FDA Announces Several Initiatives to Encourage Real World Evidence
​[Thursday, January 31, 2019}

FDA plans to implement several measures to encourage use of Real World Evidence (RWE) in clinical trials, post-market pharmacovigilance and regulatory decisions. These measures include collection of feedback from stakeholders about ways to incorporate digital technologies in clinical trials to create better data collection and increase patient access to trials; and increasing FDA’s internal capabilities to evaluate and apply digital data to regulatory decisions. FDA would encourage enhancement of traditional clinical trial processes with new technologies to collect real world data (RWD) for improved decision making both for the developers and regulators. Although benefits of the use of RWD in clinical trials are easy to appreciate, there lack scientific and technical standards for incorporating such new technologies into clinical trials. FDA plans to develop such standards in collaboration with stakeholders. The immediate impact of these measures should be on the design and logistics of clinical trials. Clinical trials should be designed with endpoints that take advantage of real-time digital data such as time to response or time to treatment discontinuation. Decentralized clinical trials using remote and risk-based monitoring, and wearable devices should be preferred as they allow timely quality checks, increased efficiency and reduced cost. Automation of data collection using digital technologies should also increase investigator participation due to reduced trial-related work burden. FDA encourages bridging the gap between clinical research and clinical care using digital technologies and RWD. Another big incentive for using RWD and decentralized trials is making it easier for patients to participate; in FDA’s words, its helps “take the trial to the patient, rather than bringing the patient to the trial”. By reducing the number of visits to the clinic during a trial and using data collected real-time automatically using wearable devices, and in the electronic health records, participating in trials can be made very convenient and non-intrusive for the patients, increasing recruitment and retention in the trials. It may also lead to increased participation from groups that normally hesitate to participate such as minorities, pediatric, and vulnerable populations. In anticipation of getting increasing number of applications using software-based machine learning algorithms and analysis tools utilizing Artificial Intelligence, FDA plans to build internal skills in these areas via specialized training programs, and a competitive fellowship to attract post-doctoral fellows from academic centers to develop high-impact AI-based regulatory science tools in collaboration with FDA mentors. FDA is already accepting RWE in support of marketing approval applications and post-market data for oncology products; it expects to use similar principles for all products in the near future.