FDA Launches the Rare Disease Framework Initiative to Develop a Holistic, Fit-For-Purpose Framework for Rare Disease Drug Development

FDA announced its Rare Disease Framework Initiative for developers of drugs and biologics for rare diseases. The agency says the planned framework will consider disease differences, patient needs, product type and evidence attainable in small populations, with input from patients, advocates and industry.

Why it matters: FDA's planned framework includes patient input at defined milestones and novel statistical approaches for rare disease development.