,i #aafth ,,congress #b;d ,session ,h4 ,r4 #gefg ,,in ,,the ,,house ,,of ,,representatives ,july #i1 #bjbj ,mr4 ,tonko 7for himself and ,mr4 ,mc,kinley7 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 to improve the treatment of rare diseases and conditions1 and for other purposes4 #a4 ,short title ,this ,act may be cited as the ,helping ,experts ,accelerate ,rare ,treatments ,act of #bjbj4 #b4 ,improving the treatment of rare diseases and conditions 7a7 ,annual report on orphan drug program ,subchapter ,b of chapter ,v of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;aa et seq47 is amended by adding at the end the following new section3 #ebi;,b4 ,annual report on orphan drug program 7a7 ,in general ,not later than #a year after the date of enactment of the ,helping ,experts ,accelerate ,rare ,treatments ,act of #bjbj1 and not less than annually thereafter1 the ,secretary shall submit to the ,congress a report summarizing the activities of the ,food and ,drug ,administration for designating drugs under section #ebf for a rare disease or condition and approving such drugs under section #eje of this ,act or licensing such drugs under section #cea of the ,public ,health ,service ,act1 including-- 7#a7 the number of applications for such drugs under sections #eje of this ,act and section #cea of the ,public ,health ,service ,act received by the ,food and ,drug ,administration1 the number of such applications considered by each ,food and ,drug ,administration division1 and the number of such applications pending and approved2 7#b7 the number of applications described in paragraph 7#a7 disaggregated by-- 7,a7 the disease or condition to be addressed2 and 7,b7 the target subpopulations identified in such applications2 7#c7 the prevalence in the ,united ,states of each disease or condition addressed by an application described in paragraph 7#a72 7#d7 integration of ,food and ,drug ,administration staff with expertise in rare diseases and conditions in each review of an application described in paragraph 7#a72 7#e7 identification of all external experts with expertise in rare diseases and conditions who are consulted in connection with1 or who otherwise participate in1 each review of an application described in paragraph 7#a72 and 7#f7 the status of each application described in paragraph 7#a74 7b7 ,public availability ,the ,secretary shall make each report under subsection 7a7 available to the public1 including by posting the report on the website of the ,food and ,drug ,administration4 4 7b7 ,study on ,european ,union safety and efficacy reviews of drugs for rare diseases and conditions 7#a7 ,in general ,the ,comptroller ,general of the ,united ,states shall conduct a study on ,european ,union safety and efficacy reviews of drugs for rare diseases and conditions1 including-- 7,a7 any differential prevalence-based mechanisms2 and 7,b7 consideration and use of supplemental data submitted during the review process1 including data associated with open label extension studies and expanded access programs4 7#b7 ,report ,not later than #a year after the date of enactment of this ,act1 the ,comptroller ,general shall-- 7,a7 complete the study under paragraph 7#a72 7,b7 submit a report on the results of such study to the ,congress2 and 7,c7 include in such report recommendations for changes to the processes and authorities of the ,food and ,drug ,administration to facilitate development of1 and access to1 treatments for rare diseases and conditions4 7#c7 ,public availability ,the ,comptroller ,general of the ,united ,states shall make each report under paragraph 7#b7 available to the public1 including by posting the report on the website of the ,government ,accountability ,office4 7c7 ,review process ,subparagraph 7,a7 of section #efi7;a77#b7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb-#h7;a77#b77 is amended to read as follows3 7,a7 ,in general 7i7 ,list ,the ,secretary shall develop and maintain a list of external experts who1 because of their special expertise1 are qualified to provide advice on rare disease and condition issues1 including topics described in subsection 7b74 7ii7 ,availability at meetings ,the ,secretary shall ensure availability1 for consultation purposes1 of an external expert on rare diseases1 as described in subparagraph 7,b71 in connection with each drug product advisory committee meeting concerning a drug or biological product for a rare disease or condition4 7iii7 ,review of applications ,the ,secretary shall ensure that each review of an application submitted under section #eje of this ,act or section #cea of the ,public ,health ,service ,act for an indication associated with a rare disease or condition includes ,food and ,drug ,administration staff with expertise on rare diseases and conditions as an integral part of the review team4 4 7d7 ,risk evaluation and mitigation strategy elements ,subsection 7a7 of section #eje-a of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee-a7 is amended by adding at the end the following new paragraph3 7#e7 ,orphan drugs ,in making any determination regarding the inclusion1 in the risk evaluation and mitigation strategy for a drug that has been designated as a drug for a rare disease or condition pursuant to section #ebf1 of an element that requires patient action1 restrictions on use of the drug1 or patient participation1 the ,secretary shall consult with patients impacted by the rare disease or condition in considering the burden of participation and likely patient compliance4 4