Biomarkers play a pivotal role in drug development, offering crucial insights into treatment effectiveness, safety, and patient response. As the regulatory landscape governing biomarkers in cell and gene therapy undergoes rapid evolution, stakeholders encounter numerous challenges in ensuring compliance with applicable regulations.
Health authorities and agencies now place heightened scrutiny on biomarker data, emphasizing its Context of Use (COU) and demanding rigorous assay validation, particularly for complex drug candidates like Cell and Gene Therapy Products (CGTPs). Determining the appropriate COU and which regulatory requirements are applicable can prove challenging, given the multitude of pathways available. Each approach carries its own distinct intent regarding the utilization of biomarker data, resulting in varying levels of data accuracy as well as appropriateness of the results in patient treatment decisions.
Learning Objectives:
Gain a comprehensive understanding of the regulatory landscape governing biomarkers in cell and gene therapy – CLIA vs FDA vs IVDR (EU) vs Companion diagnostic
Recognize areas of overlap and potential conflicts within the regulatory framework for cell and gene therapy biomarkers.
Importance of biomarkers for exclusion/inclusion for CGTPs, such as pre-existing antibodies against AAV (viral delivery) or LNPs (non-viral delivery).
