USP is an Asia-Pacific Economic Cooperation (APEC) Center of Excellence for the Advanced Therapies priority work area. This priority work area is focused on building regulatory harmonization and understanding for these emerging products. Since biological assays that measure activity or potency (known as bioassays) for these critical therapies can be one of the most challenging methods to develop and validate, USP in collaboration with the APEC Regulatory Harmonization Steering Committee and Advanced Therapy Priority Work Area champions built this training program. The trainers focused on development and validation of methods which are commonly used to analyze and control critical quality attributes of cell, gene, and tissue-based therapies.

Subject area:
Biologics

Objectives (If applicable):
Define the word “bioassay” and describe common bioassay formats for different types of advanced therapies
Describe standards that exist for bioassays and how to assess if the standard was suitably followed or variations sufficiently documented
Summarize why bioassays must be fit for purpose (e.g., for potency assignment, stability assessments, etc.)
Discuss how both science and risk play a role in determining the choice of a bioassay and why multiple formats may be beneficial during clinical development
Explain common assay parameters that are assessed and documented during bioassay development and validation
Describe common statistical approaches that support bioassay analysis
Discuss common bioassay lifecycle management challenges

Target Audience (If applicable):
Scientists, regulators, and other professionals who develop, validate, perform, supervise, manage, or review bioassays for cell and gene therapies as well as other biologics