ICH looks to revamp its guidelines on cell and gene therapies

The International Council for Harmonisation’s (ICH) Cell and Gene Therapy Discussion Group (CGTDG) recently released a discussion paper recommending a more tailored approach in its guidelines for cell and gene therapy products. ICH said the updated approach would better reflect the unique characteristics of these products in comparison to traditional drugs.

In December 2024, an analysis conducted by the CGTDG revealed that over 300 region-specific guidelines for Advanced Therapy Medicinal Product (ATMP) have been issued, either in draft or final form, by ICH members and observers represented within the CGTDG. The group emphasized that this increasing number of region-specific guidelines highlights the necessity for harmonized global regulatory and development approaches.

For the analysis, the group reviewed the existing ICH guidelines across various disciplines, including quality, safety, efficacy, and multidisciplinary guidelines, as well as those that are currently being revised. They identified and prioritized topics that should be addressed to support the advancement of the ATMP field.

The document contains a table outlining CGTDG’s recommendations for key topics and proposes methods to integrate them into the ICH framework moving forward.

The group recommended drafting of an annex to the ICH Q5E guidance on comparability assessment. The paper states that “expectations for comparability assessments are primarily addressed in ICH Q5E, which is suitable for well characterized biological products. However, the principles are difficult to strictly apply to ATMPs with greater complexity and variability and novel circumstances. There is a need for a broader risk-based approach that uses the totality of evidence to manage uncertainties and unique considerations.”

The group also recommended developing an annex to the ICH Q11 guideline on starting materials, which would provide specific guidance for process development and validation for ATMPs.

Additionally, the CGTDG suggested the creation of a new ICH safety guideline for the nonclinical evaluation of ATMPs. This guideline would address various topics such as species selection, duration and endpoints of nonclinical studies, as well as assessments for carcinogenicity, tumorigenicity, and germline transmission.

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ICH looks to revamp its guidelines on cell and gene therapies

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