On September 15, 2023, FDA published an update to the guidance document – “Breakthrough Devices Program, Guidance for Industry and Food and Drug Administration Staff.” Notably, FDA states that it may consider a medical device’s ability to improve health and healthcare disparities when deciding whether to designate a medical device as a breakthrough device. The agency may use this information to determine whether a candidate device provides more effective treatment or diagnosis when compared to the current standard of care. Below, we provide additional information regarding the breakthrough device program and the updated guidance.

What is the breakthrough device program?

As many readers of this blog are likely familiar, Congress created FDA’s Breakthrough Device Program through the 21st Century Cures Act in 2016. Under the law, certain medical devices that meet specified conditions become eligible for FDA’s prioritized review, which can result in potentially expedited development and FDA authorization of the medical device. More specifically, medical devices must meet the following conditions to be granted the breakthrough device designation:

  • Provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or conditions; and
  • Meet one of the following conditions:
    • Represent breakthrough technologies;
    • No approved or cleared alternatives exist;
    • Offer significant advantages over existing approved or cleared alternatives, including the potential, compared to existing approved alternatives, to reduce or eliminate the need for hospitalization, improve patient quality of life, facilitate patients’ ability to manage their own care (such as through self-directed personal assistance), or establish long-term clinical efficiencies; or
    • the availability of such medical devices is in the best interest of patients.

What are some of the benefits?

Applicants that can demonstrate that their device meets the above thresholds are provided access various benefits, such as:

  • Reliance of post-market data collection, where scientifically appropriate, for expedited development and review of the device for marketing authorization;
  • Interactive communications with the agency;
  • Use of more flexible clinical trial designs, such as adaptive studies, composite endpoints, or intermediate/surrogate end points, among others;
  • Prioritized review for breakthrough devices, which means that the applicable application will be placed at the top of the review queue, among others.

As of June 30, 2023, FDA has granted 839 breakthrough device designations. Many of the breakthrough devices have cardiovascular, neurology, orthopedic, or gastroenterology/urology-related indications.  But to be clear, designation as a breakthrough device does not necessarily mean that the device is likely to be cleared or approved by FDA, or granted the de novo classification. Of the 839 devices, as of June 30, 2023, FDA has cleared, approved, or granted de novo classifications for only 81 devices.

What is the significance of the September 2023 update?

This most recent update to the guidance document represents FDA’s evolving thoughts on the implementation and development of the breakthrough device designation program. Specifically, FDA notes in the guidance that the designation may be available to devices that benefit individuals who are disadvantaged by health and healthcare disparities. FDA understands that treatment outcomes may differ based on race, ethnicity, sex, age, disability, and others, and that timely access to certain breakthrough devices can help address issues with health and healthcare disparities.  The agency may review the devices’ ability to address differences arising from social factors, phenotypic variations, pathophysiology, and other factors, to determine whether the devices can provide more effective treatment or diagnosis than the current standard of care – i.e., the first criteria that FDA reviews when deciding whether to designate a device as a breakthrough device.

In addition, FDA states that the agency may consider “accessibility” when determining whether a device provides more effective treatment of diagnosis. The agency distinguishes this term from “access”; “access” describes the commercial availability of the medical device, where as “accessibility” refers to patients’ capacity to benefit from a medical device or procedure (e.g., lack of healthcare facilities in a given setting). FDA recognizes that the lack of accessibility could potentially hinder patients’ access to more effective treatment or diagnosis options. To address this, the agency states that it may consider a device’s ability to increase accessibility – for example, features that allow diverse populations’ use of such features, or enable their use in more diverse settings – when determining whether a device meets the first criteria. In short, the agency will review how a device avails its features to users and patients, in addition evaluating how effective the feature is for a given indication.

We recommend that medical device manufacturers that are currently developing medical devices and reviewing whether their devices are eligible for the breakthrough device designation consider whether the medical device has the potential to address and improve health and healthcare disparities and inequity. The guidance makes it clear that a device’s ability to address such disparity or inequity may impact FDA’s analysis and determination. If the device addresses such disparities and/or inequality, the manufacturer should highlight this aspect to FDA, and explain how this may improve outcomes for patients and users.  

Earlier, we reported that a manufacturer of radiology software solutions announced that FDA granted a breakthrough designation for the first time for its radiology triage software medical device.

Reed Smith will continue to monitor developments with regard to regulation of the breakthrough device program. If you have any questions, please reach out to the authors or to the health care lawyers at Reed Smith.