,,ii #aaith ,,congress #ast ,session ,s4 #icb ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,march #aa 7legislative day1 ,march #aj71 #bjbe,mr4 ,mullin 7for himself and ,mr4 ,bennet7 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 with respect to molecularly targeted pediatric cancer investigations1 and for other purposes4 #a4 ,short title ,this ,act may be cited as the ,give ,kids a ,chance ,act of #bjbe4 #b4 ,research into pediatric uses of drugs2 additional authorities of ,food and ,drug ,administration regarding molecularly targeted cancer drugs 7a7 ,in general 7#a7 ,additional active ingredient for application drug2 limitation regarding novel-combination application drug ,section #eje;,b7a77#c7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7a77#c77 is amended-- 7,a7 by redesignating subparagraphs 7,b7 and 7,c7 as subparagraphs 7,c7 and 7,d71 respectively2 and 7,b7 by striking subparagraph 7,a7 and inserting the following3 7,a7 ,in general ,for purposes of paragraph 7#a77;,b71 the investigation described in this paragraph is a molecularly targeted pediatric cancer investigation of-- 7i7 the drug or biological product for which the application referred to in such paragraph is submitted2 or 7ii7 such drug or biological product used in combination with-- 7,i7 an active ingredient of a drug or biological product-- 7aa7 for which an approved application under section #eje7;j7 under this ,act or under section #cea7k7 of the ,public ,health ,service ,act is in effect2 and 7bb7 that is determined by the ,secretary1 after consultation with the applicant1 to be part of the standard of care for treating a pediatric cancer2 or 7,,ii7 an active ingredient of a drug or biological product-- 7aa7 for which an approved application under section #eje7;b7 of this ,act or section #cea7;a7 of the ,public ,health ,service ,act to treat an adult cancer is in effect and is held by the same person submitting the application under paragraph 7#a77;,b72 and 7bb7 that is directed at a molecular target that the ,secretary determines to be substantially relevant to the growth or progression of a pediatric cancer4 7,b7 ,additional requirements 7i7 ,design of investigation ,a molecularly targeted pediatric cancer investigation referred to in subparagraph 7,a7 shall be designed to yield clinically meaningful pediatric study data that is gathered using appropriate formulations for each age group for which the study is required1 regarding dosing1 safety1 and preliminary efficacy to inform potential pediatric labeling4 7ii7 ,limitation ,an investigation described in subparagraph 7,a77ii7 may be required only if the drug or biological product for which the application referred to in paragraph 7#a77;,b7 contains either-- 7,i7 a single new active ingredient2 or 7,,ii7 more than one active ingredient1 if an application for the combination of active ingredients has not previously been approved but each active ingredient is in a drug product that has been previously approved to treat an adult cancer4 7iii7 ,results of already-completed preclinical studies of application drug ,with respect to an investigation required pursuant to paragraph 7#a77;,b71 the ,secretary may require the results of any completed preclinical studies relevant to the initial pediatric study plan be submitted to the ,secretary at the same time that the initial pediatric study plan required under subsection 7e77#a7 is submitted4 7iv7 ,rule of construction regarding inactive ingredients ,with respect to a combination of active ingredients referred to in subparagraph 7,a77ii71 such subparagraph shall not be construed as addressing the use of inactive ingredients with such combination4 4 7#b7 ,determination of applicable requirements ,section #eje;,b7e77#a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7e77#a77 is amended by adding at the end the following3 ,the ,secretary shall determine whether subparagraph 7,a7 or 7,b7 of subsection 7a77#a7 applies with respect to an application before the date on which the applicant is required to submit the initial pediatric study plan under paragraph 7#b77;,a744 7#c7 ,clarifying applicability ,section #eje;,b7a77#a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7a77#a77 is amended by adding at the end the following3 7,c7 ,rule of construction ,no application that is subject to the requirements of subparagraph 7,b7 shall be subject to the requirements of subparagraph 7,a71 and no application 7or supplement to an application7 that is subject to the requirements of subparagraph 7,a7 shall be subject to the requirements of subparagraph 7,b74 4 7#d7 ,conforming amendments ,section #eje;,b7a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7a77 is amended-- 7,a7 in paragraph 7#c77;,c71 as redesignated by paragraph 7#a77;,a7 of this subsection1 by striking investigations described in this paragraph and inserting investigations referred to in subparagraph 7,a72 and 7,b7 in paragraph 7#c77;,d71 as redesignated by paragraph 7#a77;,a7 of this subsection1 by striking the assessments under paragraph 7#b77;,b7 and inserting the assessments required under paragraph 7#a77;,a74 7b7 ,guidance ,the ,secretary of ,health and ,human ,services1 acting through the ,commissioner of ,food and ,drugs1 shall-- 7#a7 not later than #ab months after the date of enactment of this ,act1 issue draft guidance on the implementation of the amendments made by subsection 7a72 and 7#b7 not later than #ab months after closing the comment period on such draft guidance1 finalize such guidance4 7c7 ,applicability ,the amendments made by this section apply with respect to any application under section #eje7;b7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee7;b77 and any application under