FDA Guidance Targets Rare Disease Drug Development for Industry

The FDA finalized guidance on considerations for manufacturers of drugs and biological products treating rare diseases when conducting development programs.

The Food and Drug Administration’s guidance, issued Friday, addresses aspects of rare disease drug and biological product development to support a proposed clinical investigation, including nonclinical pharmacology and toxicology; trial design and endpoint considerations to ensure quality and interpretability of data; and standard of evidence to establish safety and effectiveness.

“Many rare diseases are serious conditions with no approved treatments, leaving substantial unmet medical need for patients,” the agency said in the guidance. “FDA recognizes that rare diseases are ...