Global Partnerships Advance the Regulatory Science That Protects Public Health

By: William Slikker, Jr., Ph.D.

In work, as in life, your success often comes down to the strength of your relationships. And as the director of FDA’s National Center for Toxicological Research (NCTR), among the most pre-eminent regulatory science centers in the world, I have found that this axiom, often so apt in daily life, is also true on a grander scale in the world of research.

William SlikkerNCTR scientists develop innovative tools and strategies to advance FDA’s mission to protect and promote public health. Our center sits on 500 acres in Jefferson, Arkansas, far from agency headquarters in the Washington, D. C., metropolitan area.

But the power of the safety assessment work done at NCTR has global reach, and it is leveraged by the global nature of partnerships we have developed across FDA and with research centers in other countries. Late this summer, Aug. 21-22, I will travel to Montreal for the Global Summit on Regulatory Science, where government, industry and academic scientists from all over the world will assess how to address emerging technologies and implement innovative ways to use them to determine the safety and effectiveness of FDA-regulated products when used in real-world applications.

If you imagine our scientific collaborations as a family tree of sorts, our international activities are one limb. In addition to the annual summit, we provide opportunities for scientists from other countries to work with experienced FDA researchers in all facets of safety assessment. NCTR also has outreach partnerships with the World Health Organization, the European Food Safety Authority and other international organizations such as the International Union of Toxicology (IUTOX).

Our internal partnerships are another limb to the science of public health. Of 200 active research projects ongoing at NCTR, over 100 are done in collaboration with scientists from other FDA centers and the Office of Regulatory Affairs (ORA). For example, we work with the Center for Drug Evaluation and Research in assessing the danger, or toxicology, of certain drugs on the most vulnerable populations— pregnant women and children.

We are partners with the ORA in the Nanotechnology Core Facility on our campus that supports the study of nanomaterials, so small that they can’t be seen with a regular light microscope, yet their effects can be profound on the increasing number of drugs, foods and cosmetics in which they are found. NCTR also works with state partners in this research.

In fact, this particular effort and other partnerships have put NCTR at the forefront of research on nanotechnology. The safety and effectiveness of nanotechnology is a focus of a Memorandum of Understanding signed by the FDA Commissioner in 2011 with the State of Arkansas that enables NCTR to collaborate with five major research institutions in the state, including the University of Arkansas for Medical Sciences.

Our state partnerships within Arkansas are invaluable as they add both laboratory and investigator expertise not normally available to FDA. In addition to work with nanomaterials, our projects with Arkansas researchers include research on the effects of anesthesia on the developing brains of young animals to emulate the possible effects in children, and the development of novel bioinformatic approaches to collect, analyze and visualize massive pharmacogenomics (the genetic response to drugs) or imaging data sets.

Our federal partners, including the National Institute of Environmental Health Sciences and the National Toxicology Program (NTP), both of which share our mission to keep you safe from chemical and environmental hazards, combine with NCTR to produce a world powerhouse for safety assessment.

This 20-year partnership between NCTR/FDA and NTP has produced numerous sets of safety data that provide the scientific foundation for FDA regulators and others around the world to establish guidance and set standards to control food contaminants and assess drugs. For example, NCTR’s work on a naturally-occurring fungal contaminant (fumonisin FB1) in the nation’s corn crop produced data for FDA’s Center of Food Safety and Applied Nutrition to provide new recommended limits for fumonisin, an action that reached across the world.

NCTR also engages in public-private partnerships to foster the development of innovative products. For example, we are working with the International Anesthesia Research Society to improve the safe use of anesthetics in children. FDA has many such partnerships to leverage the expertise and resources of industry, government, and non-profit organizations in developing tools that drive innovation.

The crux of regulatory science is this: Just as an art critic must be an expert in art, a scientist at FDA must be an expert in the science that he or she is evaluating. “It takes a village” has become almost a cliché, but in truth it does take a global village to give regulatory scientists the tools they need to ensure that the exciting new technologies will translate into products that are safe, effective and will enhance your life.

William Slikker, Jr., Ph.D., is the Director of FDA’s National Center for Toxicological Research

OpenFDA: Innovative Initiative Opens Door to Wealth of FDA’s Publicly Available Data

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

Today, I am pleased to announce the launch of openFDA, a new initiative from our Office of Informatics and Technology Innovation (OITI). OpenFDA is specifically designed to make it easier for web developers, researchers, and the public to access and use the many large, important, health data sets collected by the agency.

Taha Kass-HoutThese publicly available data sets, once successfully integrated and analyzed, can provide knowledge and insights that cannot be gained from any other single source.

Consider the 3 million plus reports of drug adverse reactions or medication errors submitted to FAERS, the FDA Adverse Event Reporting System (previously AERS), since 2004.

Researchers, scientists, software developers, and other technically-focused individuals in both the private and public sectors have always been invited to mine that publicly available data set – and others – to educate consumers, which in turn can further our regulatory or scientific missions, and ultimately, save lives.

But obtaining this information hasn’t always been easy.

In the past, these vast datasets could be difficult for industry to access and to use.  Pharmaceutical companies, for example, send hundreds of Freedom of Information Act (FOIA) requests to FDA every year because that has been one of the ways they could get this data. Other methods called for downloading large amounts of files encoded in a variety of formats or not fully documented, or using a website to point-and-click and browse through a database – all slow and labor-intensive processes.

openFDA logoOpenFDA will make our publicly available data accessible in a structured, computer-readable format. It provides a “search-based” Application Programming Interface – the set of requirements that govern how one software application can talk to another – that makes it possible to find both structured and unstructured content online.

