Moving Toward a National Medical Device Postmarket Surveillance System

By: Jeffrey Shuren, M.D., J.D. and Thomas P. Gross, M.D., MPH

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health

Despite rigorous premarket evaluation, what really counts is how well a medical device works when it’s used day-to-day by patients, caregivers and clinicians. Beyond clinical trials, real-life patient experience may reveal unanticipated device risks and confirm long-term benefits. Similar to other medical products such as drugs or vaccines, medical devices offer vital, sometimes life-saving, benefits, but they must be balanced against certain risks. A strong postmarket surveillance system can provide more robust and timely benefit-risk profiles for devices so that providers and patients can make better informed health care decisions.

In 2012, CDRH laid out a strategy to strengthen the nation’s postmarket surveillance system for devices. As described in that strategy, our vision for medical device postmarket surveillance consists of a national system that quickly identifies poorly performing devices, accurately characterizes and disseminates risk and benefit information about real-world device performance, and efficiently generates data to help support premarket clearance or approval of new devices and new uses of currently marketed devices.

Thomas Gross, MD, MPH, Director, Office of Surveillance and Biometrics in FDA’s Center for Devices and Radiological Health

Thomas Gross, MD, MPH, Director, Office of Surveillance and Biometrics in FDA’s Center for Devices and Radiological Health

We cannot create a system like this alone. Achieving our vision for a national system requires thoughtful input and active participation from many key national and international stakeholders—now and in the future.  In 2013, after receiving public input on the 2012 strategy, we published an update that described the five major steps the FDA would take to create a National Medical Device Postmarket Surveillance System (MDS):

(1) Establish a multi-stakeholder Medical Device Postmarket Surveillance System Planning Board to identify the governance structure, practices, policies, procedures, methods and business model(s) necessary to facilitate the creation of a sustainable, integrated medical device postmarket surveillance system.

(2) Establish a unique device identification (UDI) system and promote its incorporation into electronic health information.

(3) Promote the development of national and international device registries for selected products.

(4) Modernize adverse event reporting and analysis.

(5) Develop and use new methods for evidence generation, synthesis, and appraisal.

Over the past year, we’ve made tremendous progress in laying the groundwork for this national system. We have begun implementing the UDI rule, including development of a Global UDI Database (GUDID) as the repository for information that unambiguously identifies devices through their distribution and use. We continued to build registry capabilities both domestically (such as the National Breast Implant Registry) and internationally (such as the International Consortium of Vascular Registries).  And we established a Medical Device Registry Task Force consisting of key registry stakeholders under CDRH’s Medical Device Epidemiology Network (MDEpiNet) Program. Importantly, we also commissioned the Engelberg Center for Health Care Reform at the Brookings Institution to convene and oversee deliberations of the Medical Device Postmarket Surveillance System Planning Board.

Today, we are happy to announce the release of the Planning Board’s report Strengthening Patient Care: Building an Effective National Medical Device Surveillance System, which outlines recommended steps toward achieving the MDS and strategies for implementation. The report provides a pathway to realizing a national system that harnesses novel data sources, modern analytical techniques and the participation of all stakeholders to optimize patient care. Interested stakeholders will be able to share their feedback on the report through a public docket.

In the coming months, we will also get reports from the Medical Device Registry Task Force. As noted in the 2013 Update, these reports will address significant issues such as defining effective registry governance and data quality practices, which will enrich the national dialogue on development of registries as a crucial source of data on device performance.

Our vision of a National Medical Device Postmarket Surveillance System is a 21st Century solution to an age-old problem. The system relies on the experience gained by health care providers in their daily use of medical devices leveraged by modern technology. This experience, made possible by new tools and systems unimaginable a generation ago, gives us real-time data about what happens to patients in clinical practice. We will be able to leverage these capabilities not only to quickly identify poorly performing devices, but also to facilitate device approval/clearance and patient access, to reduce postmarket data collection for manufacturers, and to better inform healthcare decisions by providers and patients alike.  We look forward to overcoming the challenges and embracing the opportunities that lie ahead. We are optimistic that with the engagement of the public and private sectors, we can collectively build a medical device postmarket surveillance system that will achieve all of our goals.

Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health

Thomas Gross, MD, MPH, Director, Office of Surveillance and Biometrics in FDA’s Center for Devices and Radiological Health

Smart Ways to Manage Health Need Smart Regulation

By: Bakul Patel, M.S., M.B.A. and Jeffrey Shuren, M.D., J.D.

Engaged patients! Quantified self! Lifelogging! These buzzwords describe an exciting technology-based, patient-centered approach to living healthier. The myriad of systems that record, share, and use personal and health data have become a significant help for many of us by putting information at our fingertips to use when and where we think it might help promote a healthy lifestyle. The ultimate goal of these products is to improve our quality of life.

Bakul Patel

Bakul Patel, Associate Director for Digital Health in FDA’s Center for Devices and Radiological Health

From wearable sensors to simple tracking apps, more and more consumers are choosing to use technology to monitor their health and motivate them to engage in health-promoting activities. These products, which may count steps, calculate burned calories, or record heart rates and sleep cycles, all have the goal of helping individuals to live a healthy lifestyle.

The FDA seeks to advance public health by promoting innovation and development in this area by continually adapting our regulatory approach to technological advances to meet the needs of patients and consumers.

