Happy Summer!
Our July roundup spotlights what’s working (and what’s still missing) when it comes to real-world evidence. We’re covering trends in regulatory use, a step-by-step framework for reviewer-ready RWD, and a real-world look at Wegovy® use and outcomes after a change in public insurance coverage.
Plus, we’ll be presenting at ISPE next month—let’s connect!
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Great Potential, Limited Practice: RWE Use in FDA MAAs Limited to Orphan Drugs
Despite growing interest in using real-world evidence (RWE) in regulatory approvals, limited information is available about how these guidelines have been applied in practice. Our targeted literature review and case series of drug applications containing RWE (2021-2023) suggests RWE use is still often limited to first-in-class treatments for orphan indications. It seems that regulators are be receptive to RWE in principle, but successful drug applications appear to rely on the entire body of evidence, including indication, methodological rigor, and the use of effective, transparent, and fit-for-purpose RWE.
While the FDA has long used RWE for post-approval monitoring and evaluation, use of RWE for BLAs/NDAs has typically been limited to rare disease indications. The FDA, along with global regulatory bodies, have issued guidelines about RWE incorporation into a wider range of regulatory submissions. However, our findings suggest ongoing uncertainty about how to leverage RWD effectively in the regulatory space. This represents a clear opportunity for sponsors who proactively design for RWE acceptability to strengthen their MAAs and stand out. If you want to learn more, look for detailed case studies highlighting best practices in next month’s newsletter!
Want to unlock RWE’s full potential in regulatory submissions? Contact Landmark for support designing credible, fit-for-purpose studies that go beyond the rare, or learn more about our other RWE services.
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Reproducible, Reviewable, Regulatory-Ready: A Roadmap for Transforming RWD into RWE Reviewers Trust
There is plenty of high-level guidance on real-world evidence (RWE) use in regulatory submissions, but sponsors are often left wondering how to put those recommendations into practice. What does it take to transform complex real-world data (RWD) into regulatory-ready RWE? What can be done to help regulators trust your evidence—and approve your submission? We developed a clear step-by-step framework for the preparation of RWD for regulatory submission based on an in-depth review and synthesis of current FDA guidance and regulator feedback to real-world submissions containing RWE.
Key steps include:
Want to learn more about preparing RWD for regulatory review? Connect with our team to explore how to apply our framework to your next submission, or explore our other RWE services.
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Context is Key: Understanding Drug Use and Performance in a Changing Landscape
Drugs are developed to treat diseases in individual patients—but real-world use often depends more on systems than symptoms.
For example, Medicaid Part D does not cover Wegovy® for weight loss, but started covering it to reduce cardiovascular risk in March of 2024. Together with Humbi.ai, we presented our initial results of an in-depth analysis of characteristics, treatment patterns and outcomes among Medicare FFS beneficiaries who used Wegovy® following this policy shift. These early adopters were typically older adults with existing cardiovascular risks, but were less likely than users of other GLP-1s to have diabetes. Over 95% continued this medication without switching during the study period, suggesting higher continuation rates than have been observed in studies of real-world semaglutide use for obesity.
When prescribing becomes more (or less) accessible, it doesn’t just affect cost but also who gets treated and for what indications. RWE is needed to understand how the real-world context, including policy shifts, provider practices, new populations, and care infrastructure shape real world use—and real-world impact.
Want to get a snapshot of a dynamic market landscape or contextualize trial results? Connect with our team to get a more complete picture, or explore or explore our other RWE services.
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Want to find out more? Join Us at ISPE Next Month!
We will be presenting at the 41st annual meeting of the International Society for Pharmacoepidemiology (ISPE) in Washington DC on August 22-26. Registration is now open.
If you will be there, we’d love to meet in person! Make an appointment or drop by one of our posters. Look for our times and locations in our next newsletter.
