Regulatory & Documentation
Breakthrough Therapy Designation
Breakthrough Therapy Designation is an FDA program that expedites the development and review of drugs intended to treat serious or life-threatening conditions when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over existing therapies on a clinically significant endpoint.
Established by the 2012 FDASIA legislation, Breakthrough Designation provides intensive FDA guidance, organizational commitment from senior managers, and eligibility for rolling review and Priority Review. It can shorten development timelines by 1; 2 years.
Other expedited programs include Fast Track (earlier-stage), Priority Review (NDA/BLA review timeline), and Accelerated Approval (surrogate endpoint pathway). They are not mutually exclusive.
Related terms
Accelerated Approval
Accelerated Approval is an FDA pathway that allows earlier approval of drugs treating serious conditions and filling unmet medical need on the basis of a surrogate endpoint that is reasonably likely to predict clinical benefit, conditional on confirmatory post-marketing trials.
IND Application (Investigational New Drug)
An Investigational New Drug (IND) application is the submission a sponsor files with the FDA to obtain authorization to ship an unapproved drug across state lines and to administer it to humans in a clinical trial.
Rare Disease Clinical Trial
A rare disease clinical trial is a study evaluating a therapy for a condition affecting fewer than 200,000 people in the U.S. (per the Orphan Drug Act) or fewer than 5 in 10,000 in the EU, characterized by small patient populations, geographic dispersion, and reliance on natural history data and external controls.
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