This week, we finalized our guidance on medical device data systems (MDDS), and we recently issued two draft guidance documents that outline our thinking about low-risk devices intended to promote general wellness, and our risk classification approach to medical device accessories. We committed to issue these guidances in the FDASIA Health IT Report of April 2014.

Through these actions, we continue to clarify which medical devices are of such low risk that we will no longer focus our regulatory oversight on them or we will regulate them under a lower risk classification, narrowly tailoring our approach to the level of risk to which patients or consumers are exposed.

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health

The MDDS guidance confirms our intention to not enforce compliance with applicable regulations for technologies that receive, transmit, store, or display data from medical devices. We hope that finalization of this policy will create an impetus for the development of new technologies to better use and display this data. We also updated the Mobile Medical Apps guidance to be consistent with the MDDS final guidance. We will discuss our MDDS approach at an upcoming webinar.

Last month, the FDA also proposed to not examine regulatory compliance for low risk products that are intended only for general wellness. These products are designed to maintain or encourage a general state of health and may associate a healthy lifestyle with reducing the risk or impact of certain diseases or conditions. We hope this policy fosters the development of low-risk products intended to promote a healthy lifestyle.

And finally, we issued draft guidance proposing to regulate medical device accessories based on the risks they present when used as intended with their parent devices and on the level of regulatory controls necessary to assure their safety and effectiveness, independent of the risks of their parent devices. Some accessories can have a lower risk profile than that of their parent device and, therefore, may warrant being regulated in a lower class. For example, an accessory to a Class III parent device may pose lower risk that could be mitigated through general controls or general and special controls and thus could be regulated as Class I or Class II.

Through such smart regulation we can better facilitate innovation and at the same time protect patients.

Bakul Patel is Associate Director for Digital Health in FDA’s Center for Devices and Radiological Health

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

A Year of Significant Progress in Public Health

By: Margaret A. Hamburg, M.D.

Margaret Hamburg, M.D.A new year offers both an opportunity to look forward and an opportunity to reflect on the achievements of the previous year. And, in 2014, FDA’s accomplishments were substantial, touching on many of the agency’s broad responsibilities to protect and promote the public health.

Whether our achievements involved medical product safety and innovation, food safety and nutrition, tobacco control, or other areas of our important work, all were accomplished thanks in large part to our ability to respond to evolving needs and opportunities including the embrace of new approvals, technologies and cutting-edge science.

Consider these highlights:

Drug Approvals: This past calendar year, FDA approved 51 novel drugs and biologics (41 by CDER and 10 by CBER), the most in almost 20 years. Among CDER’s 2014 approvals are treatments for cancer, hepatitis C and type-2 diabetes, as well as the most new drugs for “orphan” diseases since Congress enacted the Orphan Drug Act over 30 years ago. Seventeen of these new approvals are “first in class” therapies, which represent new approaches in the treatment of disease. In addition, CBER approved many important biological products in 2014, including a number of groundbreaking vaccines for meningitis B, the flu, and certain types of Human Papillomavirus, the latter of which is expected to prevent approximately 90 percent of the cervical, vulvar, vaginal and anal cancers caused by HPV.

These developments are a testament not just to our expanding understanding of human biology, the biology of disease and the molecular mechanisms that drive the disease process, but also to FDA’s innovative approaches to help expedite development and review of medical products that target unmet medical needs, while adhering to the established standards for safety and efficacy. These include enhanced guidance to shape the research and development agenda, early input on clinical study needs and design, expedited review programs, targeted regulatory advice and other tools and incentives that spur investment and innovation in new medical products to address unmet medical needs.

Opioids: This past year FDA took several actions to address the abuse of opioid drugs. First, we approved abuse deterrent labeling for three opioid products that are designed to deter prescription drug abuse. These drugs used different technologies to combat the abuse problem in different ways, such as by making the product resistant to crushing or dissolving or using “aversive technology” to discourage users from taking more than the approved dosage of the drug. To help encourage the development of more drugs in abuse-deterrent forms, we are also working to provide additional advice to manufacturers. Although abuse-deterrent opioid drugs are not a silver bullet to prevent opioid abuse, we believe that our work in this area will give physicians effective new treatment options with less risk of abuse.

FDA also worked to improve the treatment of patients who overdose on opioids. We approved a new dosage form of naloxone, with an autoinjector to enable a caregiver to administer the drug in the emergency treatment of opioid overdose (as it rapidly reverses the effects of an overdose). While we continue to support development in this area, this approval offers a new valuable tool to help prevent the tragedy of opioid drug overdose.

Antibiotic Resistance: We made important strides in confronting the growing resistance of some bacteria to antimicrobial drugs. Our efforts, which are a critical part of the recently unveiled National Strategy on Combating Antibiotic Resistant Bacteria, offer a multi-pronged approach that recognizes that to effectively address this challenge means simultaneously addressing the many different causes for increasing antibiotic resistance. One important response has been efforts to expand the pipeline of new medical products, including therapeutics to treat and cure infection, diagnostics to aid in the identification of the cause of infection and of resistant infections, and vaccines to help prevent infection with bacteria in the first place.

These efforts are already having an impact. In 2014, FDA approved four novel systemic antibiotics. In contrast, only five new antibiotics had been approved in the previous ten year period.

