Keywords: meta-analysis, non-inferiority margin, equivalence margin, surrogate endpoint
In recent years, it is of increasing interest in oncology and hematology drug development to use meta-analysis to obtain information and synthesize evidence from multiple studies. Meta-analytic tools have been applied in various stages of drug development and regulatory settings, often with both unique and some common methodological challenges. In this talk we will review a number of case studies in drug applications CDER/FDA has reviewed. For example, in non-inferiority studies or biosimilar studies, meta-analysis has been used to determinate the non-inferiority margin or the equivalence margin for efficacy evaluation based on data from multiple historical studies. Meta-analytic methods have also been used to develop and validate putative surrogate endpoints for efficacy evaluation to accelerate drug development, in which case we aim to identify potential surrogate endpoints that can accurately and reliably reflect true clinical benefits while meeting the unmet medical needs in an even more timely fashion.