The use of single arm trials has become more widespread, especially as the COVID-19 has forced clinical trials to change or end early. An alternative approach is the use of synthetic control arms (SCAs), arms that can be created using Bayesian Dynamic Borrowing. In this method, data from patients who have been newly enrolled is combined with historical data, thereby reducing the number of patients that must be enrolled into a control group.
“Breakthrough treatments in oncology and rare diseases are now commonly approved based on a pivotal single arm trial – however this is not always optimal. Use of single arm trials in oncology or rare diseases requires appropriate comparisons to be developed to document the benefits of the new treatment. Deriving such comparisons from real world or historical trial data is not straightforward and requires data source and methods expertise.” Read more here.
(Source: Cytel, 2/12/21)