New Breakthrough Devices Program Guidance: Expanding Opportunities for Medical Product Innovations

October 17, 2023By Philip Won & Lisa M. Baumhardt, Senior Medical Device Regulation Expert

On September 15, 2023, FDA released a final guidance document titled “Breakthrough Devices Program.”  Compared to the previous version, the new guidance document highlights that the Breakthrough Devices Program may also be applicable to certain devices that benefit populations impacted by health and/or healthcare disparities and to certain non-addictive medical products to treat pain or addiction.  If you are developing such products, now may be the time to explore the opportunities presented by the Breakthrough Devices Program.


The Breakthrough Devices Program, established under section 515B of the Federal Food, Drug, and Cosmetic Act (FD&C Act), is a voluntary program for certain medical devices and device-led combination products.  FDA previously released the final guidance document in December 2018 outlining the program’s principles, features, designation criteria, and other considerations.  The designation criteria, as defined in section 515B(b) of the FD&C Act, have remained unchanged and continue to govern the program for devices as follows:

(1) that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or conditions; and

(2)(A) that represent breakthrough technologies;

(B) for which no approved or cleared alternatives exist;

(C) that 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

(D) the availability of which is in the best interest of patients.

In October 2022, FDA released the draft guidance entitled “Select Updates for the Breakthrough Devices Program Guidance: Reducing Disparities in Health and Healthcare,” as previously discussed in our blog.  This 2022 draft guidance proposed that certain non-addictive medical products for pain or addiction treatment could be eligible for breakthrough designation, aligning with the Agency’s obligations under the Substance Use-Disorder Prevention That Promotes Opioid Recovery and Treatment (SUPPORT) for Patients and Communities Act.  Additionally, the 2022 draft guidance suggested the addition of a new section to the 2018 guidance on reducing disparities in health and healthcare.  The new guidance does not surprise us as the scope of the changes is limited to the select updates, but it is certainly exciting that FDA expands product areas for the Breakthrough Devices Program.

Non-Addictive Medical Products to Treat Pain or Addiction

The SUPPORT Act (Public Law 115-271) mandated that FDA issue a guidance document to provide information on how the Agency can apply section 515B of the FD&C Act to non-addictive medical products intended to treat pain or addiction.  As suggested in the 2022 draft guidance, FDA included a statement in the introduction section of the guidance, indicating that “the Breakthrough Devices Program may be available for certain non-addictive medical products to treat pain or addiction (FD&C Act section 515B (21 U.S.C. 360e-3)).”  Apart from this statement in the introduction section, the new guidance document does not offer any further guidance related to non-addictive medical products for pain or addiction treatment.  This may be because FDA has already been granting breakthrough designation to devices intended to treat pain (e.g., Wysa, see here) and addiction (e.g., reSET-O, see here, here).

The readers may be wondering what kind of devices would be able to leverage the new guidance to receive breakthrough designations.  Although not directly relevant, the FDA Innovation Challenge program called “Devices to Prevent and Treat Opioid Use Disorder” may provide you with an idea.  FDA launched this challenge in 2018 to foster the development of medical devices aimed at addressing the opioid crisis, preventing, and treating Opioid Use Disorder (OUD).  FDA selected and posted eight challenge participants, which included monitoring, OUD therapy, pain therapy, medication dispensing, overdose therapy, and diagnostic devices.  FDA notes “Breakthrough Device designation will be granted to those devices that meet the statutory criteria for designation without submission of a separate application.”  It appears that the devices selected for the challenge may align with the type of non-addictive medical products for pain or addiction treatment that the new breakthrough guidance targets.

Reducing Disparities in Health and Health Care

FDA introduced a new section in the guidance document focused on reducing disparities in health and healthcare.  In this section, FDA acknowledged that treatment outcomes might vary based on factors such as race, ethnicity, sex, age, disability, and others.  Consequently, FDA underscores the importance of considering “technologies and device features tailored to address characteristic differences” such as social factors, phenotypic variations, pathophysiology, and response to treatment when evaluating the first designation criterion.  This implies that if device technologies and features can produce a clinically meaningful impact for the treatment or diagnosis of a condition in certain populations, such data could support the case that the device satisfies the first designation criterion.

The new guidance also presents an opportunity for device manufacturers working for patient populations affected by rare life-threatening or irreversibly debilitating diseases or conditions, such as sickle cell disease.  FDA will consider if technologies and device features “address unmet needs in these populations” when evaluating criterion one.  In addition, criterion one can also apply to technologies and device features aimed at improving accessibility, defined as “an individual or group’s capacity to benefit from a medical device or procedure.”  FDA recognizes the existence of barriers that may hinder underserved populations from receiving medical treatment or diagnosis.  To address these barriers, FDA promotes the development of impactful devices that can enhance accessibility for patient populations with limited or no available options.  This might include “user features that are adaptable or more easily used by diverse populations or allow for use in more diverse settings.”  With improved accessibility, one can make the argument that there is a “reasonable expectation that the device may provide for more effective treatment or diagnosis as compared to the standard of care.”

Other Updates

With respect to the first part of the first criterion (i.e., whether a device provides for more effective treatment or diagnosis), FDA introduced a paragraph explaining that “[t]he level and type of evidence needed to determine whether a device is reasonably expected to “provide for more effective treatment or diagnosis” may vary depending on the intended use of the device, its technology and features, and the available standard of care alternatives.” (Emphasis added.)  While the reasonable expectation standard remains unchanged, FDA emphasizes that it will consider the totality of information regarding the proposed device, its function, potential for technical success, potential for clinical success, potential for a clinically meaningful impact, and its potential benefits and risks.

Remaining Questions

We perceive the new guidance as an expansion rather than a restriction of the types of products that may qualify for Breakthrough Device designation.  However, the new guidance does not address certain questions that the industry may encounter when seeking the breakthrough designation.

For example, it remains uncertain whether devices indicated for addiction or pain treatment would be considered to satisfy the second part of the first criterion (i.e., whether a disease or condition is life-threatening or irreversibly debilitating), regardless of the type or severity of the underlying diseases or conditions causing pain.  Furthermore, the new guidance does not provide any indication of the level of evidence necessary to demonstrate that a candidate device can contribute to reducing disparities in health and healthcare.  There may also be questions about the level of evidence required to demonstrate that a device can address characteristic differences arising from social factors, phenotypic variations, pathophysiology, and/or response to treatment.  Although the new guidance does not explicitly state that clinical data is a mandatory requirement for a breakthrough application, it is our experience that a review division typically expects to review some level of clinical evidence to assess the reasonable expectation of clinical success.

If FDA’s evidentiary expectations are set too high, it could undermine the intent of the new guidance.  FDA has stated that it “considers the totality of available information regarding the device, including its potential for a clinically meaningful impact and its potential benefits and risks.”  We hope that “the totality of available information” that could meet FDA’s expectations will not impose an excessive burden on companies seeking breakthrough designations.

Categories: Medical Devices