shall consider any 
input from the Secretary regarding the use of funding 
related to— 
‘‘(A) best practices to increase, and provide 
for the advancement of, advanced and continuous 
pharmaceutical manufacturing through the Na-
tional Center of Excellence; and
3422 
† HR 2617 EAS2 
‘‘(B) the extent to which activities con-
ducted by the National Center of Excellence are 
consistent with the mission of the Food and 
Drug Administration. 
‘‘(3) RULE OF CONSTRUCTION.—Nothing in this 
section shall be construed as precluding a National 
Center for Excellence designated under this section 
from receiving funds under any other provision of 
this Act or any other Federal law. 
‘‘(g) ANNUAL REVIEW AND REPORTS.— 
‘‘(1) ANNUAL REPORT TO CONGRESS.—Beginning 
not later than one year after the date on which the 
first designation is made under subsection (a), and 
annually thereafter, the Secretary shall— 
‘‘(A) submit to Congress a report describing 
the activities, partnerships and collaborations, 
Federal policy recommendations, previous and 
continuing funding, and findings of, and any 
other applicable information from, the National 
Centers of Excellence designated under this sec-
tion; 
‘‘(B) include in such report an accounting 
of the Federal administrative expenses described 
in subsection (i)(2) over the reporting period; 
and
3423 
† HR 2617 EAS2 
‘‘(C) make such report available to the pub-
lic in an easily accessible electronic format on 
the website of the Food and Drug Administra-
tion. 
‘‘(2) CENTER OF EXCELLENCE REPORT.—An en-
tity receiving a grant under this section shall, not 
later than 1 year after receiving such grant, and an-
nually thereafter for the duration of the grant period, 
submit to the Secretary a summary of programs and 
activities funded under the grant. 
‘‘(3) PERIODIC REVIEW.—The Secretary shall pe-
riodically review the National Centers of Excellence 
designated under this section to ensure that such Na-
tional Centers of Excellence continue to meet the cri-
teria for designation under this section. 
‘‘(4) ADDITIONAL REPORT TO CONGRESS.—Not 
later than 1 year after the date on which the first des-
ignation is made under subsection (a), the Secretary, 
in consultation with the National Centers of Excel-
lence designated under this section, shall submit a re-
port to the Congress on the role of the Food and Drug 
Administration in supporting advanced and contin-
uous pharmaceutical manufacturing, including—
3424 
† HR 2617 EAS2 
‘‘(A) a national framework of principles re-
lated to the implementation of advanced and 
continuous pharmaceutical manufacturing; 
‘‘(B) a plan for the development of Federal 
regulations and guidance to support and facili-
tate the incorporation of advanced or continuous 
manufacturing into the development of pharma-
ceuticals; 
‘‘(C) a plan for development of Federal reg-
ulations or guidance related to the review of ad-
vanced and continuous pharmaceutical manufac-
turing, including how such manufacturing prac-
tices may be incorporated into the review of 
medical product applications; and 
‘‘(D) a summary of relevant feedback re-
lated to improving advanced and continuous 
pharmaceutical manufacturing solicited from the 
public, which may include other institutions of 
higher education, nonprofit organizations, and 
large and small pharmaceutical manufacturers, 
including generic and nonprescription manufac-
turers, and contract manufacturers, and other 
relevant entities. 
‘‘(h) DEFINITIONS.—In this section:
3425 
† HR 2617 EAS2 
‘‘(1) ADVANCED
AND
CONTINUOUS
PHARMA-
CEUTICAL
MANUFACTURING.—The term ‘advanced 
and continuous pharmaceutical manufacturing’ refers 
to a method of pharmaceutical manufacturing, or a 
combination of pharmaceutical manufacturing meth-
ods— 
‘‘(A) that incorporates a novel technology, 
or uses an established technique or technology in 
a new or innovative way, that enhances drug 
quality or improves the manufacturing process 
for a drug, including processes that may apply 
to advanced therapies and the production of bio-
logical products, such as cell and gene therapies; 
or 
‘‘(B) for which the input materials are con-
tinuously fed into and transformed within the 
process, and the output materials are continu-
ously removed from the system, utilizing an inte-
grated manufacturing process that consists of a 
series of 2 or more simultaneous unit operations. 
