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Biosimilar assessments: do we need efficacy trials? Posted 23/10/2020

Regulatory guidelines for biosimilar approval have existed in Europe since 2004. These are based on the totality of evidence from comparative studies with reference products and have always required comparative efficacy studies. However, a recent article by Bielsky et al., published in Drug Discovery Today [1], explores the increasingly questioned role of the clinical efficacy trial in biosimilar approval. 

The authors, based at the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), carried out a review of complex biosimilar applications in the EU. They found that, in many cases, efficacy trials do not act as an effective tool to discriminate between a biosimilar and its reference product and are often an inefficient use of resources [2]. With this in mind, they explain the rationale for allowing biosimilar applications without comparative efficacy trials in detail [3].

The authors then outline a blueprint for a biosimilar pathway that reduces the need for clinical efficacy trials [4]. This blueprint considers that, in most cases, extensive comparative analytical studies, together with an abbreviated clinical package consisting of a pharmacokinetic (PK) trial with simple pharmacodynamic (PD) trial (if available), safety and immunogenicity evaluation, is sufficient to assess biosimilarity. However, they do note that in some cases efficacy trials may still be required. In addition, the authors identified the key requirements for a reference product that would qualify for this pathway and the key requirements for comparing the reference and biosimilar candidate using a discriminatory range of structural and functional studies, together with crucial PK parameters. In creating this ‘streamlined’ biosimilar assessment, the authors hope to facilitate wider patient access to crucial therapies.

Conflict of interest
The authors of the paper [1] did not provide the conflict of interest statement.

The authors disclaim that the publication reflects the views of the individual authors and should not be understood to represent official views or policies of the UK Medicines and Healthcare products Regulatory Agency (MHRA).

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Clinical data requirements for biosimilars in the EU

References
1. Bielsky M-C, Cook A, Wallington A, et al. Streamlined approval of biosimilars: moving on from the confirmatory efficacy trial. Drug Discov Today. 2020. In press. doi.org/10.1016/j.drudis.2020.09.006
2. GaBI Online - Generics and Biosimilars Initiative. Role of efficacy trials in biosimilar assessments questioned [www.gabionline.net]. Mol, Belgium: Pro Pharma Communications International; [cited 2020 Oct 23]. Available from: www.gabionline.net/Biosimilars/Research/Role-of-efficacy-trials-in-biosimilar-assessments-questioned
3. GaBI Online - Generics and Biosimilars Initiative. Rationale for biosimilar assessment without efficacy trials [www.gabionline.net]. Mol, Belgium: Pro Pharma Communications International; [cited 2020 Oct 23]. Available from: www.gabionline.net/Biosimilars/Research/Rationale-for-biosimilar-assessment-without-efficacy-trials 
4. GaBI Online - Generics and Biosimilars Initiative. A blueprint for biosimilar assessment without efficacy trials [www.gabionline.net]. Mol, Belgium: Pro Pharma Communications International; [cited 2020 Oct 23]. Available from: www.gabionline.net/Biosimilars/Research/A-blueprint-for-biosimilar-assessment-without-efficacy-trials 

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