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March 29, 2006 - Scott Gottlieb, MD - Harvard University

 Speech before

The Harvard University Medical Device Compliance Congress

Remarks by

Scott Gottlieb, MD
Deputy Commissioner for Medical and Scientific Affairs

March 29, 2006

This text contains Dr. Gottlieb's prepared remarks. It should be used with the understanding that some material may have been added or deleted during actual delivery.

Thank you for inviting me to speak here today. I think we'd all agree that over the next decade, new medical technologies that integrate engineering and biological approaches, and technologies that enable increasingly precise and effective interventions, will inspire hope for better clinical outcomes with less invasive procedures and shorter recovery times.

The FDA is already seeing a steady increase in the number of requests from device developers for pre-submission meetings to seek advice on the best approaches for scientific and clinical testing and evaluation of cutting-edge technologies such as molecular medicines, including genetic and proteomic diagnostics and therapeutics, as well as products developed using nanotechnology. In fact, we are planning on holding a workshop soon to specifically look at the opportunities offered from emerging nanotechnologies and how the development process for these technologies can be better defined.

Already medical innovations that would have been considered the stuff of science fiction just a few years ago are quickly becoming the standard of care. All you need to do is look at some of the advances that the FDA has recently approved such as drug-eluting stents, and imaging devices that make use of Computer Aided Detection software to assist radiologists in the detection of cancers. Ten years ago such technologies could hardly be imagined and today they are already commonplace in medical practice.

As these technologies advance, and new ones come along, the critical path for turning promising new science and lab discoveries into practical solutions that treat patients may present greater challenges for both innovative device manufacturers and for the FDA. In some places, the current science that we use to assess the safety and effectiveness of these products may not always be optimal for the timely, efficient, and effective development and review of certain highly novel products. Similarly, the ability to develop reliable information to more effectively guide clinical practice and inform peoples' medical decisions must keep pace with the rapidly increasing complexity of the underlying products and the ways doctors and patients use them.

So while the future holds many new opportunities, it also brings with it new challenges. We need to make sure that the pace of innovation in the medical devices themselves doesn't outstrip the ability to efficiently evaluate them for safety and effectiveness and effectively inform doctors and patients about the most appropriate use of these products. We need to make sure that our own approaches to evaluating products keep pace with the expanding knowledge that is embedded in these new devices. We need to continuously develop better ways to predict earlier in the process which new products are likely to be safe and effective in patients and how to best deploy new technologies to maximize benefits and minimize harm. And we need to do all of these things efficiently, so that people are able to quickly realize the benefits from safe and effective new medical devices.

That means that, in particular, we need to ensure that clinical trials, product reviews and approvals, and manufacturing processes are conducted in the most efficient and effective ways. If we don't take steps now -- with so much complexity already entering the device pipeline -- if we don't seek ways to improve the critical path for product development, we may risk squandering some innovations and delaying other safe and effective deices from reaching patients and practitioners. And with so much suffering today that could be mitigated and perhaps even cured with devices just now in development, we simply can't afford to risk not being prepared.

And so at FDA, we plan to soon take additional proactive steps to improve the critical path for device development through an initiative aimed at facilitating technology development. Among the goals of this new initiative is the need to find new ways to move cutting-edge discoveries from the lab bench into clinical trials; the need to modernize the clinical trials process so safety and effectiveness can be assessed at the lowest cost, while protecting human subjects; and the need to apply the most advanced manufacturing methods from other industries, such as semiconductors, to the commercialization and use of medical devices. These kinds of advances have not been as widely adopted in the medical device industry as in other areas.

I want to talk today about some of these new steps we are taking to improve development and to continue to make sure that patients have available to them the latest advances in technology, both big and small, when it comes to the medical products that they use. And then I want to talk about the steps I believe we can take, working together, to continue to provide patients and doctors with timely and up-to-date information about the risks and benefits of those products -- even with so many new advances becoming available all the time.

The good news is that our approach to the regulation of medical devices -- the law that governs how we evaluate new products -- gives us a box of regulatory tools of different shapes and sizes that allows us to have a lot of flexibility to calibrate our regulatory touch to the level of potential risk posed by new products.

We classify devices by how much potential risk they pose. Taking the toolbox analogy further, instead of one regulatory wrench, we have an array of wrenches, all in different sizes, to address different risks. For less risky devices, the 510(k) process imposes a lighter regulatory burden on developers. Although some changes to 510(k)-cleared devices require submission of the more extensive PMA, many can be dealt with by another 510(k) submission. And CDRH has done a tremendous job developing alternatives to the traditional 510(k) so that sponsors have several options under this provision of the device statute.

