Abstract
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.
Key Words
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Article info
Publication history
Published online: April 19, 2017
Accepted:
March 20,
2017
Received:
January 31,
2017
Identification
Copyright
© 2017 International Society for Cellular Therapy. Published by Elsevier Inc. All rights reserved.
