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Top FDA Milestones and Controversies Under Gottlieb's Watch

Friday marks Scott Gottlieb's final day as FDA commissioner. Here is a review of the key regulatory milestones and controversies that occurred during his time as the agency's 23rd commissioner.

  • Scott Gottlieb, MD, was sworn in as the 23rd commissioner of FDA on May 11, 2017. The medical device industry was quick to throw its support behind Gottlieb after he was picked by President Trump to run the agency.  Gottlieb rattled the industry a month ago with news of his resignation, but nobody can accuse Gottlieb of phoning it in during his final weeks at the agency. He has released 18 official statements since announcing his resignation, and there has been a flurry of other agency activity in that time as well.

    MD+DI / Amanda Pedersen
  • FDA released a five-pronged Medical Device Safety Action Plan in April 2018, 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," Gottlieb said at the time.

    During the public comment period, which ended Aug. 17, 2018, AdvaMed and Johson & Johnson 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).

    Here are 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.

  • FDA came under fire earlier this year when an investigative report claimed the agency is hiding millions of adverse event reports from the public under what is known as the alternative summary reporting program.

    Kaiser Health News reported that at least 1.1 million incidents have been reported through the alternative summary reporting (ASR) repository instead of being disclosed through the public database known as MAUDE.

    But just last week Gottlieb tweeted that all of the adverse event reports would be made open to the public.

  • Image courtesy of Netflix

    Early last year Gottlieb met with a group of women informally known as the E-Sisters regarding their concerns about Bayer's Essure birth control implant.

    "Some of the women I spoke to went on to develop significant medical problems that they ascribe to their use of the product," he said in a statement issued about a month after the meeting. "As a physician, I know that patients are uniquely positioned to provide feedback to the FDA about their experiences with currently available medical products, and I appreciated the thoughtful discussion. Ensuring the safety and effectiveness of medical products is paramount to the FDA and a core part of our consumer protection role. The FDA takes concerns about Essure very seriously."

    The E-Sisters were not impressed with the commissioner's words.

    "Commissioner Scott Gottlieb penned an industry-friendly response attempting to justify the weak actions taken by the FDA," Holly Kelly Ennis, a partner at the law firm Ennis & Ennis, told MD+DI.

    In April 2018, however, FDA turned up the heat on Bayer to make sure women who are considering the implantable contraceptive device are adequately informed of its risks.

    Bayer stopped selling Essure in the United States at the end of last year, citing a decline in U.S. sales of the device and the conclusion that the Essure business is no longer sustainable.

    Although the controversial product is the subject of nearly 30,000 adverse event reports to FDA and thousands of patient lawsuits, Bayer insisted in a statement that its decision to discontinue sales of the product is for business reasons only, "not for any safety or efficacy concerns about Essure."

    FDA took steps late last year to make sure the long-term safety profile of Essure continues to be studied even after U.S. sales of the device are discontinued at the end of the year.

  • Image courtesy of Netflix

    FDA endured some pretty harsh publicity hits in 2018 under Gottlieb's watch. 

    The biggest blow came in July following the Netflix release of The Bleeding Edge documentary, which offered a poignant look at specific medical device categories that have adversely impacted patients. The film pointed to perceived faults in the way medical devices are regulated in the United States.

    Later in the year, an investigative report on the industry that blamed poorly regulated medical devices for millions of patient injuries and thousands of deaths. The report was based on a year-long investigation by the International Consortium of Investigative Journalists.

  • Gottlieb put the wheels in motion for the agency to make some long overdue changes to the 510(k) clearance program, which was adopted in 1976 and now accounts for the majority of medical devices the agency reviews. Those changes are likely to include actions to retire outdated predicates, especially in cases where safer or more effective technologies have emerged.

    In January, FDA issued final guidance establishing the framework for what is now being called the Safety and Performance Based Pathway. The guidance was previously dubbed Expansion of the Abbreviated 510(k) Program: Demonstrating Substantial Equivalence through Performance Criteria.

    Modernizing the 510(k) pathway falls under the five-pronged Medical Device Safety Action Plan described on slide 2.

  • FDA forged a new breakthrough pathway for medical devices under Gottlieb's leadership.

    What FDA says: The new pathway enables manufacturers to work with FDA on managing submissions for medical devices that are granted the new breakthrough designation. The voluntary program supersedes the agency’s existing programs, including the Expedited Access Pathway (EAP) and the Priority Review Program, while combining some aspects of the EAP and Innovation Pathway.

    Benefit: This program will allow breakthrough devices priority attention, thereby expediting their assessment and review over other devices. The priority attention given to breakthrough devices will help patients have more timely access to these products.

