Overview
Colorectal cancer is the third most common cancer globally, with rectal cancer accounting for ~30% of all colorectal cancers. Dostarlimab is a new potential treatment for patients with a rare form of rectal cancer (mismatch repair deficient/microsatellite instability-high locally advanced rectal cancer) that responds poorly to currently available chemotherapies. Due to the rarity of this disease, a traditional randomized clinical trial is not possible and therefore real-world evidence will be critical to contextualize single-arm trial results.
Researchers utilized the Flatiron Health Research Database to emulate the clinical trial population for the AZUR-1, demonstrating the feasibility for real-world data’s inclusion in a planned natural history study that will be critical for the approval of a new treatment in the early rectal space. The team found patient distributions in the feasibility cohort align with existing clinical data for this population, indicating representativeness for dMMR/MSI-H locally advanced rectal cancer.
Why this matters
These findings support real-world data’s ability to provide the evidence needed to approve new therapies for rare patient groups. Where traditional clinical trials are not feasible, rich and high-quality real-world evidence can contextualize single-arm trial results in regulatory settings, enabling better treatments to reach patients faster.