,i #aacth ,,congress #b;d ,session ,h4 ,r4 #ehje ,,in ,,the ,,house ,,of ,,representatives ,december #h1 #bjad ,mr4 ,mc,caul introduced the following bill2 which was referred to the ,committee on ,energy and ,commerce ,a ,,bill ,to amend the ,federal ,food1 ,drug1 and ,cosmetic ,act with respect to expanding access for breakthrough drugs1 and for other purposes4 #a4 ,short title ,this ,act may be cited as the ,andrea ,sloan ,compassionate ,use ,reform and ,enhancement ,act or the ,andrea ,sloan ,,cure ,act4 #b4 ,expanded access policy as condition of expedited approval ,section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb7 is amended-- 7#a7 by redesignating subsections 7d7 and 7e7 as subsections 7e7 and 7f71 respectively2 and 7#b7 by inserting after subsection 7c7 the following new subsection3 7d7 ,expanded access policy required for covered breakthrough drugs 7#a7 ,in general ,with respect to a qualified breakthrough drug1 not later than #cj days after the date on which the drug meets the definition of a covered breakthrough drug 7as specified in paragraph 7#b771 the sponsor of the covered breakthrough drug shall submit to the ,secretary and make publicly available the policy of the sponsor with respect to requests submitted under subsection 7b74 ,in the case of such a policy under which the sponsor accepts such requests1 such policy shall include-- 7,a7 a single point of contact who receives and processes such requests2 7,b7 procedures for making such requests2 7,c7 the minimum criteria for the sponsor's consideration or approval of such requests2 and 7,d7 the amount of time the sponsor anticipates will be necessary to make a decision on such requests4 7#b7 ,covered breakthrough drug ,in this subsection1 the term covered breakthrough drug means a drug-- 7,a7 that is designated as a breakthrough therapy or as a fast track product or is approved under accelerated approval under section #ejf2 7,b7 that is designated under section #eje;,e7d7 as a qualified infectious disease product2 or 7,c7 the sponsor of which is awarded a priority review voucher under section #ebd or #ebi4 4 #c4 ,notification of submitters of compassionate use requests ,section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb71 as amended by section #b1 is further amended-- 7#a7 by redesignating subsections 7e7 and 7f7 7as redesignated by section #b7a77 as subsections 7f7 and 7g71 respectively2 and 7#b7 by inserting after subsection 7d7 7as inserted by section #b7b77 the following new subsection3 7e7 ,notification of submitters of requests ,in the case of the denial by a manufacturer or distributor of a request under subsection 7b71 not later than #e days after the date of such denial1 the manufacturer or distributor1 as applicable1 shall submit to the person 7or physician7 who made the request written notice of the denial1 including an explanation for the denial4 4 #d4 ,,gao qualitative analysis on individual patient access to unapproved therapies and diagnostics ,not later than #ahj days after the date of the enactment of this ,act and each year thereafter1 the ,comptroller ,general of the ,united ,states shall submit to the ,committee on ,energy and ,commerce of the ,house of ,representatives and the ,committee on ,health1 ,education1 ,labor and ,pensions of the ,senate a report containing a qualitative analysis of the extent to which individual patients have access to investigational drugs pursuant to subsection 7b7 of section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb7 and recommendations for improving such access4 ,in preparing such report1 the ,comptroller ,general shall conduct a qualitative analysis of the following3 7#a7 ,whether there are any identifiable patterns in requests submitted under subsection 7b7 of such section1 such as the types of indications for which requests for individual patient access are sought or the reasons for the denial of such requests4 7#b7 ,what the primary barriers are to drug sponsors granting requests for individual patient access4 7#c7 ,how the ,secretary evaluates safety and efficacy data submitted in connection with such requests4 7#d7 ,the amount of time that-- 7,a7 a physician typically takes to complete the paperwork necessary to make such a request2 7,b7 a drug sponsor takes to process such a request and to issue a decision with respect to the request2 and 7,c7 the ,secretary takes to process such a request and to issue a decision with respect to the request4 7#e7 ,how regulations1 guidance1 policies1 or practices may be modified1 streamlined1 expanded1 or discontinued to reduce or prevent delays in approving such requests4 7#f7 ,the number of such requests that1 for the period covered by the report-- 7,a7 were approved by drug sponsors and the ,food and ,drug ,administration2 7,b7 were approved by drug sponsors but denied by the ,food and ,drug ,administration2 and 7,c7 were denied by drug sponsors4 7#g7 ,how to encourage drug sponsors to grant requests for expanded access under such section #efa1 including requests for emergency use1 intermediate-size patient