For an AIDS-Free Generation: Access to Drugs and Diagnostics Is Essential

By: FDA Commissioner Margaret A. Hamburg, M.D. and HHS Assistant Secretary Jimmy Kolker

Margaret Hamburg, M.D.On World AIDS Day this year, tens of millions of people with HIV are now living healthy, productive lives because of access to safe and lower priced medicines. We rejoice in this achievement, because all people, no matter how rich or poor, deserve to have the medicines they need to live their lives in the best health possible.

We can truly see in our future an AIDS-Free generation because of the wide availability of prevention and treatment tools. But the availability of these drugs and diagnostic tools, especially in Africa, was never a given. Ten years ago, in 2004, the U.S. Food and Drug Administration (FDA) committed to support the President’s Emergency Plan for AIDS Relief (PEPFAR) by introducing an expedited review process to make generic and low-cost treatment more readily available for the most affected countries. PEPFAR requires antiretroviral drugs to be safe, effective, and of high quality and supports their distribution to people needing treatment around the globe. But meeting these requirements can be costly and time-consuming. Those suffering from AIDS cannot wait. The FDA, an agency that is part of the Department of Health and Human Services (HHS), applied the tentative approval process in order to increase dramatically the number of products approved for purchase and distribution by PEPFAR.

Thanks to the commitment of FDA scientists, as of today FDA has issued expedited approval decisions for 179 products, including 39 formulations specifically designed for children that allow flexible dosing across multiple weight bands and many innovative formulations, such as fixed-dose combinations and co-packaged products that improve adherence to treatment and reduce the risk of developing resistance. The 179 tentative approvals allowed PEPFAR to purchase products at a lower cost, leading to cost savings of hundreds of millions of dollars. These savings contributed to additional patients being able to receive treatment.

Jimmy KolkerAccording to UNAIDS, by June 2014, 13.6 million people around the world had access to antiretroviral therapy. This is an important success, but many more people still need access.

Unfortunately, too many countries lack the regulatory capacity to conduct product registrations in a timely manner. This makes it difficult for these countries to provide high-quality rapid HIV tests and treatment.

The FDA and the HHS have been working with the Department of State Office of the Global AIDS Coordinator (S/GAC); the World Health Organization; the Global Fund to Fight AIDS, Tuberculosis, and Malaria; and other organizations to help countries build both their health care systems and regulatory capacities.

Importantly, FDA has partnered with host country health ministries to help strengthen regulatory capacities in support of their public health programs. PEPFAR recently contributed $1.5 million in support of this FDA partnership to further regulatory system strengthening in the East African community.

With these improvements, countries battling HIV and AIDS can build the systems necessary to ensure that patients get the high-quality treatment they need, which one day will lead to the realization of an AIDS-free generation.

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

Jimmy Kolker is Assistant Secretary for Global Affairs in the U.S. Department of Health and Human Services

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

 

FDA Approves a Vaccine to Address a Critical Public Health Need – Trumenba– for Prevention of Serogroup B Meningococcal Disease

By: Karen Midthun, M.D.

Over the last year and a half, there have been numerous reports about outbreaks of bacterial meningitis on several college campuses. Invasive meningococcal disease is a life-threatening illness caused by the bacterium Neisseria meningitidis that infects the bloodstream (sepsis) and the lining that surrounds the brain and spinal cord (meningitis). N. meningitidis is a leading cause of bacterial meningitis, which can have devastating consequences. Between 10 and 15 percent of people who develop meningococcal disease die from the infection, and another 10 to 20 percent suffer permanent complications, such as brain damage or limb loss.

Dr. Karen MidthunThere are five main serogroups of meningococcal bacteria that cause illness: A, B, C, Y and W. FDA has previously approved other vaccines to prevent invasive disease caused by serogroups A, C, Y and W, but they do not protect against meningococcal disease caused by serogroup B, which is the serogroup responsible for the recent outbreaks.  These outbreaks underscore why it’s so important to have vaccines that can prevent meningococcal disease.

FDA recently used several strategies to approve Trumenba, the first vaccine licensed in the United States to prevent invasive meningococcal disease caused by serogroup B. This included use of the accelerated approval regulatory pathway, which allows the agency to approve products that provide meaningful therapeutic benefit to patients over existing treatments for serious or life-threatening diseases, based on evidence that the product has an effect that is reasonably likely to predict clinical benefit. The use of this pathway reduces the time it takes for needed medical products to become available to patients.

