We support FDA’s ongoing work to facilitate 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 called 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. The RWE Alliance appreciates FDA’s commitment to realizing the full potential of fit-for-purpose RWD to generate RWE that will advance the development of medical products and enhance regulatory decision making. We share this goal, as these efforts will ultimately benefit patients.

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. 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 further develops RWD/E policy, and as Congress considers additional action in this space.

As part of these efforts, the RWE Alliance will work to promote utilization of RWE across therapeutic areas to ensure that more patients will benefit.

RWE can serve as a data source in the important effort to address healthcare disparities.

RWE can play a role in providing FDA, patients, healthcare providers, and other stakeholders with information on treatment effects among underrepresented populations across therapeutic areas. Clinical trial study populations frequently are not fully representative of relevant patient populations in real-world settings. We will work to support the use of RWE as a resource to help address disparities in healthcare access, treatment, and outcomes.

We seek and support increased opportunities for RWE organizations to consult with FDA on specific topics relevant to the potential uses of RWE for regulatory decision making across therapeutic areas.

Medical product sponsors and RWE organizations benefit greatly from FDA’s feedback. We will work to provide summary information and insights from our collective industry efforts to FDA. We will also work with FDA and other stakeholders to establish mechanisms and opportunities for RWE organizations to engage on RWE methods and applications.

Communication of FDA’s review of RWE in marketing applications is crucial to advancing best practices in the use of RWE for regulatory purposes and to ensuring widespread understanding of the benefits that RWE ultimately delivers to patients.

To increase this communication, we support FDA raising broader awareness about current and potential uses for RWE. For example, we support FDA making public a summary of the Agency’s review of RWE when included in original or supplemental applications.

Policies should account for the unique characteristics and potential of RWD/E so that the benefits of these high-quality sources of evidence can be fully realized by FDA and patients.

RWD differ from traditional clinical trial data in how and why they are collected, curated, and analyzed. RWD can offer unique advantages for medical product development and FDA’s regulatory decision making. Because RWD are different than traditional clinical trial data, new regulatory policies should recognize the unique aspects and opportunities for use of RWD/E. To realize the full potential of RWE to benefit patients, and because many existing regulations and guidance were developed with traditional clinical trials in mind, FDA should apply these policies in a manner that accounts for the unique characteristics of RWD/E and provide flexibility as appropriate (e.g., in data standards to support the submission of various types of data).