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Statistical analysis plan considerations for autologous cell and cell-based gene therapy clinical trials
Patricia Anderson1, Revathi Ananthakrishnan2, Zhenzhen Xu3
1Biostatistics Consulting Services, ICON plc, Raleigh, NC, USA.
Abstract:
By early 2023, there were over 100 gene, cell and RNA products approved globally (Chancellor et al. 2023). The way in which autologous cell and cell-based gene therapy products are administered can include multiple treatment stages, at times including leukapheresis, optional bridging therapy during manufacturing, conditioning regimen, and one or more doses of product. This brings important considerations in the development of the statistical analysis plan for clinical trials of autologous cell and cell-based gene therapies, including application of the estimand framework for key facets. In this article, we review all of the FDA currently approved autologous cell and cell-based gene therapy products as examples as they apply to general considerations for the statistical analysis plan as well as more in-depth discussion of treatment exposure, safety and efficacy analyses, and other aspects.
Insights
Statistical analysis plans for autologous cell and gene therapies require careful consideration due to complex treatment stages. This review examines FDA-approved products to guide trial design.
Area of Science:
- Biomedical Science
- Clinical Trials
- Gene Therapy
Background:
- Over 100 gene, cell, and RNA products approved globally by early 2023.
- Autologous cell and gene therapy administration involves complex, multi-stage treatment protocols.
- These stages include leukapheresis, bridging therapy, conditioning, and product dosing.
Purpose of the Study:
- To review FDA-approved autologous cell and gene therapy products.
- To provide general considerations for statistical analysis plans (SAP) in clinical trials.
- To discuss specific aspects like treatment exposure, safety, and efficacy analyses.
Main Methods:
- Review of all FDA-approved autologous cell and cell-based gene therapy products.
- Application of the estimand framework for key statistical facets.
- In-depth discussion of statistical considerations for clinical trial design.
Main Results:
- Complex treatment administration necessitates robust SAP development.
- The estimand framework is crucial for addressing trial complexities.
- Specific analyses for treatment exposure, safety, and efficacy are highlighted.
Conclusions:
- Statistical analysis plans for autologous cell and gene therapies must account for intricate treatment pathways.
- Utilizing FDA-approved products as examples aids in developing effective SAPs.
- Careful planning ensures rigorous evaluation of safety and efficacy in gene therapy trials.
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