FDA Encourages Development of Devices for Patients with Disabilities

By: William Maisel, M.D., M.P.H.

William Maisel, M.D., M.P.H.For people with disabilities, medical devices can offer a vital and potentially life-changing option. Take, for example, a patient who has had his arms amputated. Medications can treat phantom pain, but they can’t help that patient pick up a glass of water. But devices can—and do.

In recent months, FDA has reviewed a number of noteworthy products for people with disabilities. And it has approved, cleared or allowed manufacturers to market several new devices.

Products that have met FDA’s premarket requirements include:

  • The DEKA Arm System, the first prosthetic arm that can perform multiple, simultaneous, powered movements controlled by electrical signals from electromyogram (EMG) electrodes;
  • The Nucleus Hybrid L24 Cochlear Implant System, which can help people aged 18 and over (who don’t benefit from conventional hearing aids) with a specific kind of hearing loss; and
  • The Argus II Retinal Prosthesis System, the first implanted device to treat adult patients with vision loss from advanced retinitis pigmentosa (RP).

FDA is committed to encouraging such innovation that benefits patients. We foster an approach that enables our staff to interact with device manufacturers and clarify our agency’s expectations for product evaluation. This communication can help new devices get to market in a timely fashion. We also listen to patients’ feedback, which helps us determine which devices may be particularly useful.

When it comes to regulatory decisions, we carry out tailored reviews that protect public health while advancing innovation. Each of the products recently approved or cleared by the agency has benefits that outweigh its risks. For example, in June we allowed marketing of ReWalk, a first-of-its-kind, motorized device. Risks associated with the exoskeleton-like device include pressure sores and injuries from falls. But the big benefit is that it can help patients with complete or partial paraplegia to actually walk in their homes and communities.

We have seen amazing advances in technology in recent years. These advances make it possible for manufacturers to address longstanding disabilities in innovative ways. That said, we will continue to acknowledge that all medical therapies have benefits as well as risks. So, when making regulatory assessments, we’ll make every effort to make sure our decisions are balanced, and to ensure that approved or cleared devices can aid the patients who use them.

In addition to helping patients across the country, we are committed to empowering agency employees. For instance, FDA Commissioner Margaret A. Hamburg, M.D., recently held a meeting with the agency’s Advisory Committee for Employees with Disabilities (ACED). There, the committee provided an annual update and discussed topics that impact employees with disabilities, including making sure all buildings are accessible and facilitating access to assistive and adaptive technologies through a new Ergonomic Resource Center at our headquarters. And this month the committee held an additional, internal roundtable event to focus on continued awareness, timed to National Disability Employment Awareness Month.

People with disabilities make many important contributions to our agency and to society at large. It’s our goal and commitment to help them maintain an active lifestyle and enjoy a good quality of life.

William Maisel, M.D., M.P.H., is FDA’s Deputy Center Director for Science and Chief Scientist for its Center for Devices and Radiological Health.

FDA as part of a coordinated global response on Ebola

By: Margaret A. Hamburg, M.D.

The tragic Ebola epidemic is an extraordinary global public health crisis, and FDA is taking extraordinary steps to be proactive and flexible in our response – whether it’s providing advice on medical product development, authorizing the emergency use of new diagnostic tools, quickly enabling access to investigational therapies, or working on the front lines in West Africa.

Margaret Hamburg, M.D.FDA has an Ebola Task Force with wide representation from across FDA to coordinate our many activities. We are actively working with federal colleagues, the medical and scientific community, industry, and international organizations and regulators to help expedite the development and availability of medical products – such as treatments, vaccines, diagnostic tests, and personal protective equipment – with the potential to help bring the epidemic under control as quickly as possible.

These efforts include providing scientific and regulatory advice to commercial developers and U.S. government agencies that support medical product development, including the National Institutes of Health (NIH), the Office of the Assistant Secretary for Preparedness and Response (ASPR), the Centers for Disease Control and Prevention (CDC), and the Department of Defense (DoD). The advice that FDA is providing is helping to accelerate product development programs.

