The US Food and Drug Administration (FDA) finalized guidance on drug development programs for rare diseases, eliminating a section on natural history and providing additional considerations related to patient and caregiver input and pediatric populations.
The final guidance, issued on 26 December 2023, is aimed at assisting orphan drug sponsors and covers nonclinical pharmacology and toxicology, trial design and endpoint selection, the evidence standard for establishing safety and effectiveness, and drug manufacturing issues.
“FDA recognizes that rare diseases are highly diverse with varying prevalence, rates of progression, and degrees of heterogeneity that can affect both clinical manifestations and disease courses even within a condition. Further complexity is added depending on what is known about a disease’s natural history and pathophysiology. As such, no one program can be designed exactly like another,” agency officials wrote in the final guidance…
