Rapid growth in the availability of data—and the ability to review and manipulate datathrough emerging technologies like natural language processing and machine learning—have dramatically changed the landscape in terms of how healthcare and Medical Affairs professionals gather and consider scientific evidence to inform decisions. Augmenting the use of traditional randomized clinical trial (RCT), there is an increasing interest in the potential use of real world evidence (RWE) that raises both opportunities and challenges.

The Institute for Clinical and Economic Review (ICER) published a report in March outlining a conceptual framework for developing a common approach to using RWE to make treatment decisions and reporting on benefits and potential drawbacks of this trend.

Key Benefits

There are some significant benefits associated with the use of RWE, most significantly, the potential to shorten the time frame for making new treatments or new indications available —and associated positive impact on patient outcomes. In addition, RWE takes the evaluation of pharmaceutical efficacy and implications out of the strict clinical setting and into the lives of real people, where actual impacts can be observed. This, combined with the ability of modern technology to draw insights and aggregate data from a wide range of sources, including Electronic Health Records (EHRs), can help increase the reliability and validity of the information being gathered.

Main Drawbacks

As ICER points out, EHRs “are often incomplete, incompatible with those from other institutions, or hard to obtain given privacy concerns.” In addition, RWE gleaned through observation studies, says ICER, can be impacted by “biases introduced by unknown confounders.”

The report also mentioned additional areas of concern:

• While RWE is used quite broadly already in Europe, its adoption is slower in the U.S., driven to a large degree by the sharing of data—or, more precisely, the lack of sharing due to legal restrictions
• Healthcare professionals are concerned that because this is an area that is evolving so rapidly, standards don’t yet exist and may not be able to keep up with the pace of change
• “Innocent misinterpretation” may occur when those who don’t know the source of the data weigh in with their perspectives. Claims data (procedure codes and pharmacy data, for instance) is generally reliable; diagnosis codes, not part of the claim, may be less so. As ICER points out, when this data are interpreted by less experienced players, reliability and validity may become questionable.

The challenges associated with the use of RWE, according to ICER’s report include: bias and confounding, incomplete data, data mining, lack of access to data, lack of universally accepted methodological standards, lack of investigator expertise, and obsolete evidence hierarchies.

However, they also point to measures that can be taken to address these limitations and challenges including:

• Increasing the quality and credibility of observational RWE studies
• Establishing effective governance arrangements that clarify how much data can be shared
• Focusing RWE efforts on the development of pragmatic clinical trials

Fulfilling the Promise of RWE

Real-world evidence has caught the attention of healthcare players across the continuum from scientists and researchers, to drug and device developers, payers, clinicians, regulators—and patients.

Milind Kamkolkar, the first enterprise chief data officer in a major pharmaceutical company, Sanofi, told PharmaTimes:“We’re moving into an era of real-world evidence, and it’s going to be very interesting when we start moving from clinical endpoint to physiological endpoints to behavioral endpoints. All of those are an aggregation of different data that come from all sorts of things. You can literally store anything now.”

Senior research fellow at Tufts CSDD and lead investigator on a study conducted by Tufts about the promise of RWE, Mary Jo Lamberti, PhD, also is optimistic : “While the cost and effort to integrate data sources, and then convincing regulators and payers of the validity of that evidence, pose hurdles to expanded use of real world evidence, developers are confident that emerging technologies will help them address those issues.”

RWE is here to stay, and with the development of standards, it will gain in importance.

In Understanding the Context, Electing the Standards: A Framework for RWE, a companion paper to its report, ICER concludes: “Guided by a shared understanding of the contextual considerations, and supported by process principles of transparency, collaboration, and communication, RWE can be developed and applied as a vital complement to other evidence in improving the care for patients in the US healthcare system.”