Michele Wilson, Giancarlo Castaman, Will Thomas, Carolyn Millar, Ginés Escolar, Wolfgang Miesbach, Cheryl McDade, Radovan Tomic, Songkai Yan
{"title":"Voncento预防与按需治疗血管性血友病在英国的成本效益","authors":"Michele Wilson, Giancarlo Castaman, Will Thomas, Carolyn Millar, Ginés Escolar, Wolfgang Miesbach, Cheryl McDade, Radovan Tomic, Songkai Yan","doi":"10.1182/bloodadvances.2024014376","DOIUrl":null,"url":null,"abstract":"<p><strong>Abstract: </strong>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.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1312-1319"},"PeriodicalIF":7.4000,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Cost-effectiveness of Voncento prophylaxis vs on-demand treatment in von Willebrand disease in the United Kingdom.\",\"authors\":\"Michele Wilson, Giancarlo Castaman, Will Thomas, Carolyn Millar, Ginés Escolar, Wolfgang Miesbach, Cheryl McDade, Radovan Tomic, Songkai Yan\",\"doi\":\"10.1182/bloodadvances.2024014376\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Abstract: </strong>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. 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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.
期刊介绍:
Blood Advances, a semimonthly medical journal published by the American Society of Hematology, marks the first addition to the Blood family in 70 years. This peer-reviewed, online-only, open-access journal was launched under the leadership of founding editor-in-chief Robert Negrin, MD, from Stanford University Medical Center in Stanford, CA, with its inaugural issue released on November 29, 2016.
Blood Advances serves as an international platform for original articles detailing basic laboratory, translational, and clinical investigations in hematology. The journal comprehensively covers all aspects of hematology, including disorders of leukocytes (both benign and malignant), erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Each article undergoes a rigorous peer-review process, with selection based on the originality of the findings, the high quality of the work presented, and the clarity of the presentation.