Module 9 2021

22/03/2021

PD CLINICAL ENDPOINTS

USA – The need for efficacy studies will be determined by the degree of residual uncertainty regarding the similarity of the products – Similar clinical PK, PD, and immunogenicity profile may provide sufficient clinical data to support a conclusion that there are no clinically meaningful differences – PK and PD parameters are generally more sensitive than clinical efficacy endpoints e.g. TSH levels would provide a more sensitive comparison of two thyroxine products than an effect on clinical symptoms of euthyroidism. – Where meaningful correlation between PK and PD results and clinical effectiveness; comparative efficacy study may be unnecessary. JAPAN – If a clinically relevant pharmacodynamic marker exists and pharmacodynamic studies are sufficient to ensure the similarity in efficacy between the biosimilar and reference agent, a comparative efficacy study might not be necessary. If necessary, clinical studies to assess clinical safety including immunogenicity should be considered.

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STREAMLINED APPROVAL OF BIOSIMILARS: MOVING ON FROM THE CONFIRMATORY EFFICACY TRIAL

If relatively small effect impractically large trial needed. On review, therapeutic equivalence trials contribute little additional evidence of biosimilarity, extensive analytical testing and a pharmacokinetic study are usually sufficient

Marie-Christine Bielsky1, Anne Cook1, Andrea Wallington1, Andrew Exley1, Shahin Kauser1, Justin L. Hay1, Leonard Both1, and David Brown1 1Medicines & Healthcare Products Regulatory Agency,, UK Drug Discovery Today S1359-6446(20)30343-3 DOI: https://doi.org/10.1016/j.drudis.2020.09.006 Reference: DRUDIS 2770 h

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