We think it could.
Final guidance for FDA “Breakthrough Devices Program” was published in December 2018. As this could be potentially relevant for many medical devices in development, we thought it would be worthwhile bringing it to your attention.
Ultimately, if successfully applied, this program could bring your product to the market more quickly.
What is the FDA Breakthrough Devices Program?
The FDA Breakthrough Devices Program is a voluntary program for certain medical devices and device-led combination products that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions.
It is available for products which are subject to review under a PMA, 510(k), or De Novo request.
This program is intended to help patients have more timely access to these medical devices by expediting their development, assessment and review.
The Breakthrough Devices Program supersedes both the Expedited Access Pathway and the Priority Review Program.
Breakthrough Program Principles
The FDA Breakthrough Devices Program is comprised of two phases.
In the first phase, the sponsor of a device requests that the FDA grants that device Breakthrough Device designation.
The second phase encompasses actions to expedite the development of the device and the prioritized review of subsequent regulatory submissions.
Communication is Money
For Breakthrough Devices, the FDA intends to provide interactive and timely communication with the sponsor during the device’s development and throughout the review process.
Premarket Versus Postmarket Data
In order to enable the quick and efficient development and review of PMA devices, the FDA may consider, if scientifically appropriate, which data may be collected in the postmarket setting, rather than premarket. Please also see the FDA’s guidance, “Balancing Premarket and Postmarket Data Collection for Devices Subject to Premarket Approval”, which provides more information regarding the FDA’s current policy on balancing premarket and postmarket data collection during the FDA’s review.
Efficient and Flexible Clinical Study Design
The FDA intends to ensure that clinical trial design is efficient and flexible. This, for example, may include:
- determining endpoints regarding the minimum clinically meaningful effect or
- setting intermediate and surrogate endpoints which are likely to predict the clinical benefit of a device
All Breakthrough Devices submissions will receive priority review. The earlier interaction between the FDA and sponsors during the device development process should enable patients to have more timely access to these devices than they would otherwise have. Likewise, the sponsor’s efforts to deal with development and regulatory hurdles should match that of the FDA.
Manufacturing Considerations for PMA Submissions
A device must be conform with the Quality System regulation (21 CFR part 820).
As with other PMAs, sponsors of a medical Breakthrough Device should submit PMA information as described in the FDA guidance, “Quality System Information for Certain Premarket Application Reviews”.
In appropriate cases, the FDA may decide not to conduct an inspection of certain manufacturing sites prior to approval of a Breakthrough Device.
For example, for finished device manufacturing sites that have been inspected within a period of two to five years prior to the filing date of the PMA, for which the inspection outcome was ‘No Action Indicated’ or ‘Voluntary Action Indicated’, the FDA may determine that it is appropriate to inspect those sites after the Breakthrough Device is approved, usually 12 months after approval.
Breakthrough designation request
A designation request for a Medical Breakthrough Device is the mechanism by which sponsors request entry into the Breakthrough Devices Program. The request for designation may be made at any time prior to the submission.
Breakthrough Designation Criteria
In order to be eligible for the FDA Breakthrough Program, devices should provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or conditions;
In addition, they must meet at least one of the following criteria:
- Device represents 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, to improve patient quality of life, to facilitate patients’ ability to manage their own care (such as through self-directed personal assistance) or to establish long-term clinical efficiencies; or
- The availability of which is in the best interest of patients.
In determining whether a device meets the first criterion of providing “for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or condition,” the FDA considers the following three factors:
- Whether a Device Provides for “More Effective” Treatment or Diagnosis
A sponsor should demonstrate a reasonable expectation that the device could provide for more effective treatment or diagnosis of the disease relative to the current standard of care (SOC) in the U.S. A complete set of clinical data is not required for designation.
The mechanisms for demonstrating a reasonable expectation of technical (device could function as intended) and clinical (could more effectively treat or diagnose disease) success could include literature or preliminary data (bench, animal or clinical).
- Whether a Disease or Condition is “Life-Threatening”
Within the framework of the FDA Breakthrough Devices Program, a life-threatening disease or condition is one for which the likelihood of death is high unless the course of the disease is interrupted in a population or subpopulation. Examples of life-threatening diseases or conditions include, but are not limited to, acute stroke, myocardial infarction, cancer and trauma.
- Whether a Disease or Condition is “Irreversibly Debilitating”
Whether a disease or condition is “irreversibly debilitating” is based on its impact on such factors as survival, day-to-day functioning and the likelihood that the disease or condition, if left untreated, will progress to a more serious disease or condition. Examples include cancer, amyotrophic lateral sclerosis (ALS), stroke and large ST segment elevation myocardial infarction.
