,,ii #aaith ,,congress #ast ,session ,s4 #hbb ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,march #c1 #bjbe ,ms4 ,klobuchar 7for herself and ,mr4 ,wicker7 introduced the following bill2 which was read twice and referred to the ,committee on ,health1 ,education1 ,labor1 and ,pensions ,a ,,bill ,to amend the ,federal ,food1 ,drug1 and ,cosmetic ,act to establish a process for science-focused drug development meetings led by the ,reagan-,udall ,foundation for the ,food and ,drug ,administration with respect to drugs for rare diseases and conditions1 and for other purposes4 #a4 ,short title ,this ,act may be cited as the ,scientific ,external ,process for ,educated ,review of ,therapeutics ,act of #bjbe or the ,scientific ,,expert ,act of #bjbe4 #b4 ,science-focused drug development meetings ,the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cja et seq47 is amended by inserting after section #ggj 7#ba ,u4,s4,c4 #cgi;dd7 the following3 #ggj;,a4 ,science-focused drug development meetings 7a7 ,in general ,the ,secretary shall develop and implement a process for ,foundation led1 science-focused drug development meetings to provide an opportunity for academic researchers and medical experts1 drug sponsors1 scientific organizations1 and patient organizations to-- 7#a7 discuss science-related challenges impacting the development of drugs for rare diseases and conditions2 7#b7 identify scientific approaches and opportunities to facilitate the development1 review1 and approval of such drugs2 and 7#c7 align on novel approaches for the development of drugs for particular diseases1 including appropriate clinical trial designs and metrics1 manufacturing standards1 patient populations1 clinical endpoints1 the use of biomarkers as surrogate endpoints1 and natural history as a control1 to advance treatment options to address unmet medical needs4 7b7 ,arrangement 7#a7 ,qualified third party convenor ,the ,secretary shall enter into an arrangement with the ,foundation under which the ,foundation agrees to convene ,,el-,,sfdd meetings regarding drugs for rare diseases or conditions in accordance with this section4 7#b7 ,minimum number of meetings ,the ,foundation shall convene no fewer than #d ,,el-,,sfdd meetings each year1 with each such meeting focused on addressing a different rare disease or condition or a different group of rare diseases and conditions4 7#c7 ,steering committee 7,a7 ,in general ,the ,foundation shall establish and maintain a permanent steering committee1 to be known as the ,science-,focused ,drug ,development ,multistakeholder ,steering ,committee1 to advise the ,foundation on implementation of this section1 including by-- 7i7 establishing a process by which licensed and board-certified medical clinicians and academics with expertise in a rare disease or condition1 sponsors of drugs for a rare disease or condition1 scientific organizations1 rare disease or condition patient organizations1 and other entities can provide suggested meeting topics to the ,foundation2 7ii7 reviewing such suggested meeting topics for ,,el-,,sfdd meetings2 and 7iii7 based on the criteria under subparagraph 7,b71 recommending to the ,foundation topics for ,,el-,,sfdd meetings4 7,b7 ,criteria for meetings ,in formulating recommendations under subparagraph 7,a71 the ,foundation shall consider-- 7i7 unmet therapeutic needs2 7ii7 the size of the patient population of the rare disease or condition2 7iii7 whether there were or are multiple products in development to prevent or treat the rare disease or condition involved2 7iv7 whether there is a need for increased regulatory flexibility to facilitate the development of products2 7v7 whether the rare disease or condition involved would benefit from clarity and alignment on drug development questions 7such as clinical trial design1 natural history as a control1 appropriate clinical endpoints1 biomarkers that may serve as surrogate endpoints1 and other approaches7 to expedite drug development for such disease or condition2 and 7vi7 whether the discussions about such rare disease or condition may have broader impact on other rare diseases and conditions4 7,c7 ,membership ,the members of the ,steering ,committee shall be subject to all relevant conflict of interest policies of the ,foundation1 and shall include-- 7i7 representatives of the ,center for ,drug ,evaluation and ,research1 the ,center for ,biologics ,evaluation and ,research1 and the ,center for ,devices and ,radiological ,health2 7ii7 academic and licensed and board-certified medical clinicians2 7iii7 patient representatives2 and 7iv7 industry representatives engaged in the development of drugs for rare diseases and conditions4 7#d7 ,planning process ,in planning an ,,el-,,sfdd meeting under this section1 the ,foundation1 in consultation with the stakeholders listed in paragraph 7#e71 shall develop-- 7,a7 a list of the specific objectives of the meeting related to key drug development issues for the rare disease or condition1 or group of rare diseases and conditions1 with a goal of expediting drug development2 7,b7 a proposed agenda for the meeting2 and 7,c7 a list of medical experts1 drug sponsors1 scientific