‘‘(2) BIOLOGICAL PRODUCT.—The term ‘biologi-
cal product’ has the meaning given such term in sec-
tion 351(i) of the Public Health Service Act (42 
U.S.C. 262(i)).
3426 
† HR 2617 EAS2 
‘‘(3) DRUG.—The term ‘drug’ has the meaning 
given such term in section 201(g) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 321(g)). 
‘‘(4) INSTITUTION OF HIGHER EDUCATION.—The 
term ‘institution of higher education’ has the meaning 
given such term in section 101(a) of the Higher Edu-
cation Act of 1965 (20 U.S.C. 1001(a)). 
‘‘(5) SECRETARY.—The term ‘Secretary’ means 
the Secretary of Health and Human Services. 
‘‘(i) AUTHORIZATION OF APPROPRIATIONS.— 
‘‘(1) IN GENERAL.—There is authorized to be ap-
propriated to carry out this section $100,000,000 for 
the period of fiscal years 2023 through 2027. 
‘‘(2) FEDERAL ADMINISTRATIVE EXPENSES.—Of 
the amounts made available to carry out this section 
for a fiscal year, the Secretary shall not use more 
than 8 percent for Federal administrative expenses, 
including training, technical assistance, reporting, 
and evaluation.’’. 
(b) TRANSITION RULE.—Section 3016 of the 21st Cen-
tury Cures Act (21 U.S.C. 399h), as in effect on the day 
before the date of the enactment of this section, shall apply 
with respect to grants awarded under such section before 
such date of enactment.
3427 
† HR 2617 EAS2 
(c) CLERICAL AMENDMENT.—The item relating to sec-
tion 3016 in the table of contents in section 1(b) of the 21st 
Century Cures Act (Public Law 114–255) is amended to 
read as follows: 
‘‘Sec. 3016. National Centers of Excellence in Advanced and Continuous Pharma-
ceutical Manufacturing.’’. 
SEC. 3205. PUBLIC WORKSHOP ON CELL THERAPIES. 
Not later than 3 years after the date of the enactment 
of this Act, the Secretary, acting through the Commissioner 
of Food and Drugs, shall convene a public workshop with 
relevant stakeholders to discuss best practices on generating 
scientific data necessary to further facilitate the develop-
ment of certain human cell-, tissue-, and cellular-based 
medical products (and the latest scientific information 
about such products) that are regulated as drugs under the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et 
seq.) and biological products under section 351 of the Public 
Health Service Act (42 U.S.C. 262), namely, stem cell and 
other cellular therapies. 
SEC. 3206. CLARIFICATIONS TO EXCLUSIVITY PROVISIONS 
FOR FIRST INTERCHANGEABLE BIOSIMILAR 
BIOLOGICAL PRODUCTS. 
Section 351(k)(6) of the Public Health Service Act (42 
U.S.C. 262(k)(6)) is amended— 
(1) in the matter preceding subparagraph (A)—
3428 
† HR 2617 EAS2 
(A) by striking ‘‘Upon review of’’ and in-
serting ‘‘The Secretary shall not make approval 
as an interchangeable biological product effective 
with respect to’’; 
(B) by striking ‘‘relying on’’ and inserting 
‘‘that relies on’’; and 
(C) by striking ‘‘the Secretary shall not 
make a determination under paragraph (4) that 
the second or subsequent biological product is 
interchangeable for any condition of use’’; and 
(2) in the flush text that follows subparagraph 
(C)(ii), by striking ‘‘taken.’’ and inserting ‘‘taken, 
and the term ‘first interchangeable biosimilar biologi-
cal product’ means any interchangeable biosimilar bi-
ological product that is approved on the first day on 
which such a product is approved as interchangeable 
with the reference product.’’. 
SEC. 3207. GAO REPORT ON NONPROFIT PHARMACEUTICAL 
ORGANIZATIONS. 
(a) GAO REVIEW.—The Comptroller General of the 
United States (referred to in this section as the ‘‘Comp-
troller General’’) shall prepare a report on— 
(1) what is known about nonprofit pharma-
ceutical manufacturing organizations, including the 
impact of such organizations on the development,
3429 
† HR 2617 EAS2 
availability, and cost of prescription drugs in the 
United States, which may include information with 
respect to the capacity and capability to help prevent 
or mitigate shortages of such drugs, and any chal-
lenges to manufacturing or other operations; and 
(2) recommendations to address such challenges. 
