Breakthrough Designation Guidance Finalized

On December 18, 2018, FDA issued a final guidance document on the Breakthrough Devices Program created by the 21st Century Cures Act.  The Breakthrough Device Program is meant to speed access to new devices that treat or diagnose “life-threatening or irreversibly debilitating diseases or conditions.”  The final guidance document is largely unchanged from the October 2017 draft, which we reviewed in detail in a prior post, here.

By way of background, a device must meet two criteria to qualify for participation in the Breakthrough Devices Program:

1. The device facilitates more effective treatment or diagnosis of life-threatening or debilitating diseases or conditions; and
2. The device must meet one of the following criteria:
   • Represents breakthrough technology;
   • No cleared or approved alternatives is available on the U.S. market;
   • Offers significant advantages over existing alternative devices; or
   • Availability is in patients’ best interest.

Breakthrough designation can be sought for a device that will require clearance or approval via any premarket approval pathways and grants priority review status for designated devices.  Designated devices are placed at the top of the review queue and are assigned additional review resources, as needed.  Unlike breakthrough drug products, however, breakthrough devices are not guaranteed a faster review.

The changes to the draft guidance are minor and largely stylistic.  However, as we pointed out in our previous post, the draft guidance did not address what will happen to devices that have been granted Expedited Access Pathway (EAP) status under the existing program.  The finalized guidance clarifies that FDA considers devices granted designation under the EAP to be part of the Breakthrough Devices Program.

The finalized guidance also provides additional detail on device-led combination products seeking breakthrough designation, noting that such devices might present complex issues requiring expertise from a different Center and may, therefore, require additional time to resolve.  The guidance explains that, when CDRH or CBER receives a Q-Submission, IDE or marketing application for a device-led combination product that has been designated as a Breakthrough Device, CDRH or CBER will notify and engage, as needed, the appropriate review staff from the consulting Center(s).

The guidance elaborates on the various options on interacting with FDA available to designated products.  For example, when submitting a Q-Submission for a designated Breakthrough Device, sponsors should specify if they are requesting one of the special program features available to designated Breakthrough Devices (i.e., a sprint discussion, review of a Data Development Plan, or a Clinical Protocol Agreement).

Interestingly, the example Data Development Plan specifically states:

FDA supports the principles of the “3Rs,” to reduce, refine, and replace animal use in testing when feasible. We encourage sponsors to consult with us if it they wish to use a non-animal testing method they believe is suitable, adequate, validated, and feasible. We will consider if such an alternative method could be assessed for equivalency to an animal test method.

This addition is consistent with Commission Gottlieb’s statement in early 2018 regarding FDA’s strengthened commitment to humane and judicious animal research following the Agency’s termination of an animal study investigating the role of exposure to various levels of nicotine on the onset of addiction in adolescence and young adults.

Lastly, the finalized guidance clarifies when breakthrough designation is available for multiple devices with the same intended use.  Specifically, a Breakthrough Device designation will not be revoked solely because another designated device obtains marketing authorization.  This means that multiple Breakthrough Device designations for the same intended use may be granted, with multiple submissions pending simultaneously.   However, once a Breakthrough Device is cleared, approved, or has had a De Novo request granted, additional devices with the same intended use will only be designated as a Breakthrough Devices if they can satisfy the designation criteria considering the first Breakthrough Device’s market availability.

The finalized guidance was announced in a broader statement by FDA Commissioner Gottlieb and CDRH Director, Jeff Shuren, M.D. regarding FDA’s steps to promote innovations in medical devices that advance patient safety.  Also included in this statement was FDA’s plan for a new Safer Technologies Program (STeP), originally outlined in April 2018 as part of the Medical Device Safety Action Plan.  While additional details are forthcoming in 2019, Gottlieb and Shuren’s statement alluded to the STeP program incorporating the principles and features of the current Breakthrough Devices Program to devices with the potential for significant safety improvements as compared to available treatment or diagnostic options.  This announcement is noteworthy because it focuses on patient safety features, which is consistent with FDA’s earlier announcement regarding 510(k) devices and the Agency’s desire to “sunset” old predicates that may not incorporate the latest safety features.

While earlier expedited device programs have not succeeded at increasing speed to market for novel devices, we are optimistic that both the Breakthrough Device Program and STeP will encourage and facilitate innovative technologies that will advance patient safety.  For those interested, FDA is hosting a webinar on January 17, 2019, to help clarify the Breakthrough Devices Program final guidance.