In 2018, the United States Food and Drug Administration (FDA) established the Breakthrough Devices Program (BDP), which allows for fast-tracking the approval of certain novel devices or device-led combination products (products that are therapeutic and/or diagnostic that combine drugs, devices, or biological products.) The goal of this program is to expedite patient access to devices that provide more effective treatment or diagnosis, through a more rapid assessment and review process, while preserving the FDA’s standards of approval. This program replaces the Expedited Access Pathway (EAP) and Priority Review for medical devices programs, as the EAP did not include 510(k) applications and the priority review is now limited only to drugs, and not devices.

The BDP offers device manufacturers an opportunity to work directly with the FDA through the review stage of the pre-market approval (PMA) process. Devices that are subject to PMA, 510(k), or requests for de novo designation are eligible for Breakthrough Device designation if they meet two criteria:

  1. The device provides a more effective treatment or diagnosis for a life threatening or debilitating human disease or condition than previous therapies.
  2. The device meets at least one of the following: a) represents breakthrough technology, b) no approved or cleared alternatives currently exist, c) provides a significant advantage over current cleared or approved alternatives, d) the device’s availability is in the best interest of patients.

Chief Scientific Officer of the Focused Ultrasound Foundation Jessica Foley, PhD, recently interviewed Sean Taffler, PhD, founder and CEO of Acoustiic, to learn more about the FDA’s Breakthrough Devices Program and how Acoustiic is taking advantage of this opportunity.

Acoustiic logo 600Tell us a little bit about Acoustiic.
Acoustiic is an early-stage ultrasound therapeutics company, leveraging emission plane electronics to produce compact, MR-compatible, high power, wide area ultrasound arrays. Typical surgical arrays are in the order of 20,000 elements with a maximum acoustic power in the range of kilowatts. This dense high-power array allows Acoustiic to noninvasively treat solid mass tumors within the body. The company’s initial targets are liver and pancreatic cancers. However, the technology is versatile so that other cancers and malignancies are easily addressable. Given the noninvasive nature, this opens the door of life-changing therapy to classes of patients that previously may have been excluded due to contraindications such as age, compromised immune systems, or reluctance to undergo an invasive surgery and the potential associated complications. Beyond that, we see our technology as a platform to research future treatments, such as opening the blood-brain barrier, neuromodulation, and drug activation. As such, we are developing a line of products aimed at researchers and universities.

Why did you decide to apply for the Breakthrough Device designation?
As a company we made it a priority from day one to build our regulatory approach into the company processes and were concerned about finding out late in the cycle that we needed to make major changes. Our regulatory consultant suggested that we apply to the expedited access and that a “partnership” would be beneficial because our device was novel and complex. The integration of high intensity ultrasound (HIFU) arrays and MR imaging presents some unique challenges – more akin to imaging than traditional HIFU – so we wanted to have those discussions early, while on the fundraising side it lends credence to the technology.

What are the benefits to receiving Breakthrough Device designation?
For us, it has been the chance to work through the data collection requirements in collaboration with the FDA. As opposed to presenting them with a data set and asking if it meets their requirements, we work with the FDA to define the data that needs to be collected to support the application before we start collecting data. This – in theory – should lead to a much smoother application process, especially for novel technologies. For a small start-up, the spectre of FDA rejection, especially over paperwork rather than technical or medical causes, is very real and has a very real impact on the financing of the company. Anything that can be done to reduce that exposure is hugely beneficial. At an early stage, it forces the company to crystalize its thoughts around the marketing strategy and how the device will be indicated for use.

What is the downside if not selected?
There is no major downside if not selected apart from the time spent putting the application together – however, that is work that will ultimately need to be done at some point. A rejection does not in any way affect your application in the future.

What does the application process entail? Is it overly burdensome?
Initially, we were concerned. However, the application is not hard. It is mostly a description of the company, a description of the technology, and, of course, your Indications for Use, Intended Use, and a short justification of how the device meets the Breakthrough Device criteria. Ours ended up at about 50 pages and has been valuable for other applications, marketing, investor presentations, and general company planning. For an early-stage company, this was a great time to really crystalize ideas and decide on the focus and strategy of the company. The week or so working out the nuances of our Intended Use and Indications for Use was exceptionally useful for such an early-stage company.

Once you applied, how long did it take to receive the designation?
Our application was granted within 30 days. I hope this bodes well for all future interactions with the organization.

What was the most frustrating aspect to the application?
There were no major frustrations. Just remember that FDA guidance documents are not just “guidance;” they are the de facto standard by which you will be judged. So, you need to make sure that you are reading the latest version of the guidance document. I noticed that the Breakthrough Guidance under which we applied has been updated.

How does Breakthrough Device designation aid in reimbursement?
In January 2021, the Centers for Medicare and Medicaid Services (CMS) issued a final rule that provides immediate national Medicare coverage for four years for any new device or diagnostic designated as a breakthrough technology and approved by the FDA. This new rule – called the Medicare Coverage of Innovative Technologies (MCIT) pathway– was expected to take effect beginning March 15, but was delayed to allow the new Administration the opportunity for a more thorough review and assessment. Following an additional public comment period that ends April 16, we anticipate the new rule to take effect May 15, 2021.

During the proposed four-year coverage period, CMS is expected to specify what additional data, if any, would be needed to maintain long-term coverage. This new rule also presents a great incentive for focused ultrasound manufacturers to apply for Breakthrough Device designation by the FDA for their future or ongoing applications. Sponsors can submit a breakthrough designation request for a device at any time prior to sending their marketing submission. See CMS’s Fact Sheet for more information, including device eligibility and guidance for manufacturers.

Outside of regulatory and reimbursement perks, have you noticed other benefits to the designation?
The obvious one is credibility for fundraising and engagement of clinical partners.

Has this designation influenced fundraising at all?
It has been beneficial in the early conversations, but it is a long road from protype to device. In medical devices, investors are extra wary of the risk posed by FDA regulatory rejection, so it is a positive to be engaged so early. The fact that you have BDP designation lends credibility to the technology of your product, but this is only one dimension of the company that is investigated during due diligence.

Would you recommend that other companies apply for Breakthrough Device designation?
The Breakthrough Devices Program is not for everyone; simple 510(k)s are unlikely to meet the criteria of new or novel. However, if you have a novel technology that is serving an underserved population or a new device that dramatically improves outcomes then you absolutely should apply.

Any tips or advice to the people applying?
Like most things in life, experience counts; a consultant or employee that has experience with BDP applications can avoid many of the minor gotchas that crop up. It can be daunting, but with a trusted advisor the challenges should be surmountable.

Did you do this on your own or did a regulatory consultant help you with the process?
We engaged a consultant – Michael Drues of Vascular Sciences – he is invaluable in his experience and counsel. For a small company it was essential to manage costs; as we develop and grow, we may well bring some functions in-house.


Additional Resources
Focused Ultrasound Manufacturers
FUS Partners
Regulatory and Reimbursement

For more about Acoustiic, visit http://acoustiic.com/.

Sean Taffler, PhD, is the founder and CEO of Acoustic.
Jessica Foley, PhD, is the chief scientific officer at the Focused Ultrasound Foundation.

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