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Patients’ Preferred Endpoints May Not Satisfy Regulatory Needs, US FDA Says

 
• By Sue Sutter

Just because a measure is relevant to the patient community does not mean it is capable of capturing meaningful change in a clinical trial, US FDA officials say at a meeting on mitochondrial disease drug development.

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Sponsors may face patient community pressure to use an outcome assessment not well-suited to detecting clinically meaningful change. • Source: Shutterstock

Sponsors may need to resist pressure from patient advocates who want to see a particular clinical outcome assessment used in a drug development program if that that instrument is not well-suited to capturing meaningful change in a trial.

At a 6 September meeting on drug development for primary mitochondrial disease, US Food and Drug Administration officials said that although it is important for sponsors to incorporate the patient voice throughout drug development, including endpoint selection,

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