FDA Guidance on Treatments for Rare Diseases

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The agency issued guidance regarding common issues involved with the development of treatments for rare diseases.


FDA issued guidance on Aug. 14, 2015 to assist companies developing drugs and biologics for the treatment and prevention of rare diseases. Rare diseases often involve specific issues that are complicated by a lack of medical knowledge.  FDA, therefore, developed guidance to discuss those specific issues commonly encountered in rare disease drug development.

The guidance addresses issues that companies should consider early on during drug development of treatments for rare diseases. These considerations include the description and understanding of the history of the rare disease, understanding the pathophysiology of the disease, nonclinical pharmacotoxicology considerations to support clinical investigations, outcome assessment, standards for establishing efficacy and safety, and manufacturing considerations.


FDA states in the guidance that, “many rare disorders are serious conditions with no approved treatments, leaving substantial unmet medical needs for patients. FDA recognizes that rare diseases are highly diverse and is committed to helping sponsors create successful drug development programs that address the particular challenges posed by each disease.”

Source: FDA