■随着炎症性肠病(IBD)的治疗前景不断扩大,需要了解患者如何看待和重视与他们的疾病以及当前和新兴治疗相关的不同属性。这些见解可以为IBD的有效干预措施的开发和监管提供信息,使包括医疗保健专业人员在内的各种利益相关者受益,药物开发商,监管者,卫生技术评估机构,付款人,最终患有IBD的患者。对此,本患者偏好研究的目的是(1)确定IBD治疗和疾病相关属性的相对偏好权重,(2)解释偏好在不同特征(偏好异质性)的患者中可能存在的差异。
■患者偏好研究(PPS)是通过8步过程开发的,每一步都由顾问委员会通知。这个过程包括:(1)陈述偏好方法选择,(2)属性和层次发展(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)选择施工任务,(4)样本量估计,(5)调查实施,(6)驾驶,(7)翻译,和(8)预测试。所得的离散选择实验(DCE)调查包括14个属性,具有2到5个变化的级别。参与者将以部分轮廓设计回答15个DCE问题,其中每个选择问题都包含两个假设的治疗概况,显示四个属性。此外,关于患者的社会人口统计学和临床特征的问题,以及上下文因素的实施。该调查以15种不同的语言提供,旨在全球最低限度地招募700名患者。
■该协议为偏好研究人员和决策者提供了有关如何透明地报告PPS设计的宝贵见解,展示偏好研究中剩余差距的解决方案。PPS的结果将提供对IBD患者最重要的疾病和治疗相关特征的证据,以及这些可能在具有不同特征的患者中有所不同。这些发现将产生适用于偏好研究的有价值的见解,药物开发,监管批准,和报销流程,能够在整个药物产品生命周期中做出符合IBD患者真实需求的决策。
UNASSIGNED: 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).
UNASSIGNED: 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.
UNASSIGNED: 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.