II 117th CONGRESS 2d Session S. 4071 IN THE SENATE OF THE UNITED STATES April 7, 2022 Mr. Casey (for himself and Mr. Scott of South Carolina ) introduced the following bill; which was read twice and referred to the Committee on Health, Education, Labor, and Pensions A BILL To amend the Federal Food, Drug, and Cosmetic Act to improve the treatment of rare diseases and conditions, and for other purposes.

1. Short title This Act may be cited as the Helping Experts Accelerate Rare Treatments Act of 2022 . 2. Improving the treatment of rare diseases and conditions (a) Annual report on orphan drug program Subchapter B of chapter V of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360aa et seq. ) is amended by adding at the end the following new section: 529B. Annual report on orphan drug program (a) In general Not later than 1 year after the date of enactment of the Helping Experts Accelerate Rare Treatments Act of 2022 , and not less frequently than annually thereafter, the Secretary shall submit to Congress a report summarizing the activities of the Food and Drug Administration related to designating drugs under section 526 for a rare disease or condition and approving such drugs under section 505 of this Act or licensing such drugs under section 351 of the Public Health Service Act, including— (1) the number of applications for such drugs under section 505 of this Act and section 351 of the Public Health Service Act received by the Food and Drug Administration, the number of such applications accepted for filing, the number of such applications rejected for filing, and the numbers of such applications pending, approved, and disapproved by the Food and Drug Administration, arrayed by the review division assigned to the application; (2) the size of the affected population in the United States of each disease or condition addressed by an application described in paragraph (1), assessed taking into consideration the documentation required by section 316.20 of title 21, Code of Federal Regulations (or any successor regulation) and other relevant information available to the Secretary; and (3) the extent of consultation with stakeholders and external experts pursuant to section 569. (b) 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 drugs. (c) Public availability The Secretary shall make each report under subsection (a) available to the public, including by posting the report on the website of the Food and Drug Administration. . (b) Study on European Union safety and efficacy reviews of drugs for rare diseases and conditions (1) In general Not later than 1 year after the date of enactment of this Act, the Secretary of Health and Human Services shall seek to enter into an agreement with the National Academies of Sciences, Engineering, and Medicine (referred to in this section as the National Academies ) to examine and report on European Union safety and efficacy reviews of drugs for rare diseases and conditions, 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 reviews, and opportunities to improve such reviews in the United States. (2) Content The report developed under paragraph (1) shall— (A) assess and evaluate, with respect to drugs for rare diseases and conditions— (i) any flexibilities, authorities, or mechanisms available in the European Union; (ii) consideration and use by the European Medicines Agency of supplemental data submitted during the orphan drug application review process, including data associated with open label extension studies and expanded access programs; and (iii) each formal or informal process that the Food and Drug Administration has utilized to gather external expertise on orphan drug applications, separately considering orphan drugs for diseases or conditions that affect fewer than 20,000 individuals in the United States, compared to orphan drugs for other rare diseases or conditions; and (B) provide recommendations for changes to the processes and authorities of the Food and Drug Administration to facilitate development of, and access to, orphan drugs, which may include— (i) new tools or mechanisms to improve efforts and initiatives of the Food and Drug Administration to collect and consider external expertise on orphan drug applications, separately considering treatments for diseases or conditions that affect fewer than 20,000 individuals in the United States, including with respect to processes related to application review, including structured benefit-risk assessments, advisory committee deliberations, and postapproval safety monitoring; and (ii) 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 condition, setting forth alternative processes to address or resolve any conflicts of interest that would otherwise impede inclusion of input from such external experts. (3) Input In conducting the study and developing the report under paragraph (2), the National Academies shall— (A) consider input from the Department of Health and Human Services, and any other Federal agencies, as appropriate; and (B) consult with relevant stakeholders, which may include conducting public meetings and other forms of engagement, as appropriate, with patient groups, health care providers, medical professional societies, public health experts, medical product manufacturers, and other entities with relevant experience. (4) Definitions In this subsection— (A) the term orphan drug means a drug designated as a drug for a rare disease or condition under section 526 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bb ); and (B) the term rare disease or condition has the meaning given such term in section 526 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bb ). (c) Review process (1) Consultation with stakeholders Section 569(a)(1) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8(a)(1) ) is amended— (A) by striking at a time and inserting at any time ; (B) by striking Consistent with sections and inserting the following: (A) In general Consistent with sections ; and (C) by adding at the end the following: (B) Consultation with patients and patient groups (i) In general The Secretary may, as appropriate, consult with patients and relevant patient groups impacted by the rare disease or condition, together with at least one expert included on the list under paragraph (2)(A) and selected by such groups— (I) 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 targeted; and (II) as applicable, during meetings between the Food and Drug Administration and sponsors prior to the submission of an application for a drug described in subclause (I). (ii) Conflicts of interest For purposes of clause (i), to be eligible for consultation pursuant to clause (i), patients and relevant patient groups may not have any financial interest in the applicable drug or biological product, and external experts shall be in compliance with applicable law, including section 208 of title 18, United States Code. (C) 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 populations, the Secretary is encouraged to consult with patients of that subpopulation, or one or more patient groups that represent that subpopulation. . (2) Requiring appropriate expert consultation Section 569(a)(2) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8(a)(2) ) is amended— (A) in subparagraph (A), by striking the second sentence; and (B) by striking subparagraph (B) and inserting the following: (B) Consultation With respect to any application under section 505 of this Act or section 351 of the Public Health Service Act for a drug designated under section 526 for a rare disease or condition or a drug or biological product that is genetically targeted, the Secretary may, as appropriate, consult— (i) with an expert with respect to the disease or condition referenced in the application who appears on the list described in subparagraph (A); or (ii) if no such expert is available, including because of conflicts of interest, with an expert on the list described in subparagraph (A) in the science of small population studies. (C) Availability at meetings In connection with each drug product advisory committee meeting concerning a drug or biological product for a rare disease or condition, the Secretary may, as appropriate— (i) include— (I) an expert in the rare disease or condition; or (II) if no such expert is available, including because of conflicts of interest, an expert in the science of small population studies; and (ii) invite at least one disease or condition expert identified by the relevant patient groups to participate as a nonvoting member of the advisory committee. . (3) Additional topic for consultation Section 569(b) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8(b) ) is amended— (A) in paragraph (6), by striking ; and and inserting ; ; (B) in paragraph (7), by striking the period and inserting ; and ; and (C) by adding at the end the following: (8) the science of small population studies. .