In addition to working on the human medical product side, we also developed and, over the next two years will be implementing, an important complementary strategy to eliminate the use of medically important antibiotics for growth promotion in food-producing animals. This strategy, once fully implemented, also will bring the remaining uses of such drugs to treat, control or prevent disease in these animals under the oversight of veterinarians. All 26 animal health companies who produce those drugs have committed to participate, and 31 products already have been withdrawn from the market.

Pharmacy Compounding: We continued to respond effectively to the 2012 outbreak of fungal meningitis that was linked to contaminated compounded drugs. This included conducting more than 90 inspections of compounding facilities across the nation in the past year. As a result, numerous firms that engaged in poor sterile practices stopped making sterile drugs, and many firms recalled drugs that have been made under substandard conditions. Where appropriate, we have worked with the Department of Justice to pursue enforcement action against some of these facilities.

We also have continued to implement the compounding provisions of the Drug Quality and Security Act (DQSA), and to develop and implement policies to address compounding by state-licensed pharmacies and the new category of registered outsourcing facilities.

Food Safety: Over the past year, the Agency has made great strides in implementing the landmark FDA Food Safety Modernization Act (FSMA). Through our proposed rules for preventive controls requirements for both human and animal food, standards for produce safety, foreign supplier verification programs, third party auditor accreditation, focused mitigation strategies to prevent intentional adulteration of food aimed at causing large-scale public health harm, and requirements for sanitary transportation practices to ensure the safe transport of food, we are working to ensure the safety of American consumers related to the foods they eat.

Nutrition: Good health depends not just on food safety, but also on what we choose to eat. FDA plays an important role in promoting good nutrition and healthy food choices by helping consumers understand the importance and benefits of good nutrition – and of being able to make informed choices about what we eat.

New rules in 2014 to finalize requiring calorie information on restaurant menus and vending machines give our citizens information they need to make healthy food choices and hopefully help reduce the epidemic of obesity in the United States. We also proposed changes to the familiar “Nutrition Facts” label on packaged foods which, when finalized, will give our citizens updated nutrition information, reflecting the most current nutrition science, to help them make healthy choices when purchasing packaged foods.

Tobacco Control: There are few areas that have as profound an impact on public health as tobacco products, which is why, five years ago, Congress gave FDA the responsibility to oversee the manufacture, marketing, distribution, and sale of tobacco products.

Over the past year, we worked with state authorities to conduct more than 124,000 inspections of retailers to enforce the ban on the sale of tobacco products to children. We unveiled the first of its kind national public education campaign—The Real Cost—to reduce youth smoking. And we took the first steps towards extending the agency’s tobacco product authority over additional products such as electronic cigarettes (e-cigarettes), cigars, pipe tobacco, nicotine gels, waterpipe (hookah) tobacco, and dissolvables not already subject to such authority through our proposed “Deeming Rule.” In addition, as part of ongoing work on product review decisions, eleven tobacco products that were allowed to enter the market during a provisional period established by the Tobacco Control Act were found “not substantially equivalent” to a predicate tobacco product. As a result of this finding, these products can no longer be sold or distributed in interstate commerce or imported into the United States.

Ebola: The tragic Ebola epidemic in West Africa demonstrates that we do not have the luxury of closing our eyes – or our borders – to the public health problems that exist in the rest of the world. I’m proud that FDA has played an important role in the response to this disease, working closely with colleagues in our government as well as the scientific community, industry and a range of other organizations and nations. We have helped facilitate the development, testing, manufacture, and availability of investigational products for use in diagnosing, treating and preventing Ebola, and worked with sponsors and health care providers to facilitate access to these products as clinical circumstances warrant. In August 2014, FDA designated the drug Z-Mapp as an orphan drug for Ebola, with the hope that this would incentivize further development and study.

And I’m very pleased to report that FDA is represented on the ground in West Africa by dedicated officers of the Commissioned Corps of the Public Health Service who continue to staff and operate the Monrovia Medical Unit in Liberia that was built to treat the health workers who became ill responding to the outbreak. Like everything FDA does, both at home and abroad, our actions on Ebola represent our agency’s continuing commitment to health and safety, and the use of science to advance these important goals.

I am extremely proud of our accomplishments in 2014, and I am confident that FDA will have a successful 2015, as we continue our work to protect and promote the public health.

Margaret A. Hamburg, M.D., is Commissioner of the Food and Drug Administration

FDA’s FY 2016 Budget Request

By: Margaret A. Hamburg, M.D.

Margaret Hamburg, M.D.FDA oversees products that represent more than 20 cents of every dollar that American consumers spend. Today, FDA presented its FY 2016 budget to Congress.This sensible budget request will help ensure that FDA can continue to fulfill its vast responsibilities to protect the public health, safety, and quality of life of the American public.

I want to share the cover letter that I wrote to Congress outlining some of our specific proposals.

 

Letter from the Commissioner

I am pleased to present the FY 2016 Food and Drug Administration (FDA) Budget.

