Synthetic control arms (SCA) 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.
Cytel’s new audiobook "Demystifying synthetic control arms", provides insights on synthetic controls, suitable conditions for their use, and some common quantitative strategies for trial design and regulatory submission. Click the button download the audiobook.
The rising costs of clinical trial completion and the greater availability of data, make the use of SCA a less-costly option adopted in a number of clinical trials, owing to the absence of either a full or partial control group. In place of collecting data from patients recruited for a trial who have been assigned to the control or standard-of-care arm, a synthetic control creates a comparator arm using either real-world datasets such as electronic health records or previous clinical trials. The synthetic control offers a practical, effective way to leverage real-world evidence and has been applied in regulatory approvals. It saves the costs and times associated with patient recruitment and retention, while avoiding the need to give very ill patients an already well-tested candidate drug. Regulators in both the United States and Europe have responded positively to the use of SCAs in clinical development.
In areas of high unmet need, it is critical to bring vital new medicines to patients faster, yet conventional development pathways involving multiple stages can be slow, expensive, and inefficient. In many breakthrough treatment areas, where the patient population is small, or there is overwhelming evidence of efficacy at Phase 2, it has become common for drugs to be approved based on a pivotal single arm trial.
However, 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 "Demystifying synthetic control arms" audiobook provides an overview of these considerations, takes a closer look at the statistical technique and explains some of the complexities faced when employing these new methods. Click the button to access the audiobook. We will also provide you with the opportunity to download our related synthetic control arm content for further knowledge of this method.