EMA Proposes Ways to Improve Development and Evaluation of Biosimilars
The EMA has issued a new proposal that aims to encourage biosimilar development and improve patient access to biosimilar therapies.
Artur - stock.adobe.com
The European Medicines Agency (EMA) announced on April 1, 2025 that it is exploring ways to improve the development and evaluation of biosimilar therapies that will still uphold strict European Union (EU) safety standards and has published a proposal, a draft reflection paper (1,2). The agency has to date more than two decades of experience in evaluating biosimilars (3) and anticipates that this new proposed approach will improve access to biosimilars for patients in the EU. The agency aims to ensure that Europe is an attractive market to develop these treatments.
The draft reflection paper outlines an approach that would potentially reduce the amount of clinical data required for the development and approval of biosimilar medicines.
“This reflection paper will examine settings for biosimilars where similar clinical efficacy and safety can be inferred from a conclusion of physicochemical and biological similarity and comparable pharmacokinetics. Currently, Comparative Efficacy Studies (CES) (in which safety and immunogenicity data are also routinely captured) can already be waived in case an accepted pharmacodynamic (PD) surrogate endpoint exists, but even this prerequisite might not be needed” the paper states (2).
The scope of the reflection paper will include discussion on the necessity of CES for demonstrating biosimilarity. To give context to those reflections, the paper will first consider current practices with respect to analytical comparability exercises, including in-vitro pharmacology. The paper will also consider the predictive value of these current analytical comparability exercises. Some reflections will then be subsequently provided with regard to the contribution of the CES, other human in-vivo studies—especially pharmacokinetic/PD studies—and to the assessment of immunogenicity. EMA specifies that the reflection paper is not intended to replace current guidance or current practice regarding analytical comparability exercises (2).
The agency is inviting stakeholders to send their comments on this reflection paper through an online EUSurvey (4). The deadline to submit comments is Sept. 30, 2025.
This draft reflection paper builds on a 2024 concept paper on a tailored clinical approach in biosimilar development that was previously issued by the EMA (5).
EMA defines a biosimilar as “a biological medicine that is highly similar to another already approved biological medicine (the 'reference medicine')” (1). Biosimilars have grown in importance in the EU as therapeutic options that improve patient access to essential treatments. While used to treat a variety of diseases—such as cancer, rheumatoid arthritis, and inflammatory bowel disease—biosimilars offer the same clinical effectiveness and safety as their respective reference product. “By providing competition in the market, they can expand patient access to critical medicines” EMA stated in its press release (1).
The European Commission authorizes biosimilars based on studies comparing them to their reference medicine. These studies include a comparability exercise on quality aspects of the biosimilar’s active substance and the active substance of the reference medicine as well as a demonstration of clinical efficacy and safety of the biosimilar in confirmatory clinical trials.
“Building on extensive experience with biosimilar medicines and advances in analytical methods, the draft reflection paper suggests that demonstrated structural and functional comparability, together with comparative data on how the body interacts with the medicines (pharmacokinetic data), may be sufficient to demonstrate similarity with the reference medicine. This could potentially reduce the need for extensive clinical efficacy studies. Waiving certain clinical data requirements would simplify the development and evaluation process while maintaining the highest standards of safety and efficacy,” EMA stated in its release (1). “This more streamlined approach would ultimately ensure wider availability of biosimilar medicines to patients in the EU.”
References
1. EMA. Streamlining Development and Assessment of Biosimilar Medicines. Press Release. April 1, 2025.
2. EMA/CHMP/BMWP/60916/2025. Reflection Paper on a Tailored Clinical Approach in Biosimilar Development, Draft (April 1, 2025).
3. EMA. Biosimilar Medicines: Overview. ema.europa.eu (accessed April 8, 2025).
4. EMA. Submission of Comments on the Draft "Reflection paper on a tailored clinical approach in biosimilar development". ec.europa.eu (accessed April 7, 2025).
5. EMA/CHMP/BMWP/35061/2024. Concept Paper for the Development of a Reflection Paper on a Tailored Clinical Approach in Biosimilar Development (Feb1, 2024).
