21 U.S.C. § 360ee–1 — FDA rare neurodegenerative disease grant program

This section requires the Secretary of Health and Human Services, acting through the Commissioner of Food and Drugs, to award grants and contracts. The money goes to public and private entities to pay for research on, and development of, ways to prevent, diagnose, ease, treat, or cure amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases, in both adults and children. The funding can also cover the cost of developing and critically testing tools, methods, and processes for three things: (1) characterizing these neurodegenerative diseases and how they typically progress over time; (2) identifying molecular targets — parts of the disease process a treatment could aim at; and (3) increasing the efficiency and productivity of clinical development of therapies for these diseases. This third goal includes using "master protocols" (a single trial framework that can test multiple treatments or approaches) and adaptive or add-on clinical trial designs (trial plans that can change partway through, or add new arms, based on results), as well as efforts to build new clinical trial networks or make better use of ones that already exist.