Long-term prophylaxis in hereditary angioedema: Real-world treatment patterns and healthcare resource utilization

Background Multiple non-androgen long-term prophylaxis (LTP) therapies have been approved in the United States to prevent hereditary angioedema (HAE) attacks. Real-world data on treatment compliance, healthcare resource utilization (HRU), and costs in this population are limited. Objective Assess LTP treatment patterns, associated HRU, and costs in patients with HAE using a national claims database. Methods Commercially insured patients from the IQVIA PharMetrics® Plus Closed Health Plan Claims Database (January 2016–September 2023) had ≥1 claim for non-androgen LTP, with ≥6 months of continuous enrollment pre-index and ≥12 months post-index (first LTP claim). Patient cohorts: no/minimal refill gaps, with refill gaps, or switchers. Annualized mean on-demand therapy claims, HRU, and costs were evaluated 12 months pre- and post-index. Results A total of 328 patients were included in this analysis. Most patients (67%) had ≥1 post-index on-demand therapy claim. Mean (SD) annualized on-demand therapy doses pre- and post-LTP, respectively, were 20.8 (25.1) vs 12.4 (15.2) ( P =0.001) with no/minimal refill gaps (n=147); 18.3 (19.7) vs 18.0 (22.3) ( P =0.769) with refill gaps (n=131); and 25.7 (28.7) vs 29.2 (28.8) ( P =0.12) for switchers (n=50). During follow-up, 17% and 8% had ≥1 HAE-related ER and inpatient visit, respectively. Mean annualized total HAE-related healthcare costs per patient were $165,348 pre-LTP and $515,333 post-LTP, driven by increased LTP pharmacy costs (mean $395,845 PPPY) and partially offset by reductions in medical costs (ER/inpatient, mean $8344 PPPY). Conclusion This study found that 55% of patients had refill gaps in LTP claims, discontinued, or switched LTP within a year of initiation. Even with use of LTP, on-demand treatment utilization remained present for over two-thirds of patients. Substantial increases in total HAE-related healthcare costs were driven by LTP pharmacy costs, without significant reductions in HRU. These findings highlight the importance of monitoring and optimizing treatments for those living with HAE and ensuring access to on-demand treatment for all as the foundation for HAE management.