Petrocchi Serena, Janssens Rosanne, Oliveri Serena, Arnou Reinhard, Durosini Ilaria, Guiddi Paolo, Louis Evelyne, Vandevelde Marie, Nackaerts Kristiaan, Smith Meredith Y, Galli Giulia, de Marinis Filippo, Gianoncelli Letizia, Pravettoni Gabriella, Huys Isabelle
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 Pharmacol. 2021 Mar 4;12:602112. doi: 10.3389/fphar.2021.602112. eCollection 2021.
The potential value of patient preference studies has been recognized in clinical individual treatment decision-making between clinicians and patients, as well as in upstream drug decision-making. Drug developers, regulators, reimbursement and Health Technology Assessment (HTA) bodies are exploring how the use of patient preference studies could inform drug development, regulatory benefit risk-assessment and reimbursement decisions respectively. Understanding patient preferences may be especially valuable in decisions regarding Non-Small Cell Lung Cancer (NSCLC) treatment options, where a variety of treatment options with different characteristics raise uncertainty about which features are most important to NSCLC patients. As part of the Innovative Medicines Initiative PREFER project, this qualitative study aimed to identify patient-relevant lung cancer treatment characteristics. This study consisted of a scoping literature review and four focus group discussions, 2 in Italy and 2 in Belgium, with a total of 24 NSCLC patients (Stages III-IV). The focus group discussions sought to identify which treatment characteristics patients find most relevant. The discussions were analyzed thematically using a thematic inductive analysis. Patients highlighted themes reflecting: 1) positive effects or expected gains from treatment such as greater life expectancy and maintenance of daily functioning, 2) negative effects or adverse events related to therapy that negatively impact patients' daily functioning such as fatigue and 3) uncertainty regarding the duration and type of treatment effects. These overarching themes were consistent among patients from Belgium and Italy, suggesting that treatment aspects related to efficacy and safety as well as the psychological impact of lung cancer treatment are common areas of concern for patients, regardless of cultural background or country. Our findings illustrate the value of using qualitative methods with patients to identify preferred treatment characteristics for advanced lung cancer. These could inform a subsequent quantitative preference survey that assesses patient trade-offs regarding treatment options.
患者偏好研究的潜在价值已在临床医生与患者之间的个体化治疗决策以及上游药物决策中得到认可。药物研发商、监管机构、报销部门和卫生技术评估(HTA)机构正在探索如何利用患者偏好研究分别为药物研发、监管效益风险评估和报销决策提供信息。在非小细胞肺癌(NSCLC)治疗方案的决策中,了解患者偏好可能特别有价值,因为各种具有不同特征的治疗方案会引发关于哪些特征对NSCLC患者最重要的不确定性。作为创新药物计划PREFER项目的一部分,这项定性研究旨在确定与患者相关的肺癌治疗特征。该研究包括一项范围界定文献综述和四次焦点小组讨论,其中两次在意大利,两次在比利时,共有24名NSCLC患者(III - IV期)。焦点小组讨论旨在确定患者认为最相关的治疗特征。使用主题归纳分析法对讨论进行了主题分析。患者强调的主题反映出:1)治疗的积极效果或预期收益,如预期寿命延长和日常功能维持;2)与治疗相关的负面影响或不良事件,这些会对患者的日常功能产生负面影响,如疲劳;3)治疗效果的持续时间和类型的不确定性。这些总体主题在比利时和意大利的患者中是一致的,这表明与疗效和安全性以及肺癌治疗的心理影响相关的治疗方面是患者共同关注的领域,无论文化背景或国家如何。我们的研究结果说明了使用定性方法与患者一起确定晚期肺癌首选治疗特征的价值。这些结果可为后续的定量偏好调查提供信息,该调查将评估患者在治疗方案方面的权衡。