臺灣重度A型血友病患者接受中等劑量預防性治療相對於需要時治療之成本效果分析初探

Abstract

背景 雖然血友病預防性治療目前已知可有效降低重度血友病患者出血事件次數及嚴重程度，在許多已開發國家也成為標準療法，但過去在臺灣，並非成人患者的普遍治療方式。而中央健康保險署核定自2014年7月起，同意實施血友病患者使用中等劑量預防性治療，因此重度血友病患者接受中等劑量預防性治療相對於需要時治療的經濟評估研究，可作為健保署政策決定的參考資料。 目的 本研究旨在分析重度A型血友病且無抗體患者，接受中等劑量預防性治療相對於需要時治療之成本效果分析。 方法 為台灣血栓暨止血學會進行的多中心觀察性研究，以分析兩種治療策略對血友病患者之治療成效；從七個血友病治療中心蒐集資料，且經過IRB審查同意的研究計畫。本研究經台灣血栓暨止血學會授權取得部分資料進行分析，以2014年1月1日起至2016年7月31日止，最長計31個月之資料為基礎，並以中央健康保險署觀點（Perspective）對140位患者資料進行分析。同時依據健保署血友病治療規範，界定A型血友病患者進行中等劑量預防性治療之給藥模式（15-25 IU/kg，一週1-3次）。以年度化之總出血次數為健康效果測量指標；成本項目測量以基因工程第八凝血因子製劑為主，分析每人每年凝血因子耗用量，並換算成年度醫療成本。 結果 預防性治療組之年出血次數（Annual bleeding rate, ABR）較需要時治療組可減少6.71次的出血次數（p-value < 0.05）。接受預防性治療，每人每年凝血因子耗用量相較於需要時治療高出8.33%（預防性治療470萬元，需要時治療434萬元，高出36萬元）。平均而言，避免一次出血事件發生的凝血因子費用為53,915元，而實施預防性治療後的年度財務影響約3億元。 結論 有社會保險制度的國家，多數已核准實施預防性治療，可見積極地在出血事件發生前給予預防性治療，已成為血友病治療趨勢。血友病患的治療不只是患者個人醫療費用的問題，而是有機會讓患者避免成為肢體障礙者，減少整體社會福利成本。本研究中避免一次出血事件發生的凝血因子費用為53,915元，與國外的參考文獻具有相同或更佳的成本效果。然而受限於追蹤時間的限制，對於接受預防性治療未來可能產生的好處尚難以觀察到，例如降低未來關節置換手術的機會及產生的健保費用。需要更長的觀察期以進一步驗證患者接受預防性治療後的健康狀態及影響。

Background Although Prophylactic treatment is a standard of care in many developed countries, known to reduce the severity of hemophilic bleeding events, adult patients in Taiwan have not traditionally used prophylaxis. Recently Taiwan National Health Insurance Administration (NHIA) have announced that adult hemophilia can now adopt Intermediate-dose prophylaxis, therefore the economic evaluation of Intermediate-dose prophylaxis versus On-demand treatments for patients with severe hemophilia in Taiwan is becoming a critical research issue for NHI when making further policy decisions. Aims This study aimed to analyze the cost effectiveness of Intermediate-dose prophylaxis treatment versus On-demand treatment for patients with severe hemophilia A without inhibitor. Methods A multicenter observational study was conducted by the Taiwan Society of Thrombosis and Hemostasis to analyze the effectiveness of two treatment strategies for hemophilia patients. The effects were collected from seven centers that treat hemophilia patients using the IRB approved protocol. This study was authorized by Taiwan Society of Thrombosis and Hemostasis to use a subset of the data to conduct the analysis. Measurement of health effect outcome was the number of bleeding events during observation period collected from the study. Direct costs were estimated according to the Taiwan NHI guidelines for hemophilia treatment by using Intermediate-dose prophylaxis treatment for hemophilia A with recombinant factor VIII (15-25 IU/kg, 1-3 times per week). Results The health effect outcome (Number bleeding events) of Intermediate-dose prophylaxis treatment is lower than On-demand treatment, prophylactic treatment avoids 6.71 episodes of bleeding (p-value <0.05). The annual clotting factors consumption were 8.33% higher for prophylactic treatment than On-demand treatment (approximately NTD 360000/person per year). The incremental cost effectiveness ratio, i.e. the additional costs to avoid per bleeding event by prophylactic treatment, was NTD 53915. The budget impact of adopting prophylactic treatment was estimated to be NTD 300 million per year. Conclusions The selection of treatment methods for hemophilia not only related to cost issues but also has the potential to avoid bleeding events of hemophilia patients. The additional costs to avoid one bleeding event by prophylactic treatment were NTD 53915 per patient per year in this research. Due to the limitation of research follow up period, we were unable to collect other benefits of prophylactic treatment such as evade the total knee replacement surgery of hemophilia patient. Further study examining the impact on more precise health effects and health status over a longer observation period is warranted.