Sinai Health System and the Department of Medicine, University of Toronto, Toronto, Ontario, Canada.
Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA.
Pharmacoepidemiol Drug Saf. 2020 Oct;29(10):1273-1278. doi: 10.1002/pds.5086. Epub 2020 Aug 14.
To determine how commonly pre-approval clinical trials could potentially be replicated using real-world data from insurance claims databases.
We conducted a cross-sectional study of medications approved by the FDA in 2011. For each medication, we reviewed the drug's label and the details of the pivotal clinical trials supporting its approval. We assessed whether each clinical trial could be replicated using an insurance claims databases by determining whether the following pivotal trial features could be reliably captured in claims data: study outcome, inclusion criteria, exclusion criteria, and the presence of an appropriate active comparator.
In 2011, 28 new medications were approved. The most common disease areas were oncology (N = 8, 29%), infectious disease (N = 5, 18%), and neurology (N = 4, 14%). The primary outcome of pre-approval clinical trials was identifiable in claims databases for six (21%) of the medications. Two (ticagrelor and linagliptin) had at least 80% of inclusion and exclusion criteria that could be identified in claims databases and had an available active comparator. The non-identifiable primary outcomes were related to patient-reported symptoms (N = 9, 32%), imaging findings (N = 5, 18%), laboratory values (N = 5, 18%), or other measurements (eg, blood pressure) not typically available in insurance claims databases (N = 4, 14%).
Among drugs FDA-approved in 2011, two (7%) had a pre-approval trial that could be replicated using insurance claims databases. In such qualifying trials, replication using claims databases could be useful in assessing whether they provide concordant results.
确定使用保险索赔数据库中的真实世界数据重复进行预批准临床试验的常见程度。
我们对 2011 年 FDA 批准的药物进行了横断面研究。对于每种药物，我们审查了药物标签以及支持其批准的关键临床试验的详细信息。我们通过确定以下关键试验特征是否可以在索赔数据中可靠地捕获，来评估每个临床试验是否可以使用保险索赔数据库进行复制：研究结果、纳入标准、排除标准以及是否存在适当的活性对照。
2011 年批准了 28 种新药物。最常见的疾病领域是肿瘤学（N=8，29%）、传染病（N=5，18%）和神经病学（N=4，14%）。预批准临床试验的主要结局可在索赔数据库中识别出六种（21%）药物。两种（替格瑞洛和利格列汀）至少有 80%的纳入和排除标准可以在索赔数据库中识别出来，并且有可用的活性对照。不可识别的主要结局与患者报告的症状（N=9，32%）、影像学发现（N=5，18%）、实验室值（N=5，18%）或其他测量值（如血压）有关，这些测量值通常不在保险索赔数据库中（N=4，14%）。
在 2011 年 FDA 批准的药物中，有两种（7%）具有可使用保险索赔数据库重复进行的预批准试验。在这些合格的试验中，使用索赔数据库进行复制可能有助于评估它们是否提供一致的结果。