FDA fulfills its important mission to promote and protect health in an increasingly complex and globalized world in many ways.  The scope of our work includes assuring that foods are safe, wholesome, sanitary and properly labeled; ensuring that human and veterinary drugs, vaccines and other biological products, and medical devices intended for human use are safe and effective; and regulating tobacco products.  We also play a lead role in protecting the public from electronic product radiation and assuring that cosmetics and dietary supplements are safe and properly labeled.  Finally, we have devoted – and will continue to devote – substantial resources to advancing the public health by helping to speed product innovations.

FDA’s responsibilities continue to expand as we work to fulfill the mandates of groundbreaking legislation passed in recent years, including the Family Smoking Prevention and Tobacco Control Act of 2009, the Patient Protection and Affordable Care Act of 2010, the Food Safety Modernization Act (FSMA) of 2011, the FDA Safety and Innovation Act (FDASIA) of 2012, and the Drug Quality and Security Act of 2013.  Further, with so many FDA-regulated products manufactured in whole or in part outside of our borders, FDA is keenly focused on the complexities of regulating in a global marketplace.

In FY 2014, we took important steps to finalize a key set of proposed food safety rules; worked to improve the safety of compounded pharmaceutical products by conducting more than 90 inspections and implementing compounding legislation through proposed regulations, guidances, and other actions; published the “deeming rule” to extend FDA’s tobacco authority; and collaborated with federal, international, and industry partners to expedite the development and availability of medical products.  In addition, FDA has worked intensively to respond to the Ebola epidemic in West Africa by facilitating the development and availability of investigational diagnostics, therapeutics, and vaccines with the potential to help combat the epidemic.

FDA continues to seek new ways to obtain the most public health value for the federal dollar as we implement expanded authorities.  The products that FDA regulates are essential to public health, safety, and quality of life and represent over 20 cents of every consumer dollar spent on products in the United States.  Yet, in terms of our FDA budget, each American taxpayer contributes approximately $8 per year for the vast array of protections and services provided by FDA.

In FY 2016, we are requesting essential and timely resources to address critical food and medical product safety issues.  Mindful of the fiscal environment, we have identified targeted reductions where possible and identified long-term needs for additional user fees to balance budget authority growth.  FDA is requesting a total of $4.9 billion to support our various mandates to protect the American people.  This includes a $148 million budget authority increase to focus on the following:

  • delivering a farm-to-table system of prevention, including improved oversight of imported foods, through effectively implementing the final rules required by FSMA;
  • combating the growing threat of antibiotic resistance – in which drugs become less effective, or ineffective, against harmful bacteria;
  • promoting the development and appropriate use of reliable molecular and genetic diagnostics – precision medicine tools – to “personalize” the diagnosis, treatment, and prevention of disease;
  • implementing key FDASIA requirements to improve medical product review and inspections;
  • addressing the safety of compounded drugs;
  • continuing implementation of new requirements for review of sunscreen ingredients under the Sunscreen Innovation Act; and
  • supporting modern facilities to provide the laboratories and office space needed to meet FDA’s expanded legislative mandates.

As a science-based regulatory agency with a public health mission, FDA plays a unique and essential role in promoting and protecting public health and safety.  We are committed to meeting the needs and expectations of the American people.

Margaret A. Hamburg, M.D.

Commissioner of Food and Drugs

FDA Considering How to Tailor its Oversight for Next Generation Sequencing

By: Margaret A. Hamburg, M.D.

FDA is weighing the appropriate regulatory approach to advances in technology that allow physicians to obtain information on large segments of a patient’s genetic makeup very quickly.

Margaret Hamburg, M.D.This technology is known as next generation sequencing, where a single test potentially can be employed to identify thousands—even millions—of genetic variants carried by a single individual. The results of such tests could be used to diagnose or predict a person’s risk of developing many different conditions or diseases and potentially help physicians and patients determine what course of treatment should be used to treat specific individuals.

Reliable and accurate NGS technologies promise to accelerate “personalized” or “precision” medicine, the tailoring of medical treatment to the individual characteristics of each patient. But they also pose some novel issues for FDA in carrying out our mission of protecting and promoting public health.

Most diagnostic tests follow a one test—one disease paradigm that readily fits FDA’s current device review approaches for evaluating a test’s analytical and clinical performance. Next generation sequencing produces a massive amount of data that may be better handled using a new approach.

Last year we took steps to adapt our oversight approach to this new technology with the marketing authorization of the first NGS sequencing instrument, Illumina’s MiSeqDx Instrument and its two tests for cystic fibrosis (CF) mutations. We applied practical regulation to these products: we looked at how accurately the instrument sequenced a representative set of genetic variants across the genome rather than requiring data on every possible variant. Doing so avoided years of data gathering and unnecessary delay in the public’s access to the benefits of this technology while still assuring its accuracy and reliability.

Similar flexibility was employed in assessing the two CF tests. FDA allowed Illumina to leverage a well-curated, shared database of CF mutations to demonstrate the clinical value of its tests, rather than requiring them to independently generate data to support each mutation’s association with the disease.

In the future, next generation sequencing tests may be available to rapidly address new medical knowledge that can be applied in treating patients. Medical knowledge itself can be strengthened through creating databases of research and clinical information tied to particular genetic variants. FDA intends to develop a practical and nimble approach that will allow medical advances to be implemented as soon as possible, using its regulatory flexibility and the power of the information placed into high-quality databases.

