Schoefs Elise, Vermeire Séverine, Ferrante Marc, Sabino João, Verstockt Bram, Avedano Luisa, De Rocchis Maria Stella, Sajak-Szczerba Magdalena, Saldaña Roberto, Straetemans Noortje, Vandebroek Martina, Janssens Rosanne, Huys Isabelle
Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Department of Gastroenterology and Hepatology, University Hospitals Leuven, KU Leuven, Leuven, Belgium.
Front Med (Lausanne). 2024 Aug 14;11:1418874. doi: 10.3389/fmed.2024.1418874. eCollection 2024.
As the therapeutic landscape for inflammatory bowel disease (IBD) continues to expand, a need exists to understand how patients perceive and value different attributes associated with their disease as well as with current and emerging treatments. These insights can inform the development and regulation of effective interventions for IBD, benefiting various stakeholders including healthcare professionals, drug developers, regulators, Health Technology Assessment bodies, payers, and ultimately patients suffering from IBD. In response to this, the present patient preference study was developed with the aim to (1) determine the relative preference weights for IBD treatment and disease related attributes, and (2) explain how preferences may differ across patients with different characteristics (preference heterogeneity).
The patient preference study (PPS) was developed through an 8-step process, with each step being informed by an advisory board. This process included: (1) stated preference method selection, (2) attribute and level development (including a scoping literature review, focus group discussions, and advisory board meetings), (3) choice task construction, (4) sample size estimation, (5) survey implementation, (6) piloting, (7) translation, and (8) pre-testing. The resulting discrete choice experiment (DCE) survey comprises 14 attributes with between two and five varying levels. Participants will answer 15 DCE questions with a partial profile design, where each of the choice questions encompasses two hypothetical treatment profiles showing four attributes. Additionally, questions about patients' socio-demographic and clinical characteristics, as well as contextual factors are implemented. The survey is available in 15 different languages and aims to minimally recruit 700 patients globally.
This protocol gives valuable insights toward preference researchers and decision-makers on how PPS design can be transparently reported, demonstrating solutions to remaining gaps in preference research. Results of the PPS will provide evidence regarding the disease and treatment related characteristics that are most important for IBD patients, and how these may differ across patients with different characteristics. These findings will yield valuable insights applicable to preference research, drug development, regulatory approval, and reimbursement processes, enabling decision making across the medicinal product life cycle that is aligned with the true needs of IBD patients.
随着炎症性肠病（IBD）治疗领域的不断拓展，有必要了解患者如何看待和重视与其疾病以及当前和新兴治疗方法相关的不同属性。这些见解可为IBD有效干预措施的开发和监管提供参考，使包括医疗保健专业人员、药物研发人员、监管机构、卫生技术评估机构、支付方以及最终IBD患者在内的各方利益相关者受益。为此，开展了本次患者偏好研究，旨在（1）确定IBD治疗及疾病相关属性的相对偏好权重，以及（2）解释不同特征患者的偏好可能存在的差异（偏好异质性）。
患者偏好研究（PPS）通过一个8步骤流程制定，每一步骤都有咨询委员会提供指导。该流程包括：（1）选择陈述偏好方法，（2）属性和水平开发（包括范围界定文献综述、焦点小组讨论和咨询委员会会议），（3）选择任务构建，（4）样本量估计，（5）调查实施，（6）预试验，（7）翻译，以及（8）预测试。最终的离散选择实验（DCE）调查包括14个属性，每个属性有两到五个不同水平。参与者将回答15个采用部分轮廓设计的DCE问题，每个选择问题包含两个展示四个属性的假设治疗方案。此外，还设置了关于患者社会人口统计学和临床特征以及背景因素的问题。该调查有15种不同语言版本，目标是在全球范围内至少招募700名患者。
本方案为偏好研究人员和决策者提供了关于如何透明报告PPS设计的宝贵见解，展示了偏好研究中尚存差距的解决方案。PPS的结果将提供证据，说明对IBD患者最重要的疾病和治疗相关特征，以及这些特征在不同特征患者之间可能存在的差异。这些发现将产生适用于偏好研究、药物开发、监管批准和报销流程的宝贵见解，使整个医药产品生命周期的决策与IBD患者的真正需求保持一致。