(b) REPORT.—Not later than 2 years after the date 
of enactment of this Act, the Comptroller General shall sub-
mit the report described in subsection (a) to the Committee 
on Health, Education, Labor, and Pensions of the Senate 
and the Committee on Energy and Commerce of the House 
of Representatives. 
SEC. 3208. RARE DISEASE ENDPOINT ADVANCEMENT PILOT 
PROGRAM. 
(a) IN GENERAL.—The Secretary shall establish a 
pilot program under which the Secretary establishes proce-
dures to provide increased interaction with sponsors of rare 
disease drug development programs for purposes of advanc-
ing the development of efficacy endpoints, including surro-
gate and intermediate endpoints, for drugs intended to treat 
rare diseases, including through— 
(1) determining eligibility of participants for 
such program; and 
(2) developing and implementing a process for 
applying to, and participating in, such a program.
3430 
† HR 2617 EAS2 
(b) PUBLIC WORKSHOPS.—The Secretary shall con-
duct up to 3 public workshops, which shall be completed 
not later than September 30, 2026, to discuss topics relevant 
to the development of endpoints for rare diseases, which 
may include discussions about— 
(1) novel endpoints developed through the pilot 
program established under this section; and 
(2) as appropriate, the use of real world evidence 
and real world data to support the validation of effi-
cacy endpoints, including surrogate and intermediate 
endpoints, for rare diseases. 
(c) REPORTS.— 
(1) INTERIM REPORT.—Not later than September 
30, 2026, the Secretary shall submit to the Committee 
on Health, Education, Labor, and Pensions of the 
Senate and the Committee on Energy and Commerce 
of the House of Representatives a report describing 
the completed and ongoing activities in the pilot pro-
gram established under this section and public work-
shops described in subsection (b). 
(2) FINAL REPORT.—Not later than September 
30, 2027, the Secretary shall submit to the Committee 
on Health, Education, Labor, and Pensions of the 
Senate and the Committee on Energy and Commerce 
of the House of Representatives a report describing
3431 
† HR 2617 EAS2 
the outcomes of the pilot program established under 
this section. 
(d) GUIDANCE.—Not later than September 30, 2027, 
the Secretary shall issue guidance describing best practices 
and strategies for development of efficacy endpoints, includ-
ing surrogate and intermediate endpoints, for rare diseases. 
(e) SUNSET.—The Secretary may not accept any new 
application or request to participate in the program estab-
lished by this section on or after October 1, 2027. 
SEC. 3209. ANIMAL TESTING ALTERNATIVES. 
(a) IN GENERAL.—Section 505 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355) is amended— 
(1) in subsection (i)— 
(A) in paragraph (1)(A), by striking ‘‘pre-
clinical tests (including tests on animals)’’ and 
inserting ‘‘nonclinical tests’’; and 
(B) in paragraph (2)(B), by striking ‘‘ani-
mal’’ and inserting ‘‘nonclinical tests’’; and 
(2) by inserting after subsection (y) the fol-
lowing: 
‘‘(z) NONCLINICAL TEST DEFINED.—For purposes of 
this section, the term ‘nonclinical test’ means a test con-
ducted in vitro, in silico, or in chemico, or a nonhuman 
in vivo test, that occurs before or during the clinical trial
3432 
† HR 2617 EAS2 
phase of the investigation of the safety and effectiveness of 
a drug. Such test may include the following: 
‘‘(1) Cell-based assays. 
‘‘(2) Organ chips and microphysiological sys-
tems. 
‘‘(3) Computer modeling. 
‘‘(4) Other nonhuman or human biology-based 
test methods, such as bioprinting. 
‘‘(5) Animal tests.’’. 
(b) BIOSIMILAR
BIOLOGICAL
PRODUCT
APPLICA-
TIONS.—Item (bb) of section 351(k)(2)(A)(i)(I) of the Public 
Health Service Act (42 U.S.C. 262(k)(2)(A)(i)(I)) is amend-
ed to read as follows: 
‘‘(bb) an assessment of tox-
icity (which may rely on, or con-
sist of, a study or studies de-
scribed in item (aa) or (cc)); 
and’’. 
SEC. 3210. MODERNIZING ACCELERATED APPROVAL. 
