“Breakthrough therapy” status is much sought after, but there is confusion about its definition and impact

The experimental cancer drug ibrutinib has wowed in clinical trials, beating deadly blood cancers without the painful side effects of currently approved therapies.

And it has raced through development and regulatory hurdles, in part thanks to a US program to accelerate the development of particularly promising drugs, says its developer Pharmacyclics, based in Sunnyvale, California.

The US Food and Drug Administration (FDA) launched the ‘breakthrough therapy’ designation in 2012, and the label has been eagerly embraced by the pharmaceutical industry. Recent months have seen a steady stream of drugs being submitted for review. For some firms — particularly young ones — the designation can bring an extra boost of cash by raising investor confidence.

But for all the fanfare, the industry is also watching closely to see exactly what benefits can be gained by having a drug reviewed through this route. “It’s like winning a beauty pageant,” says Timothy Coté, a former director of the FDA’s Office of Orphan Products Development who now runs a consultancy called Coté Orphan Consulting in Silver Spring, Maryland. “It doesn’t have specific tangible outcomes, but it does appear to have enlivened the community.”

The breakthrough therapy designation was created by the FDA Safety and Innovation Act, a law that requires the agency to fast-track promising drugs for serious or life-threatening conditions. The FDA aims to do this by meeting early and often with developers, as well as working with them to design clinical trials that deliver the needed data quickly and efficiently.

The industry leapt on the opportunity, so far submitting 99 applications for the designation. But the flurry of applications may partly be a product of confusion, says Coté: the FDA has avoided laying out detailed descriptions of what constitutes a breakthrough, and some companies are unsure of the criteria. “Most biotech chief executives with something in the clinic think that they’re already there,” Coté says — but 47 of the applications submitted in the past year have been denied. In most cases the denials are due to insufficient clinical data, the FDA says.

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