Well-designed clinical trials in urological oncology help to guide treatment decisions and aid in counselling patients, ultimately serving to improve outcomes. Since the term evidence-based medicine was first used by Gordon Guyatt in 1991, a renewed emphasis on methodology, transparent trial design and study reporting has helped to improve clinical research and in turn, the landscape of medical literature. Novel clinical trial designs (including multi-arm, multistage trials, basket and umbrella studies and research from big data sources, such as electronic health records, administrative claims databases and quality monitoring registries) are well suited to advance innovation in urological oncology. Existing urological clinical trials are often limited by small numbers, are statistically underpowered and many face difficulties with accrual. Thus, efforts to improve trial design are of considerable importance. The development and use of standard outcome sets and adherence to reporting guidelines offer researchers the opportunity to guide value-oriented care, minimize research waste and efficiently identify solutions to the unanswered questions in urology cancer care.