(a) IN GENERAL.—Section 506(c) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 356(c)) is amended— 
(1) in paragraph (2)— 
(A) by redesignating subparagraphs (A) 
and (B) as clauses (i) and (ii), respectively, and 
adjusting the margins accordingly;
3433 
† HR 2617 EAS2 
(B) by striking ‘‘Approval of a product’’ 
and inserting the following: 
‘‘(A) IN
GENERAL.—Approval of a prod-
uct’’; 
(C) in clause (i) of such subparagraph (A), 
as so redesignated, by striking ‘‘appropriate 
postapproval studies’’ and inserting ‘‘an appro-
priate postapproval study or studies’’; and 
(D) by adding at the end the following: 
‘‘(B) STUDIES NOT REQUIRED.—If the Sec-
retary does not require that the sponsor of a 
product approved under accelerated approval 
conduct a postapproval study under this para-
graph, the Secretary shall publish on the website 
of the Food and Drug Administration the ration-
ale for why such study is not appropriate or nec-
essary. 
‘‘(C) POSTAPPROVAL STUDY CONDITIONS.— 
Not later than the date of approval of a product 
under accelerated approval, the Secretary shall 
specify the conditions for a postapproval study 
or studies required to be conducted under this 
paragraph with respect to such product, which 
may include enrollment targets, the study pro-
3434 
† HR 2617 EAS2 
tocol, and milestones, including the target date of 
study completion. 
‘‘(D) STUDIES BEGUN BEFORE APPROVAL.— 
The Secretary may require, as appropriate, a 
study or studies to be underway prior to ap-
proval, or within a specified time period after 
the date of approval, of the applicable product.’’; 
and 
(2) in paragraph (3)— 
(A) in the matter preceding subparagraph 
(A), by striking ‘‘(as prescribed by the Secretary 
in regulations which shall include an oppor-
tunity for an informal hearing)’’ and inserting 
‘‘described in subparagraph (B)’’; 
(B) by redesignating subparagraphs (A) 
through (D) as clauses (i) through (iv), respec-
tively and adjusting the margins accordingly; 
(C) by striking ‘‘The Secretary may’’ and 
inserting the following: 
‘‘(A) IN GENERAL.—The Secretary may’’; 
(D) in clause (i) of such subparagraph (A), 
as so redesignated, by striking ‘‘drug with due 
diligence’’ and inserting ‘‘product with due dili-
gence, including with respect to conditions speci-
fied by the Secretary under paragraph (2)(C)’’;
3435 
† HR 2617 EAS2 
(E) in clause (iii) of such subparagraph 
(A), as so redesignated, by inserting ‘‘shown to 
be’’ after ‘‘product is not’’; and 
(F) by adding at the end the following: 
‘‘(B) 
EXPEDITED
PROCEDURES
DE-
SCRIBED.—Expedited procedures described in 
this subparagraph shall consist of, prior to the 
withdrawal of accelerated approval— 
‘‘(i) providing the sponsor with— 
‘‘(I) due notice; 
‘‘(II) an explanation for the pro-
posed withdrawal; 
‘‘(III) an opportunity for a meet-
ing with the Commissioner or the Com-
missioner’s designee; and 
‘‘(IV) an opportunity for written 
appeal to— 
‘‘(aa) the Commissioner; or 
‘‘(bb) a designee of the Com-
missioner who has not partici-
pated in the proposed withdrawal 
of approval (other than a meeting 
pursuant to subclause (III)) and 
is not subordinate of an indi-
vidual (other than the Commis-
3436 
† HR 2617 EAS2 
sioner) who participated in such 
proposed withdrawal; 
‘‘(ii) providing an opportunity for 
public comment on the proposal to with-
draw approval; 
‘‘(iii) the publication of a summary of 
the public comments received, and the Sec-
retary’s response to such comments, on the 
website of the Food and Drug Administra-
tion; and 
‘‘(iv) convening and consulting an ad-
visory committee on issues related to the 
proposed withdrawal, if requested by the 
sponsor and if no such advisory committee 
has previously advised the Secretary on 
such issues with respect to the withdrawal 
of the product prior to the sponsor’s re-
quest.’’. 
