How to Seize the Coming Seismic Shift to Real-World Evidence

Clinical research carries authority from randomized clinical trials (RCT). RCTs produce believable results because they are scientifically rigorous, transparent, and largely free of bias. RCTs have demonstrated safety and efficacy in modern medical therapy development for years.?

But one downside of RCTs is that the controlled results don’t always represent the diversity of the patient populations that might benefit from the therapy. RCTs also require more time to meet specific endpoints.?

In contrast, Real-World Evidence (RWE) is becoming more popular with clinical researchers for many reasons: simplicity, the availability of diverse populations living in real-world conditions (versus a controlled setting such as hospital studies), and the volume of data available through modern technology.?

So what is RWE, and what is it good for?

A Shift Toward RWE

RWE comprises Real-World Data (RWD) collected in various ways. The U.S. Food and Drug Administration (FDA) gives these definitions:

• Real-World Evidence (RWE) is “the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.”
• Real-World Data (RWD) is “data relating to patient health status and/or the delivery of? health care routinely collected from various sources.”

These definitions stand in contrast—and as a primary distinguisher for RWE—to how randomized clinical trials collect information, through tightly controlled means, typically at hospitals staffed by clinicians.

This focus on RWE grew from the 21st Century Cures Act of 2016. That act mandated that the FDA develop a way to evaluate the use of RWE to “support new indications for already-approved drugs and fulfill post-approval study requirements.” RWE was found to complement and perhaps move toward becoming an alternative to RCTs.

But what are the characteristics of RWE? Aside from RWE not coming from a randomized clinical trial, it makes sense to think of RWE as composed of RWD. RWD has a number of characteristics:

• RWD features observational data versus data gathered in a controlled setting as the data gathered in an RCT.?
• RWD is unstructured and possibly inconsistent because of entry variations. Think of electronic health records with various fields, from text to numeric.
• RWD is generated quickly and frequently. Real-world data has ongoing volumes, making it very attractive to researchers.
• RWD may appear incomplete or lacking in key endpoints for analysis because it was originally intended for other purposes.
• RWE may be subject to bias and measurement errors (random and non-random).

Where does Real World Data Come From?

Real-world data comes from many a surprising number of sources such as lab test results, EHR data, radiology images—all the data we might think of as “clinical.” But many sources are further removed from clinical, like family history (like historical data on health conditions and smoking status), medication data (orders, prescription refills, concomitant therapies, administration information like dosage or route of admin), patient-reported information (self-reported outcomes, surveys, diaries).?

Real-world data can include information from fitness trackers and wearable devices (mobile health devices), insurance or medical claims, environmental factors like pollutants and infections, or lifestyle factors. Even molecular profiling might contribute to real-world data. Social media already has a place in real-world data, with algorithms that can come close to pseudo-diagnosis. Facebook posts, blogs, tweets—all are increasingly parsed for meaning.?

Any list of real-world data sources will be incomplete because of the ever-growing number of such sources. Every day there are breakthroughs in technology that lead to breakthroughs in tracking the body’s parameters.

Watch for RWE using RWD to play an increasingly important role in establishing credible science in FDA submissions, both with RCTs and, perhaps in the future, even replacing RCTs.?

Are you currently running RWE studies and want to learn more? Join us along with our industry experts from UBC & Datavant on May 3rd in a webinar about the role of patient-centric technologies & tokenization in RWE studies.