MacEwan Joanna P, Doctor Jason, Mulligan Karen, May Suepattra G, Batt Katharine, Zacker Christopher, Lakdawalla Darius, Goldman Dana
Precision Health Economics, Los Angeles, California.
Novartis Oncology, One Health Plaza, East Hanover, New Jersey.
MDM Policy Pract. 2019 Jun 21;4(1):2381468319855386. doi: 10.1177/2381468319855386. eCollection 2019 Jan-Jun.
Value assessments and treatment decision making typically focus on clinical endpoints, especially overall survival (OS). However, OS data are not always available, and surrogate markers may also have some value to patients. This study sought to estimate preferences for progression-free survival (PFS) relative to OS in metastatic breast cancer (mBC) among a diverse set of stakeholders-patients, oncologists, and oncology nurses-and estimate the value patients and providers place on other attributes of treatment. Utilizing a combined conjoint analysis and discrete choice experiment approach, we conducted an online prospective survey of mBC patients and oncology care providers who treat mBC patients across the United States. A total of 299 mBC patients, 100 oncologists, and 99 oncology nurses completed the survey. Virtually all patients preferred health state sequences with contiguous periods of PFS, compared with approximately 85% and 75% of nurses and oncologists, respectively. On average, longer OS was significantly ( < 0.01) preferred by the majority (75%) patients, but only 15% of nurses preferred longer OS, and OS did not significantly affect oncologists' preferred health state. However, in the context of a treatment decision, whether a treatment offered continuous periods of stable disease holding OS constant significantly affected nurses' treatment choices. Patients and providers alike valued reductions in adverse event risk and evidence from high-quality randomized controlled clinical trials. The strong preference for observed PFS suggests more research is warranted to better understand the reasons for PFS having positive value to patients. The results also suggest a range of endpoints in clinical trials may have importance to patients.
价值评估和治疗决策通常侧重于临床终点，尤其是总生存期（OS）。然而，OS数据并非总能获得，替代指标对患者可能也具有一定价值。本研究旨在估计不同利益相关者（患者、肿瘤学家和肿瘤护理人员）对转移性乳腺癌（mBC）中无进展生存期（PFS）相对于OS的偏好，并估计患者和医疗服务提供者对治疗的其他属性的重视程度。利用联合分析和离散选择实验相结合的方法，我们对美国各地治疗mBC患者的mBC患者和肿瘤护理提供者进行了一项在线前瞻性调查。共有299名mBC患者、100名肿瘤学家和99名肿瘤护理人员完成了调查。几乎所有患者都更喜欢具有连续PFS期的健康状态序列，相比之下，分别约有85%的护士和75%的肿瘤学家有此偏好。平均而言，大多数（75%）患者显著（<0.01）更喜欢更长的OS，但只有15%的护士更喜欢更长的OS，且OS并未显著影响肿瘤学家偏好的健康状态。然而，在治疗决策的背景下，一种治疗方法在OS保持不变的情况下能否提供连续的疾病稳定期，这对护士的治疗选择有显著影响。患者和医疗服务提供者都重视不良事件风险的降低以及来自高质量随机对照临床试验的证据。对观察到的PFS的强烈偏好表明，有必要进行更多研究，以更好地理解PFS对患者具有正向价值的原因。研究结果还表明，临床试验中的一系列终点可能对患者具有重要意义。
