Importance of responder criteria for reporting health-related quality-of-life data in clinical trials for advanced cancer: recommendations of Common Sense Oncology and the European Organisation for Research and Treatment of Cancer.

The goals of treatment for people with advanced cancer are to prolong survival and improve symptoms and health-related quality of life (HRQOL). Although many phase 3 randomised clinical trials seek to evaluate HRQOL during treatment, informing individual patients about expected HRQOL outcomes is challenging, as the common method of analysis and reporting compares averages for randomised groups, and clinicians find these data difficult to apply in clinical practice. Symptomatic patients with advanced cancer would like to know the probability that a proposed treatment might improve their survival or their dominant symptoms, and the probability of having treatment-related side-effects. When specifying HRQOL endpoints, we recommend that trialists develop HRQOL hypotheses about which dominant symptoms might be improved due to the initiation of the investigational treatment, and whether aspects of functioning and overall HRQOL will also improve despite the side-effects of the treatment. Validated, disease-specific, patient-reported outcome measures should be used to assess the relevant HRQOL concepts. Changes in HRQOL should be reported as the proportion of patients who have a specified improvement (or deterioration) in these relevant HRQOL scales (ie, as a response criterion), and harmonised standards for such a response criterion are needed.