FDA Authorization of Therapeutic Devices Under the Breakthrough Devices Program

Area of Science:

• Medical device regulation
• Health technology assessment
• Clinical innovation

Background:

• The US Food and Drug Administration (FDA) established the Breakthrough Devices Program (BDP) in 2016 to expedite innovative medical technologies and improve patient access.
• The program's clinical and regulatory characteristics remain largely unassessed, necessitating an evaluation of its effectiveness.

Purpose of the Study:

• To characterize the technological novelty of FDA-designated breakthrough devices.
• To analyze FDA review times and premarket/postmarket evidence requirements for therapeutic breakthrough devices.

Main Methods:

• A cross-sectional study analyzing publicly available FDA data for authorized therapeutic breakthrough devices from January 1, 2016, to September 30, 2024.
• Assessment of device novelty, FDA review durations against Medical Device User Fee Authorization (MDUFA) goals, pivotal study designs, and postmarket regulatory actions including recalls.

Main Results:

• Of 1041 devices granted breakthrough designation, 127 were authorized, including 75 therapeutic devices (59.1%).
• High-risk therapeutic devices (34/75) were often novel (first-in-kind: 67.6%; first US commercialization: 41.4%) and reviewed within MDUFA target times (73.3%).
• Premarket studies (89.3%) showed varied endpoint measures (49.4% surrogate, 18.5% lacking statistical testing) and short follow-up durations. Postmarket studies were required for 40%, with delays in 41.3%. 12.0% of devices were recalled.

Conclusions:

• Most therapeutic breakthrough-designated devices are novel and undergo timely FDA review.
• Concerns regarding evidence for some devices' benefits and risks warrant further investigation into the program's consistent impact on public health.