FDA Working to Keep Patients Well Informed

By: Steve L. Morin R.N., B.S.N.

Steve Morin_2823My job in the Food and Drug Administration’s Office of Health and Constituent Affairs (OHCA) is to serve our nation’s patients in two ways: by listening to their concerns regarding FDA’s policy and decision-making and advocating for them in our agency;  and by informing many patients and patient organizations about FDA’s mission and its work to advance the development, evaluation and approval of new therapeutic products.

This dialogue was formalized and greatly expanded in 2012 when, after a series of listening sessions with many patient advocacy organizations, OHCA created the Patient Network.

Specifically designed for patients, caregivers, patient advocates and disease-specific patient advocacy organizations and the communities that advocate on their behalf, this program serves two goals. It facilitates patient engagement with FDA policy and decision makers, and it educates its audience about the process that brings new medications – both prescription and over-the-counter ­– and medical devices from a concept to the marketplace.

Our Patient Network covers a range of FDA-specific topics and conducts numerous activities that are of interest to patients and patient advocates. One of these activities were webinars with information about upcoming public meetings hosted by FDA.

For example on March 31, 2014, OHCA was pleased to host the first-of-its-kind “LiveChat” with the diabetes community. This online discussion gave patients an opportunity to interact with FDA experts and to better understand a recently released draft guidance dealing with the studies and criteria that FDA recommends be used when submitting premarket notifications (510(k)s) for blood glucose meters.

On September 10, 2014, our Third Annual Patient Network Meeting titled “Under the Microscope: Pediatric Product Development” brought together more than 100 patients, patient advocates, representatives of academia and industry, and FDA leaders. The participants discussed pediatric product development and the ways patient advocates can participate in it.

And on September 17, 2014, our Patient Network webpages were upgraded. The “For Patients” section on FDA’s website is presented in a clear manner with easy-to-use formats. Also, a “For Patients” button is located on our homepage.

We have continued to pursue our goals of informing the public and engaging with patients by building upon the patient-centered webpages and enhancing activities that express our desire to be helpful and transparent. This is our philosophy that has helped the Patient Network evolve to what you see today.

As the Patient Network program continues to grow, I hope to expand it to have more interactive webinars like the “LiveChat” that address specific concerns  of the patient communities. Also, we will continue to make it possible for patients to learn from FDA experts who approve medical products.

The FDA realizes that listening to the “patient voice” and conducting our dialogue is important, and it continues to develop its model for patient involvement through the Patient-Focused Drug Development Meetings and other OHCA sponsored meetings and webinars. We hope patients and those who care for them will join us in that effort, and make it still more helpful in protecting and promoting the public health.

Steve L. Morin, R.N., B.S.N., is a Commander of the United States Public Health Service and the Manager of the Patient Network in FDA’s Office of Health and Constituent Affairs

Regulatory Science Collaborations Support Emergency Preparedness

By: Jean Hu-Primmer, M.S.

Scientists love a challenge. And coordinating government agencies, healthcare providers, and numerous additional partners to protect public health in emergency situations is definitely a challenge.

Jean Hu-Primmer

Jean Hu-Primmer, Director of Regulatory Science Programs in FDA’s Office of Counterterrorism and Emerging Threats.

FDA’s Medical Countermeasures Initiative (MCMi) is working with federal agencies (through the Public Health Emergency Medical Countermeasures Enterprise), product developers, healthcare professionals, and researchers, among other partners, to help translate cutting-edge science and technology into safe, effective medical countermeasures. Through these collaborations, MCMi supports research to help develop solutions to complex regulatory science challenges.

Data are critical to help FDA evaluate the safety and effectiveness of medical countermeasures—products that can save lives—during public health emergencies. But collecting data in the midst of an emergency is exceptionally challenging. Working with the Biomedical Advanced Research and Development Authority (BARDA), FDA is teaming with critical care physicians nationwide to help address these challenges.

Under a contract awarded last month, FDA and BARDA will work with the U.S. Critical Illness and Injury Trials Group (USCIITG) to gather important information about medical countermeasures used during public health emergencies. Physicians will help address challenges with collecting and sharing data rapidly in emergencies, including streamlining electronic case reporting for clinical trials and rapidly disseminating key findings to FDA and other stakeholders to support clinical decision-making.

