T1D Exchange’s 9th Annual Meeting in Cambridge, MA featured a special presentation from two officials at the U.S. Food and Drug Administration. Alain Silk, PhD, the Diabetes Diagnostic Devices Branch Chief, and Kenneth Quinto, MD, MPH, Senior Medical Officer, Real World Evidence Analytics Staff, shared their insights on how regulatory approaches are adapting to the increased sophistication of clinical devices and availability of real-world evidence.
Real world evidence (RWE) is not to be confused with real-world data (RWD), which Dr. Silk defined as data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. By contrast, RWE is clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD.
In the past, the FDA has relied heavily or exclusively on randomized controlled clinical trials when making regulatory decisions regarding both drugs and devices. While these trials represent a key component of scientific and medical research, they do face certain limitations, including the narrow sampling of participants.
However, research and trials designed around RWE and observations, with or without clinical intervention, can measure safety and performance in diverse patient populations and groups, assess longer-term outcomes, and build larger data sets that reveal rare, but critical, adverse events.
RWD sources also offer opportunities to partner with patients in novel and exciting ways, from employing wearable devices to leveraging mobile medical apps.
Drs. Quinto and Silk addressed a number of concerns and questions surrounding the changing standards and procedures regarding these two methods for gathering patient information for regulatory decisions.
The doctors referenced an important discussion happening in the industry surrounding valid scientific evidence that is fit for purpose, demonstrating both that the drug or device benefits outweigh probable risks, and that the effectiveness of the device in the target population will yield clinically significant results.
These considerations are especially important for research and development surrounding type 1 diabetes, for a number of reasons:
- The overall population of individuals with type 1 diabetes in the US, roughly 1.25M individuals, is relatively small in relation to other conditions, such as type 2 diabetes, an estimated 30M, making it more difficult to recruit the former for traditional clinical trials.
- People with type 1 diabetes generate an outsize amount of meaningful real-world data, through the usage of devices such as CGMs, BGMs, insulin pumps, and more.
- Some of the most exciting research in medical devices is being done in the diabetes space right now, and patients are eager to see FDA approvals in hopes of better treatment options.
The use of mobile technologies and apps has also been a key driver in the growing willingness to embrace RWE and RWD. In fact, the FDA now offers an open-source framework – FDA MyStudies – for researchers and companies to actually build their own apps that are secure, compliant, and meet agency standards.
To watch this presentation and others, click here.