- The FDA and Flatiron have announced a two-year extension of their Information Exchange and Data Transformation (INFORMED) Program partnership to leverage real-world evidence (RWE) and clinical trial data to improve outcomes for cancer patients.
The new agreement builds upon a project started in 2016 to help the FDA better understand how real-world evidence extracted from deidentified patient data, can support regulatory decision-making.
In 2018, Swiss pharmaceutical company Roche purchased Flatiron for $1.9 billion.
Since beginning the collaboration in 2016, Flatiron has provided the FDA with big data on individuals with advanced non-small cell lung cancer who were receiving immunotherapy treatment. The data helped researchers launch a variety of projects related to tumor progression, treatment discontinuation, and other key patient-centered factors.
“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.”
Flatiron and the FDA will continue to focus on how to apply RWE to regulatory decisions, and will also explore how to validate data, develop advanced analytics, and apply RWE to innovative clinical trial design.
“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 FDA has made integrating real-world evidence into clinical trials a high priority as it seeks to modernize the drug review process.
In December, the agency released a new framework for incorporating RWE into the drug development pipeline, noting that data derived from electronic health records, medical devices, laboratory testing, and insurance claims can offer invaluable insight into the safety and efficacy of novel therapies.
“Because they include data covering the experience of physicians and patients with the actual use of new treatments in practice, and not just in research studies, the collective evaluation of these data sources has the potential to inform clinical decision making by patients and providers, develop new hypotheses for further testing of new products to drive continued innovation and inform us about the performance of medical products,” FDA Commissioner Scott Gottlieb, MD, said at the time.
“RWE provides us with a potential source of information that can complement, augment and expand our understanding of how best to use medical products—improving what we know about our medical care.”
Gottlieb has also promoted the idea of creating “hybrid” clinical trials that incorporate RWE to create speedier, more robust pathways to market for innovative therapies.
RWE is also critical for capturing information on diverse participants, something that traditional clinical trials often struggle to accomplish.
“We believe that more accessible clinical trials can facilitate participation by more diverse patient populations within diverse community settings where patient care is delivered, and in the process can generate information that’s more representative of the real world and may help providers and patients make more informed treatment decisions,” Gottlieb said in late January in a speech to the Bipartisan Policy Center.
“This approach, called decentralized clinical trials, can help move prospective collection of data from the real-world—including randomization—outside of the brick and mortar boundaries of traditional clinical research facilities, tapping into not only EHRs but additional digital health tools like wearable devices.”
Collaborations with industry stakeholders such as Flatiron will continue to play an important role in identifying new opportunities to apply RWE to critical drug development questions, the FDA said.
The partnership has thus far led to numerous published research studies and presentations to major professional societies about treatment and outcomes for a variety of cancers.