OBJECTIVES: Vascular Ehlers-Danlos Syndrome (vEDS) is associated with significant morbidity and mortality, including often multiple, clinically severe events, starting as early as the first decade, and a reduced life expectancy to a median of 51 years of age. There is no current approved treatment for vEDS in the US. By increasing collagen production and reducing blood pressure, celiprolol has demonstrated auspicious outcomes in vEDS patients. The aim of our analysis was to quantify the value of using celiprolol on patients with vEDS from the US payer perspective over a 5 year time horizon.

METHODS: A Markov model based on clinical practice was developed to estimate costs and consequences. A retrospective claims analysis was conducted to quantify the frequencies and costs of health resources for patients with and without events. Mortality and event-free survival (EFS) estimates were from published literature and clinical trials. The number of annual events from empiric EFS clinical trial data. The celiprolol cost was set to $0 since it has not yet received market authorization.

RESULTS: With a modeled population of 1 million members, a vEDS prevalence 1/200,000-1/50,000, as a range reported in the published literature, and 84% eligibility for celiprolol based on age, it is estimated that a US payer would treat 4-17 vEDS patients with celiprolol annually. Over 5 years, conventional therapy generated 3.1-12.4 medical events and 0.2-0.9 deaths whereas only 1.1-4.4 events and 0.03-0.11 deaths occurred among patients treated with celiprolol. Furthermore, the use of celiprolol is projected to increase EFS from 37% to 78% costing an additional $16,394/patient (+9.1%) excluding celiprolol costs as a result of prolonged life.

CONCLUSIONS: Utilization of celiprolol prevents severe medical events for a small patient population where treatment options are limited; however it increases healthcare costs due to prolonged survival.