P551 Benefit–risk trade-offs and patient preferences for therapy selection in Ulcerative Colitis: a multi-country preference study

Abstract

Abstract Background The treatment landscape of ulcerative colitis (UC) is increasingly complex. In the absence of predictive biomarkers and objective parameters, shared treatment decision-making is advocated in clinical practice. This study quantified the benefit–risk trade-offs that patients with UC are willing to make when choosing a treatment. A patient-centric benefit–risk assessment of filgotinib, an oral, Janus kinase 1 preferential inhibitor, was also conducted to show how measured trade-offs can be used. Methods Patients recruited through databases, panels, social media and patient associations completed an online discrete choice experiment (DCE). Eligible patients were aged ≥18 years, had a diagnosis of UC for ≥6 months and resided in France, Germany, Italy, Spain or the UK. DCE treatment attributes were identified using a literature review, clinical data and patient interviews. Patients completed the DCE by choosing between a series of two hypothetical treatment options with varying attributes which were systematically varied to ensure trade-offs were made. Data were analysed using a mixed logit model. Relative attribute importance (RAI) scores and maximum acceptable risks were generated. Preference data were combined with SELECTION clinical trial data to evaluate the desirability of filgotinib 200 mg versus placebo from the patients’ perspective.1 Results Among 631 patients who completed the survey (8997 patients invited), 75.3% were male and the mean age (standard deviation) was 42.2 (7.7) years. Patients preferred a daily oral pill over self-injection or intravenous treatment, and a treatment that avoided steroid use (Figure). Achieving and maintaining remission (RAI 32.4%; 95% confidence interval [CI] 29.5–35.2%) was considered the most important factor when choosing treatment. To achieve a 20% increase in the chance of remission, patients were willing to accept a 1.4% (95% CI 1.2–1.6%) increase in the risk of blood clots, a 2.0% (95% CI 1.7–2.3%) increase in the risk of serious infections, and a 0.7% (95% CI 0.6–0.8%) increase in the risk of malignancies (Table). Analysing patient preference and clinical data suggests filgotinib has a 70.1% chance of being preferred over placebo (p<0.001). Conclusion Treatment preferences of patients were mostly driven by the likelihood of achieving and maintaining remission. To achieve this, they were willing to accept some increase in the risks of blood clots, serious infections and malignancies. Patients also valued avoiding steroids. A patient-centric benefit–risk assessment indicated that patients are significantly more likely to prefer filgotinib over placebo, illustrating how preference data can be used with clinical data to inform decision-making. 1. Feagan BG et al. Lancet 2021;397:2372–84.