We support FDA’s ongoing work to facilitate the use of real-world data (RWD) and real-world evidence (RWE), which will add to the understanding of medical product safety and effectiveness and benefit patients.
FDA and Congress have taken important steps to advance the use of RWD/E. The 21st Century Cures Act contains an important provision that calls on FDA to publish a framework for the use of RWE for regulatory purposes, and FDA has taken steps to implement this provision by publishing a draft framework in December 2018 and issuing valuable guidance.
As real-world data and analytics organizations with a common interest in harnessing the power of RWE, we stand ready to provide partnership, expertise, and perspective as the Agency develops RWD/E guidance and policy, and as Congress considers action in this space. We believe that fit-for-purpose RWE can play an important role in the review of medical products—and can provide or contribute to substantial evidence of effectiveness. We believe that additional FDA guidance and policies regarding various applications of RWE, including its use in creating external comparator arms (ECAs), assessing fit-for-use data, and establishing real-world endpoints, will be helpful. We aim to contribute to the Agency’s efforts to further clarify topics such as:
- how FDA will assess RWD in determining whether it is fit to support a particular regulatory submission;
- when the development and use of a real-world ECA is appropriate to provide supportive evidence on the benefit-risk profile of a product;
- when real-world endpoints may be beneficial to include in a development program; and
- how FDA will evaluate when real-world endpoints meet the standards for regulatory decision making.
These efforts will build on FDA’s existing RWE Framework and advance the use of RWE to ultimately benefit patients. The RWE Alliance will work to promote utilization of RWE across therapeutic areas, not only within oncology and rare diseases where there are published examples of the use of RWE. To ensure RWE analyses are clinically meaningful and can be used in regulatory decision making, we recommend that regulatory guidelines for RWE communicate clear expectations for using RWD, for generating RWE, and for FDA’s evaluation of RWE. At the same time, given the rapid advancement of the RWE field, we believe it is critical that regulatory guidance remain flexible to accommodate advancements in data, methods, and technology.