,,ii #aagth ,,congress #b;d ,session ,s4 #dddf ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,june #bb1 #bjbb,ms4 ,collins 7for herself and ,mr4 ,kaine7 introduced the following bill2 which was read twice and referred to the ,committee on ,health1 ,education1 ,labor1 and ,pensions ,a ,,bill ,to modernize the process of accelerated approval of a drug for a serious or life-threatening disease or condition4 #a4 ,short title ,this ,act may be cited as the ,modernizing the ,accelerated ,approval ,pathway ,act of #bjbb4 #b4 ,modernizing accelerated approval 7a7 ,in general ,section #ejf7;c7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cef7;c77 is amended-- 7#a7 in paragraph 7#b7-- 7,a7 by redesignating subparagraphs 7,a7 and 7,b7 as clauses 7i7 and 7ii71 respectively1 and adjusting the margins accordingly2 7,b7 by striking ,approval of a product and inserting the following3 7,a7 ,in general ,approval of a product 2 7,c7 in clause 7i7 of such subparagraph 7,a71 as so redesignated1 by striking appropriate postapproval studies and inserting an appropriate postapproval study or studies 7which may be augmented or supported by real world evidence72 and 7,d7 by adding at the end the following3 7,b7 ,studies not required ,if the ,secretary does not require that the sponsor of a product approved under accelerated approval conduct a postapproval study under this paragraph1 the ,secretary shall publish on the website of the ,food and ,drug ,administration the rationale for why such study is not appropriate or necessary4 7,c7 ,postapproval study conditions ,not later than the time of approval of a product under accelerated approval1 the ,secretary shall specify the conditions for a postapproval study or studies required to be conducted under this paragraph with respect to such product1 which may include enrollment targets1 the study protocol1 and milestones1 including the target date of study completion4 7,d7 ,studies ,begun ,before ,approval ,the ,secretary may require such study or studies to be underway prior to approval4 2 and 7#b7 in paragraph 7#c7-- 7,a7 by redesignating subparagraphs 7,a7 through 7,d7 as clauses 7i7 through 7iv71 respectively1 and adjusting the margins accordingly2 7,b7 by striking ,the ,secretary may and inserting the following3 7,a7 ,in general ,the ,secretary may 2 7,c7 in clause 7i7 of such subparagraph 7,a71 as so redesignated1 by striking drug with due diligence and inserting product with due diligence1 including with respect to conditions specified by the ,secretary under paragraph 7#b77;,c72 7,d7 in clause 7iii7 of such subparagraph 7,a71 as so redesignated1 by inserting shown to be after product is not2 and 7,e7 by adding at the end the following3 7,b7 ,expedited procedures described ,expedited procedures described in this subparagraph shall consist of1 prior to the withdrawal of accelerated approval-- 7i7 providing the sponsor with-- 7,i7 due notice2 7,,ii7 an explanation for the proposed withdrawal2 7,,iii7 an opportunity for a meeting with the ,commissioner or the ,commissioner's designee2 and 7,,iv7 an opportunity for written appeal to-- 7aa7 the ,commissioner2 or 7bb7 a designee of the ,commissioner who has not participated in the proposal withdrawal of approval 7other than a meeting pursuant to subclause 7,,iii77 and is not subordinate of an individual 7other than the ,commissioner7 who participated in such proposed withdrawal2 7ii7 providing an opportunity for public comment on the proposing to withdrawal approval2 7iii7 the publication of a summary of the public comments received1 and the ,secretary's response to such comments1 on the website of the ,food and ,drug ,administration2 and 7iv7 convening and consulting an advisory committee on issues related to the proposed withdrawal1 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 request4 4 7b7 ,reports of postmarketing studies ,section #ejf;,b7a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cef;b7a77 is amended-- 7#a7 by redesignating paragraph 7#b7 as paragraph 7#c72 and 7#b7 by inserting after paragraph 7#a7 the following3 7#b7 ,accelerated approval ,notwithstanding paragraph 7#a71 a sponsor of a drug approved under accelerated approval shall submit to the ,secretary a report of the progress of any study required under section #ejf7;c71 including progress toward enrollment targets1 milestones1 and other information as required by the ,secretary1 not later than #ahj days after the approval of such drug and not less frequently than every #ahj days thereafter1 until the study is completed or terminated4 ,the ,secretary shall promptly publish on the website of the ,food and ,drug ,administration the information so reported1 in an easily searchable format4 4 7c7 ,enforcement ,section #cja of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cca71 is amended by adding at the end the following3 7fff7 ,the failure of a sponsor of a product approved under accelerated approval pursuant to section #ejf7;c7-- 7#a7 to conduct with due diligence any postapproval study required under section #ejf7;c7 with respect to such product2 or 7#b7 to submit timely reports with respect to such product in accordance with section #ejf;,b7a77#b74 4 7d7 ,guidance 7#a7 ,in general ,the ,secretary of ,health and ,human ,services shall issue guidance describing-- 7,a7 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 ,administration2 7,b7 the use of novel clinical trial