II 

117TH CONGRESS 

2D SESSION S. 4446 

To modernize the process of accelerated approval of a drug for a serious 

or life-threatening disease or condition. 

IN THE SENATE OF THE UNITED STATES 

JUNE 22, 2022 

Ms. COLLINS (for herself and Mr. KAINE) introduced the following bill; which 

was read twice and referred to the Committee on Health, Education, 

Labor, and Pensions 

A BILL 

To modernize the process of accelerated approval of a drug 

for a serious or life-threatening disease or condition. 

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 ‘‘Modernizing the Accel5 erated Approval Pathway Act of 2022’’. 

6 SEC. 2. MODERNIZING ACCELERATED APPROVAL. 

7 (a) IN GENERAL.—Section 506(c) of the Federal 

8 Food, Drug, and Cosmetic Act (21 U.S.C. 356(c)) is 

9 amended— 

10 (1) in paragraph (2)— 


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1 (A) by redesignating subparagraphs (A) 

2 and (B) as clauses (i) and (ii), respectively, and 

3 adjusting the margins accordingly; 

4 (B) by striking ‘‘Approval of a product’’ 

5 and inserting the following: 

6 ‘‘(A) IN GENERAL.—Approval of a prod7 uct’’; 

8 (C) in clause (i) of such subparagraph (A), 

9 as so redesignated, by striking ‘‘appropriate 

10 postapproval studies’’ and inserting ‘‘an appro11 priate postapproval study or studies (which may 

12 be augmented or supported by real world evi13 dence)’’; and 

14 (D) by adding at the end the following: 

15 ‘‘(B) STUDIES NOT REQUIRED.—If the 

16 Secretary does not require that the sponsor of 

17 a product approved under accelerated approval 

18 conduct a postapproval study under this para19 graph, the Secretary shall publish on the 

20 website of the Food and Drug Administration 

21 the rationale for why such study is not appro22 priate or necessary. 

23 ‘‘(C) POSTAPPROVAL STUDY CONDI24 TIONS.—Not later than the time of approval of 

25 a product under accelerated approval, the SecVerDate Sep 11 2014 17:23 Jun 24, 2022 Jkt 029200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\S4446.IS S4446

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1 retary shall specify the conditions for a post2 approval study or studies required to be con3 ducted under this paragraph with respect to 

4 such product, which may include enrollment 

5 targets, the study protocol, and milestones, in6 cluding the target date of study completion. 

7 ‘‘(D) STUDIES BEGUN BEFORE AP8 PROVAL.—The Secretary may require such 

9 study or studies to be underway prior to ap10 proval.’’; and 

11 (2) in paragraph (3)— 

12 (A) by redesignating subparagraphs (A) 

13 through (D) as clauses (i) through (iv), respec14 tively, and adjusting the margins accordingly; 

15 (B) by striking ‘‘The Secretary may’’ and 

16 inserting the following: 

17 ‘‘(A) IN GENERAL.—The Secretary may’’; 

18 (C) in clause (i) of such subparagraph (A), 

19 as so redesignated, by striking ‘‘drug with due 

20 diligence’’ and inserting ‘‘product with due dili21 gence, including with respect to conditions spec22 ified by the Secretary under paragraph (2)(C)’’; 

23 (D) in clause (iii) of such subparagraph 

24 (A), as so redesignated, by inserting ‘‘shown to 

25 be’’ after ‘‘product is not’’; and 


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1 (E) by adding at the end the following: 

2 ‘‘(B) EXPEDITED PROCEDURES DE3 SCRIBED.—Expedited procedures described in 

4 this subparagraph shall consist of, prior to the 

5 withdrawal of accelerated approval— 

6 ‘‘(i) providing the sponsor with— 

7 ‘‘(I) due notice; 

8 ‘‘(II) an explanation for the pro9 posed withdrawal; 

10 ‘‘(III) an opportunity for a meet11 ing with the Commissioner or the 

12 Commissioner’s designee; and 

13 ‘‘(IV) an opportunity for written 

14 appeal to— 

15 ‘‘(aa) the Commissioner; or 

16 ‘‘(bb) a designee of the 

17 Commissioner who has not par18 ticipated in the proposal with19 drawal of approval (other than a 

20 meeting pursuant to subclause 

21 (III)) and is not subordinate of 

22 an individual (other than the 

23 Commissioner) who participated 

24 in such proposed withdrawal; 


