Bringing medical devices to market in the United States requires working through the regulatory processes overseen by the U.S. Food and Drug Administration (FDA). The FDA provides a large number of resources and programs to industry to ensure medical devices entering the market are safe and effective. One such resource is the agency’s specialized program that provides least burdensome regulatory policies to industry.
The principles of the least burdensome approach apply both to the FDA and to the MedTech industry. While the FDA focuses on the review of clinical study protocols and market access requests (IDE, 510(k), PMA, HDE), the intent to require the minimum amount of information necessary to make an informed determination—the FDA expects industry to submit clear, concise, logically structured and well thought-out information for their review.
To assist with this process, the FDA issued a final guidance on February 5, 2019, “The Least Burdensome Provisions: Concept and Principles,” which establishes the definition and requirements of the “least burdensome” regulatory approach for industry.
The U.S. FDA’s mission is to protect and promote public health in the United States. This mission embraces bringing new medical technology to the market as efficiently as possible by streamlining regulations and reducing unnecessary burdens associated with regulatory activities while upholding stringent standards of review and scientific integrity.
The least burdensome regulatory approach applies across the total product lifecycle and enables the FDA to maximize resources on the issues of the highest public health concern. Through the Least Burdensome guidance document and its predecessors, the Agency has enacted three overarching, least burdensome concepts which include provisions for 510(k) reviews, clinical study design and Pre-Market Approval (PMA) reviews. Of significance is that this approach, while intended to lower regulatory burden and speed reviews, does not change the Agency’s rigorous standards for PMA or substantial equivalence.
The Least Burdensome Core Concepts
The definition of “least burdensome” is the minimum amount of information necessary to adequately address any relevant regulatory question or issue through the most efficient manner and at the right time.
This definition has three prime concepts:
- Minimal Information (the minimum needed to make a determination)
- Efficient Manner (flexibility in answers to regulatory procedure questions)
- Burden at the Right Time (in light of the phase of the total product lifecycle)
The ‘Minimal Information’ concept supports the idea of leveraging existing data and real-world evidence, including data gathered outside of the United States. The use of non-clinical data in lieu of clinical data is also embodied by the minimal information requirement concept. In addition, non-clinical data not only means bench testing and In Vivo studies, but is also meant to include computer modeling and simulations.
The ‘Efficient Manner’ concept focuses on weighing the probability of benefits and risks (harms) of allowing a medical device into the market. This means that a positive decision may be issued with respect to a device even if it has probable risks. Efficiency also advances the idea that administrative burdens should be reduced and promotes the utilization of electronic filings.
The ‘Right Time’ concept emphasizes the importance of calculating “the right balance between clinical trial and pre-market information and post-market information needs.” This promotes the idea that in certain cases, safety and effectiveness questions may be answered appropriately through post-market data capture, analysis and reporting.
The ‘Right Time’ guidance applies to all activities pertaining to the regulation of medical devices, including both pre-market and post-market actions and also device components of combination products. However, least burdensome does not alter the statutory criteria for substantial equivalence or a reasonable assurance of safety and effectiveness. And, least burdensome does not alter the requirement for valid scientific evidence to support decisions. With this in mind, the FDA follows seven guiding principles to apply least burdensome methodologies to pre and post market oversight activities.
Seven Least Burdensome Guiding Principles
- The FDA requests the minimum information necessary to adequately address a regulatory question.
- Industry submission materials should be least burdensome for the FDA to review and must only include relevant information, utilize pertinent FDA guidance documents and be well-organized, clear and concise.
- The most efficient means will be used by the FDA to resolve regulatory questions, which include streamlining procedures, using both formal and informal interactions, use of tailored approaches to address issues and to consider resource and time requirements of information requests to industry.
- The right information should be provided at the right time (just-in-time data collection) to address the right questions (i.e., Can a post-market study reduce the pre-market study data collection burden?).
- Regulatory approaches should be designed to fit the medical device technology and account for unique innovation cycles, evidence generation needs and timely patient access.
- Data and regulatory actions from other national medical device regulatory agencies is to be leveraged by the FDA to the extent appropriate and feasible.
- FDA will converge, harmonize and engage in the development, recognition and use of voluntary consensus standards published by international and other standards development organizations.
Positive Outcomes for Industry
The Least Burdensome approach to regulatory review is a great benefit to industry, and the American public alike. Since the FDA implemented the final ‘Least Burdensome Provisions’ guidance, industry has leveraged the program through increased use of international clinical data and non-clinical data while pursuing new indications and innovations. Efforts within the FDA to allow for electronic filings and to provide routes for issue resolution and increased interactions mechanisms have been embraced by industry.
The guidance has also given industry a new “flag” tool to use during FDA review of a marketing submission. The flag allows a sponsor to request an informal review of reviewer requests for information that the Sponsor believes are not least burdensome. Although not a first line action when negotiating a market application, the use of the flag might be leveraged when regulatory reviews seem to require unreasonable data requests.
Bringing a new medical device to market is a complex and time consuming process. By the time evidence for a device market application is ready to be submitted to FDA, truly understanding how the FDA will view that evidence is critical. The principles provided in this guidance offer useful tools to assess evidence requirements and information on how to effectively navigate the regulatory review process in the United States.
How Can NAMSA Help?
Navigating the FDA regulatory landscape can be overwhelming for any medical device manufacturer—not to mention very cost-intensive. That’s why having the right partner, at the right time, can be invaluable in achieving regulatory requirements, market success and accelerated timelines.
Are You a MedTech Sponsor:
- Seeking to understand clinical strategies that utilize foreign clinical trial data?
- Wondering about the use of early feasibility IDE programs?
- Wishing to understand how to use a balanced approach of pre-market and post-market clinical data strategies?
- Wanting guidance on when to use the least burdensome flag?
NAMSA is here to help. We are the industry leader in driving successful regulatory outcomes through effective interactions with the FDA and can help guide conversations and interactions with the FDA related to the Least Burdensome pathway and test plans. 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 and SaMD/AI/ML reviews, our teams are the most experienced in industry at accelerating regulatory submissions and approvals for device manufacturers. This expertise is 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. Or, learn more about our development experts by visiting: https://namsa.com/namsa-expertise/subject-matter-experts/
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.