Regulatory Approach to Manufacturing and Control of Cell/Gene Therapies Draws International Discussion

Regulators around the world have been taking the opportunity at recent international forums to benchmark on their respective efforts to create a viable framework for regulating the manufacturing and control of cell/gene/tissue therapies and to consider harmonization possibilities. Issues under discussion include:

● autologous vs. allogeneic products and how the respective risks should be handled

● where the regulatory boundaries lie between local hospital oversight and the more formalized structures in place at the national/regional level

● how agency oversight may need to be adapted to meet the flexibility and individualized nature of products in the cell/gene/tissue arena

● what the expectations are for validating manufacturing processes and assays, and

● the importance of product characterization and comparability assessment after process changes, including scale-up, and what the assessments should include.

The search for a risk-based framework for the new therapies that would provide the needed flexibility is heightened due to the “breakthrough” nature of many of them and the expedited review status they are gaining.

The challenges of expediting the CMC development process are daunting enough for more conventional biotech products, but only expand for the CGTs, where the CMC pathways are less established. While speeding up the complex review process involved may not be realistic, what is at issue is heightened sponsor/agency knowledge sharing as early in development as possible to help untangle where the quality/clinical connections lie and how to manage the risks, so that commercialization can actually happen.

At two recent conferences sponsored by CASSS – the CMC Strategy Forum Japan in December and the Well-Characterized Biotech Products (WCBP) conference in Washington, DC in January – regulators from North America, Europe, and Asia discussed their current efforts regarding advanced therapy products and where the alignment divergences and opportunities lie.

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