Sec. 3. Intercenter Institute on Rare Diseases and Conditions
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The first sentence of section 1014(a) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 399g(a) ) is amended by inserting , at least one of which shall be focused on rare diseases and conditions before the period at the end of the sentence. Subsection
(c)of section 1014 of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 399g ) is amended to read as follows: Not later than the date that is 1 year after the date of enactment of the Speeding Therapy Access Today Act of 2021 , the Secretary shall establish, in accordance with this section and section 529B, an Institute under subsection
(a)focused on rare diseases and conditions, to be known as the Intercenter Institute on Rare Diseases and Conditions. . Subchapter B of chapter V of the Federal Food, Drug, and Cosmetic Act (relating to drugs for rare diseases or conditions) is amended by inserting after section 529A of such Act ( 21 U.S.C. 360ff–1 ) the following new section: In addition to carrying out activities listed in section 1014(a), the Intercenter Institute on Rare Diseases and Conditions shall— serve as the Food and Drug Administration’s coordinating office for engagement with rare disease and condition stakeholders, complementing but not supplanting engagement activities between stakeholders and the review divisions; build, within the Food and Drug Administration, knowledge and understanding associated with the review of medical products to treat rare diseases and conditions, including advancements in trial design, statistical analysis, regulatory science, product manufacturing, and other topics as determined by the Secretary; implement cross-center rare disease and condition-focused meetings and policy development; coordinate rare disease and condition-specific regulatory science initiatives; facilitate stakeholder engagement to the external community and international regulatory agencies on rare disease and condition product development; establish and implement the Accelerating Lifesavings Therapies in Treating Ultra-rare Disease Entities Program under subsection (b); and establish and carry out the rare disease and condition third-party payor program under subsection (d). The Intercenter Institute shall establish and implement a program, to be known as the Accelerating Lifesavings Therapies in Treating Ultra-rare Disease Entities Program, to identify and make recommendations to address current and emerging regulatory science and public policy challenges associated with developing medical products to treat rare diseases or conditions in an individual or very small populations. The program under paragraph
(1)shall focus on issues including— manufacturing standards for therapies described in such paragraph, including in non-industry settings; trial designs and metrics; regulatory flexibilities for abbreviated toxicology studies, overlapping animal studies, and patient dosing; regulatory science, chemistry, manufacturing, and other needs associated with developing such therapies; and other issues as determined by the Secretary. Not later than 180 days after the date of enactment of this section, the Intercenter Institute shall convene a meeting of stakeholders from the rare disease community, including patients, caregivers, product manufacturers, third-party payors, and others, to consider potential amendments to labels for medical products to treat rare diseases or conditions approved pursuant to a pathway under section 506. Not later than 90 days after the date of the meeting under paragraph (1), the Secretary shall issue guidance to propose changes to how the labels of medical products to treat rare diseases or conditions demonstrate clinical benefits and reflect relevant scientific data including surrogate endpoints. The Intercenter Institute shall establish and carry out a voluntary rare disease and condition early third-party payor feedback program— to inform coverage policies for rare disease therapies; and to inform clinical trial design, patient engagement, and other data collections. The program under paragraph
(1)shall— facilitate voluntary communication between sponsors of medical products to treat rare diseases and conditions and third-party payors; and require participation of the Centers for Medicare & Medicaid Services with representation from— the Center for Medicare; and the Center for Medicaid and CHIP Services. The Intercenter Institute shall— on an annual basis, submit a report to that Congress on— the participation within the program under paragraph (1); and the impacts of the program under paragraph (1); and post each such report on the public website of the Intercenter Institute. Following the approval, clearance, or authorization by the Food and Drug Administration of a medical product to treat a rare disease or condition, the Secretary shall issue a bulletin to State Medicaid directors containing information to help inform coverage decisions on the product by State Medicaid and Children’s Health Insurance programs. In this section, the terms Intercenter Institute on Rare Diseases and Conditions and Intercenter Institute refer to the Intercenter Institute on Rare Diseases and Conditions established pursuant to section 1014. .
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- 21 USC 360ff–1
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