FDA allows unique endpoint in rare disease study www.pharmavoice.com/news/fda-uni... via @PharmaVoice Using hospitalization as a primary outcome measure is another demonstration of regulators granting flexibility in clinical trial design for rare diseases.
FDA allows unique endpoint in rare disease study
Global regulators are allowing the company to use hospitalization as the primary efficacy endpoint in its registration trial for a rare neuromuscular disorder.
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