The FDA and Flatiron Health Expand Real-World Data Cancer Research Collaboration

Renewed collaboration to focus on further integrating real-world evidence derived from electronic health records into regulatory decision-making

NEW YORK, NY

Flatiron Health and the Information Exchange and Data Transformation (INFORMED) Program at the U.S. Food and Drug Administration (FDA) today announced a two-year renewal and expansion of their research collaboration agreement. The collaboration, which began in 2016, has enabled the FDA to better understand how real-world evidence (RWE), derived from de-identified patient datasets curated from electronic health records, can support regulatory decision-making.

Over the past two years, the FDA and Flatiron have explored the use of RWE to generate insights into cancer treatment trends and clinical outcomes in the United States. The expanded collaboration will continue to focus on critical topics related to the use of RWE in regulatory decision-making, including characterization of data quality, validation of reliable real-world clinical endpoints, collaboration on new analytic methodologies, and exploration of innovative applications such as real-world control arms.

Flatiron and the FDA will also evaluate real-world cancer populations that are typically underrepresented in clinical trials. Complementing these direct collaboration efforts, Flatiron will also engage with the FDA through its life sciences partners, as they increasingly incorporate RWE into regulatory filings for postmarketing studies, label expansions and other supportive use cases.

In the first phase of the partnership, Flatiron provided the FDA with de-identified datasets to provide insights on cancer patients with advanced non-small cell lung cancer being treated with immunotherapy (see here and here for the research published in The Oncologist). These data, which included real-world endpoints such as progression, tumor response and reason for therapy discontinuation, supported a variety of novel outcomes research projects.

“The FDA recognizes the tremendous importance of analyzing treatment data from the real world,” said Richard Pazdur, MD, Director of FDA’s Oncology Center of Excellence. “Traditional clinical trials have long provided the high-quality evidence the FDA needs to determine whether a product is safe and effective for its intended use, but traditional trials do not always represent the real world, lack clinical context, and may not provide sufficient follow-up to truly understand the impact of a new therapy on real-world patients. We believe that regulatory-grade real-world data can help inform our decision-making so that we can provide cancer patients with better care.”

“In order for the entire industry to benefit from RWE, we must ensure that our datasets are comprehensive and of the highest-quality. Working with the FDA enables Flatiron to both learn from their leadership and continue to contribute to standards development,” says Flatiron Health Co-Founder and President Zach Weinberg. “At the end of the day, this is how we’re closing the evidence gap in oncology: real-world patient experiences inform research, which ultimately leads to better cancer treatments and outcomes.”

The expanded research collaboration with Flatiron is aligned with the FDA’s long-term initiative to modernize the drug review process. As demonstrated in last month’s release of the strategic framework to advance RWE to support drug development and the newly established Office of Drug Evaluation Science, the agency is innovating to understand, in real-time, how treatments impact patient populations in the real world.

To date, the FDA-Flatiron collaboration has produced numerous peer-reviewed publications, as well as presentations at leading industry conferences like American Society for Clinical Oncology and American Association of Cancer Research. For the full list of Flatiron’s research publications, see here.

About FDA INFORMED

INFORMED is an incubator for collaborative regulatory science research focused on supporting innovations that enhance FDA’s mission of promotion and protection of public health. The research portfolio of INFORMED is focused on data science and health technology applications such as the use of real world data for clinical evidence generation, the utility of biosensors and the internet of things to quantify intrinsic and extrinsic factors influencing the patient’s experience, and opportunities for machine learning and artificial intelligence to augment existing practices. For more information, please visit www.fda.gov/INFORMED.