On 1 April 2025, the European Medicines Agency (EMA) published a draft reflection paper questioning whether biosimilars may be approved in future without submission of Comparative Efficacy Studies (CES). The EMA considers that “[w]aiving certain clinical data requirements would simplify the development and evaluation process” of biosimilars “while maintaining the highest standards of safety and efficacy”.
According to the EMA, advances in analytical characterisation, and its extensive regulatory experience in approving significant changes in manufacturing processes of biological medicines by confirmation of structural and functional comparability, “supports the notion that under specific prerequisites, analytical comparability exercises and pharmacokinetic (PK) data could be sufficient for demonstrating biosimiliarity”. The EMA also refers to its experience indicating that the results from CES in the past “generally did not add relevant additional information to the biosimilarity exercise”.
The reflection paper concludes that CES may no longer be required for approval of biosimilars “if similar clinical efficacy and safety pharmacology can be inferred from a sufficiently stringent evaluation of analytical comparability, in vitro pharmacology, and a comparative clinical PK trial”.
The EMA is inviting stakeholders to send their comments on the reflection paper via an online EU Survey until 30 September 2025.