Sec. 703. Improving the treatment of rare diseases and conditions
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Not later than September 30, 2026, the Secretary shall submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate a report summarizing the activities of the Food and Drug Administration related to designating drugs under section 526 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bb ) for a rare disease or condition and approving such drugs under section 505 of such Act ( 21 U.S.C. 355 ) or licensing such drugs under section 351 of the Public Health Service Act ( 42 U.S.C. 262 ), including— the number of applications for such drugs under section 505 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 355 ) or section 351 of the Public Health Service Act ( 42 U.S.C. 262 ) received by the Food and Drug Administration, the number of such applications accepted and rejected for filing, and the number of such applications pending, approved, and disapproved by the Food and Drug Administration; a description of trends in drug approvals for rare diseases and conditions across review divisions at the Food and Drug Administration; the extent to which the Food and Drug Administration is consulting with external experts pursuant to section 569(a)(2) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8(a)(2) ) on topics pertaining to drugs for a rare disease or condition, including how and when any such consultation is occurring; and the Food and Drug Administration’s efforts to promote best practices in the development of novel treatments for rare diseases, including— reviewer training on rare disease-related policies, methods, and tools; and new regulatory science and coordinated support for patient and stakeholder engagement.
The Secretary shall make the report under paragraph
(1)available to the public, including by posting the report on the website of the Food and Drug Administration. Nothing in this subsection shall be construed to authorize the disclosure of information that is prohibited from disclosure under section 1905 of title 18, United States Code, or subject to withholding under paragraph
(4)of section 552(b) of title 5, United States Code (commonly referred to as the Freedom of Information Act ). The Secretary of Health and Human Services shall enter into a contract with an appropriate entity to conduct a study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union, including— flexibilities, authorities, or mechanisms available to regulators in the United States and the European Union specific to rare diseases or conditions; the consideration and use of supplemental data submitted during review processes in the United States and the European Union, including data associated with open label extension studies and expanded access programs specific to rare diseases or conditions; an assessment of collaborative efforts between United States and European Union regulators related to— product development programs under review; policies under development recently issued; and scientific information related to product development or regulation; and recommendations for how Congress can support collaborative efforts described in subparagraph (C). The contract under paragraph
(1)shall provide for consultation with relevant stakeholders, including— representatives from the Food and Drug Administration and the European Medicines Agency; rare disease or condition patients; and patient groups that— represent rare disease or condition patients; and have international patient outreach. The contract under paragraph
(1)shall provide for, not later than 2 years after the date of entering into such contract— the completion of the study under paragraph (1); and the submission of a report on the results of such study to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate. The contract under paragraph
(1)shall provide for the appropriate entity referred to in paragraph
(1)to make the report under paragraph
(3)available to the public, including by posting the report on the website of the appropriate entity. Not later than December 31, 2023, the Secretary of Health and Human Services, acting through the Commissioner of Food and Drugs, shall convene one or more public meetings to solicit input from stakeholders regarding the approaches described in paragraph (2). The public meeting or meetings under paragraph
(1)shall address approaches to increasing and improving engagement with rare disease or condition patients, groups representing such patients, rare disease or condition expert s, and experts on small population studies, in order to improve the understanding with respect to rare diseases or conditions of— patient burden; treatment options; and side effects of treatments, including— comparing the side effects of treatments; and understanding the risks of side effects relative to the health status of the patient and the progression of the disease or condition. The Secretary of Health and Human Services shall establish a public docket to receive written comments related to the approaches addressed during each public meeting under paragraph (1). Such public docket shall remain open for 60 days following the date of each such public meeting. Not later than 180 days after each public meeting under paragraph (1), the Commissioner of Food and Drugs shall develop and publish on the website of the Food and Drug Administration a report on— the approaches discussed at the public meeting; and any related recommendations. Section 569(a)(2) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8(a)(2) ) is amended by adding at the end the following: The external experts on the list maintained pursuant to subparagraph
(A)may include experts on the science of small population studies. . The Comptroller General of the United States shall conduct a study on the use of Food and Drug Administration mechanisms and tools to ensure that patient and physician perspectives are considered and incorporated throughout the processes of the Food and Drug Administration— for approving or licensing under section 505 of the Federal Food, Drug, or Cosmetic Act ( 21 U.S.C. 355 ) or section 351 of the Public Health Service Act ( 42 U.S.C. 262 ) 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 in making any determination related to such a drug’s approval, including assessment of the drug’s— safety or effectiveness; or postapproval safety monitoring. The study under paragraph
(1)shall— identify and compare the processes that the Food and Drug Administration has formally put in place and utilized to gather external expertise (including patients, patient groups, and physicians) related to applications for rare diseases or conditions; examine tools or mechanisms to improve efforts and initiatives of the Food and Drug Administration to collect and consider such external expertise with respect to applications for rare diseases or conditions throughout the application review and approval or licensure processes, including within internal benefit-risk assessments, advisory committee processes, and postapproval safety monitoring; and examine processes or alternatives to address or resolve conflicts of interest that impede the Food and Drug Administration in gaining external expert input on rare diseases or conditions with a limited set of clinical and research experts. Not later than 2 years after the date of enactment of this Act, the Comptroller General of the United States shall— complete the study under paragraph (1); submit a report on the results of such study to the Congress; and include in such report recommendations, if appropriate, for changes to the processes and authorities of the Food and Drug Administration to improve the collection and consideration of external expert opinions of patients, patient groups, and physicians with expertise in rare diseases or conditions. In this section, the term rare disease or condition has the meaning given such term in section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bb(a)(2) ).
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- 21 USC 360bbb–8(a)(2)
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Sec. 703
Improving the treatment of rare diseases and conditions
Cite21 USC 360bbb–8(a)(2)
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