(1) to characterize such neurodegenerative diseases and their natural history;
(2) to identify molecular targets for such neurodegenerative diseases; and
(3) to increase efficiency and productivity of clinical development of therapies, including through—(A) the use of master protocols and adaptive and add-on clinical trial designs; and
(B) efforts to establish new or leverage existing clinical trial networks.
Structure US Code
CHAPTER 9— FEDERAL FOOD, DRUG, AND COSMETIC ACT
SUBCHAPTER V— DRUGS AND DEVICES
Part B— Drugs for Rare Diseases or Conditions
§ 360aa. Recommendations for investigations of drugs for rare diseases or conditions
§ 360bb. Designation of drugs for rare diseases or conditions
§ 360cc. Protection for drugs for rare diseases or conditions
§ 360dd. Open protocols for investigations of drugs for rare diseases or conditions
§ 360ee. Grants and contracts for development of drugs for rare diseases and conditions
§ 360ee–1. FDA rare neurodegenerative disease grant program
§ 360ff. Priority review to encourage treatments for rare pediatric diseases