Kadakia Kushal T, Dhruva Sanket S, Ross Joseph S, Burke James F, Johnston James L, Ramachandran Reshma, Krumholz Harlan M, Rathi Vinay K
Harvard Medical School, Boston, Massachusetts.
Philip R. Lee Institute for Health Policy Studies, University of California, San Francisco, California.
JAMA Intern Med. 2025 Jun 30. doi: 10.1001/jamainternmed.2025.2235.
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.
To characterize technological novelty, FDA review times, and premarket and postmarket evidence requirements for therapeutic breakthrough-designated devices.
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.
(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).
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.
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.
美国国会于2016年设立了美国食品药品监督管理局（FDA）突破性设备计划（BDP），以促进技术创新并便利患者获得相关设备。然而，FDA指定的突破性设备的临床和监管特征尚未得到评估。
描述治疗性突破性指定设备的技术新颖性、FDA审查时间以及上市前和上市后证据要求。
这项横断面研究分析了2016年1月1日至2024年9月30日期间获得FDA授权的所有治疗性突破性指定设备的公开可用FDA数据。
（1）设备新颖性（同类首创和首次商业化推出）；（2）FDA上市前审查时间（与医疗器械用户收费授权[MDUFA]目标相比）；（3）上市前关键研究特征；（4）上市后监管经验（FDA要求的研究和召回情况）。
在2016年1月1日至2024年9月30日期间，FDA对1041项设备授予了突破性指定，其中127项已获授权（12.2%），包括75项治疗设备（59.1%）。75项治疗设备中的34项（45.3%）被归类为高风险；在该亚组中，23项为同类首创（67.6%），14项在美国首次商业化（41.4%）。所有75项设备的平均（标准差）FDA审查时间为225.8（107.6）天，34项高风险设备的审查时间为243.3（98.4）天。MDUFA规定的FDA审查时间目标适用于30项高风险设备，其中22项（73.3%）在法定目标时间框架之前完成审查。大多数治疗设备（89.3%[75项中的67项]）进行了上市前临床试验，总共75项关键研究评估了81个主要有效性终点。40个终点（49.4%）纳入了有效性的替代指标，15个终点（18.5%）缺乏统计检验。终点的随访时间较短（植入式设备的中位数为6个月[四分位间距，3.8 - 12个月]）。FDA要求对30项突破性指定设备进行46项上市后研究（40.0%）；19项研究报告有延迟（41.3%）。截至目前，美国市场的平均（标准差）使用寿命为2.4（1.9）年，在此期间，12.0%的设备（75项中的9项）被召回，包括1项I类（1.3%）（即最严重程度）召回。
本研究发现，大多数治疗性突破性指定设备具有新颖性，且在法定目标时间框架之前接受了FDA审查。然而，一些设备的收益和风险存在不确定性，这引发了关于BDP是否始终如一地实现改善公众健康的计划目标的疑问。