During this four-year project, USCIITG will also develop and pre-position a simple influenza treatment protocol in 10 hospitals throughout the U.S. during the 2015-2016 influenza season. The project will help doctors more easily use an investigational treatment protocol for patients with severe influenza, and test the data collection and reporting system during peak times. The goal is to help streamline the process during future influenza seasons and emergencies.

When it is not ethical or feasible to test the effectiveness of products in humans—such as countermeasures for potential bioterror agents—products may be approved under the Animal Rule. When products are approved under the Animal Rule, FDA requires additional studies, called phase 4 clinical trials, to confirm safety and effectiveness. In addition to the MCMi work, BARDA is funding USCIITG to investigate conducting phase 4 clinical studies during public health emergencies. USCIITG partners will train on these protocols, have them reviewed through their Institutional Review Boards (a requirement for all human studies), and create plans for enactment. USCIITG will then conduct an annual exercise to test these plans, a unique approach to broader science preparedness.

MCMi has also recently awarded regulatory science contracts to support other aspects of emergency preparedness, including two projects to investigate decontamination and reuse of respirators in public health emergencies (awarded to Battelle and Applied Research Associates, Inc.), and an award to support appropriate public use of medical countermeasures through effective emergency communication.

Our work involves big challenges. Through regulatory science, and through new and expanding collaborations, we continue to address these challenges to better prepare our nation to use medical countermeasures in emergencies.

Want to help? We’re currently accepting submissions for additional research to support medical countermeasure preparedness. If you have an idea for a new medical countermeasure regulatory science collaboration, we’d love to hear from you.

You can also visit BARDA’s MCM Procurements and Grants page for more information.

Jean Hu-Primmer, M.S., is Director of Regulatory Science Programs in FDA’s Office of Counterterrorism and Emerging Threats.

FDA and the Cybersecurity Community: Working Together to Protect the Public Health

By: Suzanne Schwartz, M.D., M.B.A.

Cyber vulnerabilities – bugs or loopholes in software codes or other unintentional access points – are a real and constant threat to our networked laptops, mobile phones, or tablets. The Heartbleed virus and security breaches at major retailers are just a few recent examples of exploits of this hazard that have been in the news.

Suzanne SchwartzWhat you may not know is that there is a coordinated network of cybersecurity researchers, software engineers, manufacturers, government staffers, information security specialists, and others who share the responsibility of discovering and closing these security gaps. As a result, many vulnerabilities are detected and fixed before they seriously affect the public.

Medical devices that contain computer hardware or software or that connect to computer networks are subject to the same types of cyber vulnerabilities as consumer devices. The consequences of medical device breaches include impairing patient safety, care, and privacy. And as in the case of consumer devices, strengthening the cybersecurity of medical devices requires collaboration and coordination among many stakeholders, as well as a shared sense of responsibility for reducing the cybersecurity vulnerabilities.

This is why on October 21-22, 2014 the FDA, the Department of Homeland Security (DHS), and the Department of Health and Human Services (DHHS) will host a public meeting, Collaborative Approaches for Medical Device and Healthcare Cybersecurity.   The purpose of the meeting is to catalyze collaboration in the health care and public health sector to more fully address medical device cybersecurity. The meeting will bring together medical device manufacturers; health care providers; biomedical engineers; IT system administrators; professional and trade organizations; insurance providers; cybersecurity researchers; local, state and federal government staffs; and representatives of information security firms. They will explore topics such as:

The cybersecurity of medical devices is an important part of public health safety, and the FDA has a significant role. In addition to convening this meeting, the FDA entered into a partnership with the National Health – Information Sharing and Analysis Center (NH-ISAC), a non-profit organization that closely cooperates with government agencies, and numerous health care and public health organizations. The partnership will enable FDA and NH-ISAC to share information about medical device cybersecurity vulnerabilities and threats. It will foster the development of a shared risk framework where information about medical device vulnerabilities and fixes is quickly shared among health care and public health stakeholders.

In addition, on October 1 the FDA released a final guidance for the Content of Premarket Submissions for Management of Cybersecurity in Medical Devices. The guidance recommends that manufacturers consider cybersecurity risks as part of the design and development of a medical device, and submit documentation to the FDA about the risks identified and controls in place to mitigate those risks. We think this will help improve the cybersecurity of medical devices and help contribute to the strengthening of our Nation’s health care cybersecurity infrastructure.

