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An International Framework for Real-World Evidence – Day 2: Regulatory and Policy Needs for RWE

An International Framework for Real-World Evidence – Day 2: Regulatory and Policy Needs for RWE

Day 2 of the Friends of Cancer Research (Friends) meeting on real-world evidence (RWE) turned the focus from Day 1  to the regulatory and policy implications of real-world evidence.  The hour-long meeting was comprised of two parts: a keynote conversation featuring Amy Abernethy, Deputy Commissioner of the FDA and a leader in the agency on RWE, and Michelle McMurry-Heath, President and CEO of the Biotechnology Innovation and Organization, followed by a panel composed of leaders in RWE. The session focused on how the findings from collaborative efforts like the RWE Pilot Project 2.0 can inform future drug development programs and regulatory decision-making.

The keynote conversation between Abernethy and McMurry-Heath covered a wide array of topics. Of most urgency was the importance of RWE during the COVID-19 pandemic, as the pandemic has forced people to get more comfortable with remote monitoring and electronic data. They specifically looked at learnings from the COVID-19 Evidence Accelerator, a joint effort led by the Reagan-Udall Foundation and Friends, acknowledging the need to leverage real-world data quickly while still getting the science right. The discussion then turned toward the potential of policy and legislation to advance RWE, particularly when addressing the next round of Prescription Drug User Fee (PDUFA) negotiations. This included looking at diversity in clinical trials and using RWE to support less restrictive eligibility criteria. Lastly, they discussed the concerns about the FDA, including the scientific integrity, fast-moving science, and heavy workload facing the FDA. Abernethy highlighted particular areas the agency was reviewing, including an emphasis on clear, concise, consistent communication, and bringing on talent and allowing them to work at the top of their license.

This insightful keynote conversation was followed by a panel and Q&A composed of leading RWE stakeholders. The panel was moderated by Kate Rawson of Prevision Policy and featured Adrian Cassidy, Novartis; John Concato, FDA; Mark McClellan, Duke-Margolis Center for Health Policy; and Michael Vasconcelles, Flatiron Health. They began the panel discussing the successes of RWE in PDUFA VI and how to build on that in PDUFA VII, looking at what regulatory guidance stakeholders would like to see. They also discussed the importance of increasing transparency in RWE to allow regulators and policy-makers to better trust the data. Lastly, they further emphasized the point brought up in Day 1 that RWE is not an alternative to clinical trials, but rather something to synergize with and streamline clinical trials in the future.

The two-day meeting concluded with a few closing remarks from Friends President & CEO Jeff Allen. Be sure to keep an eye out for additional analyses and publications on the findings from the project. 


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