Biosimilars and Biologics

Biography

Biography: Francois Xavier Frapaise

Abstract

Most patients still have limited or no access to life-changing therapeutic proteins in the treatment of their cancer or autoimmune disorders. Th e current clinical development model of biosimilars is expensive, and in most cases, large, phase 3 trials do not provide meaningful information on the clinical equivalence of biosimilars and reference compounds. At the same time, the development of state-of-the-art orthogonal analytical methods has enabled a better understanding of the structure and structure–function relationship of biotherapeutics. Hence, we suggest here that a solid chemistry, manufacturing, and controls (CMC) package and meaningful phase 1 studies will leave limited uncertainty on biosimilarity, which can be addressed— if needed—by post-approval, long-term follow-up studies (post-approval studies, pharmacovigilance, real world evidence data and registries, and possibly new postapproval models to be developed). We believe that this new approach may be more appropriate than 600- to 1000-patient, phase 3 trials in assessing biosimilarity and therapeutic equivalence, under the condition that the administered biosimilar given to individual patients can be clearly identifi ed. Obviously, there will probably never be a “one size fi ts all” development model, and an individualized, risk-based approach to biosimilar development will always have to be considered and discussed early with regulators.