A biosimilar is a biological product that is highly similar to and has no clinically meaningful differences from a US Food and Drug Administration (FDA)‐approved reference product, in terms of safety, purity and potency of the product. Biosimilar approvals to date use comparative clinical efficacy studies which often require large sample sizes and long study durations. However, FDA guidance suggests that biosimilarity may be demonstrated based on clinical pharmacology, pharmacokinetic and pharmacodynamic data in place of clinical efficacy studies. Yet very few biosimilar approvals included PD similarity data, either due to lack of an established PD biomarker or lack of an evidentiary framework for using PD similarity data. The FDA is actively investigating how to advance the science for PD biomarkers to increase their use in human PD similarity studies, thereby mitigating the need for developers to conduct costly comparative clinical studies with efficacy end point(s). These efforts are geared towards accelerating biosimilar approvals and enhance access to cost-effective alternatives of approved biological products which are used to treat many serious and life-threatening diseases.
Learning Objectives:
Understand the role of clinical pharmacology in biologics and biosimilar approvals
Understand the Use of PD biomarkers in biosimilar development programs
Considerations for PD biomarker assessment and PK/PD similarity study design
Learn about FDA’s action to fill information gaps and inform on best practices for PD biomarkers to support biosimilar development