Vimizim Panel’s Desire For Quality-Of-Life Data Runs Into Regulatory Reality
FDA advisory committee overwhelmingly endorses BioMarin’s elosulfase alfa for Morquio A syndrome based on a walking endpoint but says future studies in the rare disease should include QoL assessments, prompting an agency rep to caution that such instruments need to be fully validated for regulatory purposes.
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When FDA’s guidance document on patient-reported outcomes was finalized five years ago, it was lauded as a milestone event for an industry interested in advancing patient-centered drug development. But implementation of the guidance hasn’t gone the way some had hoped – and actually may be dissuading sponsors from pursuing PRO labeling claims. Can FDA’s patient-centered drug development initiative help?
FDA questioned the clinical meaningfulness of BioMarin’s endpoint for its Morquio A syndrome treatment, but the company declined to conduct FDA’s suggested trial. Unmet need convinced the agency to approve Vimizim on available data while it still seeks more suitable measures of efficacy.
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