Wednesday, December 7, 2016

15 FDA Authors on Real World Evidence: NJEM December 2016

With the passage of 21st Century Cures on the Hill (by huge margins), and the likely signing by President Obama, we'll hear more about Real World Evidence.   

While there's been alarmist talk that 21CC will foist poorly validated trials with RWE onto the American health system, all that 21CC does is require the FDA to "look at" RWE and set up some guidance documents if it can.

To get a view on what the FDA leadership really thinks about RWE, see an article in New England Journal of Medicine by Sherman et al., including leadershp such as Jeff Shuren, Robert Temple, Janet Woodcock, and the administrator, Robert Califf.   The article is open access, here.  

The following week, FDA authors Califf et al. wrote a companion NEJM piece but focused on big data, coordination, and the learning healthcare system.

FDA published a guidance document on RWE last summer (guidance here, RAPS article here.)
From the opening paragraph, 
The term “real-world evidence” is widely used by those who develop medical products or who study, deliver, or pay for health care, but its specific meaning is elusive. We believe it refers to information on health care that is derived from multiple sources outside typical clinical research settings, including electronic health records (EHRs), claims and billing data, product and disease registries, and data gathered through personal devices and health applications.1,2 Key to understanding the usefulness of real-world evidence is an appreciation of its potential for complementing the knowledge gained from traditional clinical trials, whose well-known limitations make it difficult to generalize findings to larger, more inclusive populations of patients, providers, and health care delivery systems or settings that reflect actual use in practice.3'

From the conclusion,
We believe that when the term “real-world evidence” is used, the primary attribute that distinguishes it from other kinds of evidence is related to the context in which the evidence is gathered — in other words, in clinical care and home or community settings as opposed to research-intensive or academic environments. Most important, the distinction should not be based on the presence or absence of a planned intervention or the use of randomization. Real-world research and the concepts of a planned intervention and randomization are entirely compatible. Indeed, one of the most important advances in clinical trial methodology may be the broadening of the application of randomization outside more typical venues for clinical trials, such as academic research centers. But in order to gain collective confidence in the appropriate uses of this array of methods across disparate settings, we must first be clear about our terminology and its application.