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The program's popularity is perhaps understandable. A new drug receives the designation if there is 'preliminary' clinical evidence indicating a substantial improvement over existing therapies for that life-threatening condition. For the chosen candidates, the promise from the US Food and Drug Administration (FDA) is “intensive guidance on efficient drug development” as early as phase 1, plus senior manager “commitment” and all the attractive features of FDA's existing 'fast track' program, such as rolling submissions, and expedited development and review. “We're led to believe that it's fast track on steroids,” sums up Patrick McEnany, chairman and CEO of Coral Gables, Florida–based Catalyst Pharmaceuticals, whose orphan treatment for Lambert-Eaton myasthenic syndrome (LEMS) received the breakthrough stamp in August 2013.
The breakthrough designation, like fast track, accelerated approval and priority review before it (Table 1), underscores FDA's drive to make available treatments that can have a great impact on severe diseases as fast as possible. The four programs are similar in overall intent and aren't mutually exclusive. But there's little doubt that the breakthrough label tops the ranking in terms of where FDA resources are being prioritized. “The designation can lead to cleared calendars, and it also means that the senior management of the FDA division becomes involved, not just the reviewers who serve on the FDA's front lines,” said Richard Pazdur, director of FDA's Office of Oncology and Hematology Products in a June 2013 interview with Forbes' Matthew Herper. “A breakthrough designation means there are more times a company can expect to be able to pick up the phone and get an answer,” Pazdur continued. That's great news, especially for smaller biotechs whose fate may depend on just one or two product candidates. Plus, because most breakthrough candidates are likely to be orphans, there are no FDA User Fees, even when a new drug application (NDA) or biologics license application (BLA) is filed.