,,ii #aaith ,,congress #ast ,session ,s4 #gje ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,february #be1 #bjbe,mr4 ,reed 7for himself and ,mrs4 ,capito7 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 ,innovation in ,pediatric ,drugs ,act of #bjbe4 #b4 ,ensuring completion of pediatric study requirements 7a7 ,equal accountability for pediatric study requirements ,section #eje;,b7d7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7d77 is amended-- 7#a7 in paragraph 7#a71 by striking ,beginning #bgj and inserting ,noncompliance letter4--,beginning #bgj2 7#b7 in paragraph 7#b7-- 7,a7 by striking ,the drug or and inserting ,effect of noncompliance4--,the drug or2 and 7,b7 by striking 7except that the drug or biological product shall not be subject to action under section #cjc7 and inserting 7except that the drug or biological product shall be subject to action under section #cjc only if such person demonstrated a lack of due diligence in satisfying the applicable requirement72 and 7#c7 by adding at the end the following3 7#c7 ,limitation ,the ,secretary shall not issue enforcement actions under section #cjc for failures under this subsection in the case of a drug or biological product that is no longer marketed4 4 7b7 ,due diligence ,section #eje;,b7d7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7d771 as amended by subsection 7a71 is further amended by adding at the end the following3 7#d7 ,due diligence ,before the ,secretary may conclude that a person failed to submit or otherwise meet a requirement as described in the matter preceding paragraph 7#a71 the ,secretary shall-- 7,a7 issue a noncompliance letter pursuant to paragraph 7#a72 7,b7 provide such person with a #de-;day period beginning on the date of receipt of such noncompliance letter to respond in writing as set forth in such paragraph2 and 7,c7 after reviewing such written response1 determine whether the person demonstrated a lack of due diligence in satisfying such requirement4 4 7c7 ,conforming amendments ,section #cjc7;f77#d77;,a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #ccc7;f77#d77;,a77 is amended by striking or #eje-a and inserting #eje-a1 or #eje;,b4 7d7 ,transition rule ,the ,secretary of ,health and ,human ,services may take enforcement action under section #cjc of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #ccc7 for failures described in section #eje;,b7d7 of such ,act 7#ba ,u4,s4,c4 #cee;c7d77 only if such failures occur on or after the date that is #ahj days after the date of enactment of this ,act4 #c4 ,,fda report on ,,prea enforcement ,section #ejh7;b7 of the ,food and ,drug ,administration ,safety and ,innovation ,act 7#ba ,u4,s4,c4 #cee;c-#a7;b77 is amended-- 7#a7 in paragraph 7#aa71 by striking the semicolon at the end and inserting 1 including an evaluation of compliance with deadlines provided for in deferrals and deferral extensions22 7#b7 in paragraph 7#ae71 by striking and at the end2 7#c7 in paragraph 7#af71 by striking the period at the end and inserting 2 and2 and 7#d7 by adding at the end the following3 7#ag7 a listing of penalties1 settlements1 or payments under section #cjc of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cec7 for failure to comply with requirements under such section #eje;,b1 including1 for each penalty1 settlement1 or payment1 the name of the drug1 the sponsor thereof1 and the amount of the penalty1 settlement1 or payment imposed4 4 #d4 ,program for pediatric studies of drugs ,section #dji;,i7d7 of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bhdm7d77 is amended to read as follows3 7d7 ,funding ,of the amount made available for pediatric research to each national research institute and national center under this title for each of fiscal years #bjbf through #bjcj1 the ,director of ,,nih is authorized to make available up to one percent of such amount for pediatric research under this section4 4 #e4 ,pediatric studies of orphan drugs 7a7 ,application of pediatric research requirements to orphan drugs 7#a7 ,in general ,section #eje;,b7k7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7k77 is amended to read as follows3 7k7 ,relation to orphan drugs 7#a7 ,in general ,this section does not apply to a drug or biological product for an indication for which orphan designation has been granted under section #ebf unless the ,secretary determines that pediatric assessments of such drug or biological product required under this section could represent a meaningful therapeutic benefit as described in subsection 7c74 7#b7 ,deferrals and waivers ,deferrals and waivers under subsections 7a77#d7 and 7a77#e7 shall apply to assessments described in this subsection to the same extent and in the same manner as such deferrals and waivers apply with respect to the assessments under subsection 7a77#a71 and waivers under subsection 7b77#b7 shall apply to assessments described in this subsection to the same extent and in the same manner as such waivers apply with respect to the assessments required pursuant to subsection 7b77#a74 4 7#b7 ,applicability ,the amendment made by paragraph 7#a7 applies only to applications described in subparagraph 7,a7 or 7,b7 of section #eje;,b7a77#a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7a77#a77 that are submitted on or after the later of-- 7,a7 the date that is #ah months after the date of issuance of the final guidance under subsection 7b72 and 7,b7 such later date as may be specified by the ,secretary of ,health and ,human ,services 7referred to in this section as the ,secretary7 by regulation4 7b7 ,guidance 7#a7 ,issuance ,the ,secretary shall-- 7,a7 not later than #a year after the date of enactment of this ,act1 issue draft guidance describing how1 upon the applicability of the amendment made by