This week President Obama unveiled his Precision Medicine Initiative. As part of that effort, FDA has been reviewing the current regulatory landscape involving next generation sequencing as the technology moves rapidly from research to clinical practice. To get the dialogue started, FDA published a preliminary discussion paper in late December that posed a series of questions about how to best assure that tests are not only accurate and reliable, but are available for patients as soon as possible. Public comment is essential, so FDA has opened a public docket and will be holding a public meeting on NGS technology on February 20.

NGS technology is clearly integral to the future of personalized medicine. Whatever approach FDA ultimately adopts must be selected with care to ensure continued innovation in the advancement of medical care and public health for this still evolving technology.

Margaret A. Hamburg, M.D., is Commissioner of the Food and Drug Administration

Listening to Patients’ Views on New Treatments for Obesity

By: Kathryn O’Callaghan and Jeffrey Shuren, M.D., J.D.

The world was a very different place in 1976, when the Food and Drug Administration launched its medical device program.

Kathryn O'Callaghan

Kathryn O’Callaghan, Associate Director for Science and Strategic Partnerships (Acting), FDA’s Center for Devices and Radiological Health

Since Steve Jobs and Steve Wozniak were just that year launching a computer company called Apple, doctors weren’t yet able to view X-ray images or look up drug prescribing information on their iPhones. Moreover, patients couldn’t Google treatments for heart disease, nor were they able to instantly find all open U.S. clinical trials for breast cancer. Not only was patients’ access to health care information much more limited, so was their role in making their own health care decisions.

Doctors diagnosed. Doctors made treatment decisions. Patients followed directions.

It’s different now.

Patients are more empowered today. Driven in part by a need to address emerging or neglected illnesses, such as HIV/AIDS and rare disorders, patients over the past three decades have increasingly banded together, creating organizations that advocated for their interests and generated public awareness of their diseases, their needs, and the lack of effective therapies. This activity produced legions of informed and empowered patients, who today urge us to take a more active role in our own health and urge clinicians to engage patients in shared health care decision-making. Patients are now not only partners in their health care but active consumers who make choices about their doctors, treatments, diagnostics, and health care experiences, an empowerment that is affecting the development of innovative therapies and new clinical solutions.

Today, there are no health care debates, discussions and decisions without considering the patient perspective.

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health

At FDA’s Center for Devices and Radiological Health (CDRH), we have been systematically involving patients in our regulatory decision making process. Since 1999, CDRH has included a patient representative on each of our advisory panels of outside experts, giving us a better understanding of patient concerns about particular technologies. And in 2012, we began focusing our medical device approval decisions on incorporating the patients’ perspective.

Under this benefit-risk framework for high-risk and innovative, lower-risk medical devices, CDRH’s health care professionals, scientists, and engineers consider the patients’ perspective on both a product’s benefits and their tolerance for any risks when weighing the evidence to determine whether or not to approve a product.

In the past, CDRH experts may have determined that a device should not be approved because its probable risks outweighed its probable benefits. However today, under a patient-centric assessment of risk, if adequate evidence indicates that a subset of well-informed patients with a particular illness or condition would value the product’s benefits more than its risks, CDRH may approve the device for that particular group. However, if we were to approve such a device we may require appropriate product labeling that clearly defines the patient sub-population and their benefit-risk preference. That information would be included in the product’s “Indications for Use” section of the label to ensure that patients and health care practitioners are able to make well-informed decisions.

Better tools are needed to more reliably and scientifically characterize patient preferences about benefit and risks, so we launched our Patient Preferences Initiative, to identify and develop methods for assessing patient valuations of benefit and risk related to specific device types and specific illnesses and conditions.

The goal is to ensure we have sufficient confidence in these methods to rely on them to inform product approval decisions.

Earlier this month, a team of FDA scientists led by Telba Irony, PhD, Chief of General and Surgical Devices Branch in the Division of Biostatistics, published an article in Surgical Endoscopy with leading behavioral economists at RTI Health Solutions, a business unit of RTI International, illustrating how this paradigm can inform medical device approval decisions. The authors successfully tested a new method for capturing patient sentiment and translated it into a decision-making tool for incorporating patient preferences into clinical trial design for obesity treatments. They were able to estimate the tradeoffs in risks that obese patients are willing to accept in exchange for a certain amount of weight loss, and the minimum number of pounds they would have to lose to tolerate the risks of a weight loss device.

Shortly after the study was published, FDA approved a new weight loss device – the Maestro Rechargeable System, an important therapeutic option for obese patients. The decision to approve the device was based in part on the data from Irony’s study that showed a substantial portion of obese patients would accept the risks associated with a surgically implanted device if they lost a sufficient number of pounds. Maestro is the first FDA-approved obesity device since 2007.

Our Patient Preferences Initiative is testing other ways to reach out to patients and capture their views through public workshops, websites, and a new patient-focused advisory committee. CDRH is also participating in related research as a member of the Medical Device Innovation Consortium (MDIC), a non-profit partnership between the FDA, National Institutes of Health, Centers for Medicare & Medicaid Services, and 43 medical device companies, patient groups and other non-profit organizations. MDIC is developing a framework for incorporating patient preferences into the device development and assessment process, and compiling a catalog of methods for collecting patient preference information that can be used to develop, design, and market devices that meet the needs of patients. Simultaneously, CDRH is developing draft guidance outlining how data from patient preference assessment tools can inform device approvals and other regulatory decision making.

