Cost-effectiveness of Voncento prophylaxis vs on-demand treatment in von Willebrand disease in the United Kingdom.

Abstract: von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in patients with VWD treated with Voncento in the United Kingdom. A Markov structure was developed to estimate the quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon. Treatment options included ODT or LTP. For both options, we assumed plasma-derived VWF/factor VIII 2.4:1 (Voncento) as the VWF product used. Clinical parameters were obtained from published literature and Voncento's summary characteristics. Utility weights were obtained from published literature. Costs (in 2021 GBP [£]) and outcomes were discounted annually by 3.5%. Sensitivity analyses were conducted. Three baseline annual bleed rate (ABR) scenarios (11, 26.5, and 39.6) were considered. In the base-case analyses, Voncento LTP resulted in lower costs (-£831 206) and greater QALYs (6.14) vs ODT. Savings were primarily due to reductions in product use required (-£529 571) and bleed-related other medical costs (-£301 352). Compared with ODT, LTP also resulted in 322.52 fewer major bleeds and 515.68 fewer minor bleeds over a lifetime horizon. Probabilistic sensitivity analyses showed dominance in 96.12% of simulations and cost-effectiveness in 97.68% of simulations. For the 39.6 ABR scenario also, LTP was dominant compared with ODT. Results suggest that Voncento LTP is more effective and cost saving than ODT in the United Kingdom for patients with VWD with higher ABR. Prophylaxis for patients with frequent bleeds is likely to be a cost-saving and effective strategy.