Clinical Trials System “Broken”?

Clinical Trials System “Broken”?

Clinical Trials System “Broken”?

FDA’s Janet Woodcock cites need for greater use of “Real World Evidence”

An official at the Food & Drug Administration (FDA) described the clinical trials system as “broken.” Speaking at a workshop at the National Academies of Sciences, Engineering, and Medicine, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, cited the need for new ways to gather and use patient data, the use of master protocols and the development of new clinical trial networks.

As reported by Zachary Brennan in Endpoints News, a publication of the Regulatory Affairs Professionals Society (RAPS) on September 20, Woodcock believes that master protocols that “look at multiple therapies in a single disease or a single treatment in multiple diseases” need to be part of the future. She also thinks that “regulation is often just aimed at the bottom 1 percent of companies.”

While Woodcock acknowledged that the 21st Century Cures Act and the new user fee laws are likely to increase FDA’s use of what is called Real World Evidence (RWE) in some situations, she said that there has been “very little historical use of real world experience in drug regulatory decisions about effectiveness.” Using RWE to determine effectiveness is “obviously most important for incentives” for industry, according to Woodcock. However, she believes that RWE functions when there is a “big effect,” but it could be harder to determine smaller effects, because “so many biases are introduced.”

FDA Commissioner Scott Gottlieb said that the FDA must work with the healthcare system to change the way clinical information is collected in order to enable greater adoption of RWE in clinical and regulatory decisions, as reported by Brennan on September 19. As he explained, "We need to close the evidence gap between the information we use to make FDA's decisions and the evidence increasingly used by the medical community, by payers and by others charged with making healthcare decisions.  Ideally, we'd like to have a system where providers have the right incentives to enter clinically relevant information into EMRs at the point of care."

Gottlieb acknowledged the present uncertainty among sponsors on the role RWE plays in regulatory decision making. He added that FDA must think of itself as an information curator, rather than an arbiter of information, "where a single truth standard is secured to a fixed orthodoxy." He explained that electronic medical records frequently are organized in ways that are designed for billing, while clinically important information might be retained in unstructured notes.

Woodcock described instances in which drug developers might use RWE, including developing biomarkers, determining expanded indications and evaluating an investigational drug in a “hybrid model” that might use RWE with randomization. She added, “Let’s make generating this evidence a lot easier and randomizing within the care system as much as we can.”

While draft guidance on RWE and a new framework on using it are supposed to be released by FDA before 2021, Woodcock did not know when it would be available. In the interest of creating "a better evidence base to make product approval decisions," FDA has promised more guidance on RWE and real world data (RWD).

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