New Issue Brief: FDA Should Expand Use of Real-World Evidence in Drug Approvals

NEW YORK, NY — The newly confirmed commissioner of the Food and Drug Administration (FDA), Dr. Marty Makary, has been an outspoken advocate for increasing data-driven decision-making. The FDA reviews applications to market new pharmaceuticals and oversees clinical trials designed and sponsored by new drug developers. Unfortunately, the high and rising costs of the clinical trials required by FDA deters drug development and approval, hindering patients’ access to new therapies. In a new Manhattan Institute issue brief, senior fellow and former FDA regulatory advisor, Randall Lutter, highlights how leveraging real-world evidence could streamline drug development, lower costs, and expand patient access to new treatments.  

Lutter points to the success of the U.K.’s RECOVERY trial, which used real-world data to rapidly evaluate COVID-19 treatments at just a fraction of the cost of traditional trials. Unlike conventional studies, pragmatic randomized trials like RECOVERY use information gathered from routine clinical conditions, rather than relying on the controlled experimental environment alone. RECOVERY illustrates how pragmatic randomized trials can quickly and cheaply feed into regulatory decisions and improve public health. However, the U.S. has made little progress in implementing real-world evidence when appropriate despite a congressional directive in the 2016 21st Century Cures Act. 

In addition to reducing drug costs and delays, the adoption of more real-world evidence would benefit those suffering with rare diseases who are traditionally excluded from clinical trials. For these reasons, Lutter urges the FDA to adopt quantitative goals for incorporating real-world evidence into the approval process—recommending that by 2030, half of all supplemental drug applications include real-world evidence. If the FDA fails to act, Congress should require reforms.  

Click here to read the full issue brief.