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1 ‘‘(ii) providing an opportunity for 

2 public comment on the proposing to with3 drawal approval; 

4 ‘‘(iii) the publication of a summary of 

5 the public comments received, and the Sec6 retary’s response to such comments, on the 

7 website of the Food and Drug Administra8 tion; and 

9 ‘‘(iv) convening and consulting an ad10 visory committee on issues related to the 

11 proposed withdrawal, if requested by the 

12 sponsor and if no such advisory committee 

13 has previously advised the Secretary on 

14 such issues with respect to the withdrawal 

15 of the product prior to the sponsor’s re16 quest.’’. 

17 (b) REPORTS OF POSTMARKETING STUDIES.—Sec18 tion 506B(a) of the Federal Food, Drug, and Cosmetic 

19 Act (21 U.S.C. 356b(a)) is amended— 

20 (1) by redesignating paragraph (2) as para21 graph (3); and 

22 (2) by inserting after paragraph (1) the fol23 lowing: 

24 ‘‘(2) ACCELERATED APPROVAL.—Notwith25 standing paragraph (1), a sponsor of a drug apVerDate Sep 11 2014 17:23 Jun 24, 2022 Jkt 029200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S4446.IS S4446

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1 proved under accelerated approval shall submit to 

2 the Secretary a report of the progress of any study 

3 required under section 506(c), including progress to4 ward enrollment targets, milestones, and other infor5 mation as required by the Secretary, not later than 

6 180 days after the approval of such drug and not 

7 less frequently than every 180 days thereafter, until 

8 the study is completed or terminated. The Secretary 

9 shall promptly publish on the website of the Food 

10 and Drug Administration the information so re11 ported, in an easily searchable format.’’. 

12 (c) ENFORCEMENT.—Section 301 of the Federal 

13 Food, Drug, and Cosmetic Act (21 U.S.C. 331), is amend14 ed by adding at the end the following: 

15 ‘‘(fff) The failure of a sponsor of a product approved 

16 under accelerated approval pursuant to section 506(c)— 

17 ‘‘(1) to conduct with due diligence any post18 approval study required under section 506(c) with 

19 respect to such product; or 

20 ‘‘(2) to submit timely reports with respect to 

21 such product in accordance with section 

22 506B(a)(2).’’. 

23 (d) GUIDANCE.— 

24 (1) IN GENERAL.—The Secretary of Health and 

25 Human Services shall issue guidance describing— 


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1 (A) how sponsor questions related to the 

2 identification of novel surrogate or intermediate 

3 clinical endpoints may be addressed in early4 stage development meetings with the Food and 

5 Drug Administration; 

6 (B) the use of novel clinical trial designs 

7 that may be used to conduct appropriate post8 approval studies as may be required under sec9 tion 506(c)(2)(A) of the Federal Food, Drug, 

10 and Cosmetic Act, as amended by subsection 

11 (a); 

12 (C) the expedited procedures described in 

13 section 506(c)(3)(B) of the Federal Food, 

14 Drug, and Cosmetic Act; and 

15 (D) considerations related to the use of 

16 surrogate or intermediate clinical endpoints 

17 that may support the accelerated approval of an 

18 application under 506(c)(1)(A), including con19 siderations in evaluating the evidence related to 

20 any such endpoints. 

21 (2) FINAL GUIDANCE.—The Secretary shall 

22 issue— 

23 (A) draft guidance under paragraph (1) 

24 not later than 18 months after the date of en25 actment of this Act; and 


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1 (B) final guidance not later than 1 year 

2 after the close of the public comment period on 

3 such draft guidance. 

4 (e) RARE DISEASE ENDPOINT ADVANCEMENT

5 PILOT.— 

6 (1) IN GENERAL.—The Secretary of Health and 

7 Human Services shall establish a pilot program 

8 under which the Secretary will establish procedures 

9 to provide increased interaction with sponsors of 

10 rare disease drug development programs for pur11 poses of advancing the development of efficacy 

12 endpoints, including surrogate and intermediate 

13 endpoints, for drugs intended to treat rare diseases, 

14 including through— 

15 (A) determining eligibility of participants 

16 for such program; and 

17 (B) developing and implementing a process 

18 for applying to, and participating in, such a 

19 program. 

