Regulators, Industry Debate Meaning of Real-World Evidence in Trials

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There’s no shortage of buzzwords in the clinical trial industry. Patient centricity and virtual trials have been flavors of the month for some time now, for example. But for pure staying power, real-world evidence (RWE) has a pretty impressive track record, too.

Unfortunately, as evidenced by a slew of recent comment letters, regulators and industry leaders are still struggling to harmonize their own definition of RWE.

The U.S. Food and Drug Administration (FDA) planted a flag in May with draft guidance for drugs and biologics (and with final guidance for medical devices in 2017) defining RWE as clinical evidence regarding the usage and likely benefits and/or risks of using a medical product culled from an analysis of real-world data (RWD).

RWD and RWE “are playing an increasing role in healthcare decisions,” FDA said in a filing earlier this year. It notes:

  • FDA uses RWD and RWE to monitor postmarket safety and adverse events and to make regulatory decisions.
  • The healthcare community is using these data to support coverage decisions and to develop guidelines and decision support tools for use in clinical practice.
  • Medical product developers are using RWD and RWE to support clinical trial designs (e.g., large simple trials, pragmatic clinical trials) and observational studies to generate innovative, new treatment approaches.

While the agency made a bold attempt to define the terms, it’s by no means gotten the last word here. For example, Novartis and Gilead, among others, have weighed in recently to say that the agency’s work has been too narrow in scope.

Further, the Association of Clinical Research Organizations (ACRO) has called for the creation of standardized summary assessments of the quality of the actual data populating RWD submissions. “We feel this will enhance the transparency, replicability, and evaluability of real-world evidence, especially when evaluating RWE in the context of multiple submissions,” the trade group said in a July 8 comment letter.

ACRO also called on the FDA to expand its two-page glossary of terms by adding definitions of “natural history studies” and “product and/or disease registry data.”

Drilling down more deeply, Gilead and Novartis’ comments are generally granular in nature (e.g., suggested word changes or clarifications of examples).

Author: Michael Causey