When the U.S. Food and Drug Administration (FDA) grants breakthrough status to a drug, that means the drug has preliminary data demonstrating its efficacy for treating a serious condition. Drugs with breakthrough therapy designation have shown clinical evidence that they can vastly improve upon current treatments for the same condition.

The FDA allows certain drugs that present promising clinical data to expedite the approval process if they can clearly show an advantage over existing therapies. The breakthrough therapy designation is reserved for the drugs that can improve the magnitude and/or the duration of the treatment’s effect (compared to currently available treatments).

Breakthrough status is different from the FDA’s fast track designation. When a drug receives fast-track status, the drug is going through an expedited approval process, but it doesn’t have the clinical data to back up its efficacy that a breakthrough therapy does.

Breakthrough Therapy Designation Timeline

Once awarded breakthrough therapy designation, a drug could undergo shortened review time and experience a much quicker time-to-market than other drugs that take the more traditional approval route. Research indicates that drugs awarded breakthrough status can shave months off review time and years off pre-market development time.