populations1 and large patient populations under a specified indication4 7#h7 ,whether and to what extent adverse events reported to the ,secretary as a result of individual use of an investigational drug or investigational device under such section #efa affected the development or approval of any drug or device4 #e4 ,expanded access task force 7a7 ,establishment ,the ,secretary of ,health and ,human ,services shall establish a task force within the ,department of ,health and ,human ,services to explore mechanisms for improving the access individual patients have to investigational drugs pursuant to subsection 7b7 of section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb71 to be known as the ,expanded ,access ,task ,force 7in this section referred to as the ,task ,force74 ,not later than #ij days after the date on which the ,comptroller ,general of the ,united ,states submits the first report required under section #d1 the ,task ,force shall be convened4 7b7 ,membership 7#a7 ,composition ,the ,task ,force shall be composed of not more than #i voting members appointed as follows3 7,a7 ,one member to serve as ,chairman of the ,task ,force1 appointed by the ,speaker of the ,house of ,representatives4 7,b7 ,one representative from the ,department of ,health and ,human ,services1 appointed by the ,secretary of ,health and ,human ,services4 7,c7 ,four representatives appointed by the ,majority ,leader of the ,house of ,representatives1 in consultation with the ,minority ,leader of the ,house of ,representatives1 and the ,chairman and the ,ranking ,member of the ,committee on ,energy and ,commerce of the ,house of ,representatives1 including-- 7i7 one representative of a biopharmaceutical company of less than #bej full-time employees2 7ii7 one representative of the rare disease patient community2 7iii7 one representative of the health care provider community2 and 7iv7 one bioethicist4 7,d7 ,three representatives appointed by ,majority ,leader of the ,senate1 in consultation with the ,minority ,leader of the ,senate1 and the ,chairman and the ,ranking ,member of the ,health1 ,education1 ,labor and ,pensions ,committee of the ,senate1 including-- 7i7 one representative of the biopharmaceutical industry2 7ii7 one representative of the patient community2 and 7iii7 one representative of the health care payor community4 7#b7 ,compensation ,members of the ,task ,force shall serve without compensation4 7c7 ,duties ,the ,task ,force shall comprehensively evaluate the access individual patients have to investigational drugs pursuant to subsection 7b7 of section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb71 taking into account-- 7#a7 the unique challenges faced by children with likely fatal diseases for which there is not a comparable or satisfactory alternative therapy available2 7#b7 possible incentives for biopharmaceutical companies and providers to approve requests submitted under such subsection2 7#c7 how the ,secretary of ,health and ,human ,services interprets and takes into consideration adverse event data reported in the case of data from use under a request submitted under such subsection2 7#d7 ways to streamline and standardize the process for submitting requests under such subsection2 and 7#e7 the costs incurred by biopharmaceutical companies for the time1 effort1 and delivery of investigational drugs to patients for the diagnosis1 monitoring1 or treatment of a serious disease or condition under such subsection4 7d7 ,report ,not later than #ahj days after the date on which the ,task ,force is convened1 the ,task ,force shall submit to the ,committee on ,energy and ,commerce of the ,house of ,representatives and the ,committee on ,health1 ,education1 ,labor and ,pensions of the ,senate a report in an electronic format describing the specific recommendations of the ,task ,force for improving the access individual patients have to investigational drugs pursuant to subsection 7b7 of section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb74 7e7 ,termination ,the task force shall terminate upon submission of the report required under subsection 7d74 #f4 ,finalizing draft guidance on expanded access 7a7 ,in general ,not later than #ahj days after the date on which the ,expanded ,access ,task ,force established under section #e submits the report under subsection 7d7 of such section1 the ,secretary of ,health and ,human ,services shall finalize the draft guidance entitled ,expanded ,access to ,investigational ,drugs for ,treatment ,use--,qs ,as and dated ,may #bjac4 7b7 ,contents ,the final guidance referred to in subsection 7a7 shall-- 7#a7 clearly define how the ,secretary interprets and uses adverse drug event data reported by investigators in the case of data reported from use under a request submitted under section #efa7;b7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb7b772 and 7#b7 take into account the report of the ,expanded ,access ,task ,force submitted under section #e7;d7 and the first report of the ,comptroller ,general of the ,united ,states submitted under section #d4