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Back to School

Accelerated approval’s big picture: a BioCentury Back to School podcast

BioCentury’s editors assess opportunities, challenges for expedited approval pathway

BioCentury’s editors assess the challenges and opportunities for the expedited approval pathway around the world.

August 30, 2021 1:10 PM UTC

BioCentury’s 2021 Back to School focuses on accelerated approval, the pathway that helps drugs get to patients faster for severe diseases with no alternatives using less data than normal. In the first of four podcasts discussing this year’s Back to School package, BioCentury’s editors assess the opportunities and challenges of accelerated approval and what the future looks like for the pathway.

EMA and FDA introduced expedited pathways to get drugs to patients quicker in the early 1990s, with Japan, China and the U.K. following suit more recently. 

“All of these agencies are signed up to the principle of this idea of conditional approval, getting drugs to patients faster,” says Editor in Chief Simone Fishburn. “The other way of looking at it is: It’s been an underperformer. It’s been fantastic for cancer; it’s done little for almost any other disease.”

Executive Editor Selina Koch digs into the numbers to debunk an existing narrative that accelerated approval isn’t working. She says the data show that the pathway is getting used increasingly over time.

She says that the status of “the vast majority” of drugs receiving accelerated approval before 2017 has been resolved: 76% have received full approval, and 11% have been withdrawn. That leaves 13% pending.

A significant amount of credit for the success of FDA’s accelerated pathway should go to the agency’s Richard Pazdur, Washington Editor Steve Usdin says.

“He and his team have done a tremendous amount of work to make the accelerated approval pathway work for cancer drugs,” he adds. “It’s saved and extended and improved the quality of lives of hundreds of thousands, maybe millions, of people in the United States.”

At the same time, Usdin notes, Pazdur is very open to the fact that the pathway could be improved.

“He thinks that there’s a really mistaken idea about the way that people conceive of accelerated approval. One of those mistakes, he thinks, is an overemphasis on surrogate endpoints,” says Usdin. “He called for banishing the term surrogate endpoints, at least for cancer. The vast majority of accelerated approvals in cancer are based on what he considers to be interim clinical endpoints.”

Usdin adds that there’s a dispute within FDA itself over how to use validated surrogate endpoints.

Inconsistency can make drug development extremely difficult for biopharmas, says Fishburn, and looking to Congress to sort the issue out could risk going backwards.

“Back to School is going to lay out a vision for what accelerated approvals and conditional approvals around the world could do for drug development, not rein it in, but actually expand it,” Fishburn says.

BioCentury’s podcast team returns Tuesday with the second installment of its Back to School series, which focuses on the first stage of the accelerated approval pathway: creating an approvable data package. 

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