There has been growing experience and interest in the use of master protocol designs within oncology drug development. Such an approach enables efficiencies in drug development by improving recruitment and providing treatment options for patients in biomarker-defined populations. Designs, such as ‘umbrella’ trials are used to evaluate multiple investigational drugs or drug combinations in a single cancer type while ‘basket’ trials are used to evaluate a single investigational drug or drug combination in multiple cancer populations. ‘Platform’ trials incorporate both ‘umbrella’ and ‘basket’ trial design aspects. Besides advantages, such trial designs can also introduce additional levels of complexity in terms of study design, conduct and analysis. A few recent publications have reviewed this novel approach (Simon, 2017, Woodcock & Lavange, 2017 and Renfro & Sergent, 2017) and the FDA have recently published a draft guidance for industry to aid the use of such designs in drug development (FDA Guidance, 2018).
Questions: What are the challenges in adopting Master Protocol? What is the current state of statistical methodologies applied to such trial designs? What new statistical methodologies can be explored?