EXCLUSION OF OLDER PATIENTS IN CLINICAL DRUG TRIALS

描述(申请人提供)：65岁及以上的个人是美国人均医疗保健消费者的最高群体，每年在医疗保健上的支出超过5300亿美元，在处方药上的支出超过560亿美元。然而，护理这些患者的临床医生往往缺乏相关的医学证据来指导他们的临床决策，并确保提供最佳的护理。人们担心，随机对照试验(RCT)--被认为是支持医疗决策的最高水平的证据--在各种疾病的试验中持续排除和低估了老年人的代表性。因此，临床医生必须推断年轻患者的试验结果，尽管已知与年龄相关的病理生理学和治疗反应差异。虽然之前已经认识到缺乏高质量的证据来支持老年患者的医疗管理，但到目前为止，还没有足够的数据来源来全面监测与老年对象有关的研究组合。这项建议使用独特的资源--ClinicalTrials.gov--并首次开发方法来评估整个研究组合，评估研究与老年人实际疾病负担的相关性和一致性，并量化和描述将这一群体排除在研究活动之外的情况。我们建议使用该注册来分析随机对照药物试验的三个具体目标：1)衡量当前包括老年人的研究与这些患者的疾病负担的相关性；2)量化老年人被排除在整个临床研究事业中的程度；以及3)确定排除老年患者的随机对照试验的特征。我们假设，包括老年患者在内的随机对照试验的重点与全球老年人实际疾病负担所界定的高度优先领域的对应程度很低。此外，我们假设，相当大比例的药物试验不仅基于年龄，而且还通过其他类型的排斥因素间接排除老年患者。由于临床试验的主体在不断演变，这项工作不仅提供了当前药物试验的快照，而且还提供了一个框架和工具包，用于监测未来的证据库。根据这些目标开发的指标将通过ClinicalTrials.gov的基于网络的前端应用程序的原型公开提供，该应用程序自动显示与老年患者有关的临床试验的当前状态。

DESCRIPTION (provided by applicant): Individuals 65 years of age and older represent the highest per capita consumers of healthcare in the United States, with greater than $530 billion spent on medical care and $56 billion on prescription drugs annually. However, clinicians caring for these patients frequently lack relevant medical evidence to guide their clinical decision-making and ensure that the optimal care is provided. There is concern that randomized controlled trials (RCTs)-considered the highest level of evidence supporting medical decisions-persistently exclude and underrepresent older individuals in trials across a spectrum of disease conditions. As a result, clinicians must extrapolate results derived from trials on younger patients despite known age-related differences in pathophysiology and response to treatment. Though the lack of high quality evidence to support medical management in older patients has been previously recognized, there has not until now been an adequate data source for comprehensive surveillance of the research portfolio pertaining to elderly subjects. This proposal uses a unique resource-ClinicalTrials.gov-and develops methods to, for the first time, take stock of the entire research portfolio, assessing the relevance and alignment of research with actual disease burden among older individuals as well as quantifying and characterizing the exclusion of this population from research activity. We propose to use the registry to analyze randomized controlled drug trials in three specific aims: 1) To measure the relevance of current research that does include elderly persons in relation to the disease burden in these patients; 2) To quantify the extent to which elderly persons are excluded in the clinical research enterprise as a whole; and 3) To identify the characteristics of RCTs that exclude elderly patients. We hypothesize that the focus of RCTs that include older patients corresponds poorly to high priority areas defined by actual global disease burden among the elderly. Further, we hypothesize that a substantial proportion of drug trials exclude older patients not only based on age but also indirectly through other types of exclusion factors. Because the body of clinical trials is constantly evolving, this work provides not only a snapshot of current drug trials, but also a framework and toolkit for surveillance of the evidence base going forward. Metrics developed under these aims will be made publicly available through a prototype of a web- based front application to ClinicalTrials.gov that automatically displays the current status of clinical trials pertaining to older patients.