Recent early phase clinical trials and trials of targeted or immune based therapy in oncology involve innovative clinical trial designs that aim to address multiple scientific objectives simultaneously and efficiently. These designs do not have straightforward power calculations for a sample size justification, and an ideal design must align with the numerous scientific questions being asked. It is imperative that statisticians involved in such protocols play a fundamental role in the concept and design of these trials as part of a larger multidisciplinary protocol team.
As the clinical field evolves, so do the design options and thus the statisticians’ role has evolved from a collaborator to a collaborator and educator who guides the clinical team on the pros and cons and the clinical implications of various designs. In this discussion, we will cover the challenges, opportunities, and responsibilities the statisticians have as collaborators and educators when working with clinicians in protocol development while aiming to address the scientific objectives and simultaneously adhere to ethical principles for medical research in human subjects.