ABSTRACT
Increasingly, tumors are defined based on molecular subtypes, creating small subgroups that present challenges for enrollment. If molecular subtypes are shared across histologies, they may be pooled into basket trials, facilitating development of agents targeted at small molecularly defined subgroups. To date, basket trials have been used either for exploratory early development, or for confirmation only in cases where a transformational benefit is anticipated. This chapter discusses a confirmatory basket trial design that is generally applicable to all beneficial therapies. Confirmatory basket trials may improve access to therapy for both rare diseases and defined patient subpopulations in cancer by creating cost effective development options, and may enhance the ability of health authorities to evaluate the risk and benefit of targeted agents designed for these indications.
