Sec. 3202. 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, with respect to the period of fiscal years 2023 through fiscal year 2025, 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 numbers of such applications pending, approved, and for which a complete response letter has been issued by the Food and Drug Administration; the number of applications for which the sponsor requested written recommendations pursuant to section 525 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360aa ) and the number of such applications for which the sponsor received such written recommendations; 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; the number of applications for which the Secretary allowed the sponsor to rely upon data and information pursuant to section 529A of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360ff–1 ); and a description of the Food and Drug Administration’s efforts to promote best practices in the development of novel treatments for rare diseases or conditions, including— reviewer training on policies, methods, and tools related to rare diseases and conditions; 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 301(j) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 331(j) ) or 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 ). Not later than 9 months after the date of enactment of this Act, the Secretary shall publish final guidance related to the draft guidance titled, Rare Diseases: Common Issues in Drug Development , issued on February 1, 2019. The Secretary shall enter into a contract with the National Academies of Sciences, Engineering, and Medicine (referred to in this section as the National Academies ) 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 and those 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; patients with rare diseases or conditions; and patient groups that— represent patients with rare diseases or conditions; 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 National Academies to make the report under paragraph

(3)available to the public, including by posting the report on the website of the National Academies. Not later than December 31, 2023, the Secretary, 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 understanding the risks of side effects relative to the health status of the patient and the progression of the disease or condition. The Secretary 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(b) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8(b) ) is amended— in paragraph (6), by striking ; and and inserting a semicolon; in paragraph (7), by striking the period and inserting ; and ; and by adding at the end the following: the science of small population studies. . Not later than 18 months after the date of enactment of this Act, the Comptroller General of the United States shall submit to the Committee on Health, Education, Labor, and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives, a report assessing the policies, practices, and programs of the Food and Drug Administration with respect to the review of applications for approval of drugs under section 505 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 355 ) and licensing of biological products under section 351 of the Public Health Service Act ( 42 U.S.C. 262 ) intended to treat rare diseases and conditions. The report under paragraph

(1)shall— describe the activities of the Food and Drug Administration dedicated to the development and review of drugs and biological products intended to treat rare diseases and conditions under section 505 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 355 ) and section 351 of the Public Health Service Act ( 42 U.S.C. 262 ); describe challenges with developing and obtaining approval or licensure of drugs and biological products intended to treat rare diseases and conditions, such as challenges related to designing and conducting clinical trials, clinical trial subject recruitment and enrollment, study endpoints, and ensuring data quality, assessing the benefit-risk profile of drugs and biological products intended to treat rare diseases and conditions, and meeting requirements for approval or licensure; assess the effectiveness of policies and practices of the Food and Drug Administration related to the review of applications for drugs and biological products intended to treat rare diseases and conditions, including— initiatives to support the development and review of drugs and biological products intended to treat rare diseases and conditions, including initiatives related to regulatory science, clinical trial design, statistical analysis, and other relevant topics; consideration of relevant patient-focused drug development data and information, including patient experience data and the views of patients, pursuant to section 569C of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8c ); training and other efforts to ensure the expertise of personnel of the Food and Drug Administration regarding the review of applications for drugs and biological products intended to treat rare diseases and conditions; and consultations and engagement with stakeholders and external experts pursuant to section 569 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–8 ); assess the extent to which the Food and Drug Administration is applying the policies and practices described in subparagraph

(C)consistently across review divisions, and the factors that influence the extent to which such application is consistent; and include recommendations to address challenges and deficiencies identified, including recommendations to improve the effectiveness, consistency, and coordination of policies, practices, and programs of the Food and Drug Administration related to the review of applications for drugs and biological products intended to treat rare diseases and conditions. In this section, the terms rare disease or condition , rare diseases or conditions , and rare diseases and conditions have the meaning given the term rare disease or condition in section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb(a)(2) ).

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