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Published on: August 18, 2023
Understanding clinical development of chimeric antigen receptor T cell therapies
Sofieke de Wilde1, Henk-Jan Guchelaar1, Maarten Laurens Zandvliet1
1Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands.
Background Aims:
In the past decade, many clinical trials with gene- and cell-based therapies (GCTs) have been performed. Increased interest in the development of these drug products by various stakeholders has become apparent. Despite this growth in clinical studies, the number of therapies receiving marketing authorization approval (MAA) is lagging behind. To enhance the success rate of GCT development, it is essential to better understand the clinical development of these products. Chimeric antigen receptor (CAR) T cells are a GCT product subtype with promising efficacy in cancer treatment which are tested in many clinical trials, but have not yet received MAA.
Methods:
We generated an overview of the characteristics of CAR T-cell clinical development in the United States, Canada and Europe. Subsequently, the characteristics of clinical trials with CAR T-cell products that proceeded to a subsequent clinical trial, used as a proxy for success, were compared with those that did not proceed.
Result:
From the U.S. and European Union clinical trial databases, 106 CAR T-cell trials were selected, from which 49 were linked to a subsequent trial and 57 were not. The majority of the trials had an academic sponsor from which most did not proceed, whereas most commercially sponsored trials were followed by another clinical trial. Furthermore, trials with a subsequent trial more frequently recruited large patient cohorts and were more often multicenter compared with trials that were not followed up.
Discussion:
These characteristics can be used by investigators to better design clinical trials with CAR T cells. We encourage sponsors to plan clinical development ahead for a higher efficiency of product development and thereby achieving a higher success rate of development towards MAA.
Insights
Commercial sponsorship and larger, multicenter trials are associated with higher success rates in chimeric antigen receptor (CAR) T-cell clinical development. Planning ahead can improve gene- and cell-based therapy (GCT) development efficiency and marketing authorization approval (MAA) success.
Area of Science:
- Oncology
- Immunotherapy
- Clinical Trial Design
Background:
- Gene- and cell-based therapies (GCTs) show growing interest, but marketing authorization approval (MAA) rates lag.
- Chimeric antigen receptor (CAR) T-cell therapy is a promising GCT subtype for cancer treatment with numerous ongoing clinical trials.
- Understanding clinical development characteristics is crucial for improving GCT success rates.
Purpose of the Study:
- To provide an overview of CAR T-cell clinical development characteristics in the US, Canada, and Europe.
- To compare the characteristics of CAR T-cell trials that proceeded to a subsequent trial (proxy for success) versus those that did not.
Main Methods:
- Analysis of 106 CAR T-cell trials from US and EU clinical trial databases.
- Categorization of trials based on progression to a subsequent clinical trial.
- Comparison of trial characteristics including sponsor type, cohort size, and number of centers.
Main Results:
- 49 out of 106 CAR T-cell trials were linked to a subsequent trial.
- Commercially sponsored trials were more likely to proceed compared to academic trials.
- Trials that proceeded to a subsequent trial more frequently recruited large patient cohorts and were multicenter.
Conclusions:
- Investigator-identified characteristics can optimize CAR T-cell clinical trial design.
- Proactive planning by sponsors can enhance GCT product development efficiency.
- Improved trial design and planning are essential for increasing the success rate of GCT development towards MAA.
