Pharmacodynamic Biomarkers Evidentiary Considerations for Biosimilar Development and Approval

Abstract

As outlined in the FDA biosimilar guidance documents, 1,2 biosimilars may be approved based on PK and PD similarity studies without a comparative clinical study in patients. Reliance on PK and

New science, drug regulation, and emergent public health issues: The work of FDA’s division of applied regulatory science

“…A joint FDA/Duke Margolis Workshop ( 62 ) discussed initial findings and facilitated a broader discussion on use of PD biomarkers for biosimilar development. Additional details will be reported in the January 2023 themed issue on Innovations in Biosimilars in the journal Clinical Pharmacology and Therapeutics ( 61 , 63 ).…”

“…DARS is also leading the development of an evidentiary framework to advance the use of PD biomarkers for biosimilars. The draft version of this evidentiary framework was presented at a DARS-led public workshop ( 119 ) and a review describing this work was recently published ( 63 ).…”

Advancing Innovations in Biosimilars

“…As reported in this themed issue of Clinical Pharmacology & Therapeutics ( CPT ) on Innovations in Biosimilars ( Figure ), there is an opportunity to leverage innovations in clinical pharmacology and related disciplines to improve the efficiency of biosimilar development and approval ( Figure ). One way to do this is to leverage the use of pharmacodynamic (PD) biomarkers in clinical pharmacology studies in place of comparative clinical studies with efficacy end point(s), which is discussed in multiple articles in this issue 1–5 . These articles and others 6 also discuss how modeling and simulation (or model‐informed drug development (MIDD)) can be used to optimize study design and analyses.…”

“…The workshop identified opportunities for investing in PD biomarker research and clarifying regulatory expectations for PD biomarker acceptability. To this end, Strauss et al 1 . discuss evidentiary considerations with practical examples to illustrate the thinking behind the recommendations in the FDA's guidance.…”

“…Broader discussions around use of PD biomarkers occurred at an FDA and Duke Margolis Center for Health Policy public workshop in 2021, which is summarized by Florian et al 2 The workshop identified opportunities for investing in PD biomarker research and clarifying regulatory expectations for PD biomarker acceptability. To this end, Strauss et al 1 discuss evidentiary considerations with practical examples to illustrate the thinking behind the recommendations in the FDA's guidance. Arato 12 and Wang 10 discuss perspectives from Japan and Canada, respectively.…”