Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases Guidance for Industry

Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases Guidance for Industry

1. Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases - Guidance for Industry

This guidance is intended for sponsors of drugs and biological products are considering submitting requests for orphan-drug designation for their drugs under section 526 of the Federal Food, Drug, and Cosmetic Act (FD&C Act). The Food and Drug Administration (FDA) does not expect to grant any additional orphan-drug designation to drugs for pediatric subpopulations of common diseases (i.e., diseases or conditions with an overall prevalence of 200,000 or greater). Pediatric-subpopulation designations that have already been granted will not be affected by this change.

2. Microdose Radiopharmaceutical Diagnostic Drugs: Nonclinical Study Recommendations - Guidance for Industry 

This guidance is intended to assist sponsors of microdose radiopharmaceutical diagnostic drugs on the nonclinical studies recommended to support human clinical trials and marketing applications. It discusses how to refine nonclinical study recommendations for this class of drug given its unique characteristics. This guidance also is intended to help sponsors facilitate the timely conduct of clinical trials, reduce the use of animals with the 3R (reduce/refine/replace) principles, and reduce the use of drug development resources