72% of Breakthrough Drugs Win Approval: Jefferies Analysis
Nearly three out of four drug candidates that received an FDA breakthrough therapy designation (BTD) over the past decade eventually gained approval, according to a new analysis from investment firm Jefferies reported by BioSpace. The report examined 599 breakthrough designations issued between 2013 and year-to-date 2025, finding that 72% of those therapies have been approved, while an additional 13% remain under FDA review. The analysis, which used data from FDA, Friends of Cancer Research, and Biomedtracker, suggests that the agency’s expedited programs continue to function effectively amid ongoing leadership turnover and policy changes, though some observers cautioned against overinterpreting the findings.
As BioSpace reported, oncology therapies accounted for the majority of approvals—176 of the 223 therapies that secured marketing clearance after receiving breakthrough status. Cancer drugs represented 46% of all BTDs granted between 2013 and 2025, followed by infectious disease (11%), metabolic (8%), and neurological (7%) products.
Rare disease programs, including those within oncology, were the most common recipients overall, representing 383 of the 599 designations issued during the period. Among candidates that ultimately failed, 43% were discontinued during clinical trials. Rare disease drugs fared better than average, with only 13% of those discontinued.
Regulatory policy analyst Steven Grossman, who writes the FDA Matters blog, told BioSpace that the data could reflect both FDA’s ability to identify promising candidates and a degree of institutional momentum once a drug receives the designation. “On its face, you can’t tell whether the learning is that FDA has a keen ability to identify the most promising drugs early in the process — or whether FDA pushes those drugs forward in a form of confirmation bias,” Grossman said. “It could be some of both.”
Jefferies’ analysts maintained a positive outlook on the agency’s regulatory posture, despite recent leadership changes and staff upheaval. In a note to investors, they said FDA’s rate of novel drug approvals and advisory committee activity suggests that the regulatory environment “has not worsened” compared to prior years. “We remain positive on the state of the FDA,” the analysts wrote, citing continued issuance of complete response letters and steady decision-making across therapeutic areas as signs of operational continuity.