Patient burden and clinical advances associated with post-approval monotherapy cancer drug trials: a systematic review

Objectives: After regulatory approval, drug companies, public funding agencies and academic researchers often pursue trials aimed at extending the uses of a new drug by testing it in new non-approved indications. Patient burden and clinical impact of such research is not well understood. Design/setting: We conducted a retrospective cohort study of post-approval clinical trials launched within five years after the drug's first approval, testing anticancer drugs in monotherapy in indications that were first pursued after a drug's first FDA license, for all 12 anticancer drugs approved 2005-2007. (Search date: 2019-02-12.) Primary and secondary outcome measures: Our primary objective was to measure burden and clinical impact for patients enrolling in these trials. Each trial was sorted into a “trajectory” defined by the drug and cancer indication. Risk was operationalized by proportions of grade 3-4 severe adverse events and deaths. Clinical impact was measured by estimating the prop...