As part of the 21st Century Cures Act, Congress gave the US Food and Drug Administration (FDA) the authority to establish a Breakthrough Devices Program intended to expedite the development and prioritize the review of certain medical devices that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating disease or conditions. In December 2018, FDA issued a guidance document describing policies FDA intends to use to implement the Program.

There are two criteria for inclusion in the Breakthrough Device Program:

1. The device must provide for a more effective treatment or diagnosis of a life-threatening or irreversibly debilitating human disease or condition; and
2. The device must (i) represent breakthrough technology, (ii) have no approved or cleared alternatives, (iii) offer significant advantages over existing approved or cleared alternatives, or (iv) demonstrate that its availability is in the best interest of patients.

As of January 2019, FDA had granted 112 Breakthrough Designation requests. The requests must be obtained by the applicant before submitting a marketing application to FDA. Devices with a Breakthrough Designation receive priority review and digital health technologies are increasingly taking advantage of this pathway. For example, in March, FDA granted a Breakthrough Device Designation to an artificial intelligence (AI) tool intended to enable pathologists to become faster and more accurate in their diagnosis and treatment recommendations for cancer patients. In May, FDA granted a Breakthrough Device Designation for a retinal imaging platform that uses AI to analyze retinal images used in the evaluation and diagnosis of Alzheimer’s disease.

Benefits of the Designation Process

Participants granted a Breakthrough Device Designation will have opportunities to interact with appropriate, specialized FDA review team members and senior management prior to submitting their device for FDA approval and receive clear guidance on what is required for approval and how requirements can be met. Participants will benefit from being able to design efficient and flexible clinical trials and, when practical and scientifically appropriate, receive expedited review of manufacturing and quality systems compliance.

Perhaps the most significant benefit is that while Breakthrough Devices subject to a premarket approval (PMA) must still meet the statutory standard of reasonable assurance of safety and effectiveness at the time of approval, for PMAs designated as Breakthrough Devices, FDA intends to use timely post-market data collection, when scientifically appropriate. In other words, i.e., completion of a pivotal trial may not be necessary for a Breakthrough Designated device to receive premarket approval.

Challenges of the Designation Process

Additionally applicants must be prepared to engage with FDA frequently and at a high-level of detail. Many companies are not prepared to offer the detailed information FDA will request. Applicants should also be sure to have a robust compliance program and data strategies that can stand up to agency scrutiny, which again can present a challenge as many companies have not considered these issues early in the development process.

The Breakthrough Designation can significantly reduce a product’s time to market, provided that it can demonstrate the product meets the relevant inclusion criteria.