,,ii #aagth ,,congress #b;d ,session ,s4 #djga ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,april #g1 #bjbb,mr4 ,casey 7for himself and ,mr4 ,scott of ,south ,carolina7 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 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 #bjbb4 #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 #bjbb1 and not less frequently than annually thereafter1 the ,secretary shall submit to ,congress a report summarizing the activities of the ,food and ,drug ,administration related to 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 section #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 accepted for filing1 the number of such applications rejected for filing1 and the numbers of such applications pending1 approved1 and disapproved by the ,food and ,drug ,administration1 arrayed by the review division assigned to the application2 7#b7 the size of the affected population in the ,united ,states of each disease or condition addressed by an application described in paragraph 7#a71 assessed taking into consideration the documentation required by section #caf4bj of title #ba1 ,code of ,federal ,regulations 7or any successor regulation7 and other relevant information available to the ,secretary2 and 7#c7 the extent of consultation with stakeholders and external experts pursuant to section #efi4 7b7 ,manner of submission ,the information required to be reported under this section may be submitted as part of another report to ,congress related to the regulation of drugs4 7c7 ,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 ,not later than #a year after the date of enactment of this ,act1 the ,secretary of ,health and ,human ,services shall seek to enter into an agreement with the ,national ,academies of ,sciences1 ,engineering1 and ,medicine 7referred to in this section as the ,national ,academies7 to examine and report on ,european ,union safety and efficacy reviews of drugs for rare diseases and conditions1 the use and sufficiency of existing mechanisms and tools of the ,food and ,drug ,administration in ensuring that patient and physician perspectives are considered throughout such reviews1 and opportunities to improve such reviews in the ,united ,states4 7#b7 ,content ,the report developed under paragraph 7#a7 shall-- 7,a7 assess and evaluate1 with respect to drugs for rare diseases and conditions-- 7i7 any flexibilities1 authorities1 or mechanisms available in the ,european ,union2 7ii7 consideration and use by the ,european ,medicines ,agency of supplemental data submitted during the orphan drug application review process1 including data associated with open label extension studies and expanded access programs2 and 7iii7 each formal or informal process that the ,food and ,drug ,administration has utilized to gather external expertise on orphan drug applications1 separately considering orphan drugs for diseases or conditions that affect fewer than #bj1jjj individuals in the ,united ,states1 compared to orphan drugs for other rare diseases or conditions2 and 7,b7 provide recommendations for changes to the processes and authorities of the ,food and ,drug ,administration to facilitate development of1 and access to1 orphan drugs1 which may include-- 7i7 new tools or mechanisms to improve efforts and initiatives of the ,food and ,drug ,administration to collect and consider external expertise on orphan drug applications1 separately considering treatments for diseases or conditions that affect fewer than #bj1jjj individuals in the ,united ,states1 including with respect to processes related to application review1 including structured benefit-risk assessments1 advisory committee deliberations1 and postapproval safety monitoring2 and 7ii7 in the case of a need for input from external experts where there are limited clinical and research experts available with respect to a rare disease or condition1 setting forth alternative processes to address or resolve any conflicts of interest that would otherwise impede inclusion of input from such external experts4 7#c7 ,input ,in conducting the study and developing the report under paragraph 7#b71 the ,national ,academies shall-- 7,a7 consider input from the ,department of ,health and ,human ,services1 and any other ,federal agencies1 as appropriate2 and 7,b7 consult with relevant stakeholders1 which may include conducting public meetings and other forms of engagement1 as appropriate1 with patient groups1 health care providers1 medical professional societies1 public health experts1 medical product manufacturers1 and other entities with relevant experience4 7#d7 ,definitions ,in this subsection-- 7,a7 the term orphan drug means a drug designated as a drug for a rare disease or condition under section #ebf of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bb72 and 7,b7 the term rare disease or condition has the meaning given such term in section #ebf of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bb74 7c7 ,review process 7#a7 ,consultation with stakeholders ,section #efi7;a77#a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb-#h7;a77#a77 is amended-- 7,a7 by striking at a time and inserting at any time2 7,b7 by striking ,consistent with sections and inserting the following3 7,a7 ,in general ,consistent with sections 2 and 7,c7 by adding at the end the following3 7,b7 ,consultation with patients and patient groups 7i7 ,in general ,the ,secretary may1 as appropriate1 consult with patients and relevant patient groups impacted by the rare disease or condition1 together with at least one expert included on the list under paragraph 7#b77;,a7 and selected by such groups-- 7,i7 during the review process of an application for a new drug or biological product for a rare disease or condition or a drug or biological product that is genetically targeted2 and 7,,ii7 as applicable1 during meetings between the ,food and ,drug ,administration and sponsors prior to the submission of an application for a drug described in subclause 7,i74 7ii7 ,conflicts of interest ,for purposes of clause 7i71 to be eligible for consultation pursuant to clause 7i71 patients and relevant patient groups may not have any financial interest in the applicable drug or biological product1 and external experts shall be in compliance with applicable law1 including section #bjh of title #ah1 ,united ,states ,code4 7,c7 ,consultation with disproportionately affected communities ,to the extent an application for a new drug or biological product relates to a rare disease or condition that disproportionately affects communities of color or other historically underrepresented and vulnerable populations1 the ,secretary is encouraged to consult with patients of that subpopulation1 or one or more patient groups that represent that subpopulation4 4 7#b7 ,requiring appropriate expert consultation ,section #efi7;a77#b7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb-#h7;a77#b77 is amended-- 7,a7 in subparagraph 7,a71 by striking the second sentence2 and 7,b7 by striking subparagraph 7,b7 and inserting the following3 7,b7 ,consultation ,with respect to any application under section #eje of this ,act or section #cea of the ,public ,health ,service ,act for a drug designated under section #ebf for a rare disease or condition or a drug or biological product that is genetically targeted1 the ,secretary may1 as appropriate1 consult-- 7i7 with an expert with respect to the disease or condition referenced in the application who appears on the list described in subparagraph 7,a72 or 7ii7 if no such expert is available1 including because of conflicts of interest1 with an expert on the list described in subparagraph 7,a7 in the science of small population studies4 7,c7 ,availability at meetings ,in connection with each drug product advisory committee meeting concerning a drug or biological product for a rare disease or condition1 the ,secretary may1 as appropriate-- 7i7 include-- 7,i7 an expert in the rare disease or condition2 or 7,,ii7 if no such expert is available1 including because of conflicts of interest1 an expert in the science of small population studies2 and 7ii7 invite at least one disease or condition expert identified by the relevant patient groups to participate as a nonvoting member of the advisory committee4 4 7#c7 ,additional topic for consultation ,section #efi7;b7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb-#h7;b77 is amended-- 7,a7 in paragraph 7#f71 by striking 2 and and inserting 22 7,b7 in paragraph 7#g71 by striking the period and inserting 2 and2 and 7,c7 by adding at the end the following3 7#h7 the science of small population studies4 4