Module 9 2024

03/09/2024

Risk Based Approach (RBA)

What Regulatory people get asked... •

What type of studies shall we do? Which techniques should we use? How much? How many batches?

• • •

What do Authorities want to see? What Regulatory people usually answer:

Well it depends ....

• Risks are product specific, for a specific patient population • Each case is different and needs a scientific and rational approach • May also depend on the stage of development

Courtesy: Florence Salmon

The Organisation for Professionals in Regulatory Affairs

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Mapping the Risks…

Starting materials (cells and genetic modification tools) ● Origin of the donor (donor testing) ● Risks of using viral vectors are different for each type of vector (lenti, retro) with specific characteristics (integration, off-target) ● Risks with autologous cells are different from risks with allogenic cells ● Collection, logistics, storage Manufacturing process ● Risks of using gene-editing are different for each type of tool (HDR, TALENs) ● Raw materials and media (origin, impurities, biologic risk)

● Equipment (closed or not-really completely closed) ● Length of culture (what does it do to the cells) Patient population ● Age, stage of disease (residual disease, clonality) ● Healthy donors, HLA-matched or partial mismatch ● Concomitant or prior therapies (exhausted T cells)

The Organisation for Professionals in Regulatory Affairs

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