Regulatory Focus™ > News Articles > Woodcock Lauds First Submission of 'Breakthrough' Product

Woodcock Lauds First Submission of 'Breakthrough' Product

Posted 04 December 2012 | By Alexander Gaffney, RAC

The US Food and Drug Administration (FDA) has already accepted its first product under its new "Breakthrough" therapies designation, and more are soon to be on the way, a high-ranking agency official said recently.

In a 3 December 2012 announcement posted on FDA's Voice blog, Janet Woodcock, director of FDA's Center for Drug Evaluation and Research (CDER), sought to explain the law, calling it an exciting development for an agency that has long searched for effective ways to get products to the neediest patients expeditiously.

The breakthrough therapy pathway, Woodcock explained, was established by the FDA Safety and Innovation Act (FDASIA) of 2012. Passed in July, FDASIA defines eligibility for the pathway as a product that is "intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints."

"In other words," Woodcock writes, "a breakthrough drug is one that may offer important new benefits for patients with serious or life-threatening disease who are especially in need of new safe and effective treatments."

And if speed is the goal, patients may be in luck, as at least one company has already received a breakthrough product designation. "It likely won't be long before we have more," added Woodcock. "Several other drug developers have already made inquiries and there is lots of interest in the pharmaceutical industry in taking advantage of this new development tool."

The new pathway will serve as a complement to three existing pathways, Woodcock said: the Fast Track, Priority Review and Accelerated Approval pathways. Fast Track is intended to meet the needs of serious diseases for which there are few available options or for which the new product would have a meaningful impact on a metric such as survival or disease progression. The Fast Track designation gives sponsors access to FDA review staff at more frequent and earlier intervals, reducing unnecessary delays.

Fast Track products are also often given Priority Review status, which aims to get products through FDA's review process within six months, though the product must meet the same standards for efficacy and safety as non-Priority Review status drugs. Accelerated Approval, meanwhile, slightly alters the standards by which FDA approves drug products meant for serious, unmet medical needs by introducing the concept of surrogate endpoints. A drug for cancer, for example, could be approval based on surrogate endpoints instead of final survivability date based on the assumption that the drug's ability to shrink a tumor would improve life expectancy.

Until the new breakthrough products designation came along, some drug products could be ineligible for FDA's priority pathways, particularly when their efficacy is based on being used in combination with another product. The pathway also affords sponsors the ability to expedite the collection of evidence during clinical trials, giving them access to FDA reviewers to make sure the trial design is as efficient as possible.

"We're delighted now to have another tool to help expedite the development and approval of products with the 'breakthrough' designation," Woodcock wrote. "We'll continue to use our existing tools and the new 'breakthrough' authority to make our expedited drug development process even more effective, with the ultimate goal of benefiting patients with unmet medical needs."

An FDA guidance document on the breakthrough therapies pathway is expected by February 2014.

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