FDA’s new strategic framework to advance use of real-world evidence to support development of drugs and biologics

The health care system is integrating more effective ways to leverage electronic tools to gather and use vast amounts of health-related data. These tools offer new opportunities to use data collected during the routine care of patients to advance medical care. Leveraging such data – typically called real-world data (RWD) - to improve regulatory decisions is a key strategic priority for the FDA. Today, the FDA is announcing our new 2019 strategic Framework for how we’ll continue to advance these opportunities.

RWD collected from a variety of sources offer new opportunities to generate evidence and better understand clinical outcomes. These data may be derived from a diverse array of sources, such as electronic health records (EHR), medical claims, product and disease registries, laboratory test results and even cutting-edge technology paired with consumer mobile devices. These data are being used to develop information and real-world evidence (RWE) that can better inform regulatory decisions.

RWD and RWE can be especially useful for postmarket monitoring of the safety of products during their use in real world settings. These tools will gradually allow us to shift some studies and data collection to the point of care, making the collection of data and the development of actionable evidence more efficient.

Advances in technology are also promoting these opportunities. New innovations such as natural language processing (NLP) are rapidly maturing and expediting the review of inputs such as single case reports, literature, EHRs, and social media. This coincides with an increasing use of RWD and RWE to help inform health care decisions. The FDA’s Sentinel safety capability continues to expand, making active surveillance a greater reality. In more cases, active post-market risk identification and analysis (ARIA) is replacing the need for post-marketing studies as a more effective, comprehensive, and achievable tool for post-market evaluation of products. As a result of these opportunities, these tools are becoming an increasingly important part of pre-market planning for post-market safety evaluation.

The FDA has already begun to incorporate RWE in regulatory decisions. The 21st Century Cures Act required the FDA to release a comprehensive plan for how we’ll continue to advance these efforts.

In implementing this new strategic Framework, we’ll work on identifying relevant standards and methodologies for collection and analysis of RWD. This is a key strategic priority that’s already actively underway. This Framework is the culmination of a longstanding effort that’s been underway inside the FDA; and it’s another milestone in our effort to advance the use of RWD and RWE to better inform patients and providers.