20 (2) PUBLIC WORKSHOPS.—The Secretary shall 

21 conduct up to 3 public workshops, which shall be 

22 completed not later than September 30, 2026, to 

23 discuss topics relevant to the development of 

24 endpoints for rare diseases, which may include dis25 cussions about— 


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1 (A) novel endpoints developed through the 

2 pilot program established under this subsection; 

3 and 

4 (B) as appropriate, the use of real world 

5 evidence and real work data to support the vali6 dation of efficacy endpoints, including surrogate 

7 and intermediate endpoints, for rare diseases. 

8 (3) REPORT.—Not later than September 30, 

9 2027, the Secretary shall submit to the Committee 

10 on Energy and Commerce of the House of Rep11 resentatives and the Committee on Health, Edu12 cation, Labor, and Pensions of the Senate a report 

13 describing the outcomes of the pilot program estab14 lished under this subsection. 

15 (4) GUIDANCE.—Not later than September 30, 

16 2027, the Secretary shall issue guidance describing 

17 best practices and strategies for development of effi18 cacy endpoints, including surrogate and intermediate 

19 endpoints, for rare diseases. 

20 (5) SUNSET.—The Secretary may not accept 

21 any new application or request to participate in the 

22 program established by this subsection on or after 

23 October 1, 2027. 

24 (f) ACCELERATED APPROVAL COUNCIL.— 


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1 (1) GENERAL.—Not later than 180 days after 

2 the date of enactment of this Act, the Secretary of 

3 Health and Human Services shall establish an intra4 agency coordinating council within the Food and 

5 Drug Administration to ensure the consistent and 

6 appropriate use of accelerated approval across the 

7 Food and Drug Administration, pursuant to section 

8 506(c) of the Federal Food, Drug, and Cosmetic Act 

9 (21 U.S.C. 356(c)). 

10 (2) MEMBERSHIP.—The members of the Coun11 cil shall consist of the following senior officials, or 

12 a designee of such official, from the Food and Drug 

13 Administration and relevant Centers: 

14 (A) The Director of the Center for Drug 

15 Evaluation and Research. 

16 (B) The Director of the Center for Bio17 logics Evaluation and Research. 

18 (C) The Director of the Oncology Center 

19 of Excellence. 

20 (D) The Director of the Office of New 

21 Drugs. 

22 (E) The Director of the Office of Orphan 

23 Products Development. 

24 (F) The Director of the Office of Tissues 

25 and Advanced Therapies. 


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1 (G) The Director of the Office of Medical 

2 Policy. 

3 (H) At least 3 directors of review division 

4 overseeing products approved under accelerated 

5 approval, including at least one director of a re6 view division within the Office of Neuroscience. 

7 (3) DUTIES OF THE COUNCIL.— 

8 (A) MEETINGS.—The Council shall con9 vene not fewer than 3 times per calendar year 

10 to discuss issues related to accelerated approval, 

11 including any relevant cross-disciplinary ap12 proaches related to product review with respect 

13 to accelerated approval. 

14 (B) POLICY DEVELOPMENT.—The Council 

15 shall directly engage with product review teams 

16 to support the consistent and appropriate use of 

17 accelerated approval across the Food and Drug 

18 Administration. Such activities may include— 

19 (i) developing guidance for Food and 

20 Drug Administration staff and best prac21 tices for, and across, product review teams, 

22 including with respect to communication 

23 between sponsors and the Food and Drug 

24 Administration and the review of products 

25 under accelerated approval; 


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1 (ii) providing training for product re2 view teams; and 

3 (iii) advising review divisions on prod4 uct-specific development, review, and with5 drawal of products under accelerated ap6 proval. 

7 (4) PUBLICATION OF A REPORT.—Not later 

8 than 1 year after the date of enactment of this Act, 

9 and annually thereafter, the council shall publish on 

10 the public website of the Food and Drug Adminis11 tration a report on the activities of the council. 

12 (g) RULE OF CONSTRUCTION.—Nothing in this sec13 tion (including the amendments made by this section) 

14 shall be construed to affect products approved pursuant 

15 to 506(c) of the Federal Food, Drug, and Cosmetic Act 

16 (21 U.S.C. 356(c)) prior to the date of enactment of this 

17 Act. 

Æ 


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