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  1. Speeches by FDA Officials

Speech | In Person

Event Title
Advancing Our Partnership for Patient Safety and Med Tech Innovation
September 5, 2018

Speech by
Scott Gottlieb, M.D.

Medical Device Innovation Consortium 2018 Annual Public Forum

Washington, DC

(Remarks as prepared for delivery)

Thank you for having me here today.

As all of you know well, we’re seeing an unparalleled period of innovation in medical devices. These opportunities are being driven by advances in material science, in digital technology, artificial intelligence, and in cutting-edge manufacturing.  And they’re offering patients new chances to improve their health.

As one measure of this progress, las year the FDA approved a record number of novel devices. And we’re encouraged by the pace of development and innovation we are seeing this year.

We’ve advanced new policies to help efficiently promote this innovation in medical product development. And also secure device safety. Innovation and safety can and must go hand-in-hand. That’s why we’re focusing equal attention on advancing new frameworks for identifying risks and protecting patients.

Dr. Jeff Shuren, Director of the FDA’s Center for Devices and Radiological Health, recognized early on that a key to advancing these goals is through collaborations that promote efficient, science based regulation. By working with others, we can help deliver innovation more quickly, while also strengthening the FDA’s assurance that devices meet our regulatory standards.

This vision drove the launch of the Medical Device Innovation Consortium. The idea was to create a dedicated, transparent forum where public and private stakeholders can come together to advance science based principles for streamlining the development of innovative medical devices and diagnostics; to solve complex regulatory, scientific, and reimbursement challenges around med tech; and enhance our understanding of the risk-benefit and safety of these products to protect patients.

These collaborations also can advance policies that promote access. We want patients to receive the care they need, when they need it. If the right care comes from a new technology, we want our efforts to promote affordable patient access to these novel devices. That means taking steps to encourage competition that can help reduce prices. And it means taking steps to reduce the cost of product development where we can, while strengthening the FDA’s gold standard for review.

Toward fulfilling these and many other goals, the FDA remains committed to our MDIC partnership. It’s a partnership that’s already paying dividends for patients. And we have important opportunities to enhance our work together.

Today, I’d like to announce two new FDA initiatives that we believe will leverage the MDIC’s efforts, by expanding payor coverage discussions and clarifying further, our approach to benefit risk decision making around innovative med tech.

As we modernize the medtech development process, and our tools for regulatory decision making, we’re seeing that more of the barriers to access and innovation are shifting from the pre-market process to the need for efficient coverage decisions.

I’m concerned about patients not having timely access to the latest advances in medical devices because of the complexity of getting coverage in place, and the cost of paying for new innovations. Timely patient access to beneficial devices is essential to treating -- and curing -- disease and suffering.

For most Americans, however, FDA approval or clearance does not equate to access. Patients can face challenges while they wait for private insurers to make coverage and reimbursement decisions. That process can sometimes take months, or years.

These delays can reduce incentives to innovate. This is especially true when it comes to small, innovative medtech companies that can be idea rich, but cash poor. They’re not able to float themselves while they wait for long periods to gain market entry.

Now insurers have every right to demand good data to support their coverage decisions. And they have an obligation to be good stewards of the healthcare needs of their beneficiaries. Insurers should be careful how they cover new technology to make sure they’re getting the most healthcare bang for the premium money that their beneficiaries entrust them with.

But if there are steps that the FDA can take to make that coverage process more efficient, and to make sure payers have the data they need to enable efficient access to beneficial new technologies, we want to do our part to make these opportunities available. Access is a matter of public health. And by taking measure of these challenges, we believe there are ways that the FDA can better align our own regulatory processes to meet more of the needs of the broader marketplace.

So to help address these opportunities, the FDA has launched a Private Payor Program as a voluntary opportunity for manufacturers to receive feedback from the FDA and other non-governmental health technology assessors and payors. The idea is to enable manufacturers to develop a more efficient evidence generation strategy during the FDA’s pre-submission meetings.

