Sec. 4. Rare Disease and Condition Drug Advisory Committee
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Subchapter B of chapter V of the Federal Food, Drug, and Cosmetic Act is further amended by inserting after section 529B of such Act, as inserted by section 3, the following new section: The Secretary shall establish and maintain a committee, to be known as the Rare Disease and Condition Drug Advisory Committee (in this section referred to as the Advisory Committee ). The Advisory Committee shall advise the Secretary on issues associated with development of therapies to treat rare diseases or conditions.
In advising the Secretary, the Advisory Committee may address issues including— modified or new regulatory pathways to support review of therapies; clinical trial design needs, including development of innovative approaches to clinical trials; qualifications of biomarkers or other drug development tools for use in reviews; modified or new standards to support the review of already marketed drugs being evaluated for repurposing to treat a rare disease or condition; and issues— that pertain to an application for approval of a therapy to treat a rare disease or condition; and with respect to which a review division has requested that the Advisory Committee provide advice.
The Advisory Committee shall consist of— not more than 15 members appointed by the Secretary in accordance with paragraph (2); and the nonvoting ex officio members under paragraph (3). Members of the Advisory Committee appointed pursuant to paragraph (1)(A) shall serve as special Government employees (as defined in section 202(a) of title 18, United States Code). To be eligible for appointment pursuant to paragraph (1)(A), an individual shall— be eligible to serve as special Government employee (as defined in section 202(a) of title 18, United States Code); and have expertise in the fields of public policy, law, regulatory policy, economics, patient-focused product development, or patient advocacy.
Of the members of the Advisory Committee appointed pursuant to paragraph (1)(A)— up to 10 shall be selected from among experts in the disciplines relevant to the activities of the Intercenter Institute on Rare Diseases and Conditions, to include at least one expert in each of— rare disease product development; conducting clinical trials with respect to rare diseases and conditions, including with respect to very small patient populations; rare disease and condition natural history and related studies; health economics pertaining to the development of medical products for rare diseases or conditions; manufacturing and related needs associated with medical products for rare diseases or conditions; and patient experience data collection; and up to 5 shall be selected from the public, to include— at least 4 individuals who are representatives of the rare disease patient community; at least one individual who is directly impacted by a rare disease or condition; and at least one person who serves as a family caregiver to a person diagnosed with a rare disease or condition.
The nonvoting ex officio members of the Advisory Committee under paragraph (1)(B) shall consist of the following: The Secretary (or the Secretary’s designee). The Director of the Intercenter Institute on Rare Diseases and Conditions. The Director of the Center for Biologics Evaluation and Research (or the Director’s designee). The Director of the Center for Drug Evaluation and Research (or the Director’s designee). The Director of the Center for Devices and Radiological Health (or the Director’s designee).
The Director of the National Center for the Advancing Translational Sciences of the National Institutes of Health (or the Director’s designee). The Administrator of the Centers for Medicare & Medicaid Services (or the Administrator’s designee). Any additional officers or employees of the Department of Health and Human Services as the Secretary determines necessary for the Advisory Committee to effectively carry out its functions. The Chair of the Advisory Committee shall be the Director of the Intercenter Institute for Rare Diseases and Conditions.
The term of a member of the Advisory Committee appointed pursuant to paragraph (1)(A) shall be 4 years, except that any member appointed to fill a vacancy in an unexpired term shall be appointed for the remainder of that term. A member appointed pursuant to paragraph (1)(A) may continue serving as a member of the Advisory Committee for up to 180 days after the expiration of that member’s term if a successor has not been appointed. A member of the Advisory Committee who has been appointed pursuant to paragraph (1)(A) for a term of 4 years may not be reappointed to serve as a member of the Advisory Committee before the date that is 2 years after the date of expiration of that member’s term.
A majority of the appointed members of the Advisory Committee shall constitute a quorum for the conduct of business. .