PDA Letter Article

How to Best Tackle Biosimilar Challenges

by Stephan Krause, PhD, AstraZeneca Biologics, and Jens Schletter, PhD, Novartis


Regulatory approval of biosimilars remains a challenge, forcing sponsors of biosimilar products to face regulatory complexity during development.

Demonstrating analytical similarity and inclusion of appropriate information in market authorization submissions are just two of the recurring challenges manufacturers face.

Following the conclusion of the 2018 PDA/FDA Joint Regulatory Conference, the 2018 PDA Biosimilars Workshop will focus on the most common challenges identified in biosimilar applications by the U.S. FDA and other regulatory agencies, including EMA and Health Canada. Stephan Krause, PhD, Director, QA Technology, AstraZeneca Biologics, and Emanuela Lacana, PhD, Associate Director for Biosimilar and Biologics Policy, FDA, will co-chair the workshop. The workshop will offer an opportunity to gain consensus among regulators and industry on what are appropriate compliance standards on analytical similarity data along with regulatory expectations for data quality, preapproval inspections and method validation studies. The use of appropriate statistical tools for analytical similarity study design will be evaluated in great depth.

With a combination of presentations and breakout sessions and a primary focus on learning from experience, the workshop will encourage active discussion among regulators and industry with the intent to increase successful registration submissions. Starting with a session moderated by Krause on regulatory perspectives on biosimilar marketing applications, presentations in this first session will feature Steven Kozlowski, MD, Supervisory Medical Officer, CDER, FDA, R. Martijn van der Plas, PhD, Senior Assessor Biologicals, Medicines Evaluation Board (MEB), The Netherlands, and Chantal Depatie, PhD, Biologist Evaluator, Health Canada. Speakers will provide an overview of challenges encountered during the assessment of biosimilar marketing applications, with a focus on development, control strategy and commercial production. Each of the agencies will provide their expectations of the CMC information needed for a successful submission.

Joel T. Welch, PhD, Acting Review Chief, CDER, FDA, will moderate the session titled, “The Trapeze and The Trap Door: Navigating High-Level Technical Challenges in Biosimilar Development.” This session will describe approaches to addressing high-level technical challenges and avoiding pitfalls frequently encountered during biosimilar candidate development, including data quality expectations, the creation of the final control strategy, and strategic choices necessary for candidate selection and development. Both regulators and industry representatives will share their perspectives and experiences. Presentations will cover “fit-for-purpose” analytical methods, inspectional expectations, the intersection of the analytical similarity assessment with the proposal for a final control strategy and critical strategic decisions necessary for a biosimilar development program.

Another session, moderated by Bev Ingram, PhD, Senior Director, Portfolio Lead Biosimilars Regulatory Affairs, Pfizer, will address the role of statistical tools in the demonstration of analytical similarity. Presentations in this session will explore the use of statistical tools to provide meaningful contributions to the demonstration of analytical similarity, highlighting common issues that arise when applying statistical tools. Solutions that could be used to address the known challenges will be discussed, including practical alternative solutions to current approaches. Experiences from regulatory agencies outside the United States will be shared, complementing details presented in the FDA draft guideline on statistical approaches to analytical similarity.

The workshop has been structured to allow attendees to learn from presenters and each other by combining full-group presentations and small-group breakout sessions. By sharing perspectives and clarifying concerns, regulators and industry may ultimately increase the success of registration submissions.

Learn more about the 2018 PDA Biosimilars Workshop.