As noted above, to qualify for Breakthrough Device designation, in addition to meeting the first criterion, a sponsor must also demonstrate that its device meets one of the four sub-paragraphs under the second criterion.
- Device Represents Breakthrough Technology
When determining whether a device represents a “breakthrough technology,” the FDA considers whether the device represents a novel technology or the novel application of an existing technology that has the potential to lead to a clinical improvement in the diagnosis, treatment (including monitoring of treatment), cure, mitigation or prevention of the life-threatening or irreversibly debilitating disease or condition.
- No Approved or Cleared Alternatives Exist
When determining whether the device meets the criterion, the FDA considers whether there is an alternative product that the FDA has approved, cleared or licensed, or for which the FDA has granted a De Novo request at the time of the request for Breakthrough Device designation where these products reflect the current SOC for the specific indication for which the product is being developed.
- Offers Significant Advantages over Existing Approved or Cleared Alternatives
The FDA will consider the potential, compared to existing approved or cleared alternatives, “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.”
The Guidance offers several illustrative examples for such devices.
Device Availability is in the Best Interest of Patients
For example, this may apply when the device has a benefit for patients who are unable to tolerate available therapy, whose disease has failed to respond to available therapy or for whom the treatment can be used effectively with other critical agents that cannot be combined with available therapy.
This criterion may also apply if the device avoids serious harm that can occur with available therapy; avoids serious harm that causes discontinuation of treatment of a life-threatening or irreversibly debilitating disease or condition; or reduces the potential for harmful interactions with other therapies.
In addition, this criterion may apply to a device that was designed or modified to address an unanticipated serious failure occurring in a critical component of an approved or cleared device for which there are no alternatives or for which alternative treatment would entail substantial risk of morbidity for the patient.
Further, the device may also satisfy this criterion if it provides an additional benefit, such as improved patient compliance that is expected to lead to a reduction in serious adverse outcomes or when it addresses an emerging or anticipated public health need, such as a device shortage or public health emergency.
As a part of the designation request review, applicants should indicate which marketing application type (PMA, 510(k), or De Novo request) they intend to submit and include a rationale for this approach in support of their Breakthrough Device designation request. The FDA’s designation decision in a way indicates the expectations of the Agency that PMA, 510(k), or a De Novo request will be necessary for marketing authorization.
The guidance states that, although eligible for FDA Breakthrough Device designation, combination products may raise certain unique scientific and regulatory challenges and issues.
We recommend that sponsors of combination products consult with a regulatory professional as to whether this program provides substantial advantages for the product development.
Multiple Devices with the Same Intended Use
The FDA Breakthrough Device designation may be granted for multiple devices with the same proposed intended use, and a Breakthrough Device designation will not be revoked solely on the basis of another designated device obtaining marketing authorization.
As a consequence, multiple Breakthrough Device designations for the same intended use may be granted and have subsequent submissions pending simultaneously. However, when a Breakthrough Device has been approved or cleared or has had a De Novo request granted, no additional devices with the same intended use will be designated as a Breakthrough Device, unless the criteria for designation described above are still met in light of the first Breakthrough Device’s market availability.
Breakthrough devices program features
Following a request for Breakthrough Device designation, the FDA should issue a grant or denial decision within 60 calendar days. The FDA offers “sprint” discussions with the goal of reaching mutual agreement on a specific topic within a set time period (e.g., 45 days). The number, format and duration of interactions within a sprint discussion may vary based on a project’s needs and should be defined a priori by the sponsor and the FDA. During a sprint discussion, the sponsor may provide additional information or revisions to the initial proposals.
Sponsors of Breakthrough Devices may also request coordination with the FDA regarding a Data Development Plan (DDP). The DDP is a high-level document intended to help ensure predictable and efficient device assessment and review by outlining data collection expectations for the entire lifecycle of the product. It should describe the balance of premarket and, as applicable, postmarket collection of clinical and non-clinical data.
The FDA intends to work with the sponsor so that the plan is developed in a manner that is least-burdensome and predictable, while allowing for some measure of flexibility and adjustments as appropriate.
This Program also includes a provision for obtaining agreement in writing for a clinical protocol which will be considered binding on both the FDA and the sponsor.
The FDA and the sponsor of a Breakthrough Device may agree to have regular (e.g. bi-monthly) status updates, through which the general progress of the project and the next steps or plans for future discussions will be agreed. This comprehensive and well written guidance also includes an example of a Breakthrough Device Designation Request and an example Data Development Plan (DDP) which are both very helpful.
If you have any question regarding the Breakthrough Device Program or need assistance or guidance throughout the process, please don’t hesitate to contact us.
We can also help you to successfully prepare PMA, 510(k), or De Novo applications.
Always pass on what you have learned. (Yoda)