Tomorrow the FDA is wrapping up its first public comment period for its real-world evidence strategy. It’s a first step in the agency’s process to determine how new data sources that represent real patients, in the real world, can inform regulatory decision-making.
And it’s the latest sign that a massive shift is underway in health care, spurred in part by the vast amount of health data now available—and new technologies that can analyze new data sources.
Yet as we move forward, let’s not lose sight of the past.
For decades, biopharma manufacturers have developed drugs that save lives and keep us healthy. And when they succeed, it’s always been for this reason: They adhere to rigorous science and the meticulous
processes required to bring safe, effective drugs to patients.
And yet, the results are not perfect.
Minorities, people over 65, people with limited incomes, those with multiple chronic illnesses are under-represented in clinical trial results. Some off-label uses do benefit some patients, yet establishing safety and effectiveness through the gold standard of randomized trials for all populations is cost-prohibitive. And the cost of drugs is on every mind, from patients to doctors to politicians and regulators to payers.
The coming era of better, cost-effective outcomes for all patients requires biopharma to re-think its well-honed path to success. The FDA and other regulatory bodies around the world are signaling a new way forward. They’re determining how additional sources of data can better represent how real people live and respond to medications and inform regulatory decision-making.
Transforming this data into evidence is a sea change. It’s one that prudent biopharma firms may well resist. That’s perfectly understandable. Sticking to what we know works is far safer than leaping into uncharted territory.
And yet manufacturers of critical medications need not look far for the map to success in this new era. They already have it. It’s right there, in the same sound principles they have always relied on to ensure safe, effective treatments.
The principles of good science will guide the industry forward into a new era.
Working with new sources of health data—from medical claims and electronic health records to patient registries, and more—does not change the need for validated processes. Analyses of real-world data can match the rigor of “gold standard” processes like randomized clinical trials if they adhere to three key principles: transparency, auditability, and reproducibility.
All are critical to ensuring that evidence developed from new sources of data is regulatory-grade; that it matches the sound science of evidence produced by randomized controlled trials.
The FDA and European Medicines Agency in particular are moving forward to ensure that these principles guide their evaluations of real-world evidence. Indeed, the agencies are already actively figuring out how to use this evidence to accelerate drug approvals, expand a drug’s indication, evaluate the post-marketing safety of a drug, and more.
Taking new pathways does not always equate with risk taking. With biopharma’s historic dedication to scientific rigor as its north star, exploration becomes innovation.
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