Medical device and diagnostic manufacturers seeking acceleration of U.S. Food and Drug Administration (FDA) approval may benefit from one of two purpose-built FDA programs: 1) the Breakthrough Devices Program; and 2) the Safer Technologies Program (STeP). The benefits of these programs are substantial for manufacturers who apply, qualify and are granted designation by FDA.
According to the FDA, the goal of these programs is to, “provide patients and healthcare providers with timely access to innovative medical devices (that) provide more effective treatment or diagnosis of life-threatening or irreversible debilitating diseases or conditions.” In practice, patients receive innovative treatments and therapies in an accelerated manner to improve quality of life.
Below is a breakdown and comparison of the Breakthrough Devices and STeP Programs:
|Safer Technologies Program (STeP)
|“Breakthrough Designation Request”
|“STeP Entrance Request”
|The device provides for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or conditions
|Not eligible for the Breakthrough Devices Program due to the less serious nature of the disease or condition treated, diagnosed, or prevented by the device
|One or more additional Criteria that must be meet
|Also meets at least one of the following:
· Represents Breakthrough Technology
· No Approved or Cleared Alternatives Exist
· Offers Significant Advantages over Existing Approved or Cleared Alternative
· Device Availability is in the Best Interest of Patients
|Expected to significantly improve the benefit-risk:
· Reduction in the occurrence of a known serious adverse event
· A reduction in the occurrence of a known device failure mode
· A reduction in the occurrence of a known use-related hazard or use error
An improvement in the safety of another device or intervention
|Multiple Sponsors Can Be Designated
|Yes, devices with same intended use
|Yes, device with same intended use
|Any time before the PMA/510(k)/or de novo has been submitted
|Any time before PMA/510(k)/or de novo “approval”
|Initial FDA Review
|Information Request Stops FDA Review
|Review Determines Regulatory Path
|Yes, (PMA, De Novo, or 510(K))
|Yes, (PMA, De Novo, or 510(K))
|Sponsor Can Reapply
|At Any Time Sponsor can Withdrawal
How Device Manufacturers Benefit
There are many benefits for manufacturers related to timing and business operations for both FDA programs. Once a manufacturer has qualified for the Breakthrough Devices or STeP Program, they should determine what benefits they wish to gain and begin a communication strategy and plan with the FDA.
The primary (and most helpful) benefit of these programs is timely communication and interaction with FDA, as well as:
- The FDA readily makes staff available, including FDA Senior Management, to address manufacturers’ questions.Sprint discussions with the FDA are available to the manufacturer, with a more timely cadence (<45 days) than a Pre-Submission (~75 days). The Pre-Sub may still be used for complicated reviews while reserving the sprint discussions for specific questions. For example, a Pre-Sub is commonly recommended to review the clinical trial protocol while endpoint discussions may be part of the sprint conversations.
- Early Engagement on Data Development Plans (DDPs): The DDP is a “high-level document” that maps out expected non-clinical and clinical data collection needs and, as appropriate, establishes the level of uncertainty that the agency is willing to accept in the premarket phase. This could allow manufacturers to push more data requirements to the post-approval phase.
- More efficient and flexible clinical study design, which considers:
a. Pre-specified endpoints to determine the minimum clinically meaningful effect
b. Immediate and surrogate endpoints
c. Composite endpoints
d. Adaptive study designs
Note that while these program aspects do not speed up the overall “FDA clock” (a 510(k) still has a 90-day timeline); these programs may limit the “stops” to the clock along the way.
Another important note is that the traditional Pre-Sub may still be utilized for complicated reviews; one common reason for a Pre-Sub is to review the clinical trial protocol. Although specific questions (such as endpoint discussions) may be part of the sprint conversations.
On January 14, 2021, the Center for Medicare and Medicaid Services (CMS) introduced a change to reimbursement benefits for devices accepted under the Breakthrough Devices Program. This new policy, expected to cause a likely increase in adoption of the Breakthrough Devices, was sidetracked when on March 12, 2021, CMS postponed the implementation of the proposed framework. This framework, “Use of Medicare Coverage of Innovative Technologies (MCIT),” ultimately delayed the immediate Medicare reimbursement for devices authorized through the Breakthrough Devices Program. As a result of this delay, the impact on the claims process for breakthrough technologies remains to be determined.
Breakthrough devices subject to Pre-Market Approvals (PMAs) are not absolved from providing reasonable assurance of safety and effectiveness through the rigorous examination of pre-market and post-market data. However, as part of its benefits-risks determination, the FDA will accept timely post-market data for breakthrough devices to fast-track development and review.
Interest from Investors
Investors want to be assured that a company’s products have value to both patients and physicians; they also want to see a return on their investment. A Breakthrough or STeP device designation gives investors added confidence in a company, and if the FDA has recognized your product as one that will advance public health and/or offer superior treatment, investors are more likely to take a chance on your organization.
