21 U.S.C. § 360aa: Recommendations for investigations of drugs for rare diseases or conditions

it may be approved for such disease or condition under section 355 of this title , or if the drug is a biological product, it may be licensed for such disease or condition under section 262 of title 42 . The Secretary shall by regulation promulgate procedures for the implementation of subsection (a). Except as provided in subsection (b), this Act and the amendments made by this Act [amending this section, sections 360bb, 360cc, and 360ee of this title, and sections 295g–1 and 6022 of Title 42, The Public Health and Welfare, and enacting provisions set out as notes under section 301 of this title and section 236 of Title 42 ] shall take effect October 1, 1985 . The amendments made by sections 2, 3, and 6(a) [amending this section and sections 360bb and 360cc of this title] shall take effect on the date of the enactment of this Act [ Aug. 15, 1985 ]. The amendment made by section 6(b) [amending section 6022 of Title 42 ] shall take effect October 19, 1984 . The amendments made by section 7 [amending section 295g–1 of Title 42 ] shall take effect October 1, 1984 and shall cease to be in effect after September 30, 1985 .” determining eligibility of participants for such program; and developing and implementing a process for applying to, and participating in, such a program. novel endpoints developed through the pilot program established under this section; and as appropriate, the use of real world evidence and real world data to support the validation of efficacy endpoints, including surrogate and intermediate endpoints, for rare diseases. Not later than September 30, 2026 , the Secretary 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 describing the completed and ongoing activities in the pilot program established under this section and public workshops described in subsection (b). Not later than September 30, 2027 , the Secretary 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 describing the outcomes of the pilot program established under this section. Not later than September 30, 2027 , the Secretary shall issue guidance describing best practices and strategies for development of efficacy endpoints, including surrogate and intermediate endpoints, for rare diseases. The Secretary may not accept any new application or request to participate in the program established by this section on or after October 1, 2027 .” foster the development of safe and effective drugs that improve or extend, or both, the lives of people living with amyotrophic lateral sclerosis and other rare neurodegenerative diseases; and facilitate access to investigational drugs for amyotrophic lateral sclerosis and other rare neurodegenerative diseases. identify appropriate representation from within the Food and Drug Administration to be responsible for implementation of such action plan; interactions and collaboration between the Food and Drug Administration, including the review centers thereof, and stakeholders including patients, sponsors, and the external biomedical research community; consideration of cross-cutting clinical and regulatory policy issues, including consistency of regulatory advice and decisionmaking; identification of key regulatory science and policy issues critical to advancing development of safe and effective drugs; and enhancement of collaboration and engagement of the relevant centers and offices of the Food and Drug Administration with other operating divisions within the Department of Health and Human Services, the Partnership, and the broader neurodegenerative disease community; and be subject to revision, as determined appropriate by the Secretary of Health and Human Services.” The Commissioner of Food and Drugs shall establish within the Food and Drug Administration a review group which shall recommend to the Commissioner of Food and Drugs appropriate preclinical, trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of rare diseases: Provided , That the Commissioner of Food and Drugs shall appoint individuals employed by the Food and Drug Administration to serve on the review group: Provided further , That members of the review group shall have specific expertise relating to the development of articles for use in the prevention, diagnosis, or treatment of rare diseases, including specific expertise in developing or carrying out clinical trials. The Commissioner of Food and Drugs shall establish within the Food and Drug Administration a review group which shall recommend to the Commissioner of Food and Drugs appropriate preclinical, trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of neglected diseases of the developing world: Provided , That the Commissioner of Food and Drugs shall appoint individuals employed by the Food and Drug Administration to serve on the review group: Provided further , That members of the review group shall have specific expertise relating to the development of articles for use in the prevention, diagnosis, or treatment of neglected diseases of the developing world, including specific expertise in developing or carrying out clinical trials: Provided further , That for the purposes of this section the term ‘neglected disease of the developing world’ means a tropical disease, as defined in section 524(a)(3) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360n(a)(3) ). submit, not later than 1 year after the date of the establishment of review groups under subsections (a) and (b), a report to Congress that describes both the findings and recommendations made by the review groups under subsections (a) and (b); issue, not later than 180 days after submission of the report to Congress under paragraph (1), guidance based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world; and develop, not later than 180 days after submission of the report to Congress under paragraph (1), internal review standards based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world.” there are many diseases and conditions, such as Huntington’s disease, myoclonus, ALS (Lou Gehrig’s disease), Tourette syndrome, and muscular dystrophy which affect such small numbers of individuals residing in the United States that the diseases and conditions are considered rare in the United States; adequate drugs for many of such diseases and conditions have not been developed; drugs for these diseases and conditions are commonly referred to as ‘orphan drugs’; because so few individuals are affected by any one rare disease or condition, a pharmaceutical company which develops an orphan drug may reasonably expect the drug to generate relatively small sales in comparison to the cost of developing the drug and consequently to incur a financial loss; there is reason to believe that some promising orphan drugs will not be developed unless changes are made in the applicable Federal laws to reduce the costs of developing such drugs and to provide financial incentives to develop such drugs; and it is in the public interest to provide such changes and incentives for the development of orphan drugs.”