The FDA shares the responsibility of managing and reducing cybersecurity risks with many other stakeholders, and we look forward to hearing from them at the public meeting on October 21-22. We’re committed to working together to build a comprehensive cybersecurity infrastructure that can detect and respond to vulnerabilities in a timely way and that best protects the public health.

Suzanne B. Schwartz, M.D., M.B.A., is Director of Emergency Preparedness/Operations & Medical Countermeasures (EMCM) at FDA’s Center for Devices and Radiological Health.

FDA’s Program Alignment Addresses New Regulatory Challenges

By: Margaret A. Hamburg, M.D.

Over the last year, a group of senior FDA leaders, under my direction, were tasked to develop plans to modify FDA’s functions and processes in order to address new regulatory challenges. Among these challenges are: the increasing breadth and complexity of FDA’s mandate; the impact of globalization on the food and medical product supply chains; and the ongoing trend of rapid scientific innovation and increased biomedical discovery.

Margaret Hamburg, M.D.The Directorates, Centers and the Office of Regulatory Affairs (ORA) have collaborated closely to define the changes needed to align ourselves more strategically and operationally and meet the greater demands placed on the agency. As a result, each regulatory program has established detailed action plans. Specifically, each plan describes the steps in transitioning to commodity-based and vertically-integrated regulatory programs in the following areas: human and veterinary drugs; biological products; medical devices and radiological health; bioresearch monitoring (BIMO); food and feed; and tobacco.

These action plans focus on what will be accomplished in FY 2015 and outline the need to develop detailed future plans for the next five years in some cases. The plans represent what each Center and ORA have agreed are the critical actions to jointly fulfill FDA’s mission in the key areas of specialization, training, work planning, compliance policy and enforcement strategy, imports, laboratory optimization, and information technology.

Because each Center has a unique regulatory program to manage, there are understandably variations among the plans. However, there are also common features across most of the plans: the need to define specialization across our inspection and compliance functions; to identify competencies in these areas of specialization and develop appropriate training curricula; to develop risk-based work planning that is aligned with program priorities and improves accountability; and to develop clear and current compliance policies and enforcement strategies.

Below are some highlights from the plans that illustrate these features:

  • Establish Senior Executive Program Directors in ORA. In the past, for example, the Center for Drug Evaluation and Research (CDER) would work with several ORA units responsible for the pharmaceutical program. Now, the Centers will have a single Senior Executive in ORA responsible for each commodity program, allowing ORA and the Centers to resolve matters more efficiently.
  • Jointly develop new inspection approaches. The Center for Devices and Radiological Health (CDRH) and ORA plan, for example, will begin to focus some inspections on characteristics and features of medical devices most critical to patient safety and device effectiveness. ORA investigators will perform these inspections utilizing jointly developed training.
  • Invest in expanded training across ORA and the Centers. The Center for Biologics Evaluation and Research (CBER) and ORA will jointly develop a biologics training curriculum, redesign investigator certification, and cross-train Center and ORA investigators, compliance officers and managers.
  • Expand compliance tools. Field investigators will be teamed with subject matter experts from the Center for Food Safety and Applied Nutrition and the Center for Veterinary Medicine to make decisions in real time, working with firms to achieve prompt correction of food safety deficiencies and to help implement the preventive approaches outlined by the FDA Food Safety Modernization Act (FSMA). If industry does not quickly and adequately correct critical areas of noncompliance that could ultimately result in food borne outbreaks, we will use our enforcement tools, including those provided under FSMA, as appropriate.
  • Optimize FDA laboratories. ORA and the various Centers will establish a multi-year strategic plan for ORA scientific laboratory work, including hiring and training analysts, purchasing and using equipment, and allocating resources and facilities. At the same time, ORA is committed to conducting an ongoing review of its labs to ensure that they are properly managed and operating as efficiently as possible.
  • Create specialized investigators, compliance officers, and first-line managers. A bioresearch monitoring (BIMO) working group is developing a plan for a dedicated corps of ORA investigators to conduct BIMO inspections, and a dedicated cadre of tobacco investigators is being established.

Working together to implement these action plans will take time, commitment, and continued investment and we’ll need to monitor and evaluate our efforts. These plans will help us implement the new FSMA rules announced in September, as well as the Agency’s new medical product quality initiatives under the FDA Safety and Innovation Act and Drug Quality and Security Act.

