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

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

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 Flatiron

Flatiron Health is a healthcare technology and services company focused
on accelerating cancer research and improving patient care. Our platform
enables cancer researchers and care providers to learn from the
experience of every patient. Currently, Flatiron partners with over 280
community cancer practices, seven major academic research centers, and
over 15 of the top therapeutic oncology companies. For more information,
please visit
or follow us @FlatironHealth.


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


Jenny Edelston

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