Commentary on Bertagnolli et al. Leveraging electronic health record data for clinical trials a brave new world
Clinical trial research seemingly appears to be at the cusp of a paradigm shift. Big data and artificial intelligence. Real world data and real world evidence. Totality of evidence and lifecycle evaluation. These phrases are ubiquitous today. We hear them from pharmaceutical and medical device industry executives. They have been written into U.S. laws and regulations. They are used at scientific conferences, on government panels, and by consulting organizations, promising new ways to gain insights on medical product use and performance. And with their frequent use, and overuse, the clinical research community teeters between 'is there true promise that can bring actual progress? or 'is this nothing but puffery and hype? Today, we live in an age where online retailers are able to predict consumer spending through past queries and spending patterns, making iterative advances with every purchase or search. Yet at the same time, despite the increasing availability of health and clinical data from electronic health records (EHRs) to personal digital devices tracking sleep, activity, and diet and clear advances in statistical methods and computing capacity, we have not yet experienced the same transformational changes to clinical trial research. Should we not be able to use these 'real world data' to generate 'real world evidence' or increase the efficiency of clinical trials? We should. And there is likely to be actual promise and progress. In time, we will enter a brave new world of conducting clinical trials to evaluate medical product safety and effectiveness by taking advantage of realworld data sources. But we will need to learn how to appropriately use these data and understand the associated tradeoffs, not expect that real world data will be effortlessly substituted for traditional clinical trials and obviate tried and true methods of data collection. And to make progress, several steps are needed to establish standards and best practices. First, we need to agree on definitions. What are realworld data? Do they include patient reporting of satisfaction or adverse experiences on social media? Does a traditional randomized clinical trial that relies on an EHR, administrative claims or a disease registry for data collection count as 'real world' data? Realworld data have been defined by the U.S. Food and Drug Administration (FDA) as
Clinical trials