The FDA evaluation of the evidence
The regulatory pathway for biosimilar medicines is a unique and thoughtful process. It is designed to help ensure the development and approval of high-quality biosimilar medicines. Approved biosimilar medicines have no clinically meaningful differences in terms of safety and efficacy from the relevant reference product, based on the totality of evidence from analytical, nonclinical, pharmacokinetic, and clinical studies. The totality of evidence represents a new approach by the FDA to the development of a new biologic product.
Robust standards for quality, efficacy, and safety: a closer look at the totality of evidence
- Robust analytical testing, including comparative structural and functional characterization, to establish high similarity of the biosimilar and the reference product
- Nonclinical testing to evaluate the toxicity and safety profiles of the biosimilar
- Comparative human pharmacokinetic and pharmacodynamic studies and clinical immunogenicity assessment
- If residual uncertainty exists, comparative trials may be required (based on recent approvals, the FDA has required studies)