The FDA has issued its long-awaited plan on real-world evidence, a document that many in the industry read as the beginning of a conversation.
The FDA doesn’t see real-world evidence as a substitute for clinical trials but as a possible augmentation to them, the document, released last week, shows. It allows for the possibility of “hybrid” trials where data extracted from medical claims, electronic health records or lab or pharmacy databases could be used for “certain elements” of clinical trials.
Indeed, the agency has already accepted real-world data for some single-arm interventional trials, such as those that brought Amgen’s Blincyto (blinatumomab) to market as a treatment for Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia.
The agency “sees promise,” for instance, “in the opportunities created by pragmatic clinical trials, including broader inclusion/exclusion criteria and streamlined data collection,” noting in the framework that coming guidance “will explore pragmatic approaches to each stage of a clinical trials, including recruitment and enrollment of patients, strategies for facilitating interventions and approaches to assessing outcomes.”
But the FDA also makes clear that it has concerns about how data is gathered, whether it’s reliable and that there are some standard measures so that sponsors, sites and regulators alike can be sure they’re comparing like with like.
The move is important because it begins to home in on and define real-world evidence, which had threatened to become an empty buzzword, says James Bannon, WCG’s president of scientific and regulatory review.
“At the end of the day, real-world evidence is going to depend on the clinical trial situation, the reliability of the data and ... the FDA is now giving [the industry] a forum ... to identify whether there’s utility and acceptability of real-world data,” Bannon says. “I still think that it’s going to be a case-by-case discussion that will evolve over time. It will come back to the utility and relevance of the data at hand.”
Bannon is one of many who doubt that real-world evidence will ever replace or supplant traditional clinical trials. He sees real-world data as being particularly helpful for measuring safety (especially once a drug’s already on the market) but doesn’t believe it will be very helpful for measuring efficacy, especially of novel compounds.
“The opportunity to use real-world evidencefor very specific instances is going to take shape over time,” he says, “but I think the fundamental question is, ‘Can I substitute real-world data for some portion of an expensive clinical trial, or maybe cut down the size of an expensive trial?’”
Read the FDA framework here: https://bit.ly/2E5ZM68.