According to Richard Pazdur, the FDA's head of hematology and oncology products, a "breakthrough" drug offers "a significant advance over what is out there." Specifically, the designation is intended for "transformative therapies that are a marked improvement, where there is no therapy, or a drug has a significant advance over what is out there," he told the Wall Street Journal.
This week, Pazdur discussed FDA's approval process at the American Society of Clinical Oncology's annual meeting, hoping to explain features of the program that have not been "well expressed."
FDA officials like Pazdur designate drugs as "breakthrough" based on clinical evidence that the drug could offer providers a considerable improvement in treating serious or life-threatening diseases. As such, even drugs that have completed phase 2 midstage clinical trials can qualify.
The "breakthrough" designation is really all about a breakthrough in communications. It triggers more frequent communication between the drugmaker and FDA staff, Pazdur says. This allows agency officials to recommend changes to the development plan that can save time and quickly address any manufacturing or supply issues that would otherwise delay efforts to bring the drug to market.
Ideally, the heightened communication during the development stages will speed up the review process once a formal drug application is ready.
"It's a mind changer both for industry and FDA," Pazdur says. He adds that drugmakers that have received the designation of a product seem pleased with the increased communication as well.
Johnson & Johnson's Peter Lebowitz says the breakthrough status bestowed on experimental cancer drug ibrutinib likely will have a "big impact" on how quickly the drugmaker will be able to move on the drug.
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