- In an effort to use EHR data to gain insights into cancer treatment trends and health outcomes across the country, FDA has expanded its research collaboration agreement with Flatiron Health and the Information Exchange and Data Transformation (INFORMED) program.
The partnership began in 2016 to enable FDA to expand its understanding of the ways real world evidence from de-identified EHR data can inform healthcare policymaking decisions.
Two years after the program launch, FDA and health IT company Flatiron will expand the project to include characterization of data quality, validation of reliable real world clinical endpoints, collaboration on new analytic methodologies, and explorations of innovation applications.
“The FDA recognizes the tremendous importance of analyzing treatment data from the real world,” said FDA Oncology Center of Excellence Director Richard Pazdur, MD. “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,” continued Pazdur. “We believe that regulatory-grade real-world data can help inform our decision-making so that we can provide cancer patients with better care.”
FDA will also work with Flatiron to evaluate real world cancer populations that are historically underrepresented in clinical trials.
The cancer-focused health IT company will also work with FDA through its life sciences partners to integrate real world evidence into regulatory filings for post-marketing studies, label expansions, and other use cases.
“In order for the entire industry to benefit from RWE, we must ensure that our datasets are comprehensive and of the highest-quality,” said Flatiron Health Co-Founder and President Zach Weinberg. “Working with the FDA enables Flatiron to both learn from their leadership and continue to contribute to standards development.”
“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,” Weinberg added.
During the first phase of the program, Flatiron provided de-identified EHR data to FDA to offer insight into treatments for cancer patients with advanced non-small cell lung cancer treated with immunotherapy.
The data included real-world endpoints such as progression, tumor response, and reason for therapy discontinuation.
This next phase of the collaboration aligns with FDA’s efforts to modernize the drug review process.
In January 2019, FDA Commissioner Scott Gottlieb, MD, emphasized the importance of real-world evidence for informing clinical decision-making and innovation.
Gottlieb addressed the Bipartisan Policy Center to discuss its plans to make real-world evidence available for clinical study and facilitate more accessible clinical trials through a decentralized approach.
The goal of leveraging real-world evidence in clinical studies is to reduce the time between identifying a health problem and developing a solution for the problem based on reliable evidence.
“Traditional post-market studies typically require years to design and complete and cost millions of dollars,” Gottlieb noted. “By encouraging the use of RWD and RWE, we may be able to provide patients and providers with important answers much sooner by potentially identifying a broader range of safety signals more quickly.”
By using data from EHR systems and connected devices, FDA and other federal regulators would have the opportunity to monitor health IT products for efficacy. Furthermore, health IT developers can use these insights to develop new products or improve upon existing versions.