On Thursday, July 23, Flatiron Health provided invited comment to the U.S. Food & Drug Administration’s first public meeting about reauthorization of the Prescription Drug User Fee Act (PDUFA). Dr. Nicole Mahoney, Flatiron’s senior director of regulatory policy, called upon the FDA to expand and clarify how it uses real-world evidence in its decision-making. Her full remarks follow here:

Good afternoon. I am Nicole Mahoney, senior director for regulatory policy and employee of Flatiron Health and Roche stockholder. Thank you for the opportunity to comment on the success of PDUFA VI and recommend enhancements for PDUFA VII from our vantage point and area of expertise.

Flatiron Health is dedicated to advancing our understanding of how real-world data derived from electronic health records can improve patient care and inform decisions about cancer therapy development, and access. We provide electronic health record and practice management software and services to cancer clinics across the U.S., and create de-identified research datasets to accelerate cancer research. Our vision is to realize the full potential of real-world evidence to support the development of oncology treatments, including precision medicines, improve patient access to effective therapies and care, and facilitate patient enrollment in clinical trials.

Real-world evidence can complement randomized clinical trials–the “gold standard” evidence base for regulatory decisions — by filling critical information gaps for patients typically excluded from traditional clinical trials, including those with rare conditions. We expect the use of real-world evidence to grow as new sources of data become available, and methods to analyze and derive insights from them evolve.

Flatiron applauds the success of PDUFA VI in advancing patient-focused drug development, and accelerating understanding of how and when RWD from a variety of sources can support regulatory decisions. We look forward to forthcoming guidance, and continued partnership with FDA.

PDUFA VII provides opportunities to build upon the success to date, explore what is possible with respect to real-world evidence, and address challenges identified under PDUFA VI. We offer the following four recommendations for your consideration.

  1. Ensure that the learnings from RWE submissions to the Agency are captured and shared broadly, including how real-world evidence is generated and used by sponsors, and incorporated into FDA’s decision making. We encourage FDA to work with sponsors to share key takeaways, obviously in ways that protect proprietary and confidential information. 
  2. Issue targeted RWE guidance that builds on lessons of PDUFA VI, clarifies the clinical and regulatory circumstances where real-world evidence may be acceptable, and addresses current limitations on its use, including, but not limited to, methods to establish causal inference–including ways to address bias and confounding; and the development and use of real-world endpoints.
  3. Establish new pathways for FDA to work with the technology companies who are generating real-world data and analytic tools that are used by a range of sponsors to enable patient-centric drug development. Regulatory certainty through formal meetings would drive investments toward technological tools with the greatest potential to help speed patient access to effective therapies.
  4. Continue to build on FDA’s efforts to promote a learning health data ecosystem by modernizing the use of technology, and increasing capacity to ingest and analyze data as described in the Technology Modernization Action Plan, and working with external stakeholders to help establish a robust real-world data ecosystem. PDUFA VII provides an opportunity for FDA to continue promoting high-quality data capture at the point of care by setting clear expectations around the data and technology standards the agency would find most useful. 

Thank you again for this opportunity to contribute. We look forward to continued participation through the user fee reauthorization process.

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