Advancing Innovations in Biosimilars

Biologic medicines have revolutionized the treatment options for many diseases and typically carry favorable benefit– risk profiles compared with small molecule drugs. Although these innovative medicines can enable longer lifespan and improved quality of life, the high cost of these products limits access. Unlike small molecule drugs, exact copies of biologic products cannot be made, and thus it is not possible to approve “generic” biologics. To address this, legal frameworks and guidelines were established in different countries to enable approval of biosimilars (i.e., a biological product that is highly similar to, and has no clinically meaningful differences from, an approved reference product) that can rely on existing scientific knowledge about the safety, purity, and potency of the reference product. Although biosimilar development has gained momentum globally since the first approved biosimilar in Europe in 2006, as reflected in the number of reference products with approved biosimilars (Table 1), most biologics do not have approved biosimilars. As reported in this themed issue of Clinical Pharmacology & Therapeutics (CPT) on Innovations in Biosimilars (Figure 1), there is an opportunity to leverage innovations in clinical pharmacology and related disciplines to improve the efficiency of biosimilar development and approval (Figure 2). One way to do this is to leverage the use of pharmacodynamic (PD) biomarkers in clinical pharmacology studies in place of comparative clinical studies with efficacy end point(s), which is discussed in multiple articles in this issue. 5 These articles and others also discuss how modeling and simulation (or modelinformed drug development (MIDD)) can be used to optimize study design and analyses. Other articles focus on characterizing the relationship between product quality characteristics (e.g., structural and functional characterization) and clinical performance to further streamline clinical development. 9