Pharmacodynamic Biomarkers for Biosimilar Development and Approval


Public Workshop

Pharmacodynamic Biomarkers for Biosimilar Development and Approval


Day 1: Pharmacodynamic Biomarkers for Biosimilar Development and Approval

Day 2: Pharmacodynamic Biomarkers for Biosimilar Development and Approval

September 20 21, 2021 Virtual Public Workshop

Duke-Margolis is collaborating with the U.S. Food and Drug Administration (FDA) to host a two-day virtual public workshop entitled “Pharmacodynamic Biomarkers for Biosimilar Development and Approval” on September 20 21, 2021. This virtual public workshop is a forum for regulators, biopharmaceutical developers and academic researchers to discuss the current and future role of pharmacodynamic (PD) biomarkers in improving the efficiency of biosimilar product development and approval.


Biologic medicines play an important role in the treatment of many serious illnesses; however, biologic medicines can be costly. With biosimilars (biologic medicines that are highly similar to and have no clinically meaningful differences from existing FDAapproved reference products), drug developers can leverage the safety and effectiveness data of reference products to support the approval of biosimilars, potentially providing access to lower cost treatments.

Comparative clinical efficacy study(ies) may not be needed to establish biosimilarity between investigational biosimilars and their reference products if there are sufficient data from other comparative studies including human pharmacokinetic (PK) and PD studies [1,2]. When available, PD biomarkers can demonstrate the characteristics of a biosimilar’s target effects, helping reduce residual uncertainty about whether there are no clinically meaningful differences between a biosimilar and its reference product.

However, few biosimilars have been approved that leverage PD biomarker data without comparative clinical efficacy studies. Although the FDA has published guidance on PD biomarker selection and general study design considerations [2], additional regulatory clarity on the use of PD biomarkers to support demonstrations of biosimilarity might facilitate additional biosimilar development and approval. Therefore, the FDA is working to advance an evidentiary framework and methodology for identifying, characterizing, and applying PD biomarkers to assess biosimilarity so patients can access safe, effective, and lower cost biosimilars sooner.

Workshop Objectives

  • Discuss the current and potential future state of leveraging PD biomarkers for biosimilar development and approval 
  • Summarize FDA’s initiatives to advance biosimilar development 
  • Describe stakeholders’ experience with PD biomarkers in biosimilar development  
  • Explain research efforts to promote a broader application of PD biomarkers in biosimilar development

Agenda and Speakers

Speakers will be announced on a rolling-basis via updates to the workshop agenda and a speaker list will be published closer to the workshop date.


[1] US Food and Drug AdministrationFDA guidance: scientific considerations in demonstrating biosimilarity to a reference product (2015). Accessed June 25, 2021.

[2] US Food and Drug Administration. FDA guidance: clinical pharmacology data to support a demonstration of biosimilarity to a reference product (2016). Accessed June 25, 2021.

Acknowledgement of Federal Support

Funding for this event was made possible, in part, by the Food and Drug Administration through grant U01FD006807. Views expressed in written materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organization imply endorsement by the United States Government.

Duke-Margolis Team

Image of Nicholas Harrison

Nicholas R. Harrison, MPH, MA

Policy Research Associate

Erin Soule Headshot

Erin Soule, PhD

Assistant Research Director