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Back to School

Delivering the evidence for accelerated approval: a BioCentury Back to School podcast

How postmarket trials to support accelerated approval could become launchpad for innovation

How postmarket trials to support accelerated approval could become a launchpad for innovation.

September 1, 2021 1:20 PM UTC

Postmarket trials to support a therapy’s accelerated approval could become a launchpad for innovation rather than a rote exercise. In the third of four podcasts discussing this year’s Back to School package, BioCentury’s editors discuss how continuing biomarker studies after an expedited approval would have clear benefits for patients, physicians and payers, and dig into the iterations on FDA’s accelerated approval pathway by regulatory agencies in Europe, China and Japan.

On Tuesday, the editors delved into the complexities of creating approvable data packages for expedited pathways.

The latest BioCentury Back to School podcast posits that stretching the vision of  what expedited approvals achieve will require a rethink away from the standard view of confirmatory trials as little more than a box to check, arguing it could be much more.

Senior Editor Lauren Martz believes biomarker-driven drug development, which is increasingly used in clinical trials, could be used more frequently in postmarket studies, in particular to refine the patient population.

“This is a part of the pathway where you could really turn the thinking on its head right now,” says Editor in Chief Simone Fishburn.

She argues that the trials could become a “springboard for innovation” rather than an exercise in simply confirming the data that supported accelerated approval.

For example, Fishburn says, “Rather than thinking of them as confirming that tumor shrinkage actually translates to longer survival, you could use them to identify which patients are going to receive the benefit and avoid giving the drug to patients that won’t really get the benefit, but would still obviously be exposed to the side effects.”

Associate Editor Stephen Hansen compares FDA’s expedited pathway with the  EMA scheme that followed it, and the more recently created pathways in China and Japan.

One key difference between Europe and the U.S. is that EMA places more stringent requirements on sponsors to maintain their postmarket commitments.

EMA also has created tandem pathways — the exceptional circumstances pathway and conditional marketing authorization — which differ primarily on the postmarket requirements. 

Hansen also sees EMA making the pathways more efficient over time and the agency changing its attitude toward risk.

Hansen says that China and Japan have built upon the pathways developed by regulators in the West, with a critical difference being that the Asian systems place time limits on delivering supporting data.

In China, Hansen says, NMPA agrees with companies on a time period for which the conditional approval is valid. In Japan, a conditional approval pathway for regenerative therapies sets a maximum sever-year window for sponsors to generate data.

BioCentury’s podcast team returns Thursday with the fourth and final installment of its Back to School series, which focuses on the third stage of the accelerated approval process: modifying accelerated approval decisions.

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