Janet Woodcock, M.D.—Director, Center for Drug Evaluation and Research (CDER)

Real-world evidence is information on patient’s health and health care that comes from multiple sources within and outside the clinical setting. Examples include electronic health records, claims and billing data from health plans, medical product or disease registries, and data gathered from personal devices that people might wear or health applications.

Well, personal devices like fitness tracking apps can provide information about vital health measures like blood pressure and heart rate. That could add more information to the total picture of the patient’s health. Electronic health records and claims data tell us about conditions people have, the stages of their disease, the treatments they are receiving, and some about how they’re responding.

Being able to take advantage of data other than those in those traditional trials increases our options for research and helps us be able to gather more information. And access to these data could increase the speed and efficiency of setting up trials.

Right now, our Sentinel system uses claims data from hundreds of millions of Americans to look for safety signals related to marketed drugs.

The FDA is also collaborating with Google to identify whether search engine data—for example, web search logs—can identify and evaluate previously unreported adverse reactions and detect safety signals earlier than current FDA strategies.

The FDA and PatientsLikeMe, which is a large online patient network with about half a million members reporting on their experiences with more than 2,500 conditions, are collaborating to explore how patient-generated health data can give new insights into drug safety.

While real-world data can augment and increase the effectiveness of clinical research, real-world data cannot completely replace the gold standard of controlled clinical trials for assessing efficacy and safety of new drugs. The accuracy and completeness of data in electronic health record systems may be highly variable from site to site and even within a single health care institution, and we have certainly seen this.

Well, CDER believes that real-world evidence is applicable across all phases of drug development. Real-world evidence can help support new indications for existing drugs, because those drugs then would already be on the market, and real-world evidence can also help demonstrate how a drug works in populations that weren’t studied in the trial or relative to another drug not included in the study.

There’s little doubt that the new sources of data now being open to researchers, clinicians, and patients hold enormous potential for improving the quality, the safety, and the efficiency of medical care.

In December 2016, Congress provided CDER with support to continue exploring how real-world evidence can be integrated into regulatory decision making for new indications for already approved drugs and to support or satisfy postmarket studies on new drugs.

FDA will work with its stakeholders to produce a framework for a CDER real-world evidence program and guidance for when and how real-world evidence can be collected and used to support drug applications. There is no doubt that this will have a bright future, but the timing when all this will come to pass is something still unknown.