How to Successfully Obtain Breakthrough Device Designation

February 21, 2019 Becky Hurt

Authored by: Trisha Eustaquio, PhD, Senior Consultant

On December 18, 2018, the FDA finalized its guidance on the Breakthrough Devices Program, which was created by the 21st Century Cures Act and supersedes the Expedited Access Pathway (EAP) that was in place from 2011 to 2015. The purpose of the program is to help patients have more timely access to innovative devices, expedite device development and review, and foster collaboration between FDA and sponsor in defining a roadmap from proof-of-concept to FDA marketing authorization and postmarket study. Attractive features of the program include early and more frequent engagement with FDA, opportunities to shift collection of certain data from the premarket to the postmarket setting, and the potential to utilize intermediate, surrogate, and composite endpoints as well as a phased study design in human studies. Given the benefits of the program, it is not surprising that PAREXEL has received many requests from clients on how they can get their device to be designated as a Breakthrough Device. To date, PAREXEL has shepherded 8 Breakthrough Device designation requests to submission to FDA and successfully achieved designation for 7 of those requests, with many more in the pipeline. In a recent statement from FDA Commissioner Scott Gottlieb, MD, and Jeff Shuren, MD, Director of the Center of Devices and Radiological Health, 110 devices have received Breakthrough Device designation and 8 have received marketing authorization since the program’s inception at the end of 2016.

To be designated entrance into the Breakthrough Devices Program, the device must meet the following criteria:

  1. Provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or condition; and
  2. Meet at least one of the following:
  3. Represent breakthrough technology;
  4. No cleared or approved alternatives are available in the U.S. standard of care (SOC);
  5. Offers significant advantages over existing alternatives; or
  6. Availability is in patients’ best interest.

In criterion 1, the term “more effective” can be difficult to define without definitive and comprehensive data. However, sponsors should demonstrate that there is a reasonable expectation of technical success (the device performs as intended) and clinical impact (the device could more effectively treat or diagnose the target disease or condition). This typically entails summary-level presentation of nonclinical and clinical data collected to date and a scientific or clinical rationale that is well-supported by literature. PAREXEL can review the available information on your device, identify critical gaps and recommend how to address these gaps, and help you structure the information to meet this criterion.

The guidance defines a “life-threatening” disease or condition as one in which “the likelihood of death is high unless the course of disease is interrupted in a population or subpopulation” and an “irreversibly debilitating” disease or condition as one “associated with morbidity that has substantial impact on day-to-day functioning.” FDA has not enacted minimum requirements or prioritization of mortality or morbidity rates in determining which devices receive Breakthrough Device designation, and from personal experience, I have seen these definitions applied loosely with success in gaining designation. The target disease or condition can be one that is not immediately life-threatening or irreversibly debilitating on its own but, if left untreated, can lead to a disease or condition that is. As such, a device with preventative indications can be accepted into the program if the indications are worded carefully to meet the first designation criterion. PAREXEL can perform research on your target disease or condition as well as relevant marketed indications to assist you in refining your indications for use.

For criterion 2, the device must meet at least one of four subcriteria. I recommend that sponsors should address all four in the designation request, if possible, to increase the odds of success in gaining acceptance into the program. As with criterion 1, the information to support criterion 2 does not need to be extensive. Details of the four subcriteria are below. The final guidance also provides illustrative examples of devices that would meet each of the four subcriteria.

For the device to represent breakthrough technology (criterion 2a), the device should utilize recent technological advances or use existing technology in a new way, such that the device has the potential to lead to clinical improvement. Device technology need not be complicated or 100% unique to meet this criterion. For example, a legally marketed device with a strategic change in indications may be all it takes. PAREXEL can investigate indications for use and technological characteristics of your device type to maximize opportunities in promoting your device technology as breakthrough.

For criterion 2b, one should consider whether there is a cleared or approved product (drug, biologic, device, or combination product) for the same indications that is currently used within the U.S. SOC. Products that are no longer used or used only rarely do not count as a cleared or approved alternative in this case. If there are existing cleared or approved products per the above definition, then the device should demonstrate a potential to offer significant advantages over these products (criterion 2c). The statute explicitly states that these advantages could include the potential to “reduce or eliminate the need for hospitalization, improve patient quality of life, facilitate patients’ ability to manage their own care, or establish long-term clinical efficiencies.” These metrics suggest that some clinical data is needed to explain a device’s potential for offering significant advantages, but qualitative information can also be applied toward this criterion. PAREXEL has a vast network of medical experts that can assist you in assessing the U.S. SOC and evaluating your device against cleared or approved alternatives, if applicable, for your proposed indications.

For the last criterion (criterion 2d), availability is in patients’ best interest, specific public health benefits of the device should be considered. Examples include addressing a known failure mode and increasing patient compliance with treatment. Public health benefits could also be supported by patient preference information. In fact, patient preference information can also be used to address other designation criteria. How patients perceive the benefits and risks of a device and how they think about the tradeoffs of these benefits and risks can feed into justifications for the device providing “more effective” treatment or diagnosis and offering “significant advantages” over cleared or approved SOC alternatives. This suggests a device that has a less than ideal benefit-risk profile based on traditional measures may still be designated as breakthrough based on patient preference information. PAREXEL can help you obtain and apply patient preference information both quantitatively and qualitatively to meet this criterion.

PAREXEL’s subject matter experts are comprised of former regulators, industry professionals, physicians, and commercialization specialists with a proven track record of providing end-to-end solutions for our clients in global markets. Our experts have broad experience in traditional therapeutic and diagnostic medical devices as well as novel and innovative technologies such as combination products, digital health, and personalized medicine. From obtaining Breakthrough Device designation to marketing authorization and beyond, PAREXEL can help you every step of the way throughout your total product lifecycle.

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