Trumenba also received breakthrough therapy designation. This designation facilitated the development, scientific evaluation, and approval of the vaccine. In particular, it provided the manufacturer with more intensive FDA guidance on an efficient vaccine development program, and an organizational commitment involving senior FDA managers. It also provided for a “rolling” submission of the Biologics License Application, which allows for sections of the application to be submitted to FDA for review as they are completed, without waiting to submit the complete application at one time.

FDA also designated this application to receive priority review. I am very proud of the extraordinary effort by FDA review staff to thoroughly evaluate the safety and effectiveness of Trumenba and approve it in record time. Our scientific staff worked tirelessly to complete their evaluation in well under the usual six-months timeframe for priority reviews. My colleagues worked closely with Pfizer, the manufacturer, to address this critical public health need.

We are committed to making important medical products available to people who need them. The approval of Trumenba is just one example of the outstanding work by dedicated FDA staff.

Karen Midthun, M.D., is the Director of FDA’s Center for Biologics Evaluation and Research

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

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.

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’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

FDA Invents: How Technology Transfer Gets FDA Inventions from Lab to Marketplace

By: Alice Welch, Ph.D.

If you think the term “government invention” is an oxymoron—well, think again. You may be surprised to learn that many of the breakthrough technologies that shape our lives today are the brainchildren of government researchers—including those at FDA.

Alice WelchTake the Internet and that GPS in your car or on your cell phone. Both technologies were developed by the U.S. Department of Defense —as were the turbine engines that power the wind farms generating some 6% of our nation’s electrical energy. Those long-lasting radial tires on your vehicle? They’re reinforced with a material five times tougher than steel that was developed by a NASA partnership. And you can thank the government for your flu shot and the development of many other life-saving vaccines such as those for hepatitis A and B and HPV.

Government funding is also critical in supporting and accelerating research in academia and industry that leads to game-changing innovations. Technologies like bar code scanners, Internet search-engines, and the touch screens on your tablet and smartphone might not have been possible without the research funding from the National Science Foundation.

Like other government agencies, FDA drives innovation in its own mission-critical work by supporting collaborative research with external partners and by transferring our life-saving inventions to the commercial market. Making all of this happen is a dedicated team of experts from across the agency that forms FDA’s Technology Transfer Program.  Managed from within the Office of the Chief Scientist, the Technology Transfer Program means many things at FDA.

To our researchers, it means they can access unique resources, participate in scientific collaborations, or obtain the technical expertise they need to make their research possible. These resources support and complement the work underway in FDA’s research laboratories. Whether it’s conducting research into how a blood product becomes a commercially produced therapy, or how to improve vaccine manufacturing, or tracking how patients use a product, the research of FDA’s scientists is fundamental to informing FDA’s evaluation of the safety and effectiveness of our regulated products.

To FDA inventors, Technology Transfer means they can get their inventions translated into commercial products that protect and promote public health. A little known fact is that in the course of their research, FDA scientists regularly gain new scientific insights and invent novel technologies or processes. The Technology Transfer team helps move these technologies to the private sector under license agreements so that new products in areas like vaccines, food-pathogen detection systems, counterfeit drug detection, and manufacturing can be created and made available on the market. To give you a sense of what we mean when we say that “FDA drives innovation,” in the last few years alone, our researchers have produced and reported about 20 patentable inventions annually.

And for FDA’s many collaborators, Technology Transfer means they’re able to engage with our researchers to solve scientific problems and create solutions to support FDA’s regulatory mission. To establish these collaborations and get the right resources for FDA researchers, our Technology Transfer team uses special tools or legal agreements, such as Material Transfer Agreements, Confidential Disclosure Agreements, Research Collaboration Agreements, and Cooperative Research and Development Agreements.

Each of these tools is designed to meet the needs of the research project at hand. They enable FDA researchers to obtain materials not available at the agency and to establish successful scientific exchanges with experts in the scientific community—at universities, small businesses, nonprofits or for-profits, or other government agencies.

Technology Transfer’s efforts may not be the stuff of headlines, but they’ve produced huge dividends for public health. They’ve helped guide FDA researchers through negotiating agreements, to establish collaborations, and to ensure that the tools they use to report, transfer and protect the patents of technologies align with legal and policy requirements. Look for my next few blog posts, where I’ll highlight some exciting, high-impact public health contributions based on FDA inventions.

Learn more:

FDA Researchers Build Partnerships to Advance Innovations

Alice Welch, Ph.D, is Director of FDA’s Technology Transfer Program

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