国内心房颤动患者对新型口服抗凝药物的偏好和支付意愿调查

Background: Disease burden of atrial fibrillation (AF) and associated cardiovascular complications is increasing in China in the past ten years. Comparing to traditional anticoagulation therapy (warfarin), novel oral anticoagulants (NOACs) offer superior or similar efficacy in stroke prevention but are more costly which barriers of their broader utilisation in China. ..Objectives: to elicit patients preferences between warfarin and NOACs, identifying their knowledge gap for the disease and treatments, measuring the WTP for NOACs and informing NOACs pricing policy...Methods: A cross-sectional survey will be conducted in patients with AF recruiting from two hospitals in the North and South of China. Important attributes of anticoagulants were identified based on a group of clinical expert consultation. A discrete choice experiment (DCE) was applied to design an interface electronic questionnaire which simulated 8 scenarios with different combination of treatment profiles (safety, efficacy and cost et al) for the choice from patients. Random-effect probit regression models will be used to rank the importance of attributes and quantify the WTP for NOACs...Feasibility: The research team has adequate experience in conducting studies regarding the utilisation of NOACs including population-based medication safety analysis, cost-effectiveness analysis and AF disease burden studies. The DCE questionnaire had been successfully piloted in 89 patients with AF in outpatient settings. Completion time of the survey was 15-20 minutes per patient that supports the feasibility of study...Expected results and applications: DCE technic is increasingly used in the area of pharmacoeconomics globally to address a wide range of health policy related concerns. In this study, it is the first application of DCE in China in investigating the preferences and WTP of anticoagulation therapy in patients with AF. This study will provide invaluable information to facilitate improved acceptability of anticoagulation therapy. By comparing consumers’ WTP with actual medication cost, the results are expected to inform the price-negotiation from divers stakeholders and initiate drug-listing of NOACs in China.

我国房颤及相关心脑血管并发症的疾病负担逐年上升。抗凝治疗能有效预防房颤性中风，新型口服抗凝药（NOACs）疗效显著，但价格高昂，难以广泛应用。本研究在房颤患者中开展双中心横断面调查，旨在揭示患者对传统和新型药物的选择偏好，了解其对疾病和抗凝治疗认知程度，量化支付意愿（WTP），为NOACs的药物政策提供参考。课题运用离散选择性试验设计（DCE），在电子问卷中模拟8种情景，供患者在两种药物中权衡选择；运用多元指数回归模型，为药物属性（安全性、有效性和价格等）作重要性排序，并量化WTP。项目团队在NOACs安全用药、卫生经济评估和疾病负担研究方面皆有较丰富的经验。初期预实验中，已有89名门诊患者完成调查，每份问卷耗时约15-20分钟，研究具可行性。DCE是药物经济学领域的新兴技术，在抗凝意愿调查中的应用尚属国内首次。研究结果将有助于提高药物接受度，并为NOACs多方价格谈判及医保纳入提供参考。

我国房颤患者人数众多，且用药依从性不佳。新型口服抗凝药（NOAC）获批上市以来，成为除华法林之外治疗房颤的又一选择，但高昂的自付价格限制了普及。除价格因素外，患者对药物有效性、安全性的偏好同样是用药依从性的决定因素。本项目采用离散选择性试验，调查房颤患者对口服抗凝药物的选择偏好和支付意愿，旨在从患者角度揭示影响口服抗凝药使用的药物属性和经济因素，为其在抗凝及预防脑卒中领域的应用提供积极的推动作用。基于少量样本（患者和医生各40名）的预试验，我们确定了7个待研究药物属性及其水平，并应用Ngene软件的高效设计模块生成了终版问卷（A、B两份问卷各含1个测试情景与8个正式情景）。随后于中国人民解放军总医院（北京）和香港大学深圳医院（深圳）正式开展问卷调查。目标收集600份问卷，最终共完成有效问卷506份，患者平均年龄70.3岁，女性占比42.1%，住院病例占比72.3%。经混合效应模型分析后发现，中国房颤患者整体上对药物的出血风险、卒中或系统性栓塞风险以及急性心梗风险最为关心，为降低这些风险的额外支付的药费额度为每月536至798元。与其他属性相比，血液监测频率最不受到患者重视，支付意愿仅为每月244元。亚组分析结果中食物药物交互作用的偏好权重与主分析差异较大。男性、有卒中或出血疾病史、北京现场招募的患者，对该属性有较高的偏好权重，甚至超过了对出血、卒中和急性心梗风险的重视程度，他们愿意额外支付的药费也相当于整体患者人群的2倍或以上。教育水平在本科或以上的患者，以及税前个人月收入5,000元以上的患者的支付意愿波动较大。本项目深入探索了可能影响患者选择偏好的关键因素，为今后改进药物依从性、改善治疗方案及可能的医保价格调整提供了科学依据，对其他口服抗凝药纳入医保及未来医保定价具有一定的参考意义。本项目完成1次国际学术交流，1篇在审SCI文章，1篇在投SCI文章。本、硕、博士生各1名参与本项目。项目投入直接经费18万元，支出17.4万元，各项支出基本符合预算。