FDA Authorization of Therapeutic Devices Under the Breakthrough Devices Program

Importance Congress established the US Food and Drug Administration (FDA) Breakthrough Devices Program (BDP) in 2016 to promote technological innovation and facilitate patient access. However, the clinical and regulatory characteristics of FDA-designated breakthrough devices have not been assessed. Objective To characterize technological novelty, FDA review times, and premarket and postmarket evidence requirements for therapeutic breakthrough-designated devices. Design and Setting This cross-sectional study analyzed publicly available FDA data for all therapeutic breakthrough-designated devices receiving FDA authorization between January 1, 2016, and September 30, 2024. Main Outcomes and Measures (1) Device novelty (first of a kind and first commercialized launch); (2) FDA premarket review times (compared with Medical Device User Fee Authorization [MDUFA] goals); (3) premarket pivotal study characteristics; and (4) postmarket regulatory experience (FDA-required studies and recalls). Results Between January 1, 2016, and September 30, 2024, the FDA granted breakthrough designations to 1041 devices, 127 of which have been authorized (12.2%), including 75 therapeutic devices (59.1%). Thirty-four of 75 therapeutic devices (45.3%) were classified as high-risk; within this subgroup, 23 were first of a kind (67.6%) and 14 were first commercialized in the US (41.4%). The mean (SD) FDA review time was 225.8 (107.6) days for all 75 devices and 243.3 (98.4) days for the 34 high-risk devices. MDUFA goals for FDA review times applied to 30 high-risk devices, of which 22 (73.3%) were reviewed before statutory target timeframes. Most therapeutic devices (89.3% [67 of 75]) underwent premarket clinical testing, totaling 75 pivotal studies evaluating 81 primary effectiveness end points. Forty end points (49.4%) incorporated surrogate measures of effectiveness, and 15 (18.5%) lacked statistical testing. End points had short follow-up durations (median, 6 months [IQR, 3.8-12 months] for implantable devices). The FDA required 46 postmarket studies for 30 breakthrough-designated devices (40.0%); 19 studies reported delays (41.3%). The mean (SD) US market life to date was 2.4 (1.9) years, during which 12.0% of devices (9 of 75) were recalled, including 1 class I (1.3%) (ie, highest severity) recall. Conclusions and Relevance This study found that most therapeutic breakthrough–designated devices are novel and reviewed by the FDA before statutory target timeframes. However, uncertainty about benefits and risks for some devices raises questions whether BDP is consistently fulfilling program objectives to improve public health.