Patient-Reported Outcomes: Open-Label Trials Can Be Designed To Minimize Bias, US FDA Says
With the expectation for increasing amounts of patient experience data comes the need for the FDA and sponsors to consider, and account for, the degree to which open-label bias influences oncology trial PROs; agency staff suggest trial design elements and analytic approaches for dealing with bias at a meeting on cancer clinical outcomes assessments.
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Build Patient-Reported Outcomes Into Cancer Drug Dose Optimization, US FDA Says
In exploratory FDA analyses, PRO data appeared more sensitive at detecting an exposure-toxicity relationship for an oral small molecule cancer drug than clinician-reported data; Project Optimus representative dispels industry concerns that FDA wants firms to find the 'mythical' optimal dose.
Cancer Trials: Patient-Reported Outcomes Should Measure Five Core Concepts, US FDA Says
Heterogeneity in PRO assessment strategies has lessened the regulatory utility of such data from cancer trials, the agency says; new draft guidance recommends systematic assessment of a core set of PROs in registrational studies using fit-for-purpose tools and includes examples of acceptable instruments.
Sarepta’s DMD Gene Therapy Adcomm Likely To Focus On Dystrophin As A Surrogate Endpoint
Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.