Generalizing RCT Efficacy Evidence: Application to NIDA Clinical Trials Network

推广 RCT 功效证据：在 NIDA 临床试验网络中的应用

• 批准号: 8611480
• 负责人: RAMIN MOJTABAI
• 金额: $ 24.3万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-03-01 至 2017-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8611480
• 关键词: Admission activity; African American; Agonist; Alcohols; Appointment; California; Characteristics; Client; Clinical; Clinical Data; Clinical Trials Network; Data; Data Set; Data Sources; Diagnostic; Drops; Drug abuse; Eligibility Determination; Evaluation Studies; Evidence based practice; Exclusion; Exclusion Criteria; Funding; Future; Gold; Government; HIV risk; Health; Health Services Research; Individual; Intervention; Logistics; Low income; Measures; Methods; Metric; Modality; Modeling; National Institute of Drug Abuse; Observational Study; Opioid; Outcome; Outcome Study; Participant; Pharmaceutical Preparations; Policies; Policy Maker; Population; Pregnant Women; Protocols documentation; Randomized; Randomized Clinical Trials; Randomized Controlled Trials; Recruitment Activity; Research Personnel; Research Project Grants; Risk Reduction; Safety; Sampling; Sampling Studies; Scoring Method; Services; Site; Solutions; Source; Staging; Standardization; Statistical Methods; Stratification; Subgroup; Substance Use Disorder; Substance of Abuse; Surveys; Target Populations; Techniques; Treatment Efficacy; Treatment outcome; Variant; Weight; Woman; alcohol abuse therapy; arm; base; design; evidence based guidelines; follow-up; improved; public health relevance; randomized trial; treatment as usual; treatment effect; treatment site; treatment trial; volunteer

Randomized controlled trials in which individuals are randomly assigned to receive an experimental or a control treatment are generally considered the gold standard for establishing the efficacy of new treatments. Evidence-based practice guidelines and policy decisions often rely on the results of such trials. Nevertheless, participants in randomized controlled trials are often different from individuals who would eventually be the recipients of new treatments ("target population") when these treatments are disseminated in usual practice settings. This is a cause for concern because the results of randomized controlled trials may not generalize to these populations. This is partly due to the fact that individuals who are recruited into randomized controlled trials are often different from the target population in a number of ways. Strict eligibility criteria of randomized trials exclude many potential participants with severe health or psychiatric conditions or individuals who may not be able to comply with treatment or to show up for follow-up appointments. This study uses data from 23 randomized controlled trials of substance use disorder treatments currently available in the NIDA Clinical Trials Network to assess how representative the randomized controlled trial samples are of the target population. Data on the target population are obtained from other publicly available data sources on individuals entering substance use disorder treatments in the US. The study also uses new statistical methods to measure the degree of deviation from the composition of the target population and also uses methods to adjust the samples of the randomized trials to better match the target populations. As a result, subgroups that are under-represented in the randomized trial sample will be given a higher weight in the analyses. After such weighting of data, the randomized controlled trial samples will be more similar to the target population with regard to socio-demographic and clinical characteristics and their results will more generalizable to the target population. We also plan to combine randomized trials of similar types of treatments to estimate the efficacy of interventions with more accuracy and to assess whether variations in the settings where trials were conducted had an impact on the outcomes of these studies. Overall, these analyses will provide more generalizable data from the available randomized trials in the NIDA Clinical Trials Network and will provide a framework for future randomized controlled trials to use at the design stage to optimize generalizability of their results.

随机对照试验，其中随机分配了个人以接收 实验或对照处理通常被认为是 建立新疗法的功效。循证实践指南和政策 决定通常依赖于此类试验的结果。然而，随机的参与者 对照试验通常与最终成为接收者的个人不同 当这些疗法在通常的情况下传播时，新的治疗方法（“目标人群”） 练习设置。这是引起关注的原因，因为随机控制的结果 试验可能不会推广到这些人群。这部分是由于个人 被招募到随机对照试验中的人通常与目标不同 人口以多种方式。随机试验的严格资格标准不包括许多 患有严重健康或精神疾病的潜在参与者或可能 不能遵守治疗或出现进行后续约会。这项研究 使用来自23个随机对照试验的物质使用障碍治疗的数据 目前在NIDA临床试验网络中可用，以评估 随机对照试验样本是目标群体。目标数据 人口是从其他进入的个人的公开数据来源获得的 美国的药物使用障碍治疗。该研究还使用新的统计方法 衡量与目标人群组成的偏差程度以及 使用方法调整随机试验的样本以更好地匹配目标 人群。结果，随机试验中代表性不足的亚组 样本将在分析中给予更高的重量。在这样的数据加权之后， 随机对照试验样本将与目标人群更相似 关于社会人口统计学和临床​​特征及其结果将更多 可以推广到目标人群。我们还计划结合随机试验的 类似类型的治疗方法可以更准确地估算干预措施的功效 并评估进行试验的设置中的变化是否具有 对这些研究结果的影响。总体而言，这些分析将提供更多 NIDA临床试验网络中可用随机试验的可概括数据 并将为未来的随机对照试验提供一个框架 优化其结果的普遍性的阶段。