Enriching single-arm clinical trials with external controls: possibilities and pitfalls.

ABSTRACT: For the past decade, it has become commonplace to provide rapid answers and early patient access to innovative treatments in the absence of randomized clinical trials (RCT), with benefits estimated from single-arm trials. This trend is important in oncology, notably when assessing new targeted therapies. Some of those uncontrolled trials further include an external/synthetic control group as an innovative way to provide an indirect comparison with a pertinent control group. We aimed to provide some guidelines as a comprehensive tool for (1) the critical appraisal of those comparisons or (2) for performing a single-arm trial. We used the example of ciltacabtagene autoleucel for the treatment of adult patients with relapsed or refractory multiple myeloma after 3 or more treatment lines as an

SUBMITTER: Lambert J 

PROVIDER: S-EPMC10539876 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature