Beusterien Kathleen, Maculaitis Martine C, Hallissey Bernadette, Gaschler Michael M, Smith Mary Lou, Law Ernest H
Kantar, Health Division, Horsham, PA, USA.
Kantar, Health Division, New York, NY, USA.
Patient Prefer Adherence. 2021 Mar 18;15:611-623. doi: 10.2147/PPA.S298670. eCollection 2021.
Several adjuvant phase III trials are evaluating cyclin-dependent kinase 4/6 inhibitors (CDK4/6is) in combination with endocrine therapy (ET) in hormonal receptor positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) early-stage breast cancer (eBC). This study examines preferences for this combination regimen and ET alone among patients, oncologists, and payers in the United States.
A web-based questionnaire, including a discrete choice experiment (DCE), was administered to patients, practicing oncologists, and payers. In the DCE, respondents selected between hypothetical treatment profiles with attributes associated with ET monotherapy and CDK4/6i + ET regimens. Each treatment alternative was defined by the following attributes: 5-year invasive disease-free survival (iDFS), nausea, diarrhea, neutropenia, alopecia, dosing schedule, and electrocardiogram (ECG) monitoring. Payers had the additional attribute of annual per-patient treatment cost. Hierarchical Bayesian models were used to estimate relative preference weights for each attribute-level and relative attribute importance.
For patients (n=300) and oncologists (n=200), iDFS was most important (2 to 3 times more important than the next most important attribute), followed by neutropenia and diarrhea risks for patients and oncologists, respectively. Patients and oncologists required an improvement in iDFS of 8.0 and 5.6 percentage-points, respectively, to accept an increase in diarrhea risk from 11% to 81%. Payers (n=60) viewed annual per-patient cost as most important for treatment access decision-making, closely followed by iDFS. Payers required an improvement in iDFS of 21.8 percentage-points to accept an increase in cost from $5,100 to $149,400. Across all stakeholder groups, dosing schedule, alopecia risk, and ECG monitoring were perceived as least important.
Patients, oncologists, and payers expect a large absolute risk reduction in efficacy to offset the potential risks and costs of adding a CDK4/6i to current standard of care. An open discussion between all stakeholders is necessary to ensure that decision-making, whether at patient- or system-level, is informed by preferences for novel treatments, like CDK4/6is.
多项辅助性III期试验正在评估细胞周期蛋白依赖性激酶4/6抑制剂（CDK4/6i）与内分泌治疗（ET）联合用于激素受体阳性（HR+）/人表皮生长因子受体2阴性（HER2-）早期乳腺癌（eBC）的疗效。本研究调查了美国患者、肿瘤学家和支付方对这种联合治疗方案及单纯ET治疗方案的偏好。
对患者、执业肿瘤学家和支付方进行了一项基于网络的问卷调查，其中包括一项离散选择实验（DCE）。在DCE中，受访者在具有与ET单药治疗和CDK4/6i + ET方案相关属性的假设治疗方案中进行选择。每个治疗方案由以下属性定义：5年无侵袭性疾病生存率（iDFS）、恶心、腹泻、中性粒细胞减少、脱发、给药方案和心电图（ECG）监测。支付方还有每位患者每年治疗费用这一额外属性。采用分层贝叶斯模型来估计每个属性水平的相对偏好权重和相对属性重要性。
对于患者（n = 300）和肿瘤学家（n = 200），iDFS最为重要（比第二重要的属性重要2至3倍），其次对于患者和肿瘤学家分别是中性粒细胞减少和腹泻风险。患者和肿瘤学家分别需要iDFS提高8.0和5.6个百分点，才会接受腹泻风险从11%增加到81%。支付方（n = 60）认为每位患者每年的费用对治疗准入决策最为重要，其次是iDFS。支付方需要iDFS提高21.8个百分点，才会接受费用从5100美元增加到149400美元。在所有利益相关者群体中，给药方案、脱发风险和ECG监测被认为最不重要。
患者、肿瘤学家和支付方期望疗效方面有大幅的绝对风险降低，以抵消在当前治疗标准中添加CDK4/6i带来潜在风险和成本。所有利益相关者之间进行公开讨论很有必要，以确保无论是患者层面还是系统层面的决策，都能参考对CDK4/6i等新型治疗方法的偏好。