organizations1 patient organizations1 and other entities to be invited to participate in the meeting4 7#e7 ,agency and stakeholder engagement ,throughout the process of planning an ,,el-,,sfdd meeting1 the ,foundation shall consult with-- 7,a7 appropriate staff of the ,food and ,drug ,administration2 7,b7 the ,steering ,committee2 7,c7 industry representatives engaged in the development of products for rare diseases and conditions to be discussed at such ,,el-,,sfdd meeting2 7,d7 patient representatives of rare diseases and conditions under discussion in such ,,el-,,sfdd meeting2 7,e7 academics or board-certified and licensed medical clinicians with expertise in treating a rare disease or condition2 and 7,f7 other appropriate stakeholders4 7#f7 ,post-meeting reports 7,a7 ,in general ,not later than #ahj days after an ,,el-,,sfdd meeting1 the ,foundation shall make publicly available on the website of the ,food and ,drug ,administration-- 7i7 a transcript and recording of the meeting2 and 7ii7 in consultation with the stakeholders listed in paragraph 7#e71 a summary analysis of the input received during the meeting that is relevant to approval or licensing of drugs for the rare disease or condition involved4 7,b7 ,contents ,each publication under subparagraph 7,a7 shall include a clear identification of-- 7i7 areas of consensus2 7ii7 areas where additional clarification or information is needed to reach consensus2 and 7iii7 next steps agreed upon with the ,food and ,drug ,administration4 7c7 ,representatives of ,,fda review divisions ,the ,secretary shall require appropriate representatives of the review divisions of the ,food and ,drug ,administration to participate in each ,,el-,,sfdd meeting under this section4 7d7 ,rules of construction ,nothing in this section shall be construed-- 7#a7 to prevent other third-party organizations from organizing similarly structured ,,el-,,sfdd-like meetings to discuss challenges in rare disease drug development2 7#b7 to require the ,food and ,drug ,administration to participate in additional meetings described in paragraph 7#a72 7#c7 to alter the protections offered by laws1 regulations1 or policies governing disclosure of confidential commercial or trade secret information and any other information exempt from disclosure pursuant to section #eeb7;b7 of title #e1 ,united ,states ,code2 7#d7 to limit the ability of the ,secretary to consult with individuals and organizations2 7#e7 to create a legal right for consultation on any matter or require the ,secretary to meet with any particular expert or stakeholder2 7#f7 to alter agreed-upon goals and procedures identified in the letters described in section #ajja7;b7 of the ,,fda ,user ,fee ,reauthorization ,act of #bjbb2 or 7#g7 to increase the number of review cycles for drugs4 7e7 ,definitions ,in this section3 7#a7 ,the term ,,el-,,sfdd meeting means an externally led1 science-focused drug development meeting4 7#b7 ,the term rare disease or condition has the meaning given such term in section #ebf4 7#c7 ,the term ,steering ,committee means the ,science-,focused ,drug ,development ,multistakeholder ,steering ,committee established under subsection 7b77#c74 7f7 ,authorization of appropriations 7#a7 ,in general ,to carry out this section1 there is authorized to be appropriated #a1jjj1jjj for each of fiscal years #bjbe through #bjbi4 7#b7 ,rule of construction ,nothing in this section shall be construed to prohibit the ,foundation from soliciting or accepting funds pursuant to section #ggj7;i7 for the purposes of planning or operating an ,,el-,,sfdd meeting authorized by this section4 #ggj;,b4 ,required actions following ,,el-,,sfdd meetings 7a7 ,incorporation of input into risk-,benefit assessments ,in approving or licensing a drug under subsection 7c7 or 7j7 of section #eje of this ,act or subsection 7a7 or 7k7 of section #cea of the ,public ,health ,service ,act1 the ,secretary shall make public-- 7#a7 a statement of whether any ,,el-,,sfdd meeting under section #ggj;,a was held that was relevant to such approval or licensure2 and 7#b7 if a meeting described in paragraph 7#a7 was held1 a description of how the ,secretary incorporated input from such meeting in the risk-benefit assessment described in section #eje7;d74 7b7 ,annual report ,on an annual basis1 the ,secretary shall submit a report to ,congress summarizing-- 7#a7 the number and topics of ,,el-,,sfdd meetings held during the reporting period2 7#b7 the extent of participation in such meetings from the review divisions of the ,food and ,drug ,administration2 7#c7 the impact of ,,el-,,sfdd meetings on the workload and resources of the ,food and ,drug ,administration2 and 7#d7 an assessment of how the input received during such meetings was used in-- 7,a7 deliberations throughout the drug development lifecycle2 7,b7 regulatory decision-making2 and 7,c7 formulating recommendations for future meetings4 7c7 ,definition ,in this section1 the term ,,el-,,sfdd meeting has the meaning given to that term in section #ggj;,a4 7d7 ,authorization of appropriations ,to carry out this section1 there is authorized to be appropriated #a1jjj1jjj for each of fiscal years #bjbe through #bjbi4 4