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Fantastic work from Dr. Rifaquat and colleagues, who showed that carefully matched external control data produced treatment effect estimates nearly identical to those obtained using the randomized control arm of the INSIGhT glioblastoma (GBM) trial. Their findings add to the growing evidence that external controls can, in appropriate scenarios, help reduce the number of patients who must be assigned to traditional control groups while still generating reliable results.
We have long advocated for this approach. Years ago, the Musella Foundation used its brain tumor registry to show that carefully curated and well-matched real-world GBM patient cohorts could approximate outcomes seen in traditional clinical trial control arms. (See HERE) We are very happy to see the neuro-oncology field advancing this concept, as it has great potential to improve the efficiency of clinical trials, increase trial enrollment, and meaningfully benefit patients.