Clinical Benefit Scales and Trial Design: Some Statistical Issues
En prenant pour exemple l'échelle de l'"European Society for Medical Oncology " destinée à évaluer les bénéfices cliniques des anticancéreux dans les essais cliniques randomisés, cet article passe en revue les problèmes statistiques liés à l'intégration de ce type d'échelle d'évaluation lors de la conception des essais cliniques
Recently developed clinical-benefit outcome scales by the European Society for Medical Oncology (ESMO) and the American Society of Clinical Oncology (ASCO) allow standardized objective evaluation of outcomes of randomized clinical trials. However, incorporation of clinical-benefit outcome scales into trial designs highlights a number of statistical issues: the relationship between minimal clinical benefit and the target treatment-effect alternative used in the trial design, designing trials to assess long-term benefit, potential problems with using a trial endpoint that is not overall survival, and how to incorporate subgroup analyses into the trial design. Using the ESMO Magnitude of Clinical Benefit Scale as a basis for discussion, we review what these issues are and how they can guide the choice of trial-design target effects, appropriate endpoints, and pre-specified subgroup analyses to increase the chances that the resulting trial outcomes can be appropriately evaluated for clinical benefit.
Journal of the National Cancer Institute , article en libre accès, 2021