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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...

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