Komodo Health Co-Founder and President Web Sun and Slipstream Senior Vice President of Regulatory and Life Sciences Research Tracy Mayne discuss what it takes to achieve regulatory-grade status on the “Pharmaphorum” podcast.
Could real-world data (RWD) eventually replace the placebo trial as the gold standard of clinical research? A decade or more ago, that question would have been preposterous to even entertain. Now, however, as the U.S. Food and Drug Administration (FDA) increasingly embraces RWD to augment — and in some cases bypass — the randomized, controlled clinical trial, Clinical Development and Health Economics and Outcomes Research (HEOR) teams are increasingly beating a data-driven path to faster, less costly regulatory submissions.
In fact, the approach recently allowed Pfizer to win a label expansion for its breakthrough breast cancer therapy, IBRANCE®, and helped Astellas receive FDA approval for its immunosuppressant drug, PROGRAF®, based on a non-interventional study that used RWE from the U.S. Scientific Registry of Transplant Recipients.
Komodo has been working with dozens of Life Sciences teams to accumulate evidence that supports these types of RWD-driven regulatory submissions. And, while it may still be a little premature to forecast the end of the placebo trial, the progress being made toward it is incredibly encouraging.
Komodo Health Co-Founder and President Web Sun recently sat down with Tracy Mayne, Senior Vice President of Regulatory and Life Sciences Research at Slipstream, and Jonah Comstock, host of the “Pharmaphorum” podcast, to discuss this progress, the challenges that still exist, and the growing set of emerging best practices.
Here are some of the top takeaways from the discussion.
Upping the ante on RWD quality and quantity
The key breakthrough that makes it possible for Clinical Development and HEOR teams to reliably re-create clinical trial results in a real-world population and deliver the types of regulatory-grade information required to meet FDA requirements is a depth and breadth of data and a level of computing power that simply did not exist previously.
Capturing the complete patient journey across multiple data sources
The value of both the data and the analytics required to link it with other datasets is critical here. This was the central thesis behind the development of Komodo’s MapEnhance™ capability, which allows users to seamlessly integrate data from Komodo’s Healthcare Map™ with specialty datasets encompassing a comprehensive mix of claims, lab, diagnostics, genomics, and census data to provide a more complete picture of the patient journey.
Slipstream’s Tracy Mayne has been through all aspects of the process of making RWD regulatory submissions to the FDA, on both the Life Sciences side (in his time at Intercept Pharmaceuticals) and the technology side in his current role.
“Let’s start with the kinds of data that the FDA has said are acceptable. FDA said, ‘Look, there are three different ways you could get this data: One of them is disease registries; another is electronic health records; and the third is claims data’,” Tracy explained. “They expect that data to be incredibly systematic and standardized. But it is not collected in the same way a clinical trial is, so there is some additional validation that has to take place. That’s where Komodo comes in, being able to put them together in a way that the FDA finds acceptable. So, of those three sources, I have to say that the databases, just like Komodo, are the ones that are the most seamless and fit for purpose when it comes to regulatory submissions.”
Multidisciplinary collaboration
Another major benefit of this approach of standardizing RWD to develop a complete, consistent view of the entire patient journey is the development of a stronger foundation of data that can be used across multiple functions in the Life Sciences enterprise. Ultimately, as Web and Tracy discussed, the focus on both quality and quantity required for a regulatory submission is making better data more widely available throughout the organization.
“Historically, HEOR teams have always been interested in closed lives,” Web explained. “They need to track these patients longitudinally, while other functions have been perfectly happy to work with legacy aggregators — to do some pill tracking, to define disease by what types of comorbidities we see and the drugs that we're using against them.
“What we're seeing right now is Clinical Development teams, regulatory teams, Commercial teams, and others across enterprise Life Sciences, using closed lives as part of their core strategies, taking a keen eye toward quality and quantity when it comes to their data.”
Tracy added: “RWD was traditionally the purview of HEOR and Medical Affairs. And now, suddenly, Clinical Development is saying, ‘Whoa, whoa, whoa! You mean, I could do much while twice as fast and half the cost if I can use RWD?’ One of the fascinating things is that you’re now able to bridge those two worlds that don’t often talk to each other.”
Undoubtedly, the future of RWE in regulatory environments is burgeoning, bringing forth significant potential to enhance care quality and patients' accessibility to it. Crucial decision-makers, be it in government arenas, medical offices, or Life Sciences boardrooms, are compelled to base their findings on a robust foundation of superior real-world evidence. This is essential in order to leverage the potent combination of data and analytics that promises to alleviate the burden of disease.
Listen to the full “Pharmaphorum” podcast.
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