designs that may be used to conduct appropriate post-approval studies as may be required under section #ejf7;c77#b77;,a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act1 as amended by subsection 7a72 7,c7 the expedited procedures described in section #ejf7;c77#c77;,b7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act2 and 7,d7 considerations related to the use of surrogate or intermediate clinical endpoints that may support the accelerated approval of an application under #ejf7;c77#a77;,a71 including considerations in evaluating the evidence related to any such endpoints4 7#b7 ,final guidance ,the ,secretary shall issue-- 7,a7 draft guidance under paragraph 7#a7 not later than #ah months after the date of enactment of this ,act2 and 7,b7 final guidance not later than #a year after the close of the public comment period on such draft guidance4 7e7 ,rare disease endpoint advancement pilot 7#a7 ,in general ,the ,secretary of ,health and ,human ,services shall establish a pilot program under which the ,secretary will establish procedures to provide increased interaction with sponsors of rare disease drug development programs for purposes of advancing the development of efficacy endpoints1 including surrogate and intermediate endpoints1 for drugs intended to treat rare diseases1 including through-- 7,a7 determining eligibility of participants for such program2 and 7,b7 developing and implementing a process for applying to1 and participating in1 such a program4 7#b7 ,public workshops ,the ,secretary shall conduct up to #c public workshops1 which shall be completed not later than ,september #cj1 #bjbf1 to discuss topics relevant to the development of endpoints for rare diseases1 which may include discussions about-- 7,a7 novel endpoints developed through the pilot program established under this subsection2 and 7,b7 as appropriate1 the use of real world evidence and real work data to support the validation of efficacy endpoints1 including surrogate and intermediate endpoints1 for rare diseases4 7#c7 ,report ,not later than ,september #cj1 #bjbg1 the ,secretary shall submit to the ,committee on ,energy and ,commerce of the ,house of ,representatives and the ,committee on ,health1 ,education1 ,labor1 and ,pensions of the ,senate a report describing the outcomes of the pilot program established under this subsection4 7#d7 ,guidance ,not later than ,september #cj1 #bjbg1 the ,secretary shall issue guidance describing best practices and strategies for development of efficacy endpoints1 including surrogate and intermediate endpoints1 for rare diseases4 7#e7 ,sunset ,the ,secretary may not accept any new application or request to participate in the program established by this subsection on or after ,october #a1 #bjbg4 7f7 ,accelerated approval council 7#a7 ,general ,not later than #ahj days after the date of enactment of this ,act1 the ,secretary of ,health and ,human ,services shall establish an intra-agency coordinating council within the ,food and ,drug ,administration to ensure the consistent and appropriate use of accelerated approval across the ,food and ,drug ,administration1 pursuant to section #ejf7;c7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cef7;c774 7#b7 ,membership ,the members of the ,council shall consist of the following senior officials1 or a designee of such official1 from the ,food and ,drug ,administration and relevant ,centers3 7,a7 ,the ,director of the ,center for ,drug ,evaluation and ,research4 7,b7 ,the ,director of the ,center for ,biologics ,evaluation and ,research4 7,c7 ,the ,director of the ,oncology ,center of ,excellence4 7,d7 ,the ,director of the ,office of ,new ,drugs4 7,e7 ,the ,director of the ,office of ,orphan ,products ,development4 7,f7 ,the ,director of the ,office of ,tissues and ,advanced ,therapies4 7,g7 ,the ,director of the ,office of ,medical ,policy4 7,h7 ,at least #c directors of review division overseeing products approved under accelerated approval1 including at least one director of a review division within the ,office of ,neuroscience4 7#c7 ,duties of the council 7,a7 ,meetings ,the ,council shall convene not fewer than #c times per calendar year to discuss issues related to accelerated approval1 including any relevant cross-disciplinary approaches related to product review with respect to accelerated approval4 7,b7 ,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 ,administration4 ,such activities may include-- 7i7 developing guidance for ,food and ,drug ,administration staff and best practices for1 and across1 product review teams1 including with respect to communication between sponsors and the ,food and ,drug ,administration and the review of products under accelerated approval2 7ii7 providing training for product review teams2 and 7iii7 advising review divisions on product-specific development1 review1 and withdrawal of products under accelerated approval4 7#d7 ,publication of a report ,not later than #a year after the date of enactment of this ,act1 and annually thereafter1 the council shall publish on the public website of the ,food and ,drug ,administration a report on the activities of the council4 7g7 ,rule of construction ,nothing in this section 7including the amendments made by this section7 shall be construed to affect products approved pursuant to #ejf7;c7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cef7;c77 prior to the date of enactment of this ,act4