The last several years have been foundational in the effort to advance the use of real-world evidence (RWE) in regulatory decision-making, with particularly strong progress made over the last 12 months. In addition to the urgency with which the U.S. Food and Drug Administration (FDA) approached RWE generation for COVID-19, the agency has also engaged in research collaborations and demonstration projects to learn more about the role RWE can play in regulatory decisions.
As the RWE space continues to evolve, and as regulators and legislators develop standards and policies to help guide its use, it’s important that industry players collaborate and consult with the FDA and Congress on key decisions related to the use of RWE.
Enter the RWE Alliance, a coalition of five organizations leading the RWE space—Aetion, Flatiron Health, IQVIA, Syapse, and Tempus—united in the goal of advancing policies and standards for RWE.
Here, Aetion’s Lowell Schiller, Chief Legal and Regulatory Officer, and Christina Purpura, Senior Director of Upstream Marketing, discuss Aetion’s role in the RWE Alliance, including predicted impact, goals, and why this group is uniquely positioned to further the use of high quality RWE.
Responses have been edited for clarity and length.
Q: What is the RWE Alliance?
CHRISTINA PURPURA (CP): It’s the first-ever trade association dedicated to advancing use of RWE for regulatory decisions on treatments.
LOWELL SCHILLER (LS): This is an organization that is deeply committed to advancing policy around RWE, and it has an incredible amount of collective expertise on generating and using RWE. This allows the RWE Alliance to bring an expert voice to policy conversations.
Q: How did the RWE Alliance come about? Why did the group feel now is the time to take a unified approach to make its voice heard?
CP: The members identified significant opportunities to advance RWE policy and legislation within the next 12 months. First, draft FDA guidance on RWE is due by September 30, 2021. As another example, Congress will consider updates to the 21st Century Cures Act, known as “Cures 2.0,” which includes provisions to advance RWE. We realized that if we band together, we will have a better shot at ensuring RWE receives appropriate attention from policymakers. Through the Alliance, we’re expanding the voice of “good” RWE to improve patients’ lives.
LS: More broadly, there are a lot of RWE-focused policy conversations going on across various parts of government. But what we’re seeing is that there’s not always a complete, shared understanding of how and when RWE can best be deployed, or what effective use of RWE might look like.
There are still a lot of misconceptions about RWE. For example, there’s a misconception that RWE is to be used as a replacement to clinical trials in evidence packages, but we see these as complementary tools that can be used together or separately depending on the circumstance. The RWE Alliance can bring a huge amount of expertise across different parts of the data and evidence generation lifecycles to help inform these discussions.
Q: What is Aetion’s role in the Alliance?
CP: We keep a close eye on how the FDA responds to use of RWE in applications for drugs and biologics, and we’ve also worked with clients on their submissions to the FDA. We find that principled database epidemiology is key to FDA approval, and that’s the kind of lesson that we convey to other members of the Alliance.
LS: We bring the perspective of a company that has very strong platform technology and deep expertise in the analyses of RWD. We believe that is a key part of the policy conversation in the RWE space.
Q: Why is Aetion well positioned to guide collaborations with the FDA and others on RWE standards and policies?
CP: We are fortunate enough to have preexisting relationships with many members of the RWE Alliance. For example, many of these organizations participated in Friends of Cancer Research’s Pilot Project 2.0. In addition, the FDA selected Aetion’s platform for a COVID-19 research collaboration agreement, which is a vote of confidence in the consistency of our approach across the drug lifecycle. Therefore, Aetion is a uniquely positioned collaborator with Flatiron Health, IQVIA, Syapse, and Tempus.
LS: Aetion has been doing a lot of work collaborating with the FDA and others to better understand how RWE can be used as a tool alongside clinical trials, for example, through the RCT DUPLICATE initiative. Aetion can leverage that expertise and research to help inform the conversation around the use cases for RWE, what a good analysis looks like, and how we can avoid introducing bias in the analysis. We believe that we can offer a lot in these conversations.
Q: What are Aetion’s goals within the Alliance? What would a successful first year look like?
CP: Aetion believes the FDA has an important role to play in helping investigators follow the core tenets of principled database epidemiology, so we prepare policy papers to support the agency’s work. For example, we address principles for working with RWD, principles for generating RWE, and examples of specific applications of RWE for which FDA input would be useful.
We believe Congress can also support these aims and help advance RWE—for example, by investing across the federal health care landscape in generalizable evidence on care interventions and their relationships with health outcomes. The focus this year is to support the agency’s work on its forthcoming guidance documents and to help advance Congress’s investment in RWE. This impact will allow us to broaden our remit to other high-stakes decisions and markets.
LS: Another major contributor to success is our ability to help improve toward a better shared understanding in the policy community of how RWE can support knowledge of what medical interventions work best, for whom, and when, as well as a nuanced comprehension of what that entails. As we bring the collective expertise of our members to bear on those policy conversations, we look ahead to a future in which we’re all speaking with a common RWE vocabulary and a common understanding on these issues.
Q: Why is it important that Aetion and the other members are part of the RWE legislation conversation?
CP: Congress is now considering Cures 2.0 legislation that includes provisions to advance RWE. As discussions about RWE progress on Capitol Hill, there’s a great opportunity to help Congress invest in generalizable evidence across the federal health care landscape.
LS: There is much that the members of the RWE Alliance can offer in understanding the effects of different policy choices on the generation and utilization of RWE. As we think through how to build toward a future world where we are best able to leverage all the available health care information, we can help ensure that we’re making smart policy choices accordingly.
CP: Overall, FDA has signaled optimism for the appropriate use of RWE for safety and effectiveness decisions on treatments. We and the other members of the RWE Alliance feel optimistic as well.