As patient groups, industry sponsors, and others conduct more patient preference studies, we will better understand the tradeoffs that patients with medical device-treatable diseases and conditions are willing to make. This research, along with actions taken by CDRH, MDIC and others will drive more patient-centered device development and assessment. As a result, patients will play an influential role in determining which treatments and diagnostics are available in the U.S. market.

It may have taken more than 30 years, but patients are finally having their say.

We should take care to listen.

Kathryn O’Callaghan is Associate Director for Science and Strategic Partnerships (Acting), FDA’s Center for Devices and Radiological Health

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

A CDRH Priority: Clinical Trials in the U.S.

By: Owen Faris, Ph.D., and Jeffrey Shuren, M.D., J.D.

At the Center for Devices and Radiological Health (CDRH), clinical trials are the foundation for our decisions to approve the most important medical devices—products that have the potential to save or sustain life, but that also present the greatest risk to patients.

Owen Faris

Owen Faris, Ph.D., Clinical Trials Director (acting), Office of Device Evaluation in FDA’s
Center for Devices and Radiological Health

Over the past year, we saw several exciting new medical devices reach U.S. patients, including devices to treat heart disease and diabetes and diagnose cancer. Just last week, we approved a new device to treat obesity. None of these products would have come to market without clinical trials.

CDRH is committed to improving U.S. patient access to new devices by strengthening and streamlining the process of testing complex medical devices so that their clinical trials are conducted in the U.S. in a safe, efficient and cost-effective manner. In fact, this is so important for us that we made it one of our three 2014-2015 Center Strategic Priorities, along with striking the right balance between premarket and postmarket data collection and improving our customer service. Please visit our website for an update on our Strategic Priorities.

Innovative medical products begin with clinical trials – and before a clinical trial of a significant risk device begins in the U.S., a researcher, among other things, must apply for and receive FDA’s approval through the Investigational Device Exemption (IDE) process.  The FDA reviews IDE applications to determine whether the sponsor has provided enough information to be sure that the study does not present an unreasonable risk to its participants. FDA takes into account the qualifications of the clinical investigators, information about the device, the design of the clinical investigation, the condition for which the device is to be investigated, and the health status of the participating patients.

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health

FDA reviews an IDE submission within 30 days, but the review often results in questions which the study sponsor needs to answer, or changes that are needed before the study can be approved. Just a few years ago, it was therefore not uncommon for a year or more to pass before FDA could grant approval to a medical device developer to begin the trial. This type of delay was one factor that led developers to seek approval in other countries.

Over the past year, CDRH has taken a number of actions to expedite the safe initiation of clinical trials in the U.S., and we believe these policies will result in conducting clinical studies in the U.S. earlier in the device development process than was the case in the past.

Our improvements started with establishing a formal Clinical Trials Program within the Office of Device Evaluation. This program provides consistency in decision-making and encourages more interaction between FDA and the device industry during the IDE process. We also provided extensive training to CDRH review staff and the device industry. In addition, we issued numerous guidance documents, including one explaining IDE Decisions and one introducing CDRH’s new Early Feasibility Study program.

We’re excited to report that these changes have greatly shortened the time for an IDE to reach approval, so that a clinical trial can begin. From 2011 to 2014, the median number of days to full IDE approval has decreased from 442 to only 101. This cuts the time it takes to bring a new medical device to market by nearly a full year.

To learn more about CDRH’s clinical trials program, please join us for a webinar on January 22, 2105, where we will discuss the implementation of the IDE processes, our 2015 performance goals, early feasibility studies and our future plans. More information, including how to attend, is on the CDRH Webinar webpage.

The FDA is charged with the enormous task of protecting and promoting the health of the American public. To do this, we must ensure that the medical products on which Americans rely every day have been rigorously tested and are safe and effective. We are committed to making U.S. patients the first in the world to have access to safe and effective medical devices. And we’ve taken the first step to that, by helping ensure that clinical trials take place here, in the U.S.

Owen Faris, Ph.D., is Clinical Trials Director (acting), Office of Device Evaluation in FDA’s Center for Devices and Radiological Health

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

Australia, Brazil, Canada, Japan, and the US: Safeguarding Medical Devices

By: Kim Trautman, M.S.

The FDA and its regulatory counterparts abroad have the weighty responsibility of ensuring the safety of the thousands of regulated medical devices imported in their countries each year. To make this task more manageable, FDA and regulatory agencies in Australia, Brazil, Canada, and Japan embarked in 2014 on a pilot called the Medical Device Single Audit Program (MDSAP). Its goal is to develop a process that allows a single audit, or inspection to ensure the medical device regulatory requirements for all five countries are satisfied, in an efficient yet thorough manner.

Kim TrautmanOn January 1, 2015 the MDSAP pilot reached a major milestone – manufacturers around the globe interested in marketing medical devices in Australia, Brazil, Canada, and the U.S. were invited to participate in the program. This summer, when Japan enters the MDSAP as a full member, the same invitation will be issued also to medical device manufacturers interested in marketing in Japan.

Under this pilot, audits will be conducted by recognized third-party organizations, and medical device regulators in the participating countries will be able to use these inspection reports when making their regulatory decisions. Not only does this program reduce the participating regulators’ need to individually perform routine inspections; it allows them all to have the same reliable information about inspectional findings.