Our medical product reviewers have been working tirelessly with sponsors to clarify regulatory requirements, provide input on manufacturing and pre-clinical and clinical trial designs, and expedite the regulatory review of data as it is received. FDA has been in contact with dozens of drug, vaccine, device, and diagnostic test developers, and we remain in contact with more than 20 sponsors that have possible products in pipeline.

We also have been collaborating with the World Health Organization and other international regulatory counterparts—including the European Medicines Agency, Health Canada, and others—to exchange information about investigational products for Ebola in support of international response efforts.

Investigational vaccines and treatments for Ebola are in the earliest stages of development and for most, there are only small amounts of some experimental products that have been manufactured for testing. For those in limited supply, there are efforts underway to increase their production so their safety and efficacy can be properly assessed in clinical trials.

As FDA continues to work to expedite medical product development, we strongly support the establishment of clinical trials, which is the most efficient way to show whether these new products actually work. In the meantime, we also will continue to enable access to investigational products when they are available and requested by clinicians, using expanded access mechanisms, also known as “compassionate use,” which allow access to such products outside of clinical trials when we assess that the expected benefits outweigh the potential risks for the patient.

In addition, under the FDA’s Emergency Use Authorization (EUA) authority, we can allow the use of an unapproved medical product—or an unapproved use of an approved medical product—for a larger population during emergencies, when, among other reasons, based on scientific evidence available, there is no adequate, approved, and available alternative. To date, FDA has authorized the use of five diagnostic tests during this Ebola epidemic: one was developed by DoD, two were developed by CDC, and this week FDA issued EUAs for two new, quicker Ebola tests made by BioFire Defense.

To further augment diagnostic capacity, we have contacted several commercial developers that we know are capable of developing rapid diagnostic tests and have encouraged them to work with us to quickly develop and make available such tests. Several entities have expressed interest and have initiated discussions with FDA.

We also are monitoring for fraudulent products and false product claims related to the Ebola virus and taking appropriate action to protect consumers. To date, we have issued warning letters to three companies marketing products that claim to prevent, treat or cure infection by the Ebola virus, among other conditions. Additionally, we are carefully monitoring the personal protective equipment (PPE) supply chain to help ensure this essential equipment continues to be available to protect health care workers.

And at least 12 FDA employees are being deployed to West Africa as part of the Public Health Service’s team to help with medical care. We are proud that they are answering the call.

As you can see, FDA has been fully engaged in response activities and is using its authorities to the fullest extent possible to continue its mission to protect and promote the public health, both domestically and abroad. Our staff is fully committed to responding in the most proactive, thoughtful, and flexible manner to the Ebola epidemic in West Africa.

I could not be more proud of the dedication and leadership that the FDA staff involved in this response has shown. I therefore want to take this opportunity to thank more than 250 staff, including those soon to be on the ground in West Africa, who have already contributed countless hours to this important effort, and who will continue to do so in the coming days and weeks as we address this very serious situation. I am hopeful that our work and the coordinated global response will soon lead to the end of this epidemic and help reduce the risk of additional cases in the U.S. and elsewhere.

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

The more we know about rare diseases, the more likely we are to find safe and effective treatments

By: Janet Woodcock, M.D.

Janet WoodcockYou may be inclined to think that rare diseases affect only a tiny fraction of the more than 320 million people in our country. That’s true about a single rare disease. But there are about 7,000 rare diseases. If you add them all together, there are about 30 million – or almost one in ten — people in the U.S. with some form of rare disease. Sadly, although great progress has been made in some areas, many of these people have no FDA approved drug to cure their condition, help them feel better, or even slow the disease’s progress.

That’s why I am pleased about FDA’s support for an exciting new tool researchers are using to study rare diseases. It’s a new database with information about the diseases’ “natural history.”

“Natural history” is the scientific term to describe how a disease would progress with no treatment. Since a disease can affect different people differently, scientists must study many cases of a disease to acquire a thorough understanding of its natural history. Well-conducted studies of natural history can yield vital information about:

  • Biomarkers, demographic, genetic, and environmental variables that correlate with the course and stages of the disease;
  • Identification of patient subpopulations with different characteristics and effects of the disease;
  • Patient perspectives on what aspects of disease are most important to treat; and,
  • How to quantify those aspects so that they can serve as useful outcome measures for clinical trials.