Thanks to Congress, we also have a completely new route to market for medical devices that are new but not highly risky. I am referring, of course, to the "de novo" process. FDA can look at a new device and decide, for example, that a submission based on existing published literature is adequate, making expensive and time-consuming clinical trials unnecessary. This is how CDRH recently handled a novel microarray as well as a diagnostic test for avian flu.

This more modern regulatory approach enables FDA to create a regulatory framework that better accommodates the continual evolution of the underlying technology we're asked to review. But this modern approach to regulation and to the translation of science to the bedside also presents FDA and product developers with some unique challenges. I want to talk about three of them.

The first challenges are regulatory challenges unique to FDA. The constant stream of innovation that we're asked to review is resource intensive. This challenges us inside FDA to improve our own internal processes. This is a topic I also want to get back to in some thoughts I'd like to share about the Medical Device User Fee and Modernization Act. But from a regulatory standpoint, as an agency we need to pay close attention to how our regulatory tools are deployed, to make sure that we're using our tool box efficiently, and not imposing unnecessary costs or time delays on the development of potentially lifesaving new medical products. Especially now, with the cost of healthcare increasing, and with more and more people potentially priced out of the benefits of new medical innovations, we need to make sure that in everything we do, we're taking steps to make sure that our approaches are efficient.

The modern approach that we're able to take to device regulation and to the rapid translation of science to the bedside also present a number of challenges that FDA and medical device developers share -- and I'll focus on two in particular: The first is that we must do more than ever to monitor the performance of their products after they are approved, and make sure that we are quickly discovering and sharing information about problems that do arise.

Improvements in material and manufacturing and engineering make today's medical devices smaller, hardier, and more sophisticated. This is important progress that has benefited literally millions of patients who can now have unobtrusive and more functional cardiac defibrillators implanted in their chests, for example, or stronger prosthesis in their hips.

But this technical sophistication sometimes also means that there is more opportunity for small errors to have bigger consequences. This is the essential paradox. What used to be a minor engineering problem years ago, that might not have been made manifest in the overall performance of the devices or affected their safety, can become magnified and have more potential for causing health consequences as the devices themselves get smaller and more sophisticated.

So as the opportunities for significant public health benefits from new medical devices continues to dramatically expand, the margin for error in their manufacturing sometimes continues to shrink. This means we need to be looking harder, after devices and engineering changes are approved, for new potential problems with their performance.

The second challenge that we share deals with how we all interact with patients and especially the broader medical community. As scientific advances in medical devices become more rapid and commonplace, and are more rapidly translated to the bedside, FDA and device developers are challenged to make sure that we're taking new steps to clearly and effectively communicate information about their performance. We need to work harder and more creatively to provide the information and tools that people need to adequately weigh the pros and cons of these kinds of innovations as they make personal choices about their health.

I want to come back and provide just a few more thoughts on each of these three challenges that I've outlined, and to tell you what we're doing at FDA to address each of them. And I want to tell you where we are going to need your help.

First, with respect to the challenges posed to FDA and to our regulatory process: Under the leadership of Dan Schultz, the head of our device center, and his dedicated team, we have taken new steps in recent years to make our processes for evaluating medical devices in the premarket period more efficient and more predictable to help improve the opportunities to bring beneficial new technologies to patients. And we have worked to better identify the places where things can go wrong with new device development so that we're focusing our resources on places that pose the most potential risk.

We will also be announcing soon a broad new initiative aimed at taking additional steps to improve the pre-market evaluation process, to focus new efforts and resources on how we look at innovative new technologies to make sure that our approaches are as modern as the devices we're being asked to evaluate.

In the document announcing this effort, we will be making specific commitments to develop new guidance documents that we believe can help increase the transparency and predictability of the review process.

We know that taking new ideas from the notebook to the market already requires huge investments in research and development. This is a risky process, and risk is the enemy of investment and creation. Especially in an era of rising healthcare costs, to continue to make progress and to make sure patients have access to better treatments, we have to do all we can to make sure that the development process is as modern, and efficient, and as effective as science allows.

To give you an idea of just a few of the things we intend to work on -- a number of these guidances will relate to specific devices and therapeutic and product areas, while others are cross-cutting or process-oriented, which should facilitate and improve the consistency of the review process.

There are clearly many cases where having good guidances for product areas can lead to improvements in the applications we receive or in our interactions with scientists and product developers. These things can help accelerate the achievement of performance goals that measure the efficiency of the review process. For example, we plan to develop cross-cutting guidance documents in key scientific areas, such as methods for incorporating Bayesian statistical analysis in clinical trials. The use of Bayesian statistics for clinical trial design and analysis allows developers to make use of existing information in an efficient and scientifically rigorous manner.