Manufacturers, too, can benefit from the MDSAP pilot by cutting down on the number of regulatory audits they have to host, thereby minimizing manufacturing plant and personnel disruptions. This form of international and standardized oversight lessens the burden on manufacturers by bringing more consistency and transparency to the regulatory process.

The MDSAP pilot does not increase regulatory requirements for medical device manufacturers – the audits cover only existing requirements of the regulatory authorities participating. In many cases, these requirements are already harmonized or very similar to one another, such as the international standard for medical devices quality management systems (ISO 13485:2003), the Brazilian Good Manufacturing Practices (RDC ANVISA 16/2013), the U.S. Quality System Regulation (21 CFR Part 820), and other specific pre- and post-market regulatory requirements of the authorities participating in the MDSAP pilot.

The FDA will accept MDSAP audits as a substitute for routine FDA inspections, typically done every two years for all classes of medical devices and including in vitro diagnostic devices. Pre-approval inspections for devices requiring premarket approval applications (PMAs) and “for cause” compliance inspections will not be part of the MDSAP pilot.

Manufacturers that choose to participate in the pilot program will help to shape the policies and procedures of the fully operational MDSAP, which is scheduled to begin in 2017. We expect that the MDSAP pilot will enhance confidence in third party audit programs, increasing the footprint of this global endeavor.

The FDA is pleased to be part of this MDSAP pilot. International cooperation promotes global alignment of regulatory approaches and technical requirements, expanding the safety net that protects patients world-wide.

New information about how countries will participate in the MDSAP pilot is available on the FDA’s MDSAP pilot web page.  Manufacturers can find additional information on the MDSAP web pageThis MDSAP page provides information on the auditing organizations involved in the pilot for interested manufacturers to contact directly.

Kim Trautman is Associate Director of International Affairs at the FDA’s Center for Devices and Radiological Health

Implementing the Unique Device Identifier System into health care systems is critical for reaching its potential to benefit public health

By: Jeffrey Shuren, M.D., J.D.

As the FDA works with manufacturers to launch a new system of identifying medical devices using standard bar codes and numbers, we look forward to the day when the system, called the Unique Device Identifier (UDI) system, will be fully set up— with identifiers on device labels and a corresponding database of identifying information about most of the devices in the U.S. marketplace.

But why does that matter?

Jeffrey ShurenMuch like vehicle identification numbers (VINs) for automobiles, UDIs are intended to streamline the monitoring of devices, improve safety tracking and recall efficiency, and even make it easier to evaluate device performance over time. So while there’s little doubt that UDI can improve patient safety, modernize how we evaluate devices once they are in use, and facilitate future device innovation, these benefits will only become a reality when the UDI system is adopted and integrated into the health care system—when hospitals, doctors’ offices, patient registries, heath care insurance companies, and others incorporate UDI as part of their standard electronic health information systems.

Without the practical implementation on the clinical side, UDI will be codes and a database with limited utility to improve patient care or reach its other critical goals.

The FDA is thinking about this now—not later. While going full steam ahead to fulfill our responsibility for implementing UDI regulations for medical device manufacturers, we are doing everything we can to promote the widespread adoption of UDI in the U.S. health care system.

We commissioned the Brookings Institution to create a “roadmap” for provider systems, patients, payers, supply chain personnel, and many others, to adopt and utilize UDIs. This report, released on Friday, December 5, provides 17 recommendations for adopting UDIs across three major intersections of the health care system—providers (e.g., electronic health records, hospital inventory management, billing records); administrative transactions (e.g., claims data and payment information); and patient-directed tools (e.g., mobile apps and public awareness campaigns).

We’re working hard to create and populate an efficient and useful UDI system for medical devices. But even the perfect system will fail to improve patient care if it’s not properly integrated into electronic health information systems. That process has to start now.

Today, we are co-sponsoring with Pew Charitable Trusts and the Department of Health and Human Services Office of the National Coordinator (ONC) a meeting where some 400 experts are convening to discuss changes that are needed to store and share UDI information throughout the health care system, with the ultimate goal of improving patient care.

The goal is to have the UDI system not only up and running—but actually used as the key to unlock important data that can help patients.

But how does such a system really help patients and the providers who care for them? Consider a possible scenario where the connections made via UDI could make an important difference in patient care.

A patient undergoing knee surgery—we’ll call him John—has the UDI of his knee implant scanned and electronically recorded into his clinical record.

When John is discharged, he can also register the UDI into his personal health record (PHR), available from his provider, through a variety of mobile apps that can enable two-way communication with his provider.

Having the UDI recorded will help John to know if safety alerts apply to his specific implant. It will also help him accurately report any potential adverse event to the provider, the FDA, or the manufacturer, with the confidence that the UDI ensures that all parties know what the type of device may be causing John—and possibly other patients—problems. Importantly, if John hears about knee implants being recalled, he will be able to quickly pinpoint, by using his UDI, if his particular type of implant is involved in that recall. If it’s not, John may avoid needless anxiety; if it is, he can take any necessary action, such as following up with his orthopedic surgeon.