subsection 7a77#a71 the requirements of section #eje;,b of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7 will apply with respect to any drug or biological product for an indication within a disease or condition for which orphan designation has been granted under section #ebf of such ,act 7#ba ,u4,s4,c4 #cfj;bb72 and 7,b7 not later than #ah months after the date of the public meeting required by subsection 7c77#a71 finalize such draft guidance4 7#b7 ,contents ,the guidance under subsection 7b7 shall address the following3 7,a7 ,information regarding how full and partial waivers under subsections 7a77#e77;,a71 7a77#e77;,b71 and 7b77#b7 of section #eje;,b of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7 for any drug or biological product for an indication within a disease or condition for which orphan designation has been granted under section #ebf of such ,act 7#ba ,u4,s4,c4 #cfj;bb7 will be granted4 7,b7 ,application of the requirements of section #eje;,b7e7 of such ,act 7#ba ,u4,s4,c4 #cee;c7e77 to drugs or biological products for an indication within a disease or condition for which orphan designation has been granted under section #ebf of such ,act 7#ba ,u4,s4,c4 #cfj;bb71 including submission and timing of planned requests for full or partial waivers and responses by the ,food and ,drug ,administration to those requests4 7,c7 ,rare diseases and conditions 7as defined in section #ebf7;a77#b7 of such ,act 7#ba ,u4,s4,c4 #cfj;bb7a77#b77 that should be added to the lists under section #eje;,b7a77#e77;,e7 and #eje;,b7b77#b77;,e7 of such ,act1 as added by subsection 7f71 and a process for regularly updating such lists4 7,d7 ,situations where the initial pediatric study plan under section #eje;,b7e7 of such ,act 7#ba ,u4,s4,c4 #cee;c7e77 includes a plan to fulfill the requirements of section #eje;,b7a7 of such ,act 7#ba ,u4,s4,c4 #cee;c7a77 without requesting waivers in any age group4 7,e7 ,consideration of how the ,secretary will balance the unique scientific challenges of rare disease drug development with the need for improved pediatric labeling of drugs and biological products for indications within diseases or conditions for which orphan designation has been granted under section #ebf of such ,act 7#ba ,u4,s4,c4 #cfj;bb74 7,f7 ,considerations of the strengths1 weaknesses1 appropriateness1 and limitations of different types of real-world evidence specific to the fulfillment of requirements under section #eje;,b of such ,act 7#ba ,u4,s4,c4 #cee;c74 7,g7 ,consideration of input received from the public meeting set forth in subsection 7c74 7c7 ,public meeting ,the ,secretary shall-- 7#a7 not later than #f months after the date of issuance of the draft guidance under subsection 7b77#a77;,a71 hold a public meeting to inform the final guidance to be issued under subsection 7b77#a77;,b72 and 7#b7 publish prior notice of such meeting in the ,federal ,register4 7d7 ,,gao study ,not later than #d years after the applicability date described in subsection 7a77#b71 the ,comptroller ,general of the ,united ,states shall submit to the ,committee on ,energy and ,commerce and the ,committee on ,ways and ,means of the ,house of ,representatives and the ,committee on ,health1 ,education1 ,labor1 and ,pensions of the ,senate a report that-- 7#a7 addresses the impacts of this ,act on-- 7,a7 rare disease drug development in the ,united ,states2 and 7,b7 the availability of pediatric information on drugs and biological products within diseases or conditions for indications for which orphan designation has been granted under section #ebf of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bb72 and 7#b7 includes-- 7,a7 the findings of a survey of companies of varying sizes engaged in the development of orphan drugs1 which shall include questions regarding the feasibility and other challenges of conducting pediatric studies for such indications2 7,b7 input from patient groups and medical provider associations2 and 7,c7 an assessment of the impact changes to required pediatric studies had on drug development for rare diseases4 7e7 ,rule of construction ,nothing in this section shall be construed to limit requirements for investigations1 as described in section #eje;,b7a77#c7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7a77#c771 of molecularly targeted pediatric cancer drugs for which orphan designation has been granted under section #ebf of such ,act 7#ba ,u4,s4,c4 #cfj;bb74 7f7 ,certainty regarding waivers ,section #eje;,b of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7 is amended-- 7#a7 in subsection 7a77#e71 by adding at the end the following3 7,e7 ,automatic full waiver list ,the ,secretary shall maintain a list1 posted on the website of the ,food and ,drug ,administration1 of adult-related diseases and conditions-- 7i7 with respect to which the necessary studies are impossible or highly impracticable1 as described in subparagraph 7,a77i72 or 7ii7 for which a drug or biological product for such disease or condition otherwise meets the criteria described in subparagraph 7,a74 2 7#b7 in subsection 7b77#b71 by adding at the end the following3 7,e7 ,automatic full waiver list ,the ,secretary shall maintain a list1 posted on the website of the ,food and ,drug ,administration1 of adult-related diseases and conditions with respect to which the necessary studies would meet the criteria for a full waiver under subparagraph 7,a74 2 and 7#c7 in subsection 7e77#d71 by adding at the end the following3 ,if1 at the time of an applicant's submission of the initial pediatric study plan1 the disease or condition for which the drug is intended to treat appears on the list under subsection 7a77#e77;,e71 then the assessments for such disease or condition shall be waived under subsection 7a77#e744