FDA Finalizes Biosimilar Guidance

On December 29, the US Food and Drug Administration (FDA) finalized long-awaited biosimilar guidance from 2014 on the needed clinical pharmacology data from pharmaceutical sponsors.

“Clinical pharmacology studies are part of a stepwise approach for developing the data and information needed to support a demonstration of biosimilarity,” said the FDA. “These studies can reduce the residual uncertainty in assessing the biosimilarity between a proposed biosimilar product and reference product and inform the design of subsequent clinical trials to assess clinically meaningful differences.”

The Biologics Price Competition and Innovation Act of 2009 (BPCIA) created the original approval pathway for follow-on biologics, and guidance on interchangeability is expected this year, containing recommendations on how clinical pharmacology studies that assess the presence or absence of clinically meaningful differences between the proposed biosimilar and the reference product should be conducted and analyzed.

FDA Guidance Main Sections

There are four areas upon which the new guidance focuses:

1. The role of clinical pharmacology studies in the demonstration of biosimilarity
2. Critical considerations in the use of clinical pharmacology studies to support biosimilarity (with subsections on safety and immunogenicity, among others)
3. Developing clinical pharmacology data for supporting a demonstration of biosimilarity (with subsections on study design, pharmacokinetic (PK)
4. Pharmacodynamic (PD) measures, among others) and the utility of simulation tools in study design and data analysis

It has been a long 20 months for drug manufacturers who have been awaiting the update to the original draft version, and had taken issue with the FDA's four possible outcomes and terminology for the analytical comparison of the reference product and proposed biosimilar drug.