European Medicines Agency Perspective on Oncology Study Design for Marketing Authorization and Beyond

Abstract

In the development of highly active anticancer drugs, the European situation may be viewed as paradoxical. Limited data may support marketing authorization, but may be insufficient for the health economic appraisal needed for reimbursement and market uptake. To achieve this, conventional confirmatory studies may be needed. For products of special interest, studies aimed at optimizing cost-effectiveness may be warranted. Efficient designs of studies to meet these objectives constitute challenges to all stakeholders.