But when it comes to rare diseases, their natural histories frequently are not fully understood because there are simply not enough cases that have been observed and studied. This lack of knowledge limits researchers’ ability to study rare diseases and develop new treatments. Knowledge of natural history is essential for developing more efficient clinical trial designs. It also could help reduce the length and cost of drug development and, possibly, contribute toward greater predictability of clinical development programs.

Recently The National Organization for Rare Diseases (NORD), has teamed up with the patient advocacy group that represents people with the rare disease known as Von Hippel Lindau disease. This is a condition with many debilitating symptoms that also predisposes individuals to benign and malignant tumors. The Von Hippel Lindau Alliance and NORD have created an online tool that enables people with this rare disease to enter information about their experiences with the disease, such as the progression of symptoms, and to add to this information at intervals throughout their lives.

This tool is now helping researchers compile valuable data about the natural history of Von Hippel Lindau disease. The even better news is that this tool is universal.  If it can be used effectively to help researchers better understand Von Hippel Lindau disease, it can do the same for other rare diseases as well!

Importantly, this online tool was developed with direct input from patients, as well as patient organizations, researchers, FDA, and other international drug regulatory agencies.

The natural history tool has important features such as these:

  • It protects  the security and privacy of personal information, while making valuable information available to a researcher or drug developer interested in creating a new therapy for a rare disease;
  • It can be used by patients or health care professionals;
  • It helps make sure that text and online tools data are accurate.

FDA is committed to working with patient advocates and other organizations to support natural history studies for rare diseases.  We encourage the use of natural history data collection tools to describe natural history for many rare diseases. It is our deeply felt hope and wish that we can then take steps toward developing and approving new therapies for persons with rare diseases.

Janet Woodcock, M.D., is the Director of FDA’s Center for Drug Evaluation and Research

For more information about the NORD patient registry tool, visit their website: http://rarediseases.org/patient-orgs/registries

And please read: A Pivotal Moment for the Treatment of Rare Diseases — Address by Dr. Margaret A. Hamburg to the NORD Rare Diseases and Orphan Products Breakthrough Summit

FDA Invites Students to Sharpen their Research Skills

By: Nysia George, Ph.D., and Tom Powers

NCTR Intern Claire Boyle, a graduate student from Florida State University

NCTR Intern Claire Boyle, is a graduate student from Florida State University. Get this and other NCTR photos on Flickr.

Biology. Chemistry. Bioinformatics. Toxicology.

Practical, hands-on laboratory work is important for all college students who want to become scientists—but, for many of them, such experiences are out of reach.

That’s one of the reasons why every summer, our National Center for Toxicological Research (NCTR)—FDA’s internationally acclaimed toxicological research center in Jefferson, Arkansas—hosts a special internship program for science students interested in toxicology research.

The 2014 program was exceptionally successful for both the students and the Center.

Applications poured in from more than 200 students pursuing a variety of majors in universities from coast-to-coast. The competition was intense, and the 21 selected students came from schools in 13 states. But they were all alike in two fundamental ways: they were top students, and were eager to hone their scientific skills in real FDA laboratories.

NCTR Intern Luis Valencia, a senior from Texas A&M University

NCTR Intern Luis Valencia, is a senior from Texas A&M University. Get this and other NCTR photos on Flickr.

During their 10 weeks at NCTR, the students worked on projects varying from the development of bioinformatics and statistical methods for RNA sequencing data, to evaluating effects of silver nanoparticles in plastic food containers. They conducted in-vitro experiments; examined effects of nicotine treatment; gained lab experience in cell culture; and were trained in computational modeling or statistical programming.

Each student’s experience was unique and addressed the student’s interests.

The interns gave the program top grades. For example Claire Boyle, a graduate student from Florida State University, said about the lab work: “I like it a lot more than classes. There they tell you that you can do research once you get into the real world. I’ve never had an opportunity to do that before coming here [to NCTR], and that’s the aspect of the program I like best. It’s given me insight into what I want to do for the rest of my life!”

Luis Valencia, a senior from Texas A&M University, echoed similar praise. “This isn’t some pointless classroom assignment; this is the FDA. Something you discover [in this lab] could save a life.” He continued, “I’m having a great experience at NCTR. [My NCTR mentor] let me design my own experiment and helps me a lot. I’m already on my second trial and we’re getting good results.”

