Goetz Iris A, Sutter Carolyn, Abraham Traci, Kanu Chisom, Boye Kristina S, Symonds Tara
Eli Lilly and Company, Lilly Corporate Center Indianapolis, Indianapolis, IN, 46285, USA.
Clinical Outcomes Solutions, 53 West Jackson Boulevard, Chicago, IL, 60604, USA.
J Patient Rep Outcomes. 2025 Feb 20;9(1):23. doi: 10.1186/s41687-025-00841-0.
Clinical trials for obesity have traditionally focused on weight loss and resolution of comorbidities as primary outcomes. However, secondary outcomes, such as the impact of weight reduction on patient experience, like health-related quality of life (HRQoL), have increasingly been recognized as important. Therefore, a review was conducted to determine the Clinical Outcome Assessments (COAs) and Digital Health Technologies (DHTs) used in clinical trials for obesity to assess the patient experience.
Two clinical trial databases (United States & European Union) were reviewed to identify Phase 2-4 clinical trials for obesity (2018-2023). A targeted literature review was also conducted using the OVID database to identify clinical trial for obesity publications which included COAs/DHTs (2010-2023).
Trials from the databases (n = 53) and publications (n = 42) were included in data extraction (N = 73). This resulted in identification of 108 COAs, the majority being patient-reported outcome (PRO) measures (n = 83), but also 24 performance outcomes (PerfO) measures, and 1 composite PRO-clinician-reported outcomes (ClinRO) measure, as well as 2 DHTs. The most frequently identified PRO measures were the Short Form 36 and the Impact of Weight on Quality of Life Lite Clinical Trials. Twenty-four PerfO measures were also identified, with the 6-minute walk test being most common. These measures were most often used to construct secondary endpoints, with physical function (PF) being the most frequently specified domain. PRO measures assessing eating-related thoughts/behaviours, physical activity, and disordered eating were also frequently included, although individual measures varied widely across trials.
Review of COAs and DHTs in registered clinical trials and publications for obesity found that PRO measures were the most common type of COA used to develop endpoints with current use of DHTs limited. Specifically, the physical function domain of multidimensional patient-reported outcome measures assessing health-related quality of life were often used to construct secondary endpoints. Further work is warranted to assess how the COAs and DHT data collected in clinical trials are viewed by regulators and payers.
肥胖症的临床试验传统上一直将体重减轻和合并症的缓解作为主要结局。然而，诸如体重减轻对患者体验的影响（如健康相关生活质量[HRQoL]）等次要结局越来越被认为很重要。因此，进行了一项综述，以确定肥胖症临床试验中用于评估患者体验的临床结局评估（COA）和数字健康技术（DHT）。
对两个临床试验数据库（美国和欧盟）进行了审查，以确定2018 - 2023年肥胖症的2 - 4期临床试验。还使用OVID数据库进行了有针对性的文献综述，以确定包含COA/DHT的肥胖症临床试验出版物（2010 - 2023年）。
来自数据库（n = 53）和出版物（n = 42）的试验被纳入数据提取（N = 73）。这导致识别出108种COA，其中大多数是患者报告结局（PRO）测量指标（n = 83），但也有24种性能结局（PerfO）测量指标、1种综合PRO - 临床医生报告结局（ClinRO）测量指标以及2种DHT。最常识别的PRO测量指标是简明健康调查问卷（Short Form 36）和体重对生活质量的影响简易版临床试验。还识别出24种PerfO测量指标，其中6分钟步行试验最为常见。这些测量指标最常被用于构建次要终点，身体功能（PF）是最常指定的领域。评估与饮食相关的想法/行为、身体活动和饮食失调的PRO测量指标也经常被纳入，尽管各个试验中的具体测量指标差异很大。
对肥胖症注册临床试验和出版物中的COA和DHT进行综述发现，PRO测量指标是用于制定终点的最常见COA类型，目前DHT的使用有限。具体而言，评估健康相关生活质量的多维患者报告结局测量指标的身体功能领域常被用于构建次要终点。有必要进一步开展工作，以评估监管机构和支付方如何看待临床试验中收集的COA和DHT数据。