    Challenge: While breakthrough devices are granted priority review, they are not guaranteed a faster review when compared with standard review timelines. Breakthrough devices may take longer to review due to the potential scientific challenges FDA has historically encountered with novel products. Reviewers may have to invest time in understanding the mechanism of action and the appropriate evidence to demonstrate safety and effectiveness.

  • In 2017 FDA introduced the pre-cert concept, a potential regulatory program for mobile apps and medical devices connected digitally to medical technology. Gottlieb tweeted recently that the program, which FDA is currently piloting, may be better suited to technologies that receive continuous software updates.

  • During his time as commissioner, Gottlieb began posting what he dubbed Sunday Tweetorials, which were Twitter threads that discussed current issues the agency was tackling. In his final Sunday Tweetorial, posted March 31, he looked back on the agency's accomplishments over the past two years. One tweet touched on a couple of major crises situations the agency dealt with under his leadership.

    The first crisis came in 2017 when Puerto Rico’s medical device plants were heavily impacted by Hurricane Irma and Hurricane Maria, which slammed into the island in September 2017 and crippled its electric power grid. During that time, FDA worked closely with 10 medical device manufacturers to prevent shortages of about 50 devices that are life-sustaining, life-supporting, or have only one source.

    More recently, the agency found itself directly impacted by a historic government shutdown. While the agency did continue vital activities that are considered critical to ensuring public health and safety, Gottlieb was clearly frustrated by the negative impact the shutdown had on his staff.

    "You’re valued each day," Gottlieb Tweeted to FDA staff during the shutdown. "But it’s even more the case that in moments of challenge, when some of our work stops, that your critical role becomes so obvious. FDA can’t achieve its full mission without each of you; whether you’re exempt, excepted, or on furlough. We’re one FDA."

    "Thank you for your understanding and support," replied a Twitter user with the handle Larry Stringer. "I’m stuck at home, furloughed and reading your tweets. I can’t even look at e-mail to see what I’m missing. I want to come back to work. I love my job and believe in our public health mission."

  • Throughout his time as the 23rd commissioner of FDA, Gottlieb was passionate about addressing the ongoing opioid crisis in America. 

    “While we continue to pursue better medicines for the treatment of opioid use disorder, we also need to look to devices that can assist in this therapy,” Gottlieb said in November 2017.

    Gottlieb said FDA supports the development of both drug and device-based treatments that can be used to address opioid dependence or addiction, as well as new, non-addictive treatments for pain that can serve as alternatives to opioids.

  • Gottlieb has also put the wheels in motion for a new regulatory framework for reviewing medical devices that use advanced artificial intelligence algorithms.

    "Artificial intelligence has helped transform industries like finance and manufacturing, and I’m confident that these technologies will have a profound and positive impact on healthcare," Gottlieb said. "I can envision a world where, one day, artificial intelligence can help detect and treat challenging health problems, for example by recognizing the signs of disease well in advance of what we can do today. These tools can provide more time for intervention, identifying effective therapies, and ultimately saving lives."

    The first step toward developing a tailored approach to reviewing these technologies comes in the form of a 20-page discussion paper. Down the road, the industry can expect to see draft guidance based on input the agency receives on the paper.

  • As part of its effort to modernize its regulatory programs, FDA also has turned its attention to materials used in medical devices. 

    The agency released a joint statement in March from Gottlieb and Jeff Shuren, director of the Center for Devices and Radiological Health, that addresses recent advances in materials science and how these advances have impacted the safety of medical devices.

    "Materials used in today's medical devices vary as widely as the devices themselves—whether the material is metal, plastic, silicone, an animal-derived product or some combination of these," Gottlieb and Shuren noted. "Because, in the case of implantable or insertable devices, these materials come into contact with tissue or other parts of the body for sometimes extended periods of time, we do a careful evaluation during our premarket review to determine if there is a potential adverse biological response resulting from contact of the device’s component materials with the body and whether the associated risks are unacceptable."

    The vast majority of patients implanted with medical devices have no adverse reactions, according to the statement. "However, a growing body of evidence suggests that a small number of patients may have biological responses to certain types of materials in implantable or insertable devices," they said.

    These patients may develop inflammatory reactions and tissue changes causing pain and other symptoms that may interfere with their quality of life, Gottlieb and Shuren said.

    Specifically, the agency is looking at materials and implantable devices that have a troublesome track record of creating safety concerns for some patients. This includes silicone breast implants; metal; nitinol-based devices including the highly controversial Essure birth control device; and metal-on-metal hip devices.

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