FDA’s Program Alignment is a well-thought out approach that responds to the needs of a changing world. I look forward to the ways in which these action plans will ultimately enhance the FDA’s public health and regulatory mission.

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

New Data Dashboard Tool Shares FDA’s Inspection, Compliance and Recall Data

By: Douglas Stearn

Douglas StearnAs part of our commitment to transparency FDA is pleased to announce that we have released a new online tool to provide insight into our compliance, inspection, and recall activities.

This new dynamic tool represents a departure from the downloadable spreadsheet-based datasets that we have posted in the past. Instead, the FDA data dashboard presents information in an easy-to-read graphical format. It also provides access to the underlying data allowing anyone interested to see related data and trends.

Our new dashboard provides data for FY 2009 to FY 2013, and allows access to data on:

  • inspections;
  • warning letters;
  • seizures and injunctions;
  • and statistics, specifically for recalls.

We plan to update the data semi-annually.

The dashboard is staged in a cloud environment, and it allows you to:

  • download information for additional analysis;
  • manipulate what you see by selecting filters;
  • rearrange the format of datasets and the way columns are sorted;
  • drill down into data; and
  • export charts and source information for further review.

We developed this new dashboard after President Obama issued a Presidential Memorandum on Regulatory Compliance in January 2011.

The President directed federal agencies to make publicly available compliance information easily accessible, downloadable and searchable online, to the extent feasible and permitted by law. FDA formed internal working groups that same year to develop recommendations for enhancing the transparency of our operations and decision-making processes. These working groups identified an online tool as a way to present compliance and enforcement data in a user-friendly manner. The dashboard represents the latest example of our commitment to compiling and posting a wealth of FDA data  for public review and feedback.

FDA works within a global environment and is carrying out more inspections around the world. We collaborate with regulatory authorities across the globe to protect public health. Our data dashboard provides information about inspections in this global environment, and makes this information more readily accessible to the public. Now you can use the dashboard to see this kind of inspection-related information to better understand our regulatory decisions.

A “feedback mechanism” is available so you can send comments, questions or concerns directly to us at FDADataDashboard@fda.hhs.gov.

This rollout effort is part of FDA’s continuing commitment to share inspection, compliance, and recall data. We will continue to update the FDA data dashboard and provide public access to this timely and important information.

Douglas Stearn is Director of the Office of Enforcement and Import Operations within FDA’s Office of Regulatory Affairs

FDA’s New Roadmap for Progress: Strategic Priorities 2014-2018

By: Margaret A. Hamburg, M.D

The U.S. Food and Drug Administration regulates products that represent about 20 cents of every dollar American consumers spend on products. This includes the safety and effectiveness of drugs, medical devices, and vaccines, the safety of blood supply to food supply, cosmetics, dietary supplements, products that emit radiation, and more recently, tobacco. This fact can be easy to gloss over, but if one pauses for a moment to reflect on this fact, it is clear that the FDA’s regulatory role is large and truly meaningful to all of our everyday lives.

Margaret Hamburg, M.D.When the FDA was first established, our regulated industries were predominantly local, the volume of imported products was low, and even the movement of goods across country was minimal. But times have changed, and so have the strategies we employ to address those changes. Over the last five years alone, the FDA’s regulatory portfolio has increased to now include regulating tobacco products, developing a new global system for protecting food safety, and addressing challenges created by the global expansion of research, commerce and trade.

In fact, more often than not today, a drug or medical product that ends up on the shelves of an American drugstore or in our hospitals will come, at least in part, from some foreign source. Nearly 40 percent of finished medicines that Americans now take are made elsewhere, as are about 50 percent of all medical devices. Approximately 80 percent of the manufacturers of active pharmaceutical ingredients used in the United States are located outside our borders.

These and other new challenges and transformative developments in global science, technology and trade are rapidly altering the environment in which we work to fulfill our broad public health mission. In order to continue to carry out that mission, we need a set of clearly defined priorities and goals, as well as the strategies for reaching them. Therefore, I am pleased to announce the release of a revised set of FDA Strategic Priorities which will guide the agency in how we continue to promote and protect the health of the American public.

