Understanding Real-World Evidence

NPC explores the benefits and challenges with the use of real-world evidence in this first article in a series on this topic.

Amazon suggests products you might like based on your past purchases or those of buyers similar to you. Netflix recommends movies you might like, while TripAdvisor puts forward appealing travel destinations. But when it comes to health care, how can we know which treatments will work best for us? Our past medical history or that of patients like us might yield clues, but how do we get that information? That information, collected from everyday health care experiences, is known as real-world data and is growing in availability. However, advanced analytics and methods are needed to make sense of this data to create real-world evidence, or RWE. Many health care decisions are made based on randomized controlled trials, which take place in highly controlled settings and may not reflect the experience of the average patient. In other scenarios, information from randomized controlled trials may not exist. But as we seek to transition to a learning health care system, it’s critical to take lessons everywhere we can find them—in communities and clinics, in patients’ homes and nursing homes, in laboratories and living rooms.

Fortunately, more people are recognizing that valuable lessons can be learned from everyday patients and care settings to inform health care. Through such channels as observational studies and patient registries, we can discover how treatments work for patients similar to us in age, demographics and genes. We can learn about what our disease course might look like in coming years, and we can learn what treatment options might work best for us based on our own past responses. RWE aids in drug and device safety; it is commonly used to uncover potential side effects that occur with broader treatment use.

There are signs that many groups are recognizing the benefits of RWE.

  • The FDA is considering how RWE might be used in regulatory decisions about drugs and devices.
  • Health insurance companies are using RWE to design care management programs for patients and sophisticated agreements with health care providers based on outcomes and value.
  • RWE is being used to inform Clinical Practice Guidelines as they are being developed for the “typical patient.”
  • Patients are contributing RWE and are interested in understanding when it can be used to inform treatment options in consultation with their health care providers.

RWE is not without its critics. Some academics and groups have expressed concern that registries, observational studies and therapeutic use “are not as rigorous or as valid as randomized trials in assessing efficacy.” However, there are methods to ensure the validity of RWE, including using “best available information” in addition to “best evidence,” which results from clinical trials but may be too long in coming to help some patients.

It can be difficult to differentiate fact from hype. Therefore, NPC is pleased to offer this six-part blog series on real-world evidence, to provide health care payers, providers and their patients with actionable information to make informed decisions about the sources and uses of RWE.

In the upcoming five posts, we will explore:

  • What is RWE?
  • When and how has RWE been used?
  • Separating fact from fiction and knowing what data sources and studies to trust
  • How patients can use RWE
  • The future of RWE and how we can accelerate the use of high-quality information.

We’ll be highlighting best practices from the field, learning from leaders developing and using data systems to improve patient care. Because patients have characteristics and treatment experiences that often differ from the controlled environment of clinical studies, understanding how treatments work in the real world is an important and complex task to guide clinical practice. We encourage you to share your stories with us. If you know an expert we should speak with, contact us at info@npcnow.org.