Recent Developments in the Use of Clinical Trials to Support Individualizing Therapies: A Regulatory Perspective

摘要: This chapter covers a broad range of issues centered around the topic optimizing therapies for individuals and role clinical trial in reaching that goal. We describe how has been increasingly relied upon to provide evidence patient level or marker differential benefit risk study design choices can change depending various objectives. consider definition prognostic predictive classifiers markers different designs used selecting evaluating enriched populations, difference between retrospective prospective approaches treatment effects among subgroups. Some examples are given illustrate issues. Two most challenging aspects identifying validating simultaneous need quantify performance characteristics (sensitivity specificity) classifier choice whether should include all comers selection positive only subgroup. The motivation these individualizing therapy is maximize benefits minimize safety risks patients.