Department of Biomedical Informatics, The Ohio State University College of Medicine, Columbus, OH, USA.
Center for Medical Technology Policy (CMTP), Baltimore, MD, USA.
Curr Med Res Opin. 2021 Apr;37(4):643-653. doi: 10.1080/03007995.2021.1888707. Epub 2021 Mar 1.
A growing literature on patient preferences informs decisions in research, regulatory science, and value assessment, but few studies have explored how preferences vary across patients with differing treatment experience. We sought to quantify patient preferences for the benefits and risks of lung cancer treatment and test how preferences differed by line of therapy (LOT).
Preferences were elicited using a discrete choice experiment (DCE) following rigorous patient and stakeholder engagement. The DCE spanned five attributes (each with three levels): progression-free survival (PFS), short-term side effects, long-term side effects, risk of developing late-onset side effects, and mode of administration (MOA) - each defined across 3 relevant levels. A D-efficient design was used to generate 3 survey blocks of 9 paired-profile choice tasks each and respondents were asked which profile they preferred and then if they preferred to have no treatment (opt-out). A mixed logit model, controlling for opt-out, was used to estimate preferences. Preferences and trade-offs between PFS and other attributes were compared across two groups: those receiving ≤1 LOT and those receiving ≥2 LOT.
Of the 466 participants, 42% received ≤1 LOT and 58% received ≥2 LOT. Stated preferences differed between the groups overall (<.001) and specifically for 18 months of PFS (<.001), moderate short-term side effects (<.001), no long-term side effects (=.03), and 30% chance of late-onset side effects (=.02). Those receiving differing amounts of LOT were willing to trade different amounts of PFS to change from moderate to mild short-term side effects (<.001), moderate to no (<.001) and mild to no (<.001) long-term side effects. There were also differing amounts of tradeoff acceptable between the groups for a 10% decrease in risk of late-onset side effects (=.016), a decrease in MOA from infusion every 3 weeks to pills taken daily at any time (=.005) and from pills taken daily without food to pills taken daily at any time (<.001).
We demonstrate differences in preferences based on experience with LOT, suggesting that patient treatment experience may have an impact on their preferences. As patient preference data become an important component of treatment decision making, preference differences should be considered when recommending therapies at different stages in the treatment journey. Understanding patient preferences regarding treatment decisions is essential to informing shared decision-making and ensuring treatment plans are consistent with patients' goals.
越来越多的患者偏好文献为研究、监管科学和价值评估中的决策提供了信息，但很少有研究探讨不同治疗经验的患者对偏好的差异。我们旨在量化肺癌治疗的获益和风险方面的患者偏好，并检验偏好是否因治疗线数（LOT）而异。
采用离散选择实验（DCE）方法，在严格的患者和利益相关者参与后得出偏好。DCE 涵盖五个属性（每个属性有三个水平）：无进展生存期（PFS）、短期副作用、长期副作用、发生晚期副作用的风险和给药方式（MOA）-每个属性都有 3 个相关水平定义。采用 D 有效设计生成 3 个调查块，每个块包含 9 个配对的特征选择任务，要求受访者选择他们偏好的特征，并询问他们是否宁愿不治疗（选择退出）。采用混合 Logit 模型（控制选择退出）来估计偏好。比较了两组患者的 PFS 和其他属性之间的偏好和权衡：接受 ≤1 LOT 的患者和接受 ≥2 LOT 的患者。
在 466 名参与者中，42%接受 ≤1 LOT，58%接受 ≥2 LOT。总体而言，两组之间的偏好存在差异（<0.001），特别是 18 个月的 PFS（<0.001）、中度短期副作用（<0.001）、无长期副作用（=0.03）和 30%的晚期副作用风险（=0.02）。接受不同 LOT 数量的患者愿意为从中度短期副作用变为轻度短期副作用（<0.001）、从中度变为无（<0.001）和从轻度变为无（<0.001）的长期副作用而进行不同数量的权衡。两组之间在接受晚期副作用风险降低 10%（=0.016）、从每 3 周输注改为每天服药（=0.005）和从每天空腹服药改为每天任何时间服药（<0.001）方面的可接受权衡也有不同数量。
我们证明了基于 LOT 经验的偏好差异，表明患者的治疗经验可能会影响他们的偏好。随着患者偏好数据成为治疗决策的重要组成部分，在推荐不同治疗阶段的治疗方法时，应考虑偏好差异。了解患者对治疗决策的偏好对于告知共同决策和确保治疗计划与患者的目标一致至关重要。
