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In July 2012, a provision in the new law called the Food and Drug Administration Safety and Innovation Act, or FDASIA for short, gave FDA another powerful expedited development tool, known as the “breakthrough therapy” designation. This new designation is now helping FDA assist drug developers expedite the development of new drugs with preliminary clinical evidence that indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. Although the designation is not yet even a year old, FDA has received 62 requests to grant this new designation to products under development. The FDA has already granted the breakthrough designation to 20 potential innovative new drugs that have shown encouraging early clinical results.

To help industry better understand each tool, including when the tools can be used and the features of each, the FDA has just published an industry draft guidance titled Expedited Programs for Serious Conditions – Drugs and Biologics. Among other important information, the draft guidance describes FDA’s policies and the threshold criteria for each expedited program, defines and discusses important concepts, including serious condition, unmet medical need, and available therapy, and provides some general considerations for products utilizing an expedited program, such as manufacturing and product quality, nonclinical considerations, and clinical inspection considerations.

The FDA previously had three other programs for over 20 years: Fast Track, Accelerated Approval, and Priority Review. But breakthrough designation required early clinical data in people showing an “unprecedented effect.” The close relationship allows the company to “design collaborative, multidisciplinary development plans that hasten timelines to approval and minimize the number of patients exposed to less efficacious treatment or placebos.”

Yesterday, there was a congressional briefing on the status and efficacy of breakthrough status designations. Jay Siegel, Johnson and Johnson’s head of global regulatory affairs, spoke about the accelerated drug development process, and how it has shaved two years off of the close to 10 years that a typical drug takes to go to market. “This has had an enormous impact,” Siegel said. “There is a very pro active role on the part of the FDA. They’ll pick up the phone and call us and say ‘Have you thought of this way to do this faster? Have you thought about this problem?'” The FDA suggested some avenues the companies should take to seek approval that they hadn’t thought of, Siegel said, without being specific. The agency also helped J and J and Sunnyvale, California-based Pharmacyclics determine which trials must be done before submitting an approval application and which could be conducted after potential approval, he said.

Vertex Pharmaceuticals Inc., based in Cambridge, Massachusetts, was the first to receive a breakthrough designation for expanded use of its cystic fibrosis drug Kalydeco.