(b) REPORTS OF POSTMARKETING STUDIES.—Section 
506B(a) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 356b(a)) is amended— 
(1) by redesignating paragraph (2) as para-
graph (3); and 
(2) by inserting after paragraph (1) the fol-
lowing:
3437 
† HR 2617 EAS2 
‘‘(2) 
ACCELERATED
APPROVAL.—Notwith-
standing paragraph (1), a sponsor of a drug ap-
proved pursuant to accelerated approval shall submit 
to the Secretary a report of the progress of any study 
required under section 506(c), including progress to-
ward enrollment targets, milestones, and other infor-
mation as required by the Secretary, not later than 
180 days after the approval of such drug and not less 
frequently than every 180 days thereafter, until the 
study is completed or terminated. The Secretary shall 
promptly publish on the website of the Food and 
Drug Administration, in an easily searchable format, 
the information reported under this paragraph.’’. 
(c) ENFORCEMENT.—Section 301 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 331), as amended by 
title II, is further amended by adding at the end the fol-
lowing: 
‘‘(ggg) The failure of a sponsor of a product approved 
under accelerated approval pursuant to section 506(c)— 
‘‘(1) to conduct with due diligence any post-
approval study required under section 506(c) with re-
spect to such product; or 
‘‘(2) to submit timely reports with respect to 
such product in accordance with section 506B(a)(2).’’. 
(d) GUIDANCE.—
3438 
† HR 2617 EAS2 
(1) IN
GENERAL.—The Secretary shall issue 
guidance describing— 
(A) how sponsor questions related to the 
identification of novel surrogate or intermediate 
clinical endpoints may be addressed in early- 
stage development meetings with the Food and 
Drug Administration; 
(B) the use of novel clinical trial designs 
that may be used to conduct appropriate post-
approval studies as may be required under sec-
tion 506(c)(2)(A) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 356(c)(2)(A)), as 
amended by subsection (a); 
(C) the expedited procedures described in 
section 506(c)(3)(B) of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 356(c)(3)(B)); and 
(D) considerations related to the use of sur-
rogate or intermediate clinical endpoints that 
may support the accelerated approval of an ap-
plication under 506(c)(1)(A) of such Act (21 
U.S.C. 356(c)(1)(A)), including considerations in 
evaluating the evidence related to any such 
endpoints. 
(2) FINAL
GUIDANCE.—The Secretary shall 
issue—
3439 
† HR 2617 EAS2 
(A) draft guidance under paragraph (1) not 
later than 18 months after the date of enactment 
of this Act; and 
(B) final guidance not later than 1 year 
after the close of the public comment period on 
such draft guidance. 
(e) ACCELERATED APPROVAL COUNCIL.— 
(1) IN GENERAL.—Not later than 1 year after 
the date of enactment of this Act, the Secretary shall 
establish an intra-agency coordinating council (re-
ferred to in this subsection as the ‘‘Council’’) within 
the Food and Drug Administration to ensure the con-
sistent and appropriate use of accelerated approval 
across the Food and Drug Administration, pursuant 
to section 506(c) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 356(c)). 
(2) MEMBERSHIP.—The members of the Council 
shall consist of the following senior officials, or a des-
ignee of such official, from the Food and Drug Ad-
ministration and relevant Centers: 
(A) The Director of the Center for Drug 
Evaluation and Research. 
(B) The Director of the Center for Biologics 
Evaluation and Research.
3440 
† HR 2617 EAS2 
(C) The Director of the Oncology Center of 
Excellence. 
(D) The Director of the Office of New 
Drugs. 
(E) The Director of the Office of Orphan 
Products Development. 
(F) The Director of the Office of Tissues 
and Advanced Therapies. 
(G) The Director of the Office of Medical 
Policy. 
(H) At least 3 directors of review divisions 
or offices overseeing products approved under ac-
celerated approval, including at least one direc-
tor within the Office of Neuroscience. 
(3) DUTIES OF THE COUNCIL.— 
(A) MEETINGS.—The Council shall convene 
not fewer than 3 times per calendar year to dis-
cuss issues related to accelerated approval, in-
cluding any relevant cross-disciplinary ap-
proaches related to product review with respect 
to accelerated approval. 
(B) POLICY
DEVELOPMENT.—The Council 
shall directly engage with product review teams 
to support the consistent and appropriate use of 
accelerated approval across the Food and Drug