The U.S. Food and Drug Administration today released its framework for evaluating the potential use of real-world evidence (RWE) to help support the approval of a new indication for a pre-approved medicine and satisfy post-approval study requirements. Development of the framework was required under the 21st Century Cures Act.
“We’re pleased to see the FDA move forward with its efforts to harness real-world data. Understanding how treatments work in the real world is an important and complex task to guide the appropriate use of biopharmaceuticals available for typical patients in real-world clinical practice environments,” said National Pharmaceutical Council Vice President for Comparative Effectiveness Research Jennifer Graff, PharmD. “High quality, real-world data and evidence can provide meaningful information for patient care and improve the treatments brought to market. Through the framework, a set of methods and standards for collecting real-world data developed in consultation with multiple stakeholders and utilized by the FDA may encourage others to accept high-quality studies.”
There are three key takeaways from the FDA’s framework:
- In an era of growing digital data, the framework paves the way for the consideration of a broader set of data sources. These range from well-understood sources, such as medical claims, electronic health records, and patient registries, to novel data sources such as mobile technologies, wearables, and patient-reported data.
- FDA will consider a wide range of study designs to guide safety and effectiveness decisions. Study designs considered under the FDA’s RWE Program include not only randomized clinical studies using real-world data, but also single arm studies using real-world data control arms and observational study designs.
- The framework marks the first step in FDA’s efforts to use RWE in assessing efficacy. Further guidance documents and stakeholder engagement activities are planned by the agency and will be needed to make the framework actionable and advance new medical innovations faster and more efficiently.
“There is so much quality data that is available—let’s not waste it,” said Dr. Graff. “We’re looking forward to working with FDA as they continue to develop the RWE program.”
In its longstanding body of research on real-world evidence, NPC has examined many of the questions that FDA highlighted in the framework. These include standards for assessing the relevance of real-world data, standards for assessing the credibility of the methods for conducting analyses, and the need for methods transparency, among others.