The new Strategic Priorities document sets the path for our Agency over the next four years. It establishes a framework for integrating our five strategic priorities – regulatory science, globalization, safety and quality, smart regulation, and stewardship.

Although each priority is significant in and of itself, the priorities are also interconnected and must not be addressed in isolation. In addition, this new roadmap sets forth FDA’s core mission goals and objectives, such as improving and safeguarding access to the products FDA regulates – and promoting better informed decisions about their use.

The Strategic Plan has been in development for more than a year and was created by a hard-working team of talented and knowledgeable FDA employees representing programs from across the agency. While this team drove the Plan’s creation, it is backed by the commitment of all of us at the FDA. My hope is that these priorities, which will be repeatedly cited in our speeches, policies and writings, will serve as our foundational guidepost, providing the strategic direction to help the agency continue to provide the level of service and protection the American people deserve.

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

20 Years of Improving Women’s Health: 1994 – 2014

By: Margaret A. Hamburg, M.D.

Margaret Hamburg, M.D.As we celebrate the 20th anniversary of the FDA’s Office of Women’s Health, I would like to highlight some of the work we’ve done to help improve women’s health, both looking across FDA and within the office. Whether it is approving new treatments for chronic conditions like heart disease, conducting research or helping to protect pregnant women from foodborne illnesses, the work we do at FDA makes a difference throughout a woman’s life.

Consider our product approvals. In 1996, for example, our agency approved a product for use in Pap smears that revolutionized the detection of cervical cancer; ten years later we approved the first vaccine for the prevention of this cancer. We have also approved advances in breast imaging, including 3D breast tomosynthesis and automated screening ultrasound.

We have encouraged innovation in lupus treatment and approved the first new lupus drug in 50 years. And we approved the latest generation of cardiac synchronization therapy devices which our own FDA scientists have shown particularly benefit women with heart failure.

FDA has also supported research to help us better understand how medical products affect women. Since 1994, the Office of Women’s Health research program has provided $30 million to support over 300 research projects, workshops, and trainings on a wide range of topics including cancer, HIV and osteoporosis. More than 25 percent of these research dollars have been directed at cardiovascular disease, the number one killer of women, with studies examining such issues as QT interval prolongation (a disorder of the heart’s electrical activity), how breast cancer drugs can affect the heart, and sex differences in various cardiac interventional therapies. FDA’s medical product centers have also sponsored women’s health research and initiatives such as the Health of Women Program that promote a better understanding of sex differences.

The results have been impressive: OWH’s research alone has been published in over 290 articles in peer-reviewed journals and has made impact on the regulatory decision-making process, including guidance documents, label changes, and standards development. Indeed, FDA’s guidance to industry is an important way that the agency has been helping to address important issues in women’s health.

Over the years, FDA guidance has encouraged greater inclusion of women in clinical trials and the evaluation of sex differences. Our own analysis last year found that women make up about half of the representation in these studies, but the numbers are lower for medical devices. So we have more to do and recently issued guidance to medical device developers to address this concern.

We have also made great strides in our communication and outreach to women during the past two decades. OWH’s Take Time to Care Program has built partnerships with other government agencies, retailers, and national organizations that provide millions of women with FDA safety information. Over the years, we have launched other educational initiatives like the Food Safety for Moms-to-Be and expanded the women’s health resources available via our “For Women” website and social media to make sure that women have tools to help them make informed decisions about the use of FDA-regulated products.

I am pleased at how much we have done to promote and protect women’s health since 1994. At the center of much of this change has been the consistent, driving force of the Office of Women’s Health and its determined leader, Marsha Henderson. I encourage you to check out OWH’s 20th Anniversary brochure to learn more about the progress that has been made. And I hope that you will collaborate with us on the work that still needs to be done.

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

Clinical Trials: Enhancing Data Quality, Encouraging Participation and Improving Transparency

By: Margaret A. Hamburg, M.D.

Today FDA is announcing important steps that the agency plans to take to enhance the collection and availability of clinical trial data on demographic subgroups – patient populations divided by sex, race/ethnicity or age.

Margaret Hamburg, M.D.Section 907 of the 2012 FDA Safety and Innovation Act directed us to take a closer look at the extent to which clinical trial participation and the inclusion of safety and effectiveness data by demographic subgroups is included in medical product applications, report our findings, and then, within one year, produce an action plan with recommendations for improvements.

