Clinical Trials in Bladder and Upper Tract Cancer – Bladder Cancer Disease States

Abstract

One of the many challenges in clinical trial design is identifying the target patient population for a particular intervention and designing a set of inclusion criteria that are accurate and verifiable. Randomized trials stratified by progression risk and presence or absence of papillary disease or carcinoma in situ (CIS) can include heterogeneous patient populations whereas single-arm trials require CIS at entry in order to determine efficacy of an experimental treatment. In muscle invasive cancer, there are multiple challenges in defining endpoints for approval for trials testing neoadjuvant or adjuvant therapies. Recent approvals of five checkpoint inhibitors and the recent approval of an FGFR inhibitor for patients with locally advanced or metastatic disease have segmented this patient population into multiple disease states depending on prior treatment and whether or not they are eligible to receive cisplatin-based chemotherapy.