患者对转移性结直肠癌治疗的偏好:使用离散选择实验和最佳-最差缩放的多方法方法。

IF 3.1 3区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Carina Oedingen, Karen V MacDonald, Barry D Stein, Gerald Batist, Winson Y Cheung, Sharlene Gill, Benjamin A Goldenberg, Yoo-Joung Ko, Deborah A Marshall
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引用次数: 0

摘要

背景:转移性结直肠癌(mCRC)的治疗决策需要患者平衡生存获益、健康相关生活质量(HRQoL)和潜在副作用风险,同时也要考虑他们自己对不同治疗方案的偏好。尽管越来越多的兴趣,定量患者的偏好尚未纳入卫生技术评估(hta)的药物报销建议。目的:加拿大结直肠癌患者价值项目旨在探索将定量患者偏好纳入癌症治疗HTA决策过程的方法。作为第一步,我们使用多方法方法在加拿大引出mCRC患者的治疗偏好和风险耐受性。方法:我们开发了一项偏好调查,包括两个离散选择实验(DCEs)和案例1最佳-最差量表(BWS-1),以估计对mCRC治疗的偏好。DCE1包括HRQoL和总生存期的变化作为属性,DCE2的治疗属性是给药和频率、副作用(恶心、疼痛、腹泻)和无进展生存期。BWS-1包括基于癌症特异性生活质量问卷的25个潜在mCRC治疗副作用属性。该调查在加拿大范围内对年龄≥18岁的mCRC患者进行,这些患者通过患者组织、癌症中心和在线小组自我报告诊断为mCRC。使用混合logit和潜在类别模型(DCEs)和基于计数的分析(BWS-1)对数据进行分析。结果:总体而言,127例mCRC患者完成了完整调查(n = 143例完全完成DCE1, n = 108例完全完成DCE2, n = 127例完全完成BWS-1)。研究中对治疗属性的相对偏好与预期一致,即更好的临床结果优于更差的临床结果。在DCE1中,患者对总生存期(24个月vs 12个月)和HRQoL(改善至90分vs恶化至50分)的评价几乎同样重要。在DCE2中,患者更喜欢更好的结果(更长的无进展生存期和无副作用)而不是更差的结果,与静脉输注相比,口服胶囊/药片的效用更低。根据结直肠癌治疗经验、治疗副作用和健康状况,观察到显著的偏好异质性。在BWS-1中,“进食、穿衣、洗澡或上厕所需要帮助”、“呕吐”和“疼痛”被列为最不需要的副作用,而“需要休息”、“难以进行剧烈活动”和“感到疲倦”是最可容忍的副作用。结论:本研究强调了综合评估mCRC治疗偏好和风险耐受的多方法方法的价值。通过对多种偏好诱导方法进行三角测量,我们的研究结果为将患者观点整合到加拿大HTA框架中提供了更坚实的基础。这些结果将为加拿大结直肠癌患者价值项目的下一步提供信息,该项目旨在探索将患者偏好与临床和经济证据明确纳入加拿大癌症治疗HTA决策过程的方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Patient Preferences for Metastatic Colorectal Cancer Treatment: A Multi-method Approach Using Discrete Choice Experiments and Best-Worst Scaling.

Background: Treatment decisions for metastatic colorectal cancer (mCRC) require patients to balance survival benefits, health-related quality of life (HRQoL), and potential risks of side effects while also factoring in their own preferences for different treatment options. Despite growing interest, quantitative patient preferences are not yet integrated into health technology assessments (HTAs) for drug reimbursement recommendations.

Objectives: The Colorectal Cancer Canada's Patient Values Project aims to explore approaches to incorporate quantitative patient preferences into cancer treatment HTA decision-making processes. As a first step, we elicited the treatment preferences and risk tolerance of patients with mCRC in Canada using a multi-method approach.

Methods: We developed a preference survey that included two discrete choice experiments (DCEs) and case 1 best-worst scaling (BWS-1) to estimate preferences for mCRC treatments. DCE1 included change in HRQoL and overall survival as attributes, and treatment attributes in DCE2 were administration and frequency, side effects (nausea, pain, diarrhea), and progression-free survival. The BWS-1 included 25 attributes of potential mCRC treatment side effects based on the cancer-specific quality-of-life questionnaire. The survey was administered across Canada to patients with mCRC aged ≥ 18 years with a self-reported diagnosis of mCRC through patient organizations, cancer centers, and an online panel. Data were analyzed using mixed logit and latent class models (DCEs) and count-based analysis (BWS-1).

Results: Overall, 127 patients with mCRC completed the full survey (n = 143 fully completed DCE1, n = 108 fully completed DCE2, n = 127 fully completed BWS-1). Relative preferences for the treatment attributes in the study were consistent with the expectation that better clinical outcomes were preferred over worse clinical outcomes. In DCE1, patients valued both overall survival (24 vs. 12 months) and HRQoL (improvement to 90 vs. worsens to 50 out of 100) as almost equally important. In DCE2, patients preferred better outcomes (longer progression-free survival and no side effects) over worse outcomes, with a disutility for oral capsules/pills compared with intravenous infusions. Significant preference heterogeneity was observed depending on experiences with CRC treatments, treatment side effects, and health status. In the BWS-1, "need help with eating, dressing, washing yourself or using the toilet", "vomiting", and "pain" were ranked as the least and "need to rest", "trouble doing strenuous activities", and "feel tired" as the most tolerable side effects.

Conclusions: This study highlights the value of a multi-method approach in comprehensively assessing treatment preferences and risk tolerance in mCRC. By triangulating multiple preference-elicitation methods, our findings offer a more robust foundation for integrating patient perspectives into Canada's HTA framework. These results will inform the next step of the Colorectal Cancer Canada's Patient Values Project, which aims to explore approaches to explicitly incorporate patient preferences alongside clinical and economic evidence into the cancer treatment HTA decision-making process in Canada.

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来源期刊
Patient-Patient Centered Outcomes Research
Patient-Patient Centered Outcomes Research HEALTH CARE SCIENCES & SERVICES-
CiteScore
6.60
自引率
8.30%
发文量
44
审稿时长
>12 weeks
期刊介绍: The Patient provides a venue for scientifically rigorous, timely, and relevant research to promote the development, evaluation and implementation of therapies, technologies, and innovations that will enhance the patient experience. It is an international forum for research that advances and/or applies qualitative or quantitative methods to promote the generation, synthesis, or interpretation of evidence. The journal has specific interest in receiving original research, reviews and commentaries related to qualitative and mixed methods research, stated-preference methods, patient reported outcomes, and shared decision making. Advances in regulatory science, patient-focused drug development, patient-centered benefit-risk and health technology assessment will also be considered. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in The Patient may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances. All manuscripts are subject to peer review by international experts.
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