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How Are Regulatory Agencies Reacting to the Use of Real-World Evidence?

Posted on June 15th, 2020 by in Pharma R&D

As we have discussed here previously, real-world data (RWD) and real-world evidence (RWE) offer many potential benefits in every stage of the drug discovery and development process, continuing on into post-market surveillance. With drug developers and other researchers becoming more interested in using RWD and the RWE that results from analyzing it, regulatory agencies have had to step up and work on producing guidance.

There are many challenges that accompany RWD. Its various forms (e.g. EHRs, disease registries, claims data) are not necessarily subject to the same well-established regulations and protocols as clinical data. The data might be inconsistent, unstructured, in multiple formats and it may not adhere to the principles of FAIR data. As regulatory bodies consider RWE, they must think about the quality of the data underpinning it.

The FDA offers guidance

The Food and Drug Administration (FDA) took its first big step in December 2018 by publishing a framework for their real-world evidence program, which helped to lay out some of their goals and issues of importance to be addressed, such as how RWE will be used for regulatory decision-making for drugs, considerations for observational study designs and clinical trial design, data standards for submissions, regulatory issues around the use of electronic source data and more. Actual draft guidance for submitting documents using RWD and RWE for drugs and biologics then followed in May 2019.

The EMA grapples with real-world challenges

Meanwhile in Europe, the European Medicines Agency (EMA) has also had to address the intense interest in RWD and RWE, though there are clearly concerns about whether real-world evidence can be credible evidence. In an article published in the journal Clinical Pharmacology & Therapeutics in October 2019, the EMA officials who authored it noted concerns that “acceptance of non‐RCT methodologies is tantamount to lowering the quality of evidence because these methods are prone to a myriad of undetected or undetectable biases.”

They remain optimistic about the future for RWE, but are adamant about the importance of testing and validation. “The ultimate key to achieving credibility is to start with an open but ‘agnostic’ mind‐set and submit novel methods to a fair, transparent, and prospective validation exercise,” they wrote.

The pharma response

The FDA has invited comments on its draft guidance, and the pharmaceutical industry has obliged. As reported in Policy & Medicine, a number of suggestions have come in from major players. Gilead, for instance, has proposed expanding the submissions list so that supplemental new drug applications and supplemental biologics license applications are included. Gilead has also suggested lab data be considered a source of RWD, and Novartis has suggested pharmacy claims should be considered a source for RWE. 

What is quite clear is that we are in the early stages of what will be a long process, as regulators work to formulate policy and guidance for a type of data that they are still trying to fully define. Real-world data and real-world evidence have much to offer in drug development and post-market, and it will be important to have the guidance and cooperation of our most influential regulatory bodies.

In our next piece on RWE, we will discuss the role of real-world evidence in the fight against COVID-19, including a new research project spearheaded by the FDA.

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