Fischer Ann-Kathrin, Sadler Andrew, Mathey Elke, Mühlbacher Axel
Health Economics and Health Care Management, Hochschule Neubrandenburg, Neubrandenburg, Germany.
Gesellschaft für empirische Beratung mbH, An-Institut Hochschule Neubrandenburg, Berlin, Germany.
PLoS One. 2025 Sep 15;20(9):e0329743. doi: 10.1371/journal.pone.0329743. eCollection 2025.
Type 2 diabetes mellitus (T2D) represents a major public health challenge with significant effects on morbidity and mortality. Clinical guidelines provide treatment recommendations, but there is limited understanding of patients' preferences. This study aimed to elicit preferences for second-line drug treatments for T2D.
A Discrete Choice Experiment with a partial-profile design was conducted from August to November 2023, involving German patients with experience in either monotherapy or second-line drug treatment. Participants completed 12 choice tasks, each presenting three alternatives described by attributes: risk of myocardial infarction, risk of stroke, risk of nerve damage, risk of nausea, risk of severe hypoglycemia, weight change, type and frequency of intake, and schedule of intake. Statistical analyses employed the Conditional Logit and Random Parameter Logit models to assess main effects and heterogeneity.
The study encompassed 583 adult individuals with T2D, evenly divided between the two populations. Key factors influencing choice decisions included risk of nausea, risk of nerve damage, and weight change, with weekly type and frequency of intake risk of myocardial infarction followed. Less impactful but still relevant were risks of stroke, and severe hypoglycemia, while the intake schedule was least significant. Analysis of BMI categories revealed distinct preferences, particularly in weight change, with significant heterogeneity observed among respondents.
This study highlights the importance of incorporating patient preferences into clinical decision-making. By quantifying preferences for second-line drug treatments, the study underscores the need for low-risk options that also consider weight change and intake conditions, aligning with the German National Health Care Guideline for T2D objectives for shared decision-making and treatment adherence. Recognizing individual sensitivities to risks and benefits is crucial for tailoring effective T2D treatment strategies. The study bridges clinical findings with patient perspectives, offering valuable insights into clinical practice, consideration for HTA processes, and design of clinical studies.
2型糖尿病（T2D）是一项重大的公共卫生挑战，对发病率和死亡率有重大影响。临床指南提供了治疗建议，但对患者偏好的了解有限。本研究旨在了解T2D二线药物治疗的偏好。
2023年8月至11月进行了一项采用部分轮廓设计的离散选择实验，纳入了有单药治疗或二线药物治疗经验的德国患者。参与者完成了12项选择任务，每项任务呈现三种由以下属性描述的备选方案：心肌梗死风险、中风风险、神经损伤风险、恶心风险、严重低血糖风险、体重变化、摄入类型和频率以及摄入时间表。统计分析采用条件Logit模型和随机参数Logit模型来评估主要效应和异质性。
该研究纳入了583名成年T2D患者，两组人群各占一半。影响选择决策的关键因素包括恶心风险、神经损伤风险和体重变化，其次是心肌梗死风险的每周摄入类型和频率。中风风险和严重低血糖风险的影响较小但仍相关，而摄入时间表的影响最小。对体重指数类别的分析显示出不同的偏好，尤其是在体重变化方面，受访者之间存在显著的异质性。
本研究强调了将患者偏好纳入临床决策的重要性。通过量化对二线药物治疗的偏好，该研究强调了需要低风险的选择，同时也要考虑体重变化和摄入条件，这与德国T2D国家医疗保健指南中关于共同决策和治疗依从性的目标一致。认识到个体对风险和益处的敏感性对于制定有效的T2D治疗策略至关重要。该研究将临床发现与患者观点联系起来，为临床实践、卫生技术评估流程的考量以及临床研究的设计提供了有价值的见解。
