So, You Think You Have a Novel Device? Here’s What FDA Says.

Imagine you have an exciting new orthopedic device to bring to market, and you think the technology is innovative enough to be called novel. But do you know exactly how to qualify a medical device as noteworthy or novel? Do you know what the definition of a Breakthrough Device Designation is? Do you know the best type of submission for your product? Or how Breakthrough Designation compares to a submission made through other FDA pathways like the Safer Technologies Program (STeP)?

These are all excellent questions for orthopedic companies to ask internally. Further, it’s important to understand the answers to each one because that will help you determine the best regulatory submission pathway for your device.

Introduction to the Breakthrough Device Designation Program

FDA’s Breakthrough Devices Program was designed to streamline the market clearance and approval process for novel and potentially life-saving technologies. There are also significant strategic benefits to being accepted into the program.

“The whole focus of the program is to provide a high-level of engagement between FDA and device innovators, with the goal of speeding the development and evaluation of products that can make a real difference where there is no good alternative for patients,” said Owen Faris, Principal Deputy Director of the Center for Devices and Radiological Health’s Office of Product Evaluation and Quality.

The program launched in 2015 and has been steadily gaining in popularity. As of March, 31,657 devices, 71 of which were orthopedic, had been accepted into the program. However, not every application is accepted and not every accepted device makes it to clearance or approval. The acceptance rate has dropped from 70% to 40%, indicating that the requirements have become more stringent.

How do you, as a company in the orthopedic device industry, know whether a device qualifies for Breakthrough Designation? FDA’s definition of breakthrough is a novel device that “has the potential to lead to clinical improvement in the diagnosis, treatment, mitigation, cure or prevention of a life-threatening or irreversibly debilitating disease or condition.” But the device also has to offer significant advantages over available FDA-approved or cleared devices. Market availability of the novel device also must be in the best interests of the targeted patient population.

For example, a wearable device with a sensor that tracks a patient’s step count and stride after a knee replacement is not a novel device, because it is a traditional, non-implantable method of data delivery. However, a tibial extension with a sensor implanted into a patient during a knee replacement qualifies as novel. FDA made such a call when it accepted Canary’s CHIRP embedded into Zimmer Biomet’s Persona IQ for Breakthrough Device Designation and later de novo clearance.

There are other strategic advantages to Breakthrough Designation. Clinical trials do not need to prove that the device is more effective than comparable products on the market, unless the manufacturer makes a specific claim. Trials still need to demonstrate that the device is safe and effective. However, FDA may consider “the extent of uncertainty that may be appropriate” and allow the trials to continue after the device has been granted market clearance or approval. There is the option of a sprint discussion, which means the manufacturer and FDA will come to mutual agreement on specific submission-related topics within 45 days.

The data collection expectations can be defined for the entire product lifecycle, or manufacturers can opt to create a binding clinical protocol agreement that benefits both regulators and manufacturers. Pre-submissions for Breakthrough Devices are much less formal and interactive, and track more quickly through the process.

It’s important to note that Breakthrough Designation is not a lifetime get-out-of-jail-free card. FDA can revoke a Breakthrough Designation at any time. Another breakthrough device could invalidate a device’s designation, or a manufacturer could fail to meet the post-approval clinical trial endpoints or trial cohort requirements negotiated with FDA.

Medical device companies must also be careful not to make changes to the device that will invalidate the breakthrough designation and should discuss proposed changes with FDA before implementing them. Devices with a revoked or invalidated Breakthrough Designation can still pursue market clearance or approval through traditional submission pathways or through the Safer Technologies Program.

Beyond Breakthrough: The Safer Technologies Program (STeP)

The Safer Technologies Program is an alternative pathway for novel medical devices, with many similarities to the Breakthrough Devices Program. It’s a voluntary program targeting products with significant safety benefits in non-life threatening or reasonably reversible conditions that are less serious than those eligible for the agency’s Breakthrough Devices Program. The launch of STeP changed FDA’s opinion on what defines life-threatening or irreversible. But the program still offers a streamlined path to market, and manufacturers benefit from more timely and interactive communication with FDA.

To qualify for the STeP program, a device must have been deemed ineligible for the Breakthrough Devices Program due to the less serious nature of the disease or condition treated, diagnosed, or prevented by the device. It should also be reasonably expected to significantly improve the benefit/risk profile of a treatment or diagnosis through substantial safety innovations that provide for at least one of the following:

1. A reduction in the occurrence of a known serious adverse event
2. A reduction in the occurrence of a known device failure
3. A reduction in the occurrence of a known use-related hazard or use error
4. An improvement in the safety of another device or intervention

There are many similarities between the STeP and Breakthrough Devices Programs (see Table 1). Devices applying to either program are required to submit a pre-submission to FDA requesting entry. The pre-submission — also known as a Q-sub — must describe the device, its proposed indications for use, the benefit provided to the target patient population, its regulatory history, and the proposed type of marketing submission to FDA. The submission must also clearly identify how the proposed device meets the acceptance criteria and specify which of the strategic advantages — the sprint discussion, the clinical protocol agreement, etc. — the manufacturer wishes to employ.

Once the Q-sub has been submitted, FDA has 30 calendar days to request additional information and 60 days to provide a decision letter indicating whether the breakthrough request was granted or declined. If the request is granted, the manufacturer proceeds with a de novo or premarket approval (PMA) submission, starting with another Q-sub. If the request is denied, the manufacturer can elect to proceed with a standard de novo, PMA or 510(k) submission — also starting with another Q-sub — but without the benefits of a Breakthrough Device or STeP submission.

Table 1

Finding the Best Path Forward

Innovation in the medical device industry is at an all-time high, and there has been a surge in the number of novel devices vying for Breakthrough Designation in the past two years. While these novel devices may offer significant patient benefits, orthopedic manufacturers should carefully consider whether their device truly qualifies as breakthrough.

FDA is clear on the distinction between life-threatening and irreversibly debilitating diseases and those that are less severe. Manufacturers must ensure that they have reviewed the Breakthrough Device criteria before sending their application to FDA. But a denied breakthrough application does not have to derail commercialization plans, because the STeP program offers an alternative submission pathway that provides many of the same benefits.

The Breakthrough Device and STeP regulatory pathways are intended to help get novel devices to market more quickly where they can positively impact patient outcomes.

Michelle Lott is the Founder and Principal of leanRAQA. Save Supplier