section #cea7;a7 of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bfb7;a771 that is submitted on or after the date that is #c years after the date of enactment of this ,act4 7d7 ,reports to ,congress 7#a7 ,secretary of ,health and ,human ,services ,not later than #f years after the date of enactment of this ,act1 the ,secretary of ,health and ,human ,services 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 on the ,secretary's efforts1 in coordination with industry1 to ensure implementation of the amendments made by subsection 7a74 7#b7 ,,gao study and report 7,a7 ,study ,not later than #h years after the date of enactment of this ,act1 the ,comptroller ,general of the ,united ,states shall conduct a study of the effectiveness of requiring assessments and investigations described in section #eje;,b of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c71 as amended by subsection 7a71 in the development of drugs and biological products for pediatric cancer indications1 including consideration of any benefits to1 or burdens on1 pediatric cancer drug development4 7,b7 ,findings ,not later than #aj years after the date of enactment of this ,act1 the ,comptroller ,general 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 containing the findings of the study conducted under subparagraph 7,a74 #c4 ,extension of authority to issue priority review vouchers to encourage treatments for rare pediatric diseases 7a7 ,extension ,section #ebi7;b77#e7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff7b77#e77 is amended by striking ,december #bj1 #bjbd1 unless and all that follows through the period at the end and inserting ,september #cj1 #bjbi44 7b7 ,user fee payment ,subsection #ebi7;c77#d7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff7c77#d77 is amended by striking subparagraph 7,a7 and inserting the following3 7,a7 ,in general ,the priority review user fee required by this subsection shall be due upon the submission of a human drug application under section #eje7;b77#a7 or section #cea7;a7 of the ,public ,health ,service ,act for which the priority review voucher is used4 ,all other user fees associated with the human drug application shall be due as required by the ,secretary or under applicable law4 4 7c7 ,,gao report on effectiveness of rare pediatric disease priority voucher awards in incentivizing rare pediatric disease drug development 7#a7 ,,gao study 7,a7 ,study ,the ,comptroller ,general of the ,united ,states shall conduct a study of the effectiveness of awarding rare pediatric disease priority vouchers under section #ebi of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff71 as amended by subsection 7a71 in the development of human drug products that treat or prevent rare pediatric diseases 7as defined in such section #ebi74 7,b7 ,contents of study ,in conducting the study under subparagraph 7,a71 the ,comptroller ,general shall examine the following3 7i7 ,the indications for each drug or biological product that-- 7,i7 is the subject of a rare pediatric disease product application 7as defined in section #ebi of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff77 for which a priority review voucher was awarded2 and 7,,ii7 was approved under section #eje of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#db ,u4,s4,c4 #cee7 or licensed under section #cea of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bfb74 7ii7 ,whether1 and to what extent1 an unmet need related to the treatment or prevention of a rare pediatric disease was met through the approval or licensure of such a drug or biological product4 7iii7 ,the size of the company to which a priority review voucher was awarded under section #ebi of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff7 for such a drug or biological product4 7iv7 ,the value of such priority review voucher if transferred4 7v7 ,identification of each drug for which a priority review voucher awarded under such section #ebi was used4 7vi7 ,the size of the company using each priority review voucher awarded under such section #ebi4 7vii7 ,the length of the period of time between the date on which a priority review voucher was awarded under such section #ebi and the date on which it was used4 7viii7 ,whether1 and to what extent1 an unmet need related to the treatment or prevention of a rare pediatric disease was met through the approval under section #eje of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#db ,u4,s4,c4 #cee7 or licensure under section #cea of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bfb7 of a drug for which a priority review voucher was used4 7ix7 ,whether1 and to what extent1 companies were motivated by the availability of priority review vouchers under section #ebi of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff7 to attempt to develop a drug for a rare pediatric disease4 7x7 ,whether1 and to what extent1 pediatric review vouchers awarded under such section were successful in stimulating development and expedited patient access to drug products for treatment or prevention of a rare pediatric disease that wouldn't otherwise take place without the incentive provided by such vouchers4 7xi7 ,the impact of such priority review vouchers on the workload1 review process1 and public health prioritization efforts of the ,food and ,drug ,administration4 7xii7 ,any other incentives in ,federal law that exist for companies developing drugs or biological products described in clause 7i74 7#b7 ,report on findings ,not later than #e years after the date of the enactment of this ,act1 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 ,labor1 and ,pensions of the ,senate a report containing the findings of the study conducted under paragraph 7#a74