Substantially manipulated cell-based products for human use are considered medicines and therefore regulatory authorities require extensive characterisation in terms of identity, purity and potency. The latter critical quality attribute is probably the most challenging to identify and measure, requiring provision that potency assays should reflect the intended mechanism of action and demonstrate the drugs' biological effect. However, in most cases, the mechanisms involved are not fully understood, making the definition and validation of suitable potency tests difficult, a 'bugaboo' quest to be feared. Although it is evident that much work is still needed in the scientific arena, the present chapter focuses on strategies currently used by developers of cell- and gene-based therapies to demonstrate potency of innovative medicines, the regulatory framework and need for standardisation seeking to demystify critical factors to consider when designing a potency assay.
Keywords: Critical quality attributes; Gene therapy; Marketing approval; Potency; Product development; Quality and regulatory compliance; Somatic cell therapy; Suitable potency assay; Surrogate potency assay; Tissue engineering products.
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