Methods for observational risk-benefit studies of medical devices: an analysis of big data and simulation studies

医疗器械被用于外科手术的许多领域。最近公布的欧盟法规可能意味着需要进行大量的上市后设备监控研究。然而，在比较不同医疗器械的风险和收益的观察性研究中，关于现有统计模型的性能以最大限度地减少混淆的方法论文献很少。许多方法存在并被广泛应用于药物安全性和比较有效性的观察性研究。然而，对于医疗器械流行病学来说，存在着特定的挑战(例如，外科医生的特征、学习曲线、修订、设备修改)。我们的目标是评估不同统计方法在实际应用条件下和潜在的所有患者中用于医疗器械的风险/S和益处/S的观察性研究的性能。我们将使用常规收集的大型健康数据，以及模拟数据集。将使用不同的方法对这些风险进行分析，例如倾向得分分析、知识产权加权、边际结构建模、中断的时间序列、竞争风险和方法研究产生的新风险。我们将通过临床用例研究解决设备流行病学中的实际问题，并通过模拟研究评估方法在这些用例带来的挑战中的性能。通过这项研究，我们将制定最佳方法指南，以应对观察性上市后设备监测研究中的不同挑战，并回答实际的临床问题。

Medical devices are used in many areas of surgery. Recently published EU regulations will likely imply the need for numerous post-marketing device surveillance studies. There is however a scarcity of methodological literature on the performance of existing statistical models to minimise confounding in observational studies comparing the risk and benefit of different medical devices. Many methods exist and are widely applied in observational drug safety and comparative effectiveness research. However, there are challenges (e.g., surgeon characteristics, learning curves, revisions, device modifications) specific to medical device epidemiology. We aim to assess the performance of different statistical methods for the observational study of the risk/s and benefit/s of medical devices, as used in actual practice conditions and in potentially all patients. We will use routinely collected big health data, as well as simulated datasets. These will be analysed using different approaches, such as propensity score analyses, IP weighting, marginal structural modelling, interrupted time series, competing risks and novel ones resulting from methodological research. We will address practical questions in device epidemiology with clinical use case studies and assess methods performance in challenges arising from these use cases with simulation studies. With this research we will create guidance on best methods to answer different challenges in observational post-marketing device surveillance research and give answer to actual clinical questions.