Real-world utilization of top-down and step-up therapy and initial costs in Crohn disease

BACKGROUND: Medication treatment strategies for Crohn disease (CD) include step-up (SU) therapy, beginning with oral anti-inflammatory agents, and top-down (TD) therapy, beginning with biologics or immunomodulators. The real-world utilization and short-term medical costs associated with these treatment strategies are not well described. OBJECTIVE: To examine the prevalence of TD therapy use over time and compare the first-year direct medical expenditures among patients initiating CD medication treatment with SU and TD therapy in a real-world setting. METHODS: We conducted a retrospective cohort study of Optum Clinformatics Data Mart examining adult patients with CD newly initiated on medication therapy from 2010 to 2018. Included patients had a CD-indicated medication dispensed within 60 days after their initial CD diagnosis, were continuously enrolled in the health plan throughout the study period, and did not have comorbidities treated with a biologic also indicated for CD. A generalized linear model was used to quantify the differences in adjusted mean first-year CD-specific, direct nonpharmacy medical costs between users of TD and SU therapy. RESULTS: We identified 3,157 patients newly initiating medication therapy for CD (2,392 [75.8%] patients treated with SU therapy and 765 [24.2%] treated with TD therapy). The use of TD therapy over the study period increased from 17% in 2011 to 31% in 2017. TD therapy was also associated with a 149.8% (51,230) higher adjusted average per-patient first-year CD-direct nonpharmacy medical cost compared with SU therapy (adjusted ratio of cost for TD compared with SU [2.498, 95% CI = 2.12-2.95]). CONCLUSIONS: In patients newly initiating medication therapy for CD, TD therapy use increased between 2010 and 2017 and was associated with higher first-year nonpharmacy medical expenditure. These findings align with the strategy of initiating TD therapy in patients with a higher disease burden. Further research is needed to determine long-term overall health care costs and clinical outcomes associated with SU and TD strategies in a real-world setting.