Applied Research Division for Cognitive and Psychological Science, IEO, European Institute of Oncology IRCCS, Milan, Italy.
Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Front Public Health. 2021 Feb 5;9:622154. doi: 10.3389/fpubh.2021.622154. eCollection 2021.
Lung cancer is the deadliest and most prevalent cancer worldwide. Lung cancer treatments have different characteristics and are associated with a range of benefits and side effects for patients. Such differences may raise uncertainty among drug developers, regulators, payers, and clinicians regarding the value of these treatment effects to patients. The value of conducting patient preference studies (using qualitative and/or quantitative methods) for benefits and side effects of different treatment options has been recognized by healthcare stakeholders, such as drug developers, regulators, health technology assessment bodies, and clinicians. However, evidence-based guidelines on how and when to conduct and use these studies in drug decision-making are lacking. As part of the Innovative Medicines Initiative PREFER project, we developed a protocol for a qualitative study that aims to understand which treatment characteristics are most important to lung cancer patients and to develop attributes and levels for inclusion in a subsequent quantitative preference survey. The study protocol specifies a four-phased approach: (i) a scoping literature review of published literature, (ii) four focus group discussions with stage III and IV Non-Small Cell Lung Cancer patients, (iii) two nominal group discussions with stage III and IV Non-Small Cell Lung Cancer patients, and (iv) multi-stakeholder discussions involving clinicians and preference experts. This protocol outlines methodological and practical steps as to how qualitative research can be applied to identify and develop attributes and levels for inclusion in patient preference studies aiming to inform decisions across the drug life cycle. The results of this study are intended to inform a subsequent quantitative preference survey that assesses patient trade-offs regarding lung cancer treatment options. This protocol may assist researchers, drug developers, and decision-makers in designing qualitative studies to understand which treatment aspects are most valued by patients in drug development, regulation, and reimbursement.
肺癌是全球最致命和最常见的癌症。肺癌的治疗方法具有不同的特点，并与一系列患者获益和副作用相关。这些差异可能会使药物开发者、监管机构、支付方和临床医生对这些治疗效果对患者的价值产生不确定性。医疗保健利益相关者（如药物开发者、监管机构、卫生技术评估机构和临床医生）已经认识到，对不同治疗选择的获益和副作用进行患者偏好研究（使用定性和/或定量方法）的价值。然而，在药物决策中如何以及何时进行和使用这些研究的循证指南仍然缺乏。作为创新药物倡议 PREFER 项目的一部分，我们制定了一项定性研究的方案，旨在了解哪些治疗特征对肺癌患者最重要，并开发纳入后续定量偏好调查的属性和水平。该研究方案规定了四阶段方法：（i）对已发表文献进行范围广泛的文献综述，（ii）对 III 期和 IV 期非小细胞肺癌患者进行四次焦点小组讨论，（iii）对 III 期和 IV 期非小细胞肺癌患者进行两次名义小组讨论，以及（iv）涉及临床医生和偏好专家的多方利益相关者讨论。本方案概述了如何应用定性研究来识别和开发属性和水平，以便纳入旨在为整个药物生命周期的决策提供信息的患者偏好研究。该研究的结果旨在为随后的定量偏好调查提供信息，该调查评估患者对肺癌治疗选择的权衡取舍。本方案可能有助于研究人员、药物开发者和决策者设计定性研究，以了解患者在药物开发、监管和报销方面最看重哪些治疗方面。
