Understanding how treatments work in the “real world” can help improve and inform our health care decision-making, such as determining which treatments physicians prescribe, what treatments clinical guidelines recommend, or what treatments payers will cover. As real-world evidence (RWE) becomes more broadly used in health care, it’s important to ensure that patients understand what RWE is, determine and address the questions and concerns patients have, and determine when RWE evidence can be trusted to guide patient treatment decisions.
That’s why on July 31, the National Health Council (NHC), with support from the National Pharmaceutical Council (NPC), Johnson and Johnson, Eli Lilly and the Pharmaceutical Manufacturers of America (PhRMA), brought together representatives from the patient community for roundtable discussions about how to address these issues.
“We have a real opportunity to make sure that the patient voice is heard on real-world evidence,” said Marc Boutin, JD, NHC CEO. “Patients have a general notion of what RWE is and assume it’s already being used, but the challenge is that it’s not very well understood.”
Real-world evidence has been in use for a long time, but it has grown in importance through improved information technology and our ability to collect and analyze data. This evidence can show, for example, how treatments work among similar patients based on their age, demographics and genes; what a disease course might look like in the coming years; or what treatment options might work best in diverse patient populations. RWE is meant to complement randomized clinical trials—studies that are most commonly used for Food and Drug Administration (FDA) approval of a treatment—and add to what we already know about how a treatment works.
“Patients are comfortable with the use of RWE to improve quality, identify the side effects of drugs after they are on the market, and improve the way treatments are used,” said Eleanor Perfetto, PhD, NHC senior vice president of strategic initiatives.
“To be clear, we aren’t talking about throwing away clinical trials, but supplementing that information and making it better to inform our health decisions. We want to work with patients to figure out how we can do this best,” said Jennifer Graff, PharmD, NPC vice president of comparative effectiveness research.
During the roundtable discussions, participants explored how patients define and use RWE, communicate about it, consider their questions and concerns, and understand the kinds of tools and skills patient groups need to review and use RWE, among other topics. In particular, participants noted that patients are willing to share their data, but need to understand how it is being used. They also believe that the data should be accessible to a broad range of stakeholders who can analyze and learn from the data and use it to improve health care outcomes. Yet in order for this information to be utilized by patients and others who are making treatment decisions, the data needs to come from a trusted source and be translated into understandable language.
Having patient input on the use of RWE is especially important as the FDA takes steps to establish a program to evaluate the potential uses of RWE in regulatory decisions, such as amending a product label or monitoring the safety of a treatment. FDA, in conjunction with the Duke-Margolis Center for Health Policy, is hosting the first of several meetings on this topic beginning September 13. The National Academy of Medicine is also planning meetings on this topic.
The output from these conversations will be incorporated into an NHC white paper and could be used to help inform the appropriate use of RWE to inform health decisions. The NHC white paper will be submitted to the Duke-Margolis Center for Health Policy for use at its upcoming FDA meeting.