Getting With The PRO-gram: Making Patient-Reported Outcomes Work
This article was originally published in RPM Report
Executive Summary
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?
You may also be interested in...
Unvalidated PROs In Rare Diseases: US FDA May Have To ‘Work Through’ Them, CBER’s Marks Says
Given the challenges of validating patient-reported outcome instruments specific to very rare diseases, agency is probably going to have to accept the use of unvalidated measures in some cases, Peter Marks tells American Society of Gene and Cell Therapy’s annual meeting.
Patient-Reported Outcome Data Helps Dermira’s Hyperhidrosis Drug Qbrexza Win US FDA Nod
Co-primary endpoint was Axillary Sweating Daily Diary, a novel PRO instrument developed by Dermira with FDA input, assessed sweating severity and impact of sweating on a person’s daily activities; results complemented quantitative measure of sweat production in Phase III trials.
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.