The FDA has partnered with Flatiron Health to assess the use of real-world data to demonstrate the safety and efficacy of immunotherapies and other emerging anticancer drugs. Flatiron, an oncology analytics shop that has received funding from Google Ventures and Roche ($RHHBY), is applying its cancer data digging capabilities to the task.
The first publicly disclosed project to be undertaken by the collaborators is focused on the use of immunotherapies to treat advanced non-small cell lung cancer, an indication in which Bristol-Myers Squibb’s ($BMY) Opdivo and Merck’s ($MRK) Keytruda are approved. Specifically, the FDA and Flatiron intend to evaluate “analytic approaches, clinically-relevant endpoints and safety assessment methods” that use real-world evidence to better understand the effects of the immunotherapies.
In the case of advanced non-small cell lung cancer, such analyses of real-world evidence could give the FDA and the wider industry a more detailed, nuanced picture of the safety and efficacy of Opdivo and Keytruda than can be seen in the clinical trial data.
“With only 4% of the adult U.S. cancer population enrolled in clinical trials, we as providers are currently limited to making clinical decisions based upon study results that represent only a small number of patients,” Flatiron CMO and Oncology SVP Amy Abernethy said in a statement. “This makes it nearly impossible to estimate whether a treatment will work, anticipate all of the potential side effects of therapies or even decide which treatment should precede another.”
The situation outlined by Abernethy fits squarely with the problem Flatiron founders Nat Turner and Zach Weinberg set out to solve when they left Google ($GOOG), which bought their first startup for $81 million. When Turner and Weinberg looked for opportunities to apply their tech skills to the improvement of cancer treatment, they quickly hit on clinical trial enrollment as a major problem, only to shift focus to address the unstructured, siloed nature of oncology care data. The FDA alliance taps into Flatiron’s ability to analyze cancer care data to mitigate the enrollment issue.
For the FDA, the alliance is in keeping with the vision for the future of drug regulation put forward by Commissioner Robert Califf at a conference attended by Regulatory Focus this month. Califf called the use of real-world evidence to support FDA decision-making the “top programmatic priority” for his early days at the helm of the regulator. Specifically, Califf wants the FDA to establish a real-world evidence system that can support its work for decades to come.
Where Flatiron fits into this vision is unclear, but the FDA has shown a willingness to work with industry to establish regulatory IT systems. Notably, the FDA has worked with DNAnexus to create precisionFDA, a platform designed, in part, to support regulatory understanding of molecular diagnostics.
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