Researchers assessed the value of using real-world evidence (RWE) to perform studies on ulcerative colitis (UC) by comparing RWE-based findings with results from the SUCCESS trial, which found that infliximab plus thiopurines improved remission compared with infliximab monotherapy. Findings based on RWE were consistent with findings from the randomized controlled trial, the researchers reported. Their assessment was published in Pharmacoepidemiology and Drug Safety.

The study used the US IBM MarketScan and Optum claims databases as well as SNDS, the French administrative health care database, to compare treatment efficacy based on RWE with results from SUCCESS. The treatment failure end point of SUCCESS was emulated by hospitalization related to UC or colectomy, switch to another biologic or immunosuppressant, or use of corticosteroids at 16 weeks post-infliximab initiation. Risk ratios were estimated in 620 propensity score-matched pairs.

The investigators reported that RWE treatment failure was observed in 124 (20%) of patients receiving infliximab plus thiopurines compared with 170 (27%) of those receiving infliximab alone. Notably, the risk of treatment failure was reduced with the combined regimen in the claims data, similar to the findings from SUCCESS (risk ratio [RR], 0.73; 95% CI, 0.60-0.90). Results were comparable across the 3 databases (IBM MarketScan RR, 0.76; 95% CI, 0.57-1.02; Optum RR, 0.82; 95% CI, 0.54-1.24; SNDS RR, 0.61; 95% CI, 0.41-0.90).

Given the consistency between RWE and randomized clinical trial findings, the authors concluded that studies using RWE were valid for the evaluation of treatment efficacy in patients with UC.

Reference: Kirchgesner J, Desai RJ, Schneeweiss MC, Beaugerie L, Kim SC, Schneeweiss S. Emulation of a randomized controlled trial in ulcerative colitis with US and French claims data: Infliximab with thiopurines compared to infliximab monotherapy. Pharmacoepidemiol Drug Saf. 2022;31(2):167-175. doi:10.1002/pds.5356