Patients on RWE: WIIFM? (What’s In It for Me?)

Patient-Community Perspectives on Real-World Evidence: Enhancing Engagement, Understanding, and Trust

Treatments can work differently in different types of patients, based on factors like age, gender, other conditions or medications, or socioeconomic differences. That’s why expanding use of real-world evidence (RWE), which captures how treatments work in patients in real-world care settings, has increasingly been the subject of discussion among clinicians, regulators and payers. But one group of stakeholders has been less vocal on RWE: the patient community.

Since patients – and their interactions with the health care system – are the source of real-world data and the basis for RWE, what does the patient community think about RWE’s potential impact on treatment, regulatory or coverage decision-making? 

New research, conducted in collaboration between the National Pharmaceutical Council and the National Health Council and published in the peer review journal, The Patient - Patient-Centered Outcomes Research, sought to explore the question by convening representatives from patient organizations to assess what patients currently know about RWE, their level of trust in RWE and interest in using it in decision-making.

During the day-long roundtable, key themes emerged. While most participants were initially unaware of RWE and its actual or potential uses, the patient community sees great potential in using RWE. “The term real-world evidence has not yet made it into the everyday vocabulary of patient organizations, but they have a general notion of what RWE is and are surprised that it isn’t already deeply embedded in health care decision-making,” said Eleanor Perfetto, PhD, MS, Executive Vice President, Strategic Initiatives, National Health Council.  “What we heard loud and clear is that patients recognize the ability of  RWE to answer the question that matters most to them – ‘How does this treatment work in patients who look like me?’”

Though the patient community agreed that RWE should inform decision-making, they flagged key areas of need:

  • Awareness and capacity: Patients need deeper awareness of RWE and its use, common definitions, skills and tools to support uptake of RWE and partnerships with scientists who can help identify high-quality evidence.
  • Ensuring optimal use of RWE: Patients think RWE should be used in decision-making but look to the clinician community to champion RWE as part of the evidence for decision-making.  Any communications with patients need to be balanced, empowering and trustworthy.
  • Data sources and ownership: Patients also want clarity around privacy and ownership of data from patient encounters with the health system and agreed that patient-generated data should be included in the development of RWE.  Patient-provided information is an important part of RWE and patients consider the information they provide is more “real” than from sources such as claims data and electronic health records.

The use of RWE in decision-making is expected to build traction among clinicians and payers. The framework for using RWE issued recently by the U.S. Food and Drug Administration was an important step forward and is likely to further entrench use of RWE for regulatory decision-making.

“The roundtable and this study underscore the importance of keeping patients front and center, to  ensure RWE answers their most pressing questions, high-quality RWE is easy to spot, and that findings from RWE promote patient understanding, trust, and optimal treatment decisions,” said Jennifer Graff, PharmD, NPC’s vice president of comparative effectiveness research. “The patient community is ready to be part of the dialogue because they know if RWE is well-developed and well-applied in decision-making, there’s something in it for them. Knowing what works for most people is good. But knowing what’s likely to work best for you, based on your individual factors, can make a world of difference.”