With accelerated approval under the microscope, experts—including FDA nominee Dr. Robert Califf and BIO’s Dr. Michelle McMurry-Heath—discussed yesterday how we can protect accelerated approval and get the data we need to provide better health outcomes.
So, what does FDA nominee Dr. Robert Califf think about accelerated approval? “I think there was wisdom in what’s been done with accelerated approval,” especially when there’s no effective treatment—but we “need a better system” to evaluate drugs after they come to market, he said during yesterday’s CERSI Innovations in Regulatory Science Summit on the margins of the virtual J.P. Morgan HealthCare Conference.
But: There’s been a “failure to produce confirmatory evidence quickly and in a way that really gives us the information we need…to decide which [drugs] are most effective and in which order,” as well as a lack of coordination between agencies, he continued.
Accelerated approval is “one of the most important innovations in regulatory policy in a while,” said Dr. Hal Barron, Chief Scientific Officer at GlaxoSmithKline—but there’s a need for “more rigor” and speed around confirmatory trials, more ex-U.S. data, and greater focus on areas in addition to oncology where there’s unmet need, such as lupus.
“The overall success…gets lost in recent controversies,” said BIO President and CEO Dr. Michelle McMurry-Heath, noting 8% of drugs approved through the accelerated pathway are withdrawn for safety or efficacy, compared to 5% approved through the usual process. “It’s not that the bar is so much lower than overall standards that we are presenting undue risk.”
The speakers agreed: a more holistic, centralized system for collecting data is needed—and legislation could help. All stakeholders—CMS, FDA, payors, industry—need to come together to figure out how to get data “faster, cheaper, and with less of a burden on patients,” added Dr. Michelle.
The bottom line: “This is not the time to retreat on accelerated approval,” said Dr. Michelle. “This is the time to double down but also modernize evidence collection techniques,” to capture evidence as quickly and accurately as possible—for accelerated approval or otherwise.