The UDI from John’s surgery is also available to be transmitted to a total joint replacement registry, without any of his personal information. Data from the registry may then be used to support the development of innovative implants and reduce the data requirements for — or replace altogether — postmarket studies conducted by the device manufacturer to demonstrate long-term performance.

The possibilities of UDI are exciting—better and more precise information can lead to better care and better awareness of how medical devices work in the general population. The FDA is working to set up the system, but implementation and integration are critical. The question is—if we build it, will people adopt it?

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

China Journal: strengthening relationships to protect public health

By: Margaret A. Hamburg, M.D.

I am just about to wrap up a jam-packed five-day visit to China, a fascinating country with a dramatically growing economy and with an increasingly significant impact on the products that Americans consume. Indeed, a key reason for my trip is the important and growing collaboration between FDA and our counterpart agencies in China to ensure the safety of the large volume of foods and medical products exchanged between our two nations.

Margaret Hamburg, M.D.Of the 200 countries that export their products to the United States, China ranks first in exports (in dollar value) to our nation. It is the sixth largest provider of food and the sixth largest provider of drugs and biologics. Only the United States has more FDA-registered drug establishments than China. And these numbers are growing. Between 2007 and 2013, China’s annual exports of FDA-regulated products to the U.S. nearly quadrupled, reaching 5.2 million “lines” (portions of a shipment) of imported goods in 2013.

Ensuring the safety and quality of these and other U.S.-destined FDA-regulated goods is a major challenge. To meet it, FDA has transformed itself— from a domestic agency that focused primarily on products manufactured in the U.S. to a truly global agency grappling with the many challenges of globalization.

Among the many efforts in this area, an important component is the FDA’s establishment of permanent outposts staffed by FDA experts in all major exporting regions, including in China. We have 13 FDA staff members currently stationed in the country, primarily in Beijing. Their job is to help ensure that the food and medical products being exported from China meet our standards. FDA’s China Office does this by providing significant support for the Agency’s inspections in China, by strengthening our relationships with Chinese regulators, by working with industry and other stakeholders, by providing important information and technical assistance to all interested parties, and by analyzing trends and events that might affect the safety of FDA-regulated products exported from China to the United States.

Given the volume of U.S. trade with China, we are working to more than triple the number of American staff we place in China. Placing more FDA experts in China will allow FDA to increase significantly the number of inspections it performs in this dynamic, strategic country, as well as to be more effective partners with our colleagues here in China. Such dramatic staffing increases will also allow FDA to enhance its training efforts and technical collaboration with Chinese regulators, industry and others.

This week, we took an important step forward in strengthening our relationship with China when we signed an Implementing Arrangement with the China Food and Drug Administration (CFDA). We expect to sign a similar Implementing Arrangement with the General Administration of Quality Supervision, Inspection and Quarantine (AQSIQ) in the coming weeks. These documents, which build on 2007 agreements with the same two agencies, help to frame the work our inspectors will do in China and create mechanisms for collaboration on inspections.

FDA is also engaging with other stakeholders to create sustainable models for training future champions of regulatory science and quality. Here in China, we helped to create a world-class graduate degree program in international pharmaceutical engineering management (IPEM) at Peking University (PKU), an institution renowned for educating Chinese leaders and thinkers.

This partnership with PKU began in 2005 with just two courses on current good manufacturing practices. These proved hugely successful, and drew attention from Chinese drug companies and regulatory agencies, as well as industry and regulators in neighboring countries. The following year, PKU established a master’s degree program in IPEM, with support from FDA and multinational pharmaceutical companies. The program was formally launched in March 2007, with courses in regulatory science, pharmaceutical science, engineering, and more.

One of the highlights of my trip this week was speaking to more than 200 PKU students, future leaders who will help to accelerate the modernization of this nation’s pharmaceutical industry. I discussed not only FDA’s growing regulatory cooperation with China but the importance of strengthening regulatory science in China to ensure that the highest standards are used to support the development, review, and approval of new medical products, as well as the manufacturing and safety monitoring of medical products. All of this can make an enormous difference in the lives of patients in China, the U.S. and beyond.

Also this week, I met with top Chinese regulatory officials, toured CFDA’s mobile laboratories that test for counterfeit drugs and contaminants in food, and attended the 9th International Summit of Heads of Medicines Regulatory Authorities in Beijing.

Throughout the week, we addressed tough problems that require global solutions. Our discussions ranged from how best to advance biomedical product innovation, expand access to important pharmaceuticals through generic and biosimilar regulatory pathways, and how coordinated action, along with using new, state-of-the art technologies and analytical methods, will more effectively protect the public from substandard or counterfeit products. We are also making tangible progress in strengthening FDA’s partnership with our Chinese counterparts to better oversee the increasingly complex international supply chain and to prevent problems before they occur.

As I prepare for the journey home, I am encouraged by what we accomplished. And all of this bodes well for our ability to promote and protect protect public health in the future.

Margaret A. Hamburg, M.D., is the Commissioner of the Food and Drug Administration

View Photos from China:

Commissioner Margaret A. Hamburg, M.D., tours an FDA China Office mobile lab that tests for counterfeit OTC drugs and contaminants in food

Commissioner Margaret A. Hamburg, M.D., meets with Chinese pharmaceutical executives

Commissioner Margaret A. Hamburg, M.D., with students of Peking University