What I learned from former FDA leaders during Aetion’s RWE Summit

Carolyn Magill
CEO, Aetion

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Aetion hosted our annual RWE Summit in New York last month, and I am still inspired by the content and discussion. This is our third consecutive year of convening the most knowledgeable minds in healthcare for in-depth discussions on how real-world evidence (RWE) is transforming healthcare delivery and payments—and how to advance its use in improving patient outcomes. After a year and a half of video calls and attending conferences on Zoom, it was delightful to engage in person, and to have other attendees active with questions and observations via the chat function on Zoom.

We were honored to have two of healthcare’s foremost thought leaders attend, Former Commissioner of the U.S. Food and Drug Administration (FDA) Dr. Scott Gottlieb and Dr. Mark McClellan, who was head of both the FDA and the Centers for Medicare and Medicaid Services (CMS). My fireside chats with both of them reinforced the FDA’s commitment to integrating RWE as a key factor in decision making. They both issued the call to action for sponsors to include RWD and RWE in submissions—even when they have conducted a traditional trial, as supplements—so that reviewers can gain increased familiarity with when both the data and the insights derived from them are most useful.

Dr. Gottlieb, who led the FDA during the emergence of COVID-19, has been candid and vocal about the national response to the pandemic, including challenges with testing and developing real-time insights about COVID. As he has emphasized, it’s imperative to reflect on the lessons learned from this pandemic. We can improve upon how we respond to the next one—or, even sooner, outbreaks of variants like Omicron—while also improving health outcomes today. Aetion is rising to the challenge of supporting this agenda, both directly and indirectly. We are partnering with FDA to design a framework and system of studies that enable real-time evidence generation for COVID-19 inpatient treatments, which the agency can apply to make swifter decisions during future public health emergencies. Manufacturers of vaccines and treatments for COVID-19 are using the Aetion Evidence Platform® to assess safety and effectiveness, especially when time is of the essence, and for patient populations underrepresented in clinical trials.

Dr. Gottlieb reminded us that RWE “was extremely important” as a tool and early warning system for waning vaccine effectiveness. For example, RWE from Israel suggested that Pfizer–BioNTech’s COVID-19 vaccine became less effective a few months after the second dose, especially in older populations. While the data wasn’t enough to drive a decision on its own, Dr. Gottlieb said, it was the impetus for Pfizer to launch a large study that gauged the effectiveness of a third (booster) shot. The trial demonstrated that a three-dose regimen had higher efficacy than the two shots. Anecdotes from doctors and hospitals in NYC in recent weeks tell of positive cases of COVID having higher prevalence in the un-boostered population.

RWE can also play a critical role in clinical studies. One of the bottlenecks during the pandemic that slowed drug development was the challenge of running an in-person randomized controlled trial (RCT) amidst lockdown. As RWE advances, Dr. Gottlieb expects to see it more frequently replace placebo arms in clinical trials.

Dr. McClellan echoed Dr. Gottlieb’s sentiments on RWE’s key role in decisions on booster shots and to gauge the effects of new variants. During our discussion, he also emphasized how RWE can be of service to health care payers, who can use RWE to connect members to the right drug at the right time and in the right clinical context. This will be hugely important, he said, for improving outcomes in the everyday setting of combating chronic health conditions. For payers, that means providing the best possible outcomes to their members at the lowest possible cost.

To demonstrate to payers that RWE is in their best interest and the best interest of their members, Dr. McClellan advised we need to show evidence that RWE works to improve outcomes broadly, rather than focusing on one-off studies demonstrating reduced cost on a particular product.

Dr. McClellan said that the real value for payers is in the knowledge wrapped around the real-world use of a product—what patterns can we observe in data about the ways an intervention affects a patient’s health? RWE can help unlock that knowledge.

This observation speaks to one of the primary reasons I joined Aetion 4.5 years ago: the notion that over time Aetion’s technology will advance understanding of which combinations of interventions are most impactful in shifting cost curves for high quality care rather than simply bending curves at the margins. Is this medication plus this telephonic care management program as effective as that same medication with in-person support? What is the impact of better sleep or nutrition or socio-economic factors on outcomes? It’s imperative we become more targeted on the most impactful interventions.

We’ll be sharing more from the Summit in the coming weeks: how sponsors are turning to RWE across the development lifecycle and obstacles to enterprise adoption; the extent to which RWE can inform value-based collaborations; optimization of data strategies; the role of RWE in achieving health equity for payers, providers and sponsors; and de-mystifying artificial intelligence and machine learning.

Stay tuned!

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