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Back to School

Standards of evidence for accelerated approval: a BioCentury Back to School podcast

The complexities of creating an approvable data package for accelerated approval

BioCentury’s editors analyze the complexities of creating an approvable data package for accelerated approval and assess emerging surrogate endpoints.

By Jeff Cranmer, EXecutive Editor
August 31, 2021 1:12 PM UTC

In the second of four podcasts discussing this year’s Back to School package, BioCentury’s editors discuss the complexities of creating an approvable data package for accelerated approval as well as emerging surrogate endpoints.

On the first Back to School podcast, BioCentury’s editors assessed the opportunities and challenges of accelerated approval and what the future looks like for the pathway.

In Tuesday’s discussion, the editors home in on the standards of evidence required for an accelerated or conditional approval.

“We’ve looked at this from the angle that accelerated and conditional approval pathways should actually be a springboard for innovation and in the stretch vision that would mean you need to create many novel surrogate endpoints to enable the use of the pathways in more indications and more diseases,” says BioCentury Editor in Chief Simone Fishburn.

In the U.S., FDA can grant accelerated approval, if a therapy meets a surrogate endpoint that is deemed “reasonably likely” to predict clinical benefit.

“The problem is there’s no real standard for what ‘reasonably likely’ means,” Fishburn says.

The goal should be a “rational standard that’s reliable and applied routinely,” says Executive Editor Selina Koch, adding that a regulatory agency could use different standards for different diseases or mechanisms of action.  

A solution, she says, could be putting a number behind “reasonably likely” that’s based on a regulatory agency’s risk tolerance. For example, an agency could say “reasonably likely” means a 70% chance to predict clinical benefit.

The near-term breakthroughs in surrogate endpoints are coming from measures already used in clinical practice in fields such as nephrology, cardiovascular disease and blood cancers, says Senior Editor Karen Tkach Tuzman. 

In the longer term, Tkach Tuzman believes the pace of development in surrogate endpoints will continue to quicken.

“I think we’ll see industrialization of omics technologies bringing a lot richer data to the table on this and allowing exploration of potential surrogates that aren’t currently under the microscope of what clinicians look at every day,” Tkach Tuzman says.

BioCentury’s podcast team returns Wednesday with the third installment of its Back to School series, which focuses on the second stage of the accelerated approval process: generating confirmatory evidence.