The internship program, which was partly funded by the FDA’s Office of Minority Health, is one of the many ways NCTR reaches far and wide to strengthen the scientific foundations of our agency. We engage with scientists within FDA and across other government agencies, industry, and academia to develop scientific information that is vital for sound regulatory policy. We cooperate with colleagues abroad to advance international standardization of regulatory science. And we’re mindful that all quest for knowledge starts with education.

If you are a science student interested in toxicology research, or if you know someone who is, it’s not too early to consider the NCTR’s 2015 internship program. To qualify for admission, a candidate must meet the GPA requirements and provide evidence of success in science courses. He or she will also need letters of recommendation and a personal statement describing his or her research interests.

If you believe you have what it takes, you could be among the select few chosen to join us in the summer of 2015. Applications are accepted throughout the month of February. We look forward to your application!

For more information about the program go to: Summer Student Research Program (NCTR)

For more information about the FDA Office of Minority Health go to: Minority Health

Nysia George, Ph.D., is the National Center for Toxicological Research’s Intern Program Coordinator.

Tom Powers is the National Center for Toxicological Research’s Communication Officer.

Two FDA drug approvals for idiopathic pulmonary fibrosis (IPF)

By: Badrul A. Chowdhury, M.D., Ph.D.

Badrul ChowdhuryPulmonary fibrosis is a disease in which tissue deep inside the lungs becomes thick, stiff, and scarred, decreasing the lungs’ ability to expand to take in air, and making it difficult to breathe. This is a progressive disease in which scarring and lack of elasticity in the lungs continues to increase until the patient can no longer breathe enough to sustain life.

Until recently, patients in the U.S. suffering from idiopathic pulmonary fibrosis (IPF), a form of pulmonary fibrosis in which the cause is unknown, had no drug treatment  approved by FDA for this debilitating, incurable, and terminal condition. However, this month, FDA approved Ofev (nintedanib) and Esbriet (pirfenidone), two important new therapies for the treatment of patients with IPF. Both drugs are “first-in-class” products that offer new hope for patients in the U.S. with IPF.

Researchers don’t understand exactly how Ofev and Esbriet work in the body against IPF, but the drugs seem to inhibit important pathways that help to prevent scarring. Neither drug is a cure. IPF may still progress after patients use these drugs. However, each drug has been shown to significantly slow the progression of the disease.

There is much work to be done, but this is a valuable start. In our continuing efforts to advance drug development for IPF, FDA recently hosted a Public Meeting on Idiopathic Pulmonary Fibrosis Patient-Focused Drug Development to obtain patients’ input on the impact of IPF on their daily life and their views on currently available therapies to treat the condition.

Many patients in the U.S. with IPF will now have effective treatments for their condition. We are addressing the input received from our public meeting on IPF and will continue to support the development and approval of new drugs, especially those that help patients with serious or life-threatening conditions for which no drug treatments are available.

Badrul A. Chowdhury, M.D., Ph.D., is Director, Division of Pulmonary, Allergy, and Rheumatology Products in FDA’s Center for Drug Evaluation and Research

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

FDA’s giant NMR magnet puts more imaging power into our regulatory science

By: Carolyn A. Wilson, Ph.D.

Carolyn A. WilsonThe word spin might make you think of someone trying to influence your opinion. But in physics, spin refers to an intrinsic property of certain subatomic particles that make some nuclei act like small magnets. That kind of spin is key to a powerful laboratory technique scientists use to study complex, carbon-based biological products at the atomic and molecular level.

This technique, called nuclear magnetic resonance (NMR), uses a strong, external magnetic field and radio waves to trigger the release of electromagnetic energy from atoms with nuclei that have spin. Computers convert these data into contour plots that resemble topographic land maps. Scientists use these data to determine the locations of atoms in relation to each other in molecules. This enables them to create three-dimensional models they can hold in their hands and study, or 3D images they can rotate on a computer screen.

Scientists in FDA’s Center for Biologics Evaluation and Research (CBER) are using NMR to study two types of molecules relevant to the vaccines against bacterial and viral disease that we regulate: polysaccharides (long chains of sugar molecules), which occur in either the cell wall or the capsule surrounding some disease-causing bacteria, and shorter chains of sugars called oligosaccharides. Oligosaccharides are found in viruses and are also part of bacterial vaccines.

