December 14, 2018
By: Scott Gottlieb, M.D. and Jeffrey Shuren, M.D., J.D.
Scott Gottlieb, M.D.
Commissioner of the U.S. Food and Drug Administration
In recent days, the Food and Drug Administration (FDA) has committed to several new policies that will modernize the agency’s approach to regulation in the medical device system.
For instance, we announced our intention to propose an alternate approach to the traditional 510(k) clearance process, which will involve the use of modern, science-based, consensus standards and FDA-developed performance criteria as the comparator for device review for certain well-understood technologies. FDA currently compares new devices to predicate devices that can in many cases be as much as 40 years old. By modernizing the standards that FDA uses to assess some new devices, it will make it easier to innovate these products, and adopt more up-to-date and rigorous benchmarks for evaluating their safety and effectiveness.
Today, FDA is taking another step to modernize its regulatory framework and make its review processes more efficient. FDA is issuing a new, draft guidance that describes how it intends to implement provisions enacted by Congress to pursue the “least burdensome” means of assuring the safety and effectiveness of new devices so that innovations that benefit patients can reach them more efficiently.
Timely patient access to high quality, safe and effective medical devices requires that FDA reduce or reform those outdated, unnecessary burdens in our regulatory approaches that can add to development costs or forestall beneficial innovation without also enhancing device safety and effectiveness.
The least burdensome approach is not new; however, recent legislation, as well as the opportunities offered by advances in science, call for updating this framework.
Since the passage of the Food and Drug Administration Modernization Act (FDAMA) in 1997, FDA has been required to take a least burdensome approach to premarket review of devices that are subject to a premarket approval application (PMA) or premarket notification (510(k)). FDA issued guidance and began to train staff on the least burdensome requirements shortly after FDAMA’s enactment.
Through the application of the least burdensome approach, we’ve moved much closer to achieving our vision of patients in the U.S. having access to high-quality, safe and effective medical devices of public health importance first in the world.
While these initial efforts first established the principles of this least burdensome framework for device premarket review, subsequent agency efforts – and especially new laws such as the 21st Century Cures Act in 2016 — clarified and expanded the least burdensome concept. As a result, the concept has been increasingly integrated into FDA’s culture and operations, and has become a guiding principle for the device center’s efficient, science-based regulation.
Today, in new draft guidance on the Least Burdensome Provisions, FDA defines least burdensome to be the “minimum amount of information necessary to adequately address a regulatory question or issue through the most efficient manner at the right time.” The new draft guidance also explains FDA’s regulatory approach and delineates guiding principles that we propose to apply across the total product lifecycle – beyond what Congress has required – including all device-related applications and interactions with medical device industry.
By making sure our processes are efficient and our regulatory frameworks are better tailored to technology type and patient needs, we believe we can garner information about the safety and effectiveness of new devices in the pre- and post-marketing setting with less time and cost. Because we believe the application of these least burdensome principles will help drive the development of, and timely patient access to, safe and effective devices, we are committed to training all device center staff on the implementation of these principles, not just those involved in pre-market review as required by the Cures Act.
Jeffrey Shuren, M.D., J.D.
Director of FDA’s Center for Devices and Radiological Health
This balanced approach will allow for FDA to focus its resources on issues of highest public health concern. In just the past few years, we’ve seen notable results of our application of the least burdensome principles on medical device review, including reduction in review times and improved quality of applications. At the same time, we’re advancing FDA’s gold standard for safety by taking additional steps such as enhancing our pre- and post-market safety tools, including the establishment of a new pre- and post-market evaluation system (NEST).
The Cures Act extended the least burdensome provisions and their application to the pre-market review process in new ways. It streamlined FDA’s ability to remove the 510(k) submission burden where no longer needed for certain lower risk devices. This allowed the agency to free up review resources to focus on higher risk devices. The application of the goals of the least burdensome approach also has resulted in new efforts to implement risk-based compliance policies. For example, through the implementation of a variety of new digital health policies since 2013 – including those involving mobile medical apps and medical device data systems – the agency generally does not oversee low risk products that are part of the fitness/wellness industry.
The successes in the pre-market arena have encouraged FDA to apply the least burdensome approach in many other device policies and programs. This includes the development of a more flexible framework for making benefit-risk determinations in support of PMA approvals and De Novo classifications; a new policy on early feasibility studies; and the wider use of Real-World Evidence, such as data gathered as a part of routine clinical care, such as in electronic health records and registries. We’ve also developed and incorporated new regulatory science tools in our review process, such as computer models to make device development, assessment and review safer, faster and more efficient.
Each of these exemplifies the advances we’ve made in creating a more efficient regulatory process – and incorporating more modern evaluative tools and approaches – while enabling our scientists to get the safety and effectiveness information that they need to inform their decisions.
In extending these same principles, FDA has embraced and led international harmonization efforts to identify areas where regulation is not achieving its intended goal, and may be adding undue burden and presenting an obstacle to beneficial innovation. For example, FDA is a founding member of the International Medical Device Regulators Forum and led the forum’s working group that established the Medical Device Single Audit Program. Under this program, participating countries share their own inspections and rely on surveillance inspections conducted by or on behalf of other participating countries. The program potentially eliminates the need for participating device companies to prepare for multiple inspections while allowing government agencies to make better use of their limited resources. It shows how adopting the most efficient approach has set FDA on a course to generate high quality information at a potentially lower cost and burden to product developers and to the agency.
The resulting impact on public health from FDA’s implementation of these and similar principles has been significant. In 2016, we approved 91 innovative devices as compared to 24 in 2009, the highest number since the start of the user fee program in 2003. This year, we may achieve a modern record when it comes to the number of innovative medical device approvals. In 2016, we also saw an increase in the number of high quality, safe and effective devices of public health importance approved in the U.S. as the first commercial market, meaning that U.S. patients now have early access to critically important technologies, such as the first “artificial pancreas.”
Through the application of the least burdensome approach, we’ve moved much closer to achieving our vision of patients in the U.S. having access to high-quality, safe and effective medical devices of public health importance first in the world. At the same time, we’re setting FDA on a modern framework for making sure it continues to strengthen and secure its gold standard for medical product review.
Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration
Jeffrey Shuren, M.D., J.D. is Director of FDA’s Center for Devices and Radiological Health
Follow Commissioner Gottlieb on Twitter @SGottliebFDA