The essence of real-world study in cancer research

Abstract

BackgroundDespite the rapid development of gene engineering and other novel therapies, advanced cancer is still a largely intractable disease, and its high mortality necessitates the development of better treatment (1). In general, the regulatory approval of new cancer treatment is built on the objective criteria of efficacy and safety derived from randomized controlled trials (RCTs). However, with advances in genetic and molecular treatment, the number of patients available for a specific clinical trial may be too small for proper assessment of a benefit-risk paradigm (2). As a result, drug developers and regulators have become increasingly interested in data external from clinical trials, and there is growing need to rethink the traditional approaches to treatment development and approval (3). One potential solution to this may be to further extend and validate the results from RCTs with the wider experience of real-world data (RWD) or real-world evidence (RWE).Compared to the rigorous admission criteria and strict research conditions of RCTs, RWD is collected from the real world to provide evidence for clinical and policyrelevant questions that cannot be addressed with data from RCTs, such as patient health status, cancer incidence and mortality, quality of and access to care delivered in routine practice, rare cancers, and rare adverse events and toxicities