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AdminApr 3, 20243 min read

Key Takeaways from the public workshop, “Advancing Real-World Data to Generate Regulatory Grade Real-World Evidence in Oncology”

By Inish O’Doherty and Purva Jain

On February 20, Friends of Cancer Research, Duke-Margolis Center for Health Policy, Reagan-Udall Foundation for the FDA, and Aetion co-hosted  "Advancing Real-World Data to Generate Regulatory Grade Real-World Evidence in Oncology." This collaborative workshop convened a wide array of stakeholders, including regulatory experts, academia, clinicians, patients, advocates, data vendors, and biopharma representatives, who gathered to discuss pressing gaps in real-world data (RWD) collection and accessibility. The event also dove into the importance of ensuring that real-world evidence (RWE) generation is relevant to patient care. 

Two members of the Aetion CARE Initiative, Inish O’Doherty (Aetion Engagement Lead) and Purva Jain (Aetion Senior Scientist), had the privilege of attending the workshop last week in Washington D.C. Here, they share their three key takeaways from the event and how Aetion is prepared to help our customers meet this crucial moment in optimizing the use of RWD and RWE for the improvement of oncology patient care and treatment

  1. ECA’s aren’t always the best option: One of the key themes from the day centered on the importance of taking a principled, grounded approach to using RWE in regulatory packages. While there is much excitement about using external control arms (ECAs) in regulatory submissions, these approaches are difficult and not always the most effective approach. In fact, as Ulka Campbell, Aetion’s Head of Scientific Strategy, noted at the meeting, there’s never been a successful patient-matched (ECA) in an FDA oncology approval. Sponsors should consider other ways RWD can augment their application packages, such as providing critical information regarding treatment utilization and natural history.

    Aetion believes sponsors should be thinking critically and creatively when it comes to RWE for regulatory uses. An external control arm is a high degree of difficulty, and there are many other roles RWE can play in a product application. 

  2. Data quality is a core, fundamental principle of epidemiology:  Across sessions devoted to patient-centered RWD and improving data collection and standardization, it was clear that fit-for-purpose, high-quality data remains a topic of utmost concern. Regarding the importance of fit-for-purpose data selection, Paul Kleutz, Deputy Director, Oncology Center of Excellence, FDA, noted, “There’s no question that doesn’t need high-quality data.” As many of the speakers repeated, many of these challenges can be addressed through collaborations and partnerships that aim to improve data quality and advance interoperability standards. 

    Aetion agrees with the importance of data fitness for purpose and is an industry leader in assessing the relevance and reliability of data sources for all types of studies. Whether the study supports a regulatory application or seeks to improve point-of-care treatment, it deserves the highest-quality data available.

  3. Decision-grade evidence: A wide variety of stakeholders stressed the importance of “decision-grade” evidence - that is, evidence that meets the decision-grade standard, as we will only improve patient outcomes if decisions are informed by causal inference and high-quality data. In other words, high-quality evidence should always be the goal, regardless of the setting or intended use.

As an industry leader in decision-grade evidence, Aetion agrees with the importance of quality in all aspects of a study and has built frameworks and processes designed to generate evidence you can trust.

Aetion was honored to participate in this day of meaningful conversations dedicated to highlighting the crucial role of high-quality data in shaping clinical decisions in the rapidly evolving field of oncology. We remain committed to advocating for the thoughtful and principled generation of RWE to improve the treatment and lives of cancer patients. To speak to one of our experts about any of the solutions mentioned above, click here.