Device experts discuss strategies for breakthrough, STeP submissions at Convergence

Regulatory NewsRegulatory News | 26 September 2022 |  By 

PHOENIX, AZ – Device manufacturers planning to submit applications through the US Food and Drug Administration (FDA)’s two novel device programs, the breakthrough devices program and Safety Technologies Programs (SteP) for Medical Devices, should consider submitting clinical data with their applications and executive summaries of how their devices work, according to experts who spoke on a panel at RAPS 2022 Convergence on 13 September.
They also offered advice on the best timing for requesting authorizations, and whether it is feasible to use these pathways as opposed to the more traditional route of a premarket approval (PMA) or a 510(k).
Kathy Herzog and Lisa Pritchard, regulatory consultants with DuVal & Associates, gave an update on these programs and imparted some best practices for submitting applications through both programs.
Dramatic increase in breakthrough designations
Since the launch of the breakthrough program in 2018, the agency has granted breakthrough status to nearly 700 devices, with FDA granting these requests 70% of the time, said Herzog.
FDA data shows the number of breakthrough designations increased dramatically since 2018, growing from 55 designations in FY2018 to 206 in FY2021. In the first three quarters of FY2022, FDA has granted 129 breakthrough device designations.
Herzog attributes the huge spike in 2020 to 2021 to a proposed rule from the Center for Medicare and Medicaid Services (CMS) that would have reimbursed breakthrough devices for four years. This rule, however, was withdrawn, and a new rule to replace it is expected.
Most of these designations, 687, were granted by FDA’s Center for Devices and Radiological Health (CDRH) and six were granted by the Center for Biologics Evaluation and Research (CBER).
Of the total number of breakthrough devices, only a small number, 54 or 8%, have been authorized or approved, including 20 premarket approval applications (PMAs), 17 de novo classifications and 15 510(k)s.
Herzog called this number “astoundingly low,” and attributed this to FDA still working through the process of reviewing the bolus of applications.
Most applications have mature clinical data
Herzog said that while the breakthrough program does not require sponsors to submit clinical data, three quarters of these applications have clinical data, based on a review of company press releases.
This clinical data was “mature data” for breakthrough applications and pulled from randomized clinical trials and pilot trial data.
STeP considered ‘stepchild’ of breakthrough approvals
Pritchard explained said the STEP program is considered the “stepchild” of the breakthrough program in that it covers devices that are not eligible for breakthrough designation. Products covered under this program include those that are less serious in nature of the disease or condition being treated, diagnosed, or prevented by the device than those included in the breakthrough program. To be eligible, the device should also be “reasonably expected” to improve the benefit-risk profile of a treatment or diagnostic through substantial safety innovations.
She said that FDA has provided “remarkably little data” about STEP, and that interest in this program is “nowhere near what we have seen with the breakthrough program.” (RELATED: Safer technologies program finalized by FDA, Regulatory Focus 5 January 2021)
Submit executive summaries in STeP applications
Pritchard said that SteP applications are usually submitted in the form of a Q-submission requests and added that executive summaries are often overlooked when making such submissions.
She added that it is “absolutely critical” to provide these summaries as “they tell the story of your submission. You are not simply providing the evidence that the guidance document tells you to have … FDA reviewers have so many files going on at one time it is impossible for them to be an expert on your product. You know your product and what it does, and you need to share this passion with the reviewer and share this advocacy with the reviewer.”
When to submit
Pritchard said a common question she hears from sponsors about both programs is when to submit applications. She advised manufacturers to wait until their device is “no longer a prototype” and wait until they have performance and characterization data to support the product.
She also told manufacturers to know the competitive landscape to help inform the timing of applications. “If there are competitors running neck and neck with you have to demonstrate you are more efficient than them.”
Alternatively, if there are no similar devices on the market, it may be better to wait and gather more evidence to support the request.
Pritchard said that from a risk perspective, it may be better to consider undergoing the traditional 510(k) or PMA approval route instead of submitting for breakthrough approval. When seeking approval through breakthrough approval, manufacturers often have less pre- and post-market data about their products and assume more risk.
RAPS Convergence


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