External Control Arms: Better Than Single-Arm Studies But No Replacement For Randomization
Synthetic control group derived from historical clinical trial data could augment smaller randomized trials and yield better information than single-arm studies, but this approach should not be viewed as a substitute for randomized trials where feasible, US FDA officials said at a Friends of Cancer Research meeting.
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Is it real, or is it synthetic? When it comes to drug development data in the future, the answer could be the latter. Datasets that replicate the statistical patterns of real patient populations but carry no risk of privacy disclosures could enable the use of synthetic control arms. However, the biopharma industry and regulators will need assurance that these datasets accurately reflect the population from which they were derived.
Agency’s approach to assessing real-world evidence for effectiveness will be guided by three key principles: fitness for use of the underlying data, adequacy of the study design, and satisfactory monitoring and data collection.
CDC’s advisory committee also approves a VFC program update allowing for the rare, off-label use of tetanus toxoid- and diphtheria toxoid-containing vaccine in young children, and plans to move up timing of its COVID-19 vaccine recommendations for the 2024-2025 formulation.