Innovative statistical methodologies to subgroup analysis in clinical trials

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FDA PA 19‐306 Bierer, Barbara E., M.D. Innovative statistical methodologies to subgroup analysis in clinical trials Project Summary The Multi‐Regional Clinical Trial (MRCT) Center of Brigham and Women's Hospital and Harvard (MRCT Center) is a research and policy center created to address the conduct, oversight, ethics and regulatory environment of clinical trials, with a focus on multinational trials. To do the work, we function as a independent convener to engage diverse stakeholders from industry, CROs, academia, patients and patient advocacy groups, non‐profit organizations, and global regulatory agencies to address problems in rigor and integrity of trials. In this proposal, we propose to convene a public conference of statisticians, clinical trialists, regulators, and patient/patient advocates to discuss innovative statistical methodologies to subgroup analysis in clinical trials. This conference is highly relevant to the FDA's efforts to promote inclusion of individuals of diverse backgrounds and characteristics in clinical trials and to considerations of regional differences in multi‐national trials. Different subgroups appear to necessitate different approaches. That is, continuous (e.g. age), categorical (e.g. sex) , and overlapping (e.g. co‐morbidity, polypharmacy) variables differ, and each may command different statistical analyses. The conference will address traditional approaches, Bayesian methods, and other innovative models and explore the advantages and limitations of each. Further, the role of visualization and graphical representation will be discussed. Not all subgroup analyses must be performed at the level of the individual clinical trial; analyses of post‐approval observational data may illuminate important differences across subgroups that were not discoverable during product development. For the clinician and for the patient, the important factor is not the average treatment effect, but rather whether the benefit of the product or intervention will outweigh the risks for the individual likely to take the product.