There has been an increased use of synthetic control arms for regulatory submissions in recent years, with three rare disease submissions and 22 medical device submissions approved using these techniques.
Synthetic control arms are virtual trial arms that use historical claims data and observational data to simulate the control arm of a study. When enrollment targets are low and large amounts of data already exist about the performance of a control, then in many cases using quantitative techniques to simulate a control arm of a trial will expedite timelines and serve as a more optimal use of resources.
Just as various adaptations suit different needs in an adaptive trial, the availability of data, the desired sample size and the anticipated length of a trial will influence how a synthetic control arm should be constructed. Cytel’s ebook gives an overview of these considerations, and takes a closer look at statistical techniques familiar to those using synthetic controls.
Download to learn more about synthetic controls, suitable conditions for their use, and some common quantitative strategies for trial design and regulatory submission.