Reflection paper on a tailored clinical approach in biosimilar development

The European Medicines Agency (EMA) is exploring an improved approach for biosimilar medicine development and evaluation, while upholding strict European Union (EU) safety standards. The is outlined in a draft reflection paper biosimilar development. Its aim is to reduce the amount of clinical data required for the development and approval of biosimilar medicines in the EU.

A biosimilar is a biological medicinal product that contains a version of the active substance of an already authorised original biological medicinal product (Reference Medicinal Product, RMP), where similarity to the reference medicinal product based on a comprehensive comparability exercise has been established. Biosimilars have become important therapeutic options, improving patient access to essential treatments. Therefore, CHMP (EMA) acknowledges the significance of biosimilars.

Currently, the required comparability exercise comprised quality data (analytical comparability exercise), in vitro and in vivo non-clinical data, and comparative pharmacokinetic, pharmacodynamic, safety and efficacy studies. However, considering the advances in the analytical sciences and the extensive regulatory experience gained, in vivo non-clinical data and, at least for some less complex biologicals with a straightforward mechanism of action, the importance of dedicated clinical efficacy and safety data should be re-evaluated. Currently, the need for Comparative Efficacy Studies (CES) is increasingly questioned in general.

Keywords:  reflection paper, biosimilar, comparative efficacy study, tailored clinical approach

Current version

Reflection paper on a tailored clinical approach in biosimilar development

Draft: consultation closed Consultation dates: 01/04/2025 to 30/09/2025 Reference Number: EMA/CHMP/BMWP/60916/2025

Document history

Concept paper for the development of a reflection paper on a tailored clinical approach in biosimilar development

Draft: consultation closed Consultation dates: 01/02/2024 to 30/04/2024 Reference Number: EMA/CHMP/BMWP/35061/2024