Epidemiology, Biostatistics and Prevention Institute, University of Zurich, Zurich, Switzerland.
Division of Geriatric Medicine and Gerontology, The Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Value Health. 2020 May;23(5):616-624. doi: 10.1016/j.jval.2019.11.007. Epub 2020 Mar 20.
In a previous project aimed at informing patient-centered care for people with multiple chronic conditions, we performed highly stratified quantitative benefit-harm assessments for 2 top priority questions. In this current work, our goal was to describe the process and approaches we developed and to qualitatively glean important elements from it that address patient-centered care.
We engaged patients, caregivers, clinicians, and guideline developers as stakeholder representatives throughout the process of the quantitative benefit-harm assessment and investigated whether the benefit-harm balance differed based on patient preferences and characteristics (stratification). We refined strategies to select the most applicable, valid, and precise evidence.
Two processes were important when assessing the balance of benefits and harms of interventions: (1) engaging stakeholders and (2) stratification by patient preferences and characteristics. Engaging patients and caregivers through focus groups, preference surveys, and as co-investigators provided value in prioritizing research questions, identifying relevant clinical outcomes, and clarifying the relative importance of these outcomes. Our strategies to select evidence for stratified benefit-harm assessments considered consistency across outcomes and subgroups. By quantitatively estimating the range in the benefit-harm balance resulting from true variation in preferences, we clarified whether the benefit-harm balance is preference sensitive.
Our approaches for engaging patients and caregivers at all phases of the stratified quantitative benefit-harm assessments were feasible and revealed how sensitive the benefit-harm balance is to patient characteristics and individual preferences. Accordingly, this sensitivity can suggest to guideline developers when to tailor recommendations for specific patient subgroups or when to explicitly leave decision making to individual patients and their providers.
在之前旨在为患有多种慢性疾病的患者提供以患者为中心的护理的项目中，我们对两个首要问题进行了高度分层的定量获益-危害评估。在当前的这项工作中，我们的目标是描述我们开发的过程和方法，并从中定性地收集解决以患者为中心的护理的重要要素。
我们让患者、护理人员、临床医生和指南制定者作为利益相关者代表参与定量获益-危害评估的全过程，并调查获益-危害平衡是否因患者的偏好和特征（分层）而有所不同。我们改进了选择最适用、最有效和最精确证据的策略。
在评估干预措施的获益和危害平衡时，有两个过程很重要：（1）让利益相关者参与，（2）根据患者的偏好和特征进行分层。通过焦点小组、偏好调查和共同研究者的方式让患者和护理人员参与其中，为确定研究问题、识别相关临床结局以及阐明这些结局的相对重要性提供了价值。我们选择分层获益-危害评估证据的策略考虑了结局和亚组之间的一致性。通过定量估计因偏好的真实差异而导致的获益-危害平衡范围，我们澄清了获益-危害平衡是否受偏好影响。
我们在分层定量获益-危害评估的所有阶段让患者和护理人员参与的方法是可行的，并揭示了获益-危害平衡对患者特征和个体偏好的敏感性。因此，这种敏感性可以向指南制定者提示何时为特定患者亚组量身定制建议，或者何时明确将决策留给个体患者及其提供者。
