Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China

Abstract

Introduction: Multiple studies have demonstrated the effectiveness of pharmacokinetic (PK)-guided individualized prophylaxis with human coagulation factor VIII (FVIII) compared with standard prophylaxis, but no studies have evaluated the economics of PK-guided prophylaxis in China. Hence, we conducted this study to assess the cost-effectiveness of PK-guided prophylaxis with recombinant FVIII (rFVIII) versus standard prophylaxis in Chinese adult patients with severe hemophilia A. Methods: A discrete event simulation model was developed to simulate 10,000 patients with hemophilia A who received rFVIII treatment over a 1-year time horizon. The standard prophylaxis rFVIII dose was 30 IU/kg by intravenous injection. The PK-guided prophylaxis dosage was adjusted for each patient to maintain FVIII trough level at 1-5 IU/dL. Dosing interval for both approaches was kept fixed at 48 h. The health outcomes included annual joint bleed rate (AJBR) and quality-adjusted life years (QALYs). The model considered the costs of drug. Incremental cost-effectiveness ratio (ICER) was estimated and scenario analysis was performed.Results: A total of 94.3% of patients receiving PK-guided individualized prophylaxis achieved the goal of maintaining the trough concentration at 1-5 IU/dL compared with 62.7% on standard prophylaxis. AJBR and QALYs gained in PK-guided and standard prophylaxis were 1.527 vs 1.601, and 0.8384 vs 0.8383, respectively. Costs of drug prophylaxis and costs of treatment for bleeding events in PK-guided prophylaxis (148,641.47 USD; 4546.43 USD) were lower than those in standard prophylaxis (159,620.93 USD; 4753.39 USD). An average saving of USD 11,186.47 was obtained by the PK-guided approach. The prophylaxis treatment scenarios were the most influential factors. Conclusion: PK-guided individualized prophylaxis appeared to be a dominant treatment compared with standard prophylaxis, with slightly higher QALYs but lower total costs.

Personalized Prophylaxis with myPKFiTCE: A Real-World Cost-Effectiveness Analysis in Haemophilia A Patients

“…Our ICERs are in line with those observed by Gringeri and colleagues, who suggested an incremental cost of EUR 7537.00 per avoided bleed event during prophylaxis [29]. In addition, the PK-guided dosing prophylaxis resulted as cost-effective compared to standard prophylactic approaches in two other studies [25,45]. Specifically, Iannazzo and colleagues performed a cost-effectiveness analysis by comparing PK-driven prophylaxis with standard prophylaxis [25].…”

“…In their study, the authors found that the use of the new device generated an overall cost saving of about EUR 5000 per patient-year and an ICER of about EUR −30,000 per bleed avoided [25]. Similarly, a cost-effectiveness analysis conducted in Gu and colleagues, in 2022, found that the costs associated with treatment injections were lower in PK-guided individualized prophylaxis compared with the standard one [45]. Similarly, the costs for the treatment of bleedings were also lower with the use of PK-guided prophylaxis compared with standard prophylaxis [45].…”

“…Similarly, a cost-effectiveness analysis conducted in Gu and colleagues, in 2022, found that the costs associated with treatment injections were lower in PK-guided individualized prophylaxis compared with the standard one [45]. Similarly, the costs for the treatment of bleedings were also lower with the use of PK-guided prophylaxis compared with standard prophylaxis [45]. The authors found that the PK-guided prophylaxis was dominant (lower costs and more effective) compared with standard prophylaxis [45].…”

“…Similarly, the costs for the treatment of bleedings were also lower with the use of PK-guided prophylaxis compared with standard prophylaxis [45]. The authors found that the PK-guided prophylaxis was dominant (lower costs and more effective) compared with standard prophylaxis [45]. Although having some differences in study design, these findings corroborate the hypothesis that PK-guided individualized prophylaxis represents an opportunity to enhance the management of patients with HA, potentially leading to an improvement in their quality of life.…”

A Response to: Letter to the Editor Regarding “Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China”

“…We would like to express our gratitude to Dr. Huang for his interest and commentary on our publication, “Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China” [ 1 ]. We hope the following information will be useful in addressing his questions and comments.…”