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疾病活动度对难以获取昂贵创新药物国家中类风湿关节炎治疗选择的影响:风湿病学家的离散选择实验

Influence of disease activity on RA treatment choices in countries with restricted access to expensive, innovative drugs: a discrete choice experiment among rheumatologists.

作者信息

Hifinger Monika, Hiligsmann Mickael, Ramiro Sofia, Watson Verity, Berghea Florian, Péntek Márta, Keat Andrew, Severens Johan L, Fautrel Bruno, Boonen Annelies

机构信息

CAPHRI Research Institute,Maastricht University, Maastricht, The Netherlands.

Department of Rheumatology, Maastricht University Medical Center, Maastricht, The Netherlands.

出版信息

RMD Open. 2017 Sep 4;3(2):e000453. doi: 10.1136/rmdopen-2017-000453. eCollection 2017.

Abstract

OBJECTIVE

To assess the influence of disease activity of patients with rheumatoid arthritis on treatment choices of rheumatologists in countries with restricted access to expensive, innovative drugs.

METHODS

Rheumatologists from Hungary, Romania and UK were invited to complete two consecutive discrete choice experiments with hypothetical drug treatments for two different patient profiles: high and moderate disease activity. Rheumatologists were asked to choose repeatedly between two unlabelled treatment options that differed in five attributes: efficacy (expected improvement and achieved disease activity state), safety (probability of serious adverse events), patient's preference (level of agreement), total medication costs and cost-effectiveness. A heteroscedastic discrete choice model using interaction terms between attribute levels and patient profiles (binary variable) was used to assess the preferences of rheumatologists towards each attribute and the influence of the patient profile.

RESULTS

Overall, 148 rheumatologists completed the survey (46% females, mean age 49 years, 49% academic). For both patient profiles, efficacy dominated the treatment choice over patient's preference, safety and economic aspects. However, for patients with high compared with moderate disease activity, the importance of drug efficacy significantly increased (from 48% for moderate to 57% for high disease activity), whereas the importance of patient's preference significantly decreased (from 15% to 11%). No significant differences were observed for economic and safety considerations.

CONCLUSION

Rheumatologists were willing to give up some efficacy to account for patient's preference when choosing treatments for patients with moderate compared to high disease activity. Disease activity however did not influence importance of economic aspects in treatment choices.

摘要

目的

评估在难以获得昂贵创新药物的国家中,类风湿关节炎患者的疾病活动度对风湿病学家治疗选择的影响。

方法

邀请来自匈牙利、罗马尼亚和英国的风湿病学家,针对两种不同患者情况(高疾病活动度和中度疾病活动度),用假设的药物治疗方案完成两个连续的离散选择实验。要求风湿病学家在两个未标记的治疗方案之间反复进行选择,这两个方案在五个属性上存在差异:疗效(预期改善情况和达到的疾病活动状态)、安全性(严重不良事件的概率)、患者偏好(认同程度)、总药物成本和成本效益。使用属性水平与患者情况(二元变量)之间的交互项的异方差离散选择模型,来评估风湿病学家对每个属性的偏好以及患者情况的影响。

结果

总体而言,148名风湿病学家完成了调查(46%为女性,平均年龄49岁,49%为学术人员)。对于两种患者情况,疗效在治疗选择中比患者偏好、安全性和经济方面更为重要。然而,与中度疾病活动度的患者相比,高疾病活动度患者的药物疗效重要性显著增加(从中度的48%增至高疾病活动度的57%),而患者偏好的重要性显著降低(从15%降至11%)。在经济和安全性考量方面未观察到显著差异。

结论

与高疾病活动度的患者相比,在为中度疾病活动度的患者选择治疗方案时,风湿病学家愿意为顾及患者偏好而牺牲一些疗效。然而,疾病活动度并未影响治疗选择中经济方面的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1ee4/5588932/d7c554a4196e/rmdopen-2017-000453f01.jpg

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