Timing and data are critical to winning breakthrough therapy designation for rare disease drugs

Many companies developing rare disease drugs have one urgent regulatory question: "How—and how soon—can we get FDA Breakthrough Therapy designation (BTD)?"

Because BTD targets drugs intended for serious conditions with unmet medical needs, it is frequently central to rare disease drug development. However, obtaining BTD can be challenging; success depends on timing and the data supporting the application. 

Companies know BTD confers valuable benefits, including increased FDA interactions and intensive guidance that could expedite drug development. They also know it often provides external validation to a company’s investors that an investigational product has—based on preliminary clinical data—shown significant potential to the FDA and for patients.

BTD has become a core component of the regulatory strategy for rare disease drugs. A Parexel analysis of FDA novel drug approvals from 2017-2022 found that orphan drugs comprised 53 percent (164/307) of all approvals and 72 percent (86/119) of BTD approvals. Additionally, 93 percent of BTD orphan products received an expedited review under Priority Review.