Achondroplasia Trial Design: US FDA Panel Moves Patient-Centered Development To The Forefront
Advisory committee’s recommendations on secondary endpoints for pivotal studies reflects patient and caregiver comments during the open public hearing about the disease’s clinical complications, including ear infections and sleep apnea, and challenges performing activities of daily living.
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Baby Steps To Real-World Evidence Of Efficacy: External Controls Gain Popularity In Rare Disease Trials
The Pink Sheet takes a closer look at recent clinical announcements showing how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.
Recent and upcoming US FDA advisory committee meetings and a summary of topics covered.
BioMarin's vosoritide was to be discussed in closed session during advisory committee on drugs for most common form of dwarfism; public session would have addressed general trial design issues, including endpoints, subpopulations and study duration, but inclement weather in the Washington, DC area postponed meeting.