We’ve been encouraged by the early results of this program. And today I’m pleased to announce that CareFirst and United Health Group -- two of America’s largest private insurers -- have joined the FDA’s growing list of private payors available to participate in these pre-submission meetings with device manufacturers.

By enabling early dialogue between device makers and payors, the FDA hopes to shorten the time between FDA approval and coverage decisions, particularly for new and innovative devices.

There are currently eight private payor or health technology assessment organizations participating in this program. They include CareFirst BlueCross BlueShield, the BlueCross BlueShield Association, Duke Evidence Synthesis Group, the ECRI Institute, Humana, Kaiser Permanente, the National Institute for Health and Care Excellence in the U.K., and United Health Group. This program continues to gain momentum. We now receive, on average, one product inquiry a week as part of this effort.

We continue to support and encourage the collection of clinical evidence that’ll not only support regulatory decisions but also help inform coverage decisions. And we continue to pursue policy efforts that simultaneously advance these twin goals.

To further promote timely coverage decisions, earlier this year we decided to include medical devices in our final guidance on Communications with Payors, Formulary Committees, and Similar Entities.  We wanted to ensure that device manufacturers are included in the FDA’s policies that address the sharing of important economic information about medical products.

But as I said at the outset, our policies serve a twin  purpose. We’re focused on ensuring efficient, timely access to new technologies; but also to making sure that the same policies that promote these goals also make our regulatory programs more rigorous, more science based, and more effective. Promoting product safety is a touchstone for all of our new policy efforts.

I want to highlight some other new efforts we’re announcing today to concurrently achieve these twin goals.

When it comes to new device innovations, we know that we can never have absolute certainty about all aspects of how a new product will perform. We know that no reasonably sized pre-market trial can ever be expected to reveal everything that could eventually become known about a novel medical product.

To address these truths, the FDA is releasing a new draft guidance, that’ll lay out our framework for how we systematically address uncertainty in making benefit and risk determinations in our pre-market approval process, in de novo classifications, and evaluating humanitarian device exemptions.

Along with other similar and recent FDA initiatives; the new draft guidance issued today advances a rigorous, methodical, and science-based approach for how the FDA considers uncertainty as part of its benefit-risk determinations. The aim is to support premarket decisions that are based on the totality of scientific evidence available at the time of a device’s market entry.

By providing a more systematic review to how we evaluate and address uncertainty -- and how we define a clear, consistent, and science-based extent of uncertainty -- we believe that this sort of transparency can help improve device development programs. This is especially true when it comes to innovation in treatment options for small patient populations, such as medical devices targeted to rare conditions or pediatric patients. It also could lead to more first-in-the-world approvals for U.S. patients.

We believe that the  path to achieving all of these goals lies in acknowledging and appropriately addressing uncertainty in benefit-risk determinations to support certain FDA premarket decisions. It lies in acknowledging that FDA decisions operate in the context of a broader healthcare system, where there’s uncertainty in the provision of health care, including how the general benefit-risk profile of a given medical product or procedure will translate to an individual patient’s health outcome, and a continually evolving standard of care.

Building on work that the FDA’s device center has already done to bring clarity and consistency to premarket benefit-risk decisions, the guidance describes the factors for evaluating uncertainty when we apply the standard of reasonable assurance of safety and effectiveness. These can include the availability of alternative treatments; public health need; feasibility of evidence generation premarket; the ability of a sponsor to be able to reduce or resolve uncertainty in the post-market setting; patient perspectives; and other factors.

For instance, consider a device intended to treat a relatively rare condition, where it’s infeasible or highly resource intensive to generate extensive clinical evidence in the premarket setting.

What if the disease is serious, but not life threatening or irreversibly debilitating? What if there are no alternative treatments available? Considering the valid scientific evidence within this framework, greater uncertainty about the benefit-risk profile of the device may be appropriate for premarket approval, provided appropriate post-market data collection, accountability, and other robust post-market controls are in place. The draft guidance we’re releasing today will bring greater transparency, predictability, consistency, and efficiency to this type of pre-market analysis and decision-making. We’ve long made these kinds of considerations as part of pre-market review. The aim of this new guidance is to distill these considerations into an objective framework. And to write it down.

