For novel cancer treatments, effectiveness in clinical practice is not always aligned with clinical efficacy results. As such it is important to understand a treatment's real-world effectiveness. We examined real-world population-based comparative effectiveness of second-line ipilimumab versus non-ipilimumab treatments (chemotherapy or targeted treatments). Methods We used a cohort of melanoma patients receiving systemic treatment for advanced disease since April 2005 from Ontario, Canada.

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... nts were identified from provincial drug databases and the Ontario Cancer Registry who received second-line ipilimumab from 2012 to 2015 (treated) or second-line nonipilimumab treatment prior to 2012 (historical controls). Historical controls were chosen, to permit the most direct comparison to pivotal trial findings. The cohort was linked to administrative databases to identify baseline characteristics and outcomes. Kaplan-Meier curves and multivariable Cox regression models were used to assess overall survival (OS). Observed potential confounders were adjusted for using inverse probability of treatment weighting. Results We identified 329 patients with MM who had received second-line treatments (189 treated; 140 controls). Patients receiving second-line ipilimumab were older (61.7 years vs 55.2 years) compared to historical controls. Median OS were 6.9 (95% CI: 5.4-8.3) and 4.95 (4.3-6.0) months for ipilimumab and controls, respectively. The crude 1year, 2-year, and 3-year OS probabilities were 34.3% (27%-41%), 20.6% (15%-27%), and 15.2% (9.6%-21%) for ipilimumab and 17.1% (11%-23%), 7.1% (2.9%-11%), and 4.7% (1.2%-8.2%) for controls. Ipilimumab was associated with improved OS (IPTW HR=0.62; 95% CI: 0.49-0.78; p <0.0001). Conclusions This real-world analysis suggests second-line ipilimumab is associated with an improvement in OS for MM patients in routine practice.