When to Submit for Breakthrough or STeP
NAMSA has helped many companies seek Breakthrough and STeP statuses, and assisted them with regulatory strategy to assess the correct timing for seeking designation. While the main cornerstone for these programs is the demonstration of significant benefit to the standard of care treatment, a device must demonstrate these benefits through some level of testing.
Often, it is helpful to have an initial Pre-Sub with FDA to introduce them to the device and to address any high-level concerns with regulatory and testing strategies. In parallel, the Breakthrough or STeP Pre-Sub meetings can be built using the FDA guidance as the framework.
A Breakthrough or STeP Pre-Sub will go through an acceptance review, beginning with an initial review within 30 days of receipt. It is during this time that the FDA seeks additional information, if needed, to provide a final determination within 60 days.
Based on the 30-day review from FDA, a manufacturer will gain a clearer understanding regarding the viability of their request.
- STeP is very new and not all FDA divisions have experience with this program; therefore, the process may initially be more burdensome.
- A manufacturer can choose to withdraw from the program at any time, including after the initial 30-day review.
- As long as there are no cleared/approved devices available, you can seek Breakthrough status. For example, there are multiple breakthrough devices for the same indication, but all of these devices are in the IDE stage.
- Manufacturers must demonstrate with robust data that a device does what it claims before submitting. Preliminary bench data with feasibility units may not be robust enough for FDA.
- The door is not closed if you do not qualify for Breakthrough or STeP Programs as a manufacturer can reapply when they have access to more data or modify an indication for use, etc.
- A manufacturer cannot bounce between the Breakthrough Devices and STeP Programs. For example, if an organization doesn’t qualify for the Breakthrough Devices Program, they do not automatically qualify for the STeP Program. It is recommended that manufacturers request a breakthrough designation before starting the pivotal clinical trial to take full advantage of flexible study design opportunities.
The FDA reserves a Breakthrough Devices designation and STeP for select medical devices and diagnostics that serve an unmet need and/or provide substantially more effective treatment or diagnosis for debilitating and life-threating conditions. The path to approval requires intense focus on data and interaction with the FDA, and the benefits via expedited review and improved interaction are worth the effort.
How Can NAMSA Help?
NAMSA is the industry leader in driving successful regulatory outcomes through effective interactions with the FDA. In fact, our internal teams of medical device development experts communicate with the FDA nearly every day. From Pre-Submission meetings – to Pre-IDE preparation – and FDA inspection preparation, our teams are the most experienced in industry at accelerating regulatory submissions and approvals for device manufacturers. This expertise has been proven to save medical device organizations up to $17M in costs and 23 months in development timelines.
If you are interested in speaking with us about FDA-related activities or other global regulatory strategies, please contact us at: https://www.namsa.com/contact-us.
Kirk currently serves as a Principal Product Development Strategist at NAMSA. He is responsible for providing support and consulting to clients pursuing development of innovative medical technologies. With over 20 years’ experience in the medical technology industry, including multiple start-ups, Kirk’s background includes expertise on all phases of device development, global regulatory strategy and market access, including preclinical, clinical, approval and post-market. He has built and supported numerous regulatory submissions, new product development strategies, presentations and market assessments.
Monica R. Montanez
Monica R. Montanez, MS, RAC, CQA currently serves as NAMSA's Principal Strategy Consultant. Monica has over twenty years’ experience in the medical device industry in Regulatory Affairs and Quality Assurance. Her primary focus is navigating the regulatory pathways for electro-mechanical and software driven medical devices worldwide. She has received clearance of many 510(k)s and approval of new indications for PMA device(s) of which 90% involved software. She has broad regulatory expertise in several areas of digital health, including: Software in a Medical Device (SiMD), Software as a Medical Device (SaMD), mobile medical apps, clinical decision support software, telehealth, artificial intelligence, machine learning, interoperability, cybersecurity and human factors engineering, including wireless medical devices -radio frequency (RF), electromagnetic compatibility (EMC) and electromagnetic interference (EMI). While in industry, she assisted in the development of FDA 510(k) guidance and FDA Software guidance directly with FDA. Monica holds a Masters of Science (MS) degree in Regulatory Science (RS) from the University of Southern California (USC) School of Pharmacy. Currently. she holds Regulatory Affairs Certification (RAC) from the Regulatory Affairs Professionals Society (RAPS) and Certified Quality Auditor (CQA) from the American Society for Quality (ASQ).
Angela Mallery, EdD, is a Principal Product Development Strategist at NAMSA with over 25 years of experience working in device regulatory affairs in small, medium, and large sized companies. Angela has experience in project management, regulatory strategies, pre-submission meetings; and has hands-on experience developing, writing, and receiving clearance/approval for 510(k), IDE submissions and reports, PMAs, CEP/CERs, and Technical File/Design Dossier submissions. Angela’s therapeutic experience includes cardiology, neurology, peripheral vascular devices, general surgical devices and orthopedic implants.