DSC_1820

Scientists at FDA discuss construction of the facility that will house the new NMR. From left to right: Hugo F. Azurmendi (CBER), Kang Chen (CDER), Darón I. Freedberg (CBER) & Marcos D. Battistel (CBER). Get this and other FDA photos on Flickr.

The microbes need these molecules to cause disease, so CBER scientists are using NMR to study how the structure of such polysaccharides and oligosaccharides triggers production of antibodies against the microbes that carry these molecules. The methods developed by CBER scientists will allow evaluation of licensed and investigational polysaccharide vaccines by using NMR to determine if those vaccines were developed in a manner consistent with these insights into how the structure of these molecules triggers antibody production. In addition, the outcomes of these studies might provide information that manufacturers could use to design novel polysaccharide vaccines that are safe and effective.

Insights into the structures of polysaccharides that play critical roles in generating protective immune responses would be especially useful in confronting dangerous pathogens for which there are no vaccines. Two such pathogens, the bacteria Neisseria meningitidis B and Escherichia coli K1, cause meningitis (a potentially fatal inflammation of the brain and spinal cord). The capsules surrounding these bacteria contain a polysaccharide called polysialic acid. This molecule is unusual because it doesn’t trigger antibody production when injected by itself into adult humans, but people infected with bacteria that have polysialic acid in their cell walls or capsules do produce antibodies against it. One logical explanation for this difference is that “free” polysialic acid has a somewhat different structure than polysialic acid on bacterial cell walls. But, using NMR, CBER scientists found that polysialic had the same structure whether free or as part of the pathogen. Figuring out why only bound polysialic acid triggers antibody production might help researchers develop much needed vaccines for these bacteria. Soon they will have a new NMR facility at the White Oak campus that could help them solve that puzzle.

NMR "Stick" Model; NMR studies at CBER are providing insights into the atomic and molecular ins and outs of polysialic acid, a molecule found on the surface of bacteria, including some that cause meningitis. This work is aimed at helping researchers develop safe and effective vaccines against such bacteria that are based polysialic acid. Using the NMR data from their studies, the CBER scientists created two models of polysialic acid, a “stick” model and a “solid spheres” model, shown in the two short animated videos (above and below).

NMR “Stick” and “Solid Spheres” Models: NMR studies at CBER are providing insights into the atomic and molecular ins and outs of polysialic acid, a molecule found on the surface of bacteria, including some that cause meningitis. This work is aimed at helping researchers develop safe and effective vaccines against such bacteria that are based polysialic acid. Using the NMR data from their studies, the CBER scientists created two models of polysialic acid, a “stick” model (above) and a “solid spheres” model (below), shown in these two short animations.

4SiA-rotation-dots

NMR “Solid Spheres” Model

The NMR spectrometer in the new facility will have a magnet that is much stronger than those previously used at FDA. The stronger the magnet, the more precise the data generated by NMR and the more precise the models that can be developed from this data.

To put this into perspective, the clinical application of NMR, called magnetic resonance imaging (MRI), uses magnets with strengths of 1.5 to 3 units of magnetic power called Tesla. The strength of NMR magnets at CBER is now about 16.4 Tesla (700 megahertz). The new NMR facility at White Oak will have a strength of 19.9 Tesla (850 megahertz)—about 6 times that of hospital MRI machines. In fact, the magnet is so powerful that the machine is isolated in a special room with walls thick enough to block its magnetic field from pulling unsecured metallic objects toward it. In the photograph you can see the NMR team visiting the facility as it is being prepared for the arrival of the machine.

CBER will share the new NMR spectrometer with the Center for Drug Evaluation and Research (CDER), which will use it to do extremely sensitive assessments of the purity of heparin and of the structures and properties of protein therapeutics.

This powerful magnetic molecular “microscope” is one way that FDA incorporates new technology into its regulatory science work to protect and promote the nation’s health.

Carolyn A. Wilson, Ph.D., is Associate Director for Research at FDA’s Center for Biologics Evaluation and Research.

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