Minimal Residual Disease Endpoint Would Aid Leukemia Drug Development, Experts Say
An international panel of hematology experts tells an FDA/ASCO workshop Feb. 27 that minimal residual disease would be a useful surrogate endpoint for assessing the effects of chronic lymphocytic leukemia therapies because it has been strongly correlated with longer-term outcomes in the disease.
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More work needs to be done to validate MRD as predictive of clinical outcomes in hematological malignancies before it can be used as the basis for accelerated approval, FDA and other stakeholders say.
Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.
Clinical Trial Diversity Planning Requires Prevalence-Based Enrollment Goals, Metrics For Gauging Success
Academic and industry experts caution against relying on historical enrollment numbers when setting new targets for under-represented populations and tout real-time monitoring dashboards, as well as a role for data monitoring committees, in assessing whether targets are being met in ongoing studies.