The FDA is working continuously to modernize our organization and review processes. We want to ensure that we remain a bridge, not a barrier, to beneficial medtech innovation. We’ve modernized our organizational structure to take a Total Product Life Cycle strategy for assuring product safety and effectiveness. Using the least burdensome approach, and new authorities like the breakthrough device designation, we’ve approved, cleared, or authorized more novel devices that can improve the lives of patients. We’re exploring how we can broaden and improve the use of RWE for expanding additional device indications. And in the past year, we were also the first regulatory agency in the world to issue a guidance to advise manufacturers on the technical aspects of 3D printing, called additive manufacturing. And we’re taking other new steps toward achieving these goals.

To advance these goals, tomorrow we’ll also be releasing a new “QUiK Review” pilot. Under this new approach, we expect to reduce our device center’s review time for certain well understood, lower-risk 510(k) submissions by as much as 30%  -- from a maximum of 90 days down to 60 days – by allowing us to receive and evaluate information much more efficiently.

This new approach is like a Turbo Tax for information submitted in 510ks. It will modernize the premarket submission process for medical devices by allowing applicants to submit information to us using a more streamlined electronic format. The structured format will also improve the consistency of our review.

Taken as a whole, this new process will improve our overall productivity, enabling the agency’s review staff to put more of our time and resources into evaluating applications for devices that pose the highest potential risks to patients.

One of those high priority areas of risk that the FDA is actively confronting, in both the pre- and post-market settings, is medical device cybersecurity. In recent years, the FDA, manufacturers, and healthcare organizations have made significant progress to address and improve the cybersecurity of medical devices. But this is a rapidly evolving space. We know we all must keep pace.

In the coming months, the FDA will share our progress on a number of actions to help strengthen device cybersecurity. Our new efforts are aimed at both protecting against attacks and enhancing response to cyber vulnerabilities or actual incidents.

This fall, the FDA plans to update our cybersecurity pre-market guidance with recommendations for how manufacturers can protect against moderate and major risks. These include things such as ransomware campaigns that disrupt clinical operations, as well as exploits involving a remote, multi-patient attack.

In addition to these actions, we have several other initiatives already underway with MDIC that we believe will have a meaningful impact on medtech development and patient safety.

One of those initiatives is how MDIC is helping us advance the use of real world evidence. Building consensus standards on how best to translate real world data into real world evidence for regulatory decision making can lead to earlier patient access to safe and effective technologies. And so to help support the integration of RWE throughout the total product life cycle, we’ve awarded a contract to MDIC to manage the National Evaluation System for health Technology coordinating center (NESTcc).

This is a key effort for the FDA.

NEST’s objective is to increase the value and use of real world evidence to address the needs of all stakeholders.

As we’ve outlined in the FDA’s Medical Device Safety Action Plan, one important role for NEST is to serve as a robust patient safety net for med tech in the U.S. through active surveillance.

The NESTcc has established relationships with 11 data partners, 150 hospitals, and thousands of outpatient clinics. Taken together, they represent nearly 470 million patient records.

NEST’s data partners will conduct testing to assess their system’s capabilities for addressing critical RWE questions across the total product life cycle. We believe manufacturers will be able to recoup their investments through a competitive marketplace that values safety and quality. And so, toward achieving this end, MDIC and CDRH are working together to create a “Case for Quality” as part of FDA’s Medical Device Safety Action Plan.

Clear performance metrics for safety outcomes and organizational quality maturity can help pro-active manufacturers gain greater market share, while pushing the rest of the industry to catch up to the best-in-class quality products.

This is how our focus on safety and quality can be a win-win, advancing the health and safety of patients and helping to promote a more inclusive business model for innovators.

This new project will be supported by the FDA’s Voluntary Manufacturing and Product Quality pilot that was launched earlier this year. This product pilot uses a maturity model approach to advance device quality that complements the agency’s current quality systems and inspection framework.

