Potency analytical control strategies are critical to support biopharmaceutical drug and vaccine development throughout the product lifecycle. For biopharmaceutical drugs, it is generally expected that a quantitative potency assay be included in the control strategy and that the assay adequately reflects the physiological Mechanism of Action (MOA). Most often these assays are developed using cell lines that mimic MOA-relevant, physiological responses. However, the development, validation, and transfer of cell-based potency assays can be challenging due to multiple factors inherent to working with living systems/live cells. Therefore, non cell-based alternatives are of great interest to the more complex cell-based potency assays to ensure the consistent generation of accurate results for drug substance/drug product release and stability specifications.

Current day vaccine potency analytical control strategies typically do not involve cell-based assays, but generally rely on binding, chromatographic or physical chemical methods. However, many legacy vaccines still utilize in vivo potency assays; these assays are inherently more variable, require weeks to perform, and are more prone to invalid test results, necessitating repeat testing. The transition to in vitro alternatives has been challenging due to less robust characterization of vaccine products, compounding the establishment of in vitro alternatives, as well as regulatory acceptance of new methods. Fortunately, recent industry driven collaboration and novel regulatory approaches have accelerated the development of in vitro alternative assays and regulatory acceptance.

In this session, we plan to discuss the following topics:

- Successful and/or failed biopharmaceutical case studies regarding strategies used to support the phase-appropriate introduction of a new non-cell-based potency assay as a replacement to an existing cell-based potency assay in the lot release and stability program,

- Successful and/or failed biopharmaceutical case studies regarding strategies used to support the phase-appropriate removal of an existing cell-based potency assay from the lot release and stability program while leveraging an existing non-cell-based potency assay.

- Vaccine industry collaboration and regulatory innovation that has accelerated the transition from in vivo to in vitro potency and safety assays for legacy vaccines.

Session Speakers:

Successful Implementation of Potency Control Strategy - Two Binding Assays Rather Than One Cell-based Assay

Catherine Shoemaker-Ramsey, Biogen

How Industry and Regulatory Innovation and Collaboration Can Promote the Transition From in Vivo to In Vitro Potency Testing for Human Legacy Vaccines

Emmanuelle Coppens, Sanofi

Replacement of in Vivo Immunogenicity Assay With in Vitro Antigenicity ELISA for Pertactin Antigen in Acellular Pertussis Combination Vaccines

Belma Ljutic, Sanofi

Additional Panelists:

Elena Grabski, Paul-Ehrlich-Institut

Emily Shacter, ThinkFDA LLC

Jayda Siggers, Health Canada

Yasuhiro Kishioka, Pharmaceuticals and Medical Devices Agency (PMDA)

Dean Smith

Appearances