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How real-world evidence is changing the game

I spend most of my work life talking to people about real-world evidence (RWE). And, in most cases, it’s pretty straightforward — whether I’m talking to inVentiv Health clients about strategies for RWE generation and dissemination or to payers and other healthcare system stakeholders about how RWE can inform decision-making. These conversations almost always rest on a common foundational understanding of what RWE is and why it’s important.

But recently, I had the opportunity to lead a panel discussion on RWE for a different kind of audience—one that may not have heard of the term before walking into the room and probably didn’t expect to care much about it by the time they left. The audience was comprised mostly of investors and mergers & acquisitions types attending a conference to hear company presentations by scores of small biotech start-ups wooing new investors.

So, how do you set the stage for a productive discussion about RWE when the people you are speaking with are much less steeped in it than you are? Why should they — or anyone — care about RWE?

A reasonable place to start is with a definition of terms. A basic definition for RWE that we commonly use is that it’s what you get when you apply rigorous analytics to real-world data (RWD). RWD refers to all data collected outside the traditional clinical realm of randomized controlled trials (RCTs). Common approaches include retrospective analyses of claims databases and medical charts, prospective observational research, and pragmatic clinical trials, among others.

Seeing RWE defined in relation to RCTs seemed to put people’s minds at ease, as RCTs are things they understand very well—after all, they’re the ones who are bankrolling clinical development for many biotech start-ups.

But it isn’t enough to define what RWE is; it is equally important to explain why it matters.

In my panel discussion, I resorted to a football analogy. Historically, before spiraling costs in the health sector garnered as much attention as they currently have, it was reasonable for a manufacturer to focus primarily on clinical development. Because once all those RCTs had been successfully executed and regulatory approval had been achieved, it was like the ball had been placed on the opponent’s two-yard line and all they had to do was mount a sales offensive to punch it over the goal line to attain commercial success. The prescriptions would come—physicians would write them, patients would fill them and insurers would pay them.

But those days are long gone. While Phase I-III trials are still the only series of plays leading to regulatory approval, the ensuing placement of the yard marker is nowhere near the end zone—it’s more likely to be around midfield. Making matters worse, several defensive reinforcements enter the playing field. The regulatory authorities are still in play, requesting post-marketing research, but they are joined by a variety of other healthcare system stakeholders, including health insurers, health technology assessment authorities, patient advocacy groups and provider associations. And what these players are interested in, above and beyond how the product performs in clinical trials, is how it performs in real-world use—in other words, they are demanding RWE.

So, there is little hope of mounting a simple sales offensive based on the Xs & Os of safety and efficacy data alone to score commercial success—the amount of yardage is too great and the defense too formidable. The offensive playbook has to change from RCTs to RWE. 

Investors are placing large bets on small biotechs and, as part of their due diligence, need to think about how these companies will transition from clinical evidence development to real-world evidence development in order to yield the expected return on investment. It’s necessary for companies to be thinking much further ahead about the range of data requirements that will be needed by payers and other key stakeholders; this can’t be delayed until the peri-launch period. As my colleagues and I at inVentiv Health like to say, “Late phase is too late.”

The bottom line is that RWE is important to everyone who has a touchpoint to the healthcare system, including traditional healthcare system stakeholders and those who might have financial (pig) skin in the game. 

For more information, download inVentiv Health’s white paper. And join me at ISPOR where we’ll be taking a closer look at how the changing regulatory environment is impacting RWE development. 

Filed Under: Clinical Trials Regulatory / Compliance