Through the pilot, we’ve already seen a ROI for participants who invest in quality systems, ranging from $65,000 to $1.2 million. To take one example I know of; a small manufacturer improved clinical trial intake times from six months to just 28 days.

That brings me to one of the biggest perceived bottlenecks in device innovation: the high cost and administrative inefficiencies associated with modern clinical research. Too many clinical trials have become overly complex and cumbersome, driving up costs without offering commensurate improvements in safety. 

This can push clinical trials outside the U.S., forcing American patients to wait longer for treatment with cutting edge medical devices.  It can also put the U.S. at risk of losing its leadership in this fast-moving sector. We need to modernize clinical research, making it more patient friendly and more efficient.

The FDA’s device center has already made important strides in this area. And we’ve seen a 50% increase in Investigational Device Exemption submissions from 2015 to 2017. This owes to the Device Center’s improved processes and policies.

But there’s a lot more work to be done. We want to continue to position the U.S. as the world’s leader in establishing standards for clinical excellence and medical technology innovation by encouraging improvements in the way medical research is conducted. And the MDIC’s Clinical Trials Sciences initiative is focused on reducing barriers to early feasibility studies — the most cutting edge clinical research — to bring this critical early experience back to U.S. healthcare facilities. 

Earlier clinical experience translates into earlier patient access to safe, effective, and innovative medical technology.

To streamline EFS studies and promote U.S. patient access to novel devices, MDIC is launching a clinical trial site consortium to improve the process and help reduce barriers to conducting EFS studies. Consortium sites are committing to a 60-60-60 campaign—completing site contracts, IRB reviews, and enrolling their first patients within 60 days of a sponsor EFS submission.

This will allow research facilities to stand up these studies much more efficiently. By committing to timeframes and standardizing best practices, U.S. research institutions will be more attractive to innovators looking to launch studies of their products.

Of course, not every question surrounding a device or diagnostic must be answered with a clinical study. Computer modeling and simulation has the potential to revolutionize clinical testing and product development by developing in silico models that balance the desire for certainty in evaluating device performance with limiting associated delays in patient access.

To advance these goals, MDIC Virtual Patient project has already demonstrated savings by limiting the size of a clinical trial that may be needed to demonstrate safety and effectiveness. It uses computational modeling to create virtual patients to be used as Bayesian priors. A retrospective analysis showed a potential reduction in clinical study size of 15 to 50% across different device and diagnostic areas using in silico patient models.

To help the broader community gain familiarity with these models, the MDIC Virtual Patient team sent a mock submission to FDA to get official feedback on the proposed method. They posted their entire submission and FDA review memos online.

The model was also published in peer-reviewed literature to explain the statistical methodology behind the Virtual Patient, and the underlying software is publicly available.

Finally, to promote efficiency in clinical development, MDIC is also helping the FDA advance the Science of Patient Input.

MDIC shares our goal of improving patient health and quality of life by enabling us to better understand  patients’ needs, experiences, and preferences across a variety of disease states.

MDIC’s work in this area includes three projects that’ll expand our early success in Patient-Centered Benefit Risk. FDA guidance on incorporating patient preferences into benefit-risk determinations was bolstered by the rich scientific resource MDIC provided to the MedTech ecosystem in its accompanying Framework. It included the creation of a compendium of methods for assessing patient preferences. This framework has been downloaded more than 1,000 times. It’s been cited across the field. And it’s impacted the FDA’s decision making for products related to obesity, renal disease, and diabetes.

These are just a few of the examples of our shared work with MDIC, and our shared public health values. And the new initiatives that I announced today are just some of the steps that we’re taking at the FDA to build on these universal goals.

Together, the FDA and our public and private sector partners like MDIC are creating a modern framework for medtech innovation.

Our shared vision is an ecosystem where we provide patients with timely and efficient access to safe, effective innovations – advances that can help them restore health and regain function.

Thank you.

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