Biosimilar Drug Development and Approval Process
The U.S. Food and Drug Administration (FDA) is responsible for advancing the public health by helping to review and evaluate biosimilar innovations that make medicines safer and more effective and by helping the public get the accurate, science-based information it needs to use medicines to maintain and improve public health. This presentation emphasizes quality system approaches to the development and availability of new drug information presented in the premarket applications. For approvals of biosimilars, the sponsors of premarket applications must present analytical characterization, pharmacokinetic (PK) and pharmacodynamic (PD) profiles, and comparative clinical studies to demonstrate that a proposed biosimilar is highly similar to a licensed reference product. It is recommended that a proposed bio similar product be tested for intended clinical use described in the licensed reference product’s labeling. The protocol requires a sponsor to describe the bio similar product’s PK/PD clinical data comparing its safety, efficacy, and immunogenicity to that of the reference product. FDA reviews clinical safety and efficacy of the biosimilar product and it is essential that any residual risks and hazards are mitigated to acceptable levels. FDA emphasizes the quality system approach to design and development studies by ensuring that organized data and appropriate labeling are presented in support of the new biosimilar’s intended clinical use. Emphasis is placed on quality-by-design (QbD) approach to design of studies by providing guidances for analysis and expected clinical PK/PD data for the use of biosimilars in appropriate patient population studies. FDA field investigators evaluate the biosimilar product’s c-GMP risk-based requirements and make recommendations based on whether the manufacturer has the required checks and balances in place and whether the manufacturer verifies and validates the implementation of critical quality attributes (CQAs) of the proposed biosimilar product. Biosimilar applications are approved based on totality of evidence described in U.S. FDA guidance documents.