The world’s greatest chess players understand that every variable (analytic, contextual, social) is interdependent and relevant. When it comes to healthcare, the goal is value and the denominator Positive Clinical Outcomes “Value” is what you want to pay for. And Real World Evidence helps us to define, capture and understand “value” for patients, payers, and providers. And these truths are as self-evident for drugs as for medical devices.
In the 21st century, the information revolution will shift from the generation of data to figuring out the meaning, purpose and value of the data with the patient’s perspective in mind. The FDA has described factors it would consider when evaluating the relevance, reliability and quality of real world evidence, and also suggests when it might use such data to make decisions about the products it regulates.
Real World Evidence (clinical outcomes data not collected in conventional randomized controlled trials) is the new star on the precision medicine horizon. However, “Big Data” and “Valid Evidence” are not the same thing. It is an important distinction that illuminates a crucial difference. Real World Evidence requires 21st Century Regulatory Science to determine and define new thinking on evidentiary standards. Nobody said it was going to be easy.
The concept driving the future is interoperability; the idea that different systems used by different groups of people can be used for a common purpose because those systems share standards and approaches. For medical device manufacturers who have heretofore devised development strategies based on predicate-based thinking, interoperability will not be as foreign a concept as it might otherwise be to manufacturers of drugs and biologics. And if you’re wondering about the future of combination products, now’s the time to put on your thinking cap.
To paraphrase Winston Churchill, when it comes to actualizing the use of real world evidence, we are nearing the end of the beginning. The road to the use of Real World Evidence is a trail that regulator and regulated must blaze together sometimes heroically and at other times with greater caution.
In the words of the British pundit Ernest Benn,
“Politics is the art of looking for trouble, finding it whether it exists or not, diagnosing it incorrectly, and applying the wrong remedy.“
Regulators and Device Developers don’t have that option.