We also intend to release several cross-cutting guidance documents that will greatly aid the process for device development by providing additional regulatory clarity and predictability. These include guidance documents for modifications to PMA devices, PMA Annual Reports, Real-time PMA supplements, and a revision of the 510(k) Paradigm guidance document.

We will continue to take steps to improve how we evaluate the clinical aspects of device innovation, especially for novel devices and approaches and clinical spaces that haven't seen a lot of technological innovation in a long time. We will be building on our use of fellows for very novel technologies and outside consults for example, an approach that has worked well for a number of reviews already.

We're also taking new steps to continue to calibrate our inspections to the level of potential risk, by developing risk assessment criteria. This criterion takes into consideration probability and the potential severity for harm from things that can go wrong, as well as focusing our field inspection resources on medical devices and on manufacturers that have the greatest likelihood of encountering problems that could pose public health harm.

And we're also taking new steps to adapt quality systems to assure the predictability of our review process, as well as making use of retrospective peer assessments and using quality assessment teams to provide a comprehensive supervision of our process. To these ends, we have instituted an on-going quality review program for premarket submissions that evaluates the quality of our scientific review in several key scientific areas.

The idea here is to build on our professional staff expertise to identify and apply best management practices internally to our review processes. CDRH wants to make sure we are doing all we can not only to continue to reduce the time it takes to review new treatments and diagnostics, but also to help product developers get their applications right the first time -- to do the R&D work that demonstrates safety and effectiveness as quickly and efficiently as possible.

We have recently completed our first round of quality reviews in the areas of biocompatibility, sterilization and statistical analysis, three cross-cutting areas that are common to many medical device submissions. For example, establishing the safety of new materials for use in implants or in combination drug-device products is paramount to efficiently assessing the new technological characteristics of these future innovations. This internal review process has pinpointed areas for improvement in the training of our reviewers and the need to develop up-to-date and clear guidance for industry. In the near future, we intend to expand the internal quality review program to additional scientific areas, including software validation.

Finally, and to take just one more example from many of the things we intend to do, we have developed milestones for the review of premarket submissions as well as improved the management and oversight of premarket review to ensure predictability and accountability in the device review process. We believe that continuing and expanding this effort will allow us to improve the quality and efficiency of our reviews, thus ensuring that we consistently ask developers the right questions at the right times and that the right resources are used in the right way to enhance the timeliness and predictability of device review.

There's no question that in all of these efforts the opportunities and resources that have been made available to the agency under the Medical Device User Fee and Modernization Act has been an important catalyst in enabling us to adopt new tools and implement fundamentally better ways for developing the science that guides all of our device review process.

Part of our commitment under MDUFMA was to meet certain performance goals that reflected, in part, the process improvements that we were able to make as a result of these additional resources. People inside our Center for Devices and Radiological Health have been working extremely hard on these goals, and I'm pleased to say they are meeting all of them.

But as we work with sponsors to re-authorize this important legislation, there is also concern inside FDA that not all of the goals have been as closely aligned as others with the bottom line that we all share: making the process for evaluating products as predictable and scientifically rigorous and efficient as possible. Some of these concerns were borne out in a recent report by the Lewin Group. It's clear, I believe, that user fees have enabled our drug program to make great strides, by gaining efficiencies and resources that enabled us to modernize the management and the science of drug review. User fees can have the same long-term benefits for the device review, but only if we work together, and only if our device center is able to benefit from the same kind of collaborative discussion with sponsors and academic and management experts that our drug program has benefited from. The interactions we have with drug sponsors during PDUFA reauthorization, during the negotiation process, have enabled us to incorporate modern management tools into our drug review process, and the same kind of interactions should provide equivalent benefits when it comes to device review. But once again, we need to work together.

This leads me to the other two challenges, the ones where I think we have some additional shared obligations, and where we need your help and we need to work together. The first is that today we must do more than ever to monitor the performance of medical products after they are approved, and make sure that we are quickly discovering any potential problems that might arise. Innovation for medical devices differs from innovation for other medical products such as drugs. Device innovation includes both giant leaps and incremental changes with life cycles as short as 18 months. Unlike many drugs, the effectiveness of such improvements depend on a continuing dialogue between product developers and the ultimate end-users about what works, what doesn't, and what the best solution to problems might look like because devices generally require the skill of the end-user.

In short, unlike drugs, medical devices are tools in the hands of physicians and surgeons, rather than therapeutic agents that work independent of physician involvement. So when it comes to devices, post-market systems that enable constant learning and feedback not only help support best medical practices to ensure safe use of devices with maximum effectiveness but they also spur continued innovation. FDA Acting Commissioner Dr. Andrew von Eschenbach has a wonderful line that I will try to quote. He says that delivery inspires new discovery. This is especially true when it comes to medical devices. Therefore, opportunities exist to enhance medical device innovation at every phase of the total product life cycle. Post-market learning becomes an essential part of the process for innovation.