Our report, issued on August 20, 2013, found that the agency’s statutes, regulations, and policies generally give product sponsors a solid framework for providing data in their applications on the inclusion and analysis of demographic subgroups. Overall, sponsors are describing the demographic profiles of their clinical trial participants, and the majority of applications submitted to FDA include demographic subset analyses. We also found that FDA shares this information with the public in a variety of ways. Now, one year later, we’re releasing the FDA Action Plan to Enhance the Collection and Availability of Demographic Subgroup Data, which we developed after extensive interaction with stakeholders.

The action plan includes 27 action items that are designed to meet three overarching priorities – improving the completeness and quality of demographic subgroup data collection, reporting and analysis (quality); identifying barriers to subgroup enrollment in clinical trials and employing strategies to encourage greater participation (participation); and, making demographic subgroup data more available and transparent (transparency).

In addition to the action plan, we’re publishing a final guidance entitled, “Evaluation of Sex-Specific Data in Medical Device Clinical Studies.” It was written in response to the fact that certain medical devices may yield different responses in women than men, and yet women are under-represented in some medical device studies. This has led to less information for women regarding the risks and benefits of using these devices.

The guidance includes recommended methods for clinical study design and conduct to increase enrollment of men and women, if needed, and ways to analyze data for sex differences. FDA has held a series of public workshops to raise awareness about common strategies for enhancing recruitment and retention of women in medical device clinical trials. Fully integrating this final guidance into the templates used by FDA’s reviewers of medical devices, and providing a webinar for industry on how to use the guidance, comprise one of the 27 items in our action plan.

I hope you’ll find that the action plan is responsive and pragmatic and, most importantly, when fully implemented, it will improve medical care and public health. Many of the steps it outlines will have a broad impact on the work of FDA’s medical product centers and will require great thought and planning as they are implemented, depending on current evidence and available resources. The action items range from relatively short-term goals that can be achieved in a year, to others that will take 1-3 years, to a small number that will require a longer period, 3-5 years, to achieve.

Although the plan certainly places significant responsibilities on FDA’s medical product centers and other FDA offices, it also engages our partners inside and outside of government to share the responsibility for this important mission. For example, industry is being asked to help develop and share best practices for encouraging broad clinical trial participation, and the National Institutes of Health will be participating in several research projects with FDA.

We know that richer information is collected when different subgroups are enrolled in pivotal studies for medical products. This kind of enrollment in turn gives us greater assurance in the safety and effectiveness of the medical products used by a diverse population.

To set the plan in motion quickly, FDA is setting up a steering committee that will oversee implementation, come up with metrics for measuring progress and be responsible for planning a public meeting to be held within 18 months after release of the plan. FDA has already set up a website where the public will be able to track the agency’s implementation progress. That website will be updated on a regular basis.

Also, we’re reopening our Section 907 public docket to solicit comments for the action plan. I encourage everyone to review the document and consider how you might be able to partner with FDA and others in encouraging necessary and appropriate demographic subgroup diversity and representation.

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

Providing Easy Access to Medical Device Reports Submitted to FDA since the Early 1990s

By: Taha A. Kass-Hout, M.D., M.S. and Jeffrey Shuren, M.D., J.D.

Taha Kass-Hout

Taha A. Kass-Hout, M.D., M.S.

In addition to food and drugs, FDA has regulatory oversight of tens of thousands of medical devices ranging from bandages and prosthetics to heart valves and robotics. These products are used by millions of Americans, and they are essential, well-performing tools of modern healthcare, but occasionally they present a safety issue due to risks not identified in prior studies, a malfunction, a problem with manufacturing, or misuse.

These incidents are collected in a publicly available FDA database called MAUDE – short for Manufacturer and User Facility Device Experience. As part of the openFDA project, there is now an Application Programming Interface (API) for this dataset, which provides a way for software to interact directly with the data. This API will allow developers and researchers to easily query thousands of reports dating back to the early 1990s.

The API can be a powerful tool for generating hypotheses for further investigation or inquiry and can inform the development of safer, more effective technologies. For example, it can help identify new, potential safety signals as well as which classes of devices may be associated with particular adverse events.

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D.

