In April, FDA released a five-pronged Medical Devices Safety Action Plan, full of rhetoric aimed at convincing the public that this new plan will enhance the agency's commitment to patient safety and recognition that safety and innovation should go hand in hand.
Some aspects of the plan call for new ways to use FDA's existing tools, while others identify areas where the agency "may need additional authority," according to FDA Commissioner Scott Gottlieb, MD.
As with most draft guidance FDA puts forth, the release of the Medical Devices Safety Action Plan came with a public comment period, which ended Aug. 17. AdvaMed and Johson & Johnson were among the industry leaders that raised concerns with some of the specifics of the plan, as did the American Association of Neurological Surgeons (AANS) and Congress of Neurological Surgeons (CNS).
The following highlights five key areas of the plan, what Gottlieb has said about it, plus the specific concerns of AdvaMed, J&J, AANS, and CNS.
1. Establish a Robust Medical Device Safety Net in the U.S.
Gottlieb: We'll establish a robust medical device patient safety net by continuing our work on NEST, or the National Evaluation System for Health Technology. NEST links data from registries, EHRs and billing claims. NEST will facilitate timely detection of potential safety risks that wouldn’t otherwise be identified as quickly, or at all. But it requires addressing variations in data standards and ensuring systems talk to each other.
AANS and CNS (jointly): we were concerned about the somewhat dismissive tenor of comments regarding the existing Medical Device Reports (MDRs) submitted to the Manufacturer and User Facility Device Experience Database (MAUDE) program, which are required of manufacturers, but are voluntary for individual clinicians. The report states, “MDRs rely on a person, often a clinician, to identify that a problem occurred, to realize that the problem may have been associated with the use of a device, and to take the time to report the incident to FDA or the manufacturer.” The AANS and CNS have encouraged its members to submit MDRs when appropriate, and we would argue that individual physicians are uniquely qualified to provide such data to the FDA. We urge the FDA to continue the program and enhance the voluntary mechanism for individual physicians to directly report device safety concerns to the FDA.
2. Explore Regulatory Options to Streamline and Modernize Timely Implementation of Postmarket Mitigations
Gottlieb: We'll consider regulatory options to speed up requiring companies to mitigate safety problems identified postmarket. Currently, mitigations such as new user training or new labeling can require rulemaking, which takes time and resources.
We’re exploring ways to use umbrella regulation or seek authority to address new risks faster. We're pursuing ways to spur innovation in safer devices; creating a Breakthrough Devices-like path and providing streamlined paths for comparative safety claims.
AdvaMed: We understand that FDA’s proposal to impose special controls through an umbrella regulation is preliminary and contains limited details. Still, this proposal raises important concerns. First, it is unclear to us if FDA can implement such an effort under existing authorities. And assuming that FDA has the requisite authority, the merit of FDA’s proposal is unclear. Notice-and-comment rulemaking serves a critical and long-recognized purpose: where agencies propose actions that may have far-reaching and unexpected effects, those agencies must solicit and consider input from stakeholders who will bear the effect of those actions and whose insights may otherwise be unavailable. This is a virtuous process that does not prevent agency action; it simply predicates action on development and consideration of available relevant data.
We, of course, understand that rulemaking may be time-consuming and complicated by bureaucratic process. But we would suggest that the solution is not to unilaterally end rulemaking. Rather, FDA should identify and overcome whatever internal obstacles prevent efficient practice. Our concerns in previous policies proposed by FDA, such as the release of draft guidance on the use of emerging signals in regulatory decision-making, is illustrative. In that case, we raised numerous issues with the proposed policy, including FDA’s failure to seek input from the device manufacturer to evaluate an emerging signal.
Without the opportunity to comment, the evaluation of emerging signals may not have included criteria that would permit the manufacturer to provide the agency with important information. We note that at the time of this submission, our members’ experience indicates that FDA’s current use of emerging signals is subjective and, at times, lacks appropriate input from the manufacturer.
Should FDA proceed with its umbrella-regulation proposal, it should first explain to stakeholders what this proposal entails and how it differs from current practice. We also ask FDA to provide a formal mechanism for stakeholders to comment on the proposal–and for FDA to consider those comments–before proceeding.
3. Spur Innovation Towards Safer Medical Devices
Gottlieb: Some devices come to market after FDA determines that they're substantially equivalent to another legally marketed device called the predicate – this is the 510(k) pathway typically used for moderate risk devices. But predicates may be decades old. Our new “expanded abbreviated” 510(k) path to market may drive greater market competition for safer devices. It explains how objective performance criteria may be used to demonstrate substantial equivalence.
