Clinical Trial Design for Rare Disease Treatments Should Include Patient Voice

Patient voice is a key ingredient for both sponsors and regulators in developing new therapeutics for rare diseases, according to the FDA.

Patient listening sessions are crucial in mid- and late-stage drug development for these disorders, FDA officials said at a public meeting sponsored by the Margolis Center for Health Policy at Duke University. Their lived experiences can shape entire clinical trials, influence approval decisions and guide long-term follow-up plans.

By listening to the experiences and concerns of patients with rare diseases as well as their caregivers and families, the FDA can get a better picture of the most troublesome symptoms; the daily physical, social and economic burdens that might affect clinical trial participation; and the risks patients are willing to accept for a treatment that could confer benefit. These are often different from the experiences of patients with more common disorders and sometimes quite surprising, Stein said.

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