To improve our post market data collection, at FDA we are using a lifecycle approach to how we look at medical devices, and focusing more attention on the kinds of systems and processes we have in place to monitor products after they are approved. Now I have spoken to people in our device center about our efforts to improve our post-market processes, and there is consensus that our pre-market review efforts and our post-market resources are inextricably linked. The more that we can develop confidence that our post-market tools will be able to quickly and accurately discover problems that arise, and before patients are harmed, the more confident we can be in our pre-market approval process and fulfill our desire to efficiently move promising new technologies to the bedside.

So we cannot sacrifice post market safety to support premarket review one bit, and we don't intend to. The two are inextricably linked. It is essential for the public health that, as the nature of medical devices becomes more complex, FDA is well-positioned to identify risks and work with industry to manage those risks once a device comes on the market. As the complexity of new technologies continues to increase, along with their lifesaving benefits, it becomes more challenging to reveal all of the subtle things that can go wrong in any reasonably sized clinical study. Because as these devices get more sophisticated, and their lifesaving benefits more numerous, the margin for engineering errors also shrinks, and the ability for things to go awry sometimes increases. So the only way we are going to be able to maintain the efficiency our review process over these innovations is by building more rigorous tools for monitoring them after they are cleared or approved.

And so at FDA we are also taking new steps to improve our post-market system when it comes to medical devices. Under our MedSun and LabSun initiatives, where we actively gather data about side effects from hospitals and other healthcare establishments, we are developing new tools and methods to improve the number and type of reports we receive. We are moving toward obtaining "real time" data from these networks, and are increasing the number of laboratory reports we get from participating hospitals and targeting new kinds of reports such as pathology and in vitro diagnostic tests.

We are also working to improve access to additional information and new electronic reporting tools and will be implementing changes to our own internal processes for reviewing these data and analyzing them for signals and for safety problems. For example, we've developed an internal tracking system for new devices and for the information we get from the different kinds of reports we receive about manufacturing changes and other measures of post-market performance. And as I mentioned, we are also working on new guidance to improve the format of the information that we ask sponsors to submit for certain kinds of devices, so that it is easier to integrate this information into our post-market review processes.

We are mindful that we depend on product developers to collect this information and to help analyze it and to make sure that it continues to be submitted to us in a timely and complete way. We appreciate all of the collaborative work we do with sponsors every day. It's often hard to uncover the patterns that might suggest that a new innovation or manufacturing improvement has led to some new and unwanted side effect or safety problem and sometimes a device developer is in a good position to uncover these kinds of links. So we need your help in working on these challenges.

The third and final challenge that we at FDA share with all of you is in how we communicate with the broader medical community. Communicating medical information, and especially risk communication, is a difficult but increasingly important challenge, especially as devices evolve more rapidly and innovations hopefully become available over shorter time frames. We need to make sure we are helping doctors incorporate these new benefits into their medical decisions, and that they understand where they need to be looking harder for new potential problems that might also arise as a result of innovations or engineering changes.

We're taking new steps to work more regularly and collaboratively with health professional societies, to improve the information we receive as part of our post-market monitoring process but also to make sure that we're communicating effectively with health professionals and with patients. For example, we recently held a day-long conference in collaboration with the Heart Rhythm Society to look specifically at communication challenges around implantable defibrillators and pacemakers. This format worked extremely well, and could be used in other product areas. As a result of the HRS Policy Conference, an ICD Working Group was created both at FDA and HRS.

Based on the input we receive from these and other interactions with stakeholders, we are undertaking an assessment and improvements upon the manner in which we communicates critical public health information, including public health notifications, and recalls.

This effort is aimed at making sure that regulatory actions are accompanied by the most complete and appropriate information for helping patients and providers put actions into medical context and make effective, individualized decisions about the medical products they use.

Among the things we are working on is a new approach to how we issue recall notifications, to make sure that we are communicating in a way that patients and clinicians can rapidly distill the most important public health information.

The bottom line is this: as technology improves, and as device developers and FDA work to continue to make new innovations and even incremental improvements available to patients more quickly, we also have certain new responsibilities that come with the sheer speed of technological change and the complexity of the innovations themselves. All of this improvement has resulted in obvious and measurable gains in health for patients, but all of this improvement doesn't come without its own burdens and obligations.

And so we need to work together to continue to make sure that the patients remain well informed about health issues, and whenever they need it, they get access to the best, highest quality healthcare around, the most innovative health care system in the world. I want to say that we're looking forward to working with you on these very important issues. Thank you all very much.