J&J: We support the establishment of a voluntary, more modern 510(k) pathway for demonstrating safety and effectiveness for certain moderate-risk devices ...However, one issue raised in the action plan does not reflect our perspective or experience on device safety. ... FDA states that “[t]he marketplace . . . does not provide strong incentives to make an established device safer in the absence of a new or greater-than-previously-understood safety concern.” For Johnson & Johnson, safety is a significant area of focus in everything we do across our entire product portfolio – including medical devices. As an enhancement to the strong safety practices of all Johnson & Johnson companies, the Johnson & Johnson Office of the Chief Medical Officer (OCMO) was established in 2013. It is a global team encompassing safety colleagues from across all three sectors of Johnson & Johnson: consumer, pharmaceuticals, and medical Device. Functionally independent from commercial and development portfolio interests, the OCMO focuses on the safety of all products across the Johnson & Johnson family of companies. The OCMO takes an evidence- and science-based, ethics- and values-driven approach to medical safety — with an emphasis on transparency. This has led to the development of policies and initiatives that keep the needs of patients and consumers at the center of our efforts. Our approach is reinforced by a proprietary, single medical safety standard, establishing procedures to which all our products are held.
AdvaMed: Safety is the number one priority for the medical device industry. We, therefore, disagree with the plan’s statement that “[t]he marketplace . . . does not provide strong incentives to make an established device safer in the absence of a new or greater-than-previously-understood safety concern.” The plan also suggests that FDA may explore actions that would provide more streamlined pathways for comparative safety claims. Because FDA is not typically involved in reviewing or approving comparative safety claims, it is unclear why a streamlined pathway is needed.
FDA also states that it will seek to establish a voluntary 510(k) pathway for certain moderate-risk devices that would allow manufacturers to use objective performance criteria to demonstrate substantial equivalence. We are concerned with the agency’s implication that this proposed, voluntary program may signal that certain devices are “safer” simply because they use this program. FDA fails to show how use of objective performance criteria, compared to use of a predicate device, would enable comparative claims of better performance or a safer device. Indeed, ample evidence shows the safety of devices marketed based on substantial equivalence to a predicate. We would anticipate that objective performance criteria would reflect appropriate clinical need, that there would be a documented, publicly available record of the basis/support for FDA’s determination of what constitutes appropriate clinical need, and that exceeding performance criteria would not necessarily indicate that a device provides a greater clinical benefit.
We are also concerned that any claims of superior performance based solely on performance criteria, without comparable increases in clinical performance, could be misleading. Accordingly, we believe that, while a voluntary 510(k) program may enhance efficiencies in some cases, such a program must be transparent and consistent and make no qualitative distinctions between devices cleared via predicates or performance criteria.
4. Advance Medical Device Cybersecurity
Gottlieb: We will advance medical device cybersecurity to keep pace with emerging threats and vulnerabilities. This includes updating premarket guidance and considering new post-market authority. We'll have an announcement soon on major new policy. Among our efforts, one approach: the possible development of a public-private partnership – a CyberMed Safety Analysis Board - to complement existing device vulnerability and response mechanisms and serve as a resource for device makers and FDA.
AdvaMed: FDA’s guidance on cybersecurity for premarket submissions states that manufacturers are to submit a summary plan for providing updates and patches when the device is in the market. As a result, device manufacturers already consider these issues during the premarket phase, and FDA reviews the plan prior to clearing or approving the product. It is unclear what more FDA seeks to require considering the existing guidance and expectations that are in place. Nevertheless, we agree that a manufacturer’s risk management process should include steps to evaluate security risks and, when appropriate, implement security risk control measures that may include design features that support updates and patches. To the extent the existing policy described in the premarket guidance is insufficient, FDA should publish revised guidance to inform industry of the agency’s policy. Statutory and/or new legal authorities are not needed.
5. Advance the Use of a Total Product Life Cycle (TPLC) Approach to Device Safety
Gottlieb: We'll integrate FDA’s premarket and post-market activities through a reorganization in our medical device centers, creating a broader and deeper view of device safety, effectiveness and quality across the total product life cycle. Patient safety is at the center of these efforts. Our aim: ensure that devices not only meet the gold standard for getting to market but continue to meet this standard as we get more data and learn more about their benefit-risk profile in real-world settings. These new approaches will also help advance beneficial innovation.