There are some necessary caveats to this API. The dataset is a record of reports submitted to FDA, and not a definitive accounting of every incident with every device. It may contain incomplete, inaccurate, unverified, or biased data. Thus, it cannot be used to determine incidence. And the appearance of a device in a report does not mean that cause-and-effect has been determined. Therefore, these data should be used in the context of other available information. It’s also important to note that the data made available under this initiative do not contain anything that potentially could be used to identify individuals or reveal other private information.

This API is the latest in a series of openFDA releases that have made publicly available data more easily accessed and queried. We believe that these tools can be used by developers and researchers to make insights that fuel new, innovative products (such as mobile apps and websites), and that help protect and promote the public’s health. Over the last two months, openFDA has released several APIs related to drugs, food, and devices. Together, they help provide perspective on the work FDA is doing, and make the public health data the agency is developing easier to access and utilize.

By design, openFDA is a research and development project that draws on community involvement. We are actively involved in the openFDA communities on GitHub and StackExchange, and encourage people interested in the project to participate in those communities. Together, we can make openFDA into a more useful, more powerful resource for the protection and advancement of the public health.

In addition to providing datasets, openFDA encourages innovative use of the agency’s publicly available data by highlighting potential data applications, and providing a place for communities to interact with one another and with FDA domain experts.

Taha A. Kass-Hout, M.D., M.S., is FDA’s Chief Health Informatics Officer and Director of FDA’s Office of Informatics and Technology Innovation

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

See more at: http://blogs.fda.gov/fdavoice/#sthash.COpthK14.dpuf

Curbing Risk, Not Medical Innovation, in Personalized Medicine

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

Innovative new tests are routinely submitted to the Food and Drug Administration to assure they are safe and effective. They include genetic tests that help oncologists decide whether a patient is a good candidate for a drug that treats melanoma as well as tests that are capable of sequencing the entire human genome.

Jeffrey ShurenBut many tests never undergo FDA premarket review to determine whether they are accurate, reliable, and clinically meaningful. These are laboratory developed tests (LDTs) designed, manufactured and intended to be used in a single laboratory.

FDA has exercised enforcement discretion over LDTs since 1976, when the agency first obtained comprehensive authority to regulate all in vitro diagnostics as medical devices. In those early days, LDTs were relatively simple, low risk, often for rare conditions, and generally only available on a limited basis.

But LDTs have evolved and proliferated because of advances in technology and evolving business models. Today, many LDTs are more complex, have a nationwide reach and have higher-risk uses such as detection of risk for breast cancer and Alzheimer’s disease. And yet they don’t undergo premarket review – or have adequate controls in place to assure proper test design and development, even when they compete with FDA-approved IVD test kits that conventional manufacturers market.

That’s concerning. Without appropriate safeguards, neither patients nor their health care providers can be assured that these tests are safe and effective. This is particularly troubling when an FDA-approved test is available, because it puts patients at unnecessary and avoidable risk. It also stifles innovation by creating disincentives for conventional manufacturers to invest in developing new, medically important tests.

We believe that LDTs serve an important role in health care and that there are many good tests on the market. Unfortunately, FDA is also aware of faulty or unproven LDTs, including ones that could cause patients to be inappropriately treated for heart disease; cancer patients to be exposed to inappropriate therapies or not get effective therapies; incorrect diagnosis of autism; and unnecessary antibiotic treatments.

That’s why FDA intends to propose a risk-based oversight framework that would appropriately balance assuring that patients and providers receive safe and effective tests with promoting innovation.

It would phase in enforcement of premarket review, quality systems, and adverse event reporting requirements for high- and moderate-risk LDTs over many years, beginning with the highest-risk tests (which include companion diagnostics—crucial to personalized medicine by targeting treatments for cancer, heart disease and other conditions) to give laboratories time to comply. Moreover, we intend to leverage existing programs, such as third party review and third party inspection as appropriate, and explore opportunities to work with entities that have experience with labs, thereby creating more efficiencies for labs to meet applicable FDA requirements.

On the other hand, under our upcoming proposed framework, we intend to continue exercising enforcement discretion with respect to the premarket review requirements for tests that labs make for rare diseases, to address an unmet need, or that are low risk.

Labs and conventional manufacturers serve as vitally important sources of innovative test development. Through smart, appropriately tailored oversight, we can best promote product development by all test developers and best serve patients and their healthcare providers.

When everyone plays by the same rules, innovation and society benefit.

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