Real world evidence: Not just “big data”

Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today's challenges and opportunities. The Catalyst welcomes guest contributors including patients, stakeholders, innovators and others to share their perspectives and point of view. Like in our Conversations series, views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today. 

To continue the dialogue about Real World Evidence and what it can mean for new therapy options for patients, we're pleased to host a guest blog from Tom Hubbard, vice president of policy research, The Network for Excellence in Health Innovation.

Over the last several years, interest in using data from the “real world” has blossomed into its own, movement for the use of Real World Evidence (RWE). Definitions of what constitutes RWE vary but in general it entails data collection and analysis about the use, benefits and risks of medicines that fall outside the bounds of the classic Randomized Clinical Trial, including use of data that is routinely collected in the daily practice of medicine, and thus reflective of the heterogeneous patients seen in real world practice settings.

Experts from many sectors, from patient advocacy groups to clinical leaders, share a sense that appropriate use of RWE can be a very important, potentially transformative force in health care decision making in the years ahead, including on the approval, adoption and safe and effective use of therapies.For example, it was retrospective analysis of 40 years of use of beta blockers among heart attack patients that created the basis for using beta blockers more broadly to control hypertension – a development that has played a part in the continuing reduction of cardiovascular disease and strokes. In recent months, RWE has yielded the suggestion that metformin, the widely used diabetes medication, may have cancer-fighting properties.  A health care system that relied solely on randomized clinical trials might have taken years to generate such insights, if it generated them at all.

Real World Evidence holds promise as a transformative force but there’s no guarantee it will be used in a manner that leads to improved health care unless key stakeholders share common standards, principles and approaches to the collection, analysis, and replicability of RWE, and regulations allow evidence based on these data to be used and communicated appropriately with all decision makers, including health care professionals to enhance patient care.