Software developers can now build their own applications (such as a mobile phone app or an interactive website) that can quickly search, query or pull massive amounts of public information instantaneously and directly from FDA datasets in real time on an “as-needed” basis. Additionally, with this approach, applications can be built on one common platform that is free and open to use. Publicly available data provided through openFDA are in the public domain with a CC0 Public Domain Dedication.

Drug adverse events is the first dataset – with reports submitted from 2004 through 2013 available now.

Using this data, a mobile developer could create a search app for a smart phone, for example, which a consumer could then use to determine whether anyone else has experienced the same adverse event they did after taking a certain drug.

As we focus on making existing public data more easily accessible, and providing appropriate documentation and examples to developers, it’s important to note that we will not release any data that could be used to identify individuals or reveal other private information.

OpenFDA uses cutting-edge technologies deployed on FDA’s new Public Cloud Computing infrastructure enabled by OITI, and will serve as a pilot for how FDA can interact internally and with external stakeholders, spur innovation, and develop or use novel applications securely and efficiently. As we move forward with the early stages of openFDA, we will be listening closely to the public, researchers, industry and all other users for their feedback on how to make openFDA even more useful in promoting and protecting the public health.

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.

Building Expertise and Crossing Boundaries to Improve Oversight

By: Howard Sklamberg, J.D.

To keep the food supply safe, have safe, effective, and high quality medical products, and decrease the harms of tobacco product use, we have to work with the rest of the world.

Howard SklambergAs FDA’s Deputy Commissioner for Global Regulatory Operations and Policy (GO), I oversee FDA’s efforts to further advance its thinking and strategies from a primarily domestic to a globally focused regulator.

GO coordinates the efforts of FDA’s Office of Regulatory Affairs (ORA) and the Office of International Programs (OIP), and works with all of FDA’s product centers on scientific, manufacturing or other regulatory challenges. The highly skilled and dedicated workforce in ORA and OIP is responsible for conducting domestic and foreign inspections, deepening collaborations with local, state and foreign regulatory partners, helping these regulatory partners to strengthen their regulatory systems, and fostering the use of science-based standards and regulatory coherence around the globe to promote the public health of our citizens.

We have to be able to share information with our regulatory partners. We need their help to implement new regulations that have worldwide impact on the oversight of food under the Food Safety Modernization Act and medical products under the Food and Drug Administration Safety and Innovation Act. Working together with these partners, we can ensure an effective public health safety net for our citizens and communities.

An important new priority for FDA is to make fundamental changes in the way we operate in today’s world by aligning our efforts across the agency to keep pace with the acceleration of scientific innovation and the global expansion of the markets. So much of FDA’s work cuts across multiple product areas. How do we make that work as a large and complex agency?

Commissioner Hamburg and senior leaders across the Agency are committed to strengthening our ability to do just that and are collaborating to achieve greater operational and program alignment across the Centers and ORA.

A key part of this process is to enhance specialization across FDA. For ORA, enhanced specialization means that investigators, compliance officers, import reviewers, laboratory personnel, managers and others will have increased technical expertise in a specific commodity area and will work closely with subject matter experts in FDA’s centers. Over time, ORA’s geographic-based model will evolve to a commodity-specific, program-based model that will provide ORA staff the opportunity to gain increased expertise in specific product areas, such as pharmaceuticals, food, animal feed, medical devices, biologics, and tobacco. They will work as part of a team with the staff from other centers, collaborating, for example, with the Center for Drug Evaluation and Research on pharmaceutical oversight or with the Center for Food Safety and Applied Nutrition on food safety issues.

It is especially important that experts in the centers and ORA be engaged in helping to develop compliance policies and priorities. Working with the centers on these broader concerns puts ORA investigators and compliance officers in a better position to implement the preventive approaches contained in new statutes and work together more seamlessly with the centers.

These and other changes that are part of the agency’s focus on program alignment will deepen our knowledge and make us more effective and efficient, with more clarity and coherence in our communications and actions.

This process is still in its early stages. There is a lot of planning still to be done, and we will work to ensure a transparent and inclusive process. Nevertheless, change is coming and I am excited and proud to be part of a transformation that can only strengthen our efforts to safeguard the foods and medical products that are so important in the lives of people all over the world.

Howard Sklamberg, J.D., is FDA’s Deputy Commissioner for Global Regulatory Operations and Policy

Johns Hopkins and UCSF-Stanford join FDA’s Centers of Excellence in Regulatory Science and Innovation

By: Stephen M. Ostroff, M.D.

If you’ve been following my blog series about the Office of the Chief Scientist (OCS), you know about a critical component of nearly all FDA efforts to promote innovative approaches to developing and evaluating our regulated products – collaboration! This week FDA made two new additions to its network of academic partnerships known as Centers of Excellence in Regulatory Science and Innovation (CERSIs).

Stephen OstroffThe first partner brings together a team of leading scientists at the University of California at San Francisco (UCSF) in a joint effort with Stanford University. The second, Johns Hopkins University, builds on a long history of collaboration with FDA. Both partners received FDA funding through a competitive application process to establish CERSIs that will promote cross-disciplinary regulatory science training, scientific exchanges, and leading-edge research focused on FDA science priority areas.

This latest expansion of our CERSI network is an exciting development. The specialized, cutting-edge science required for FDA’s increasingly complex mission makes it imperative that we leverage available knowledge and infrastructure from collaborative partners in academia. These partnerships enrich the breadth and depth of FDA expertise, enabling us to base our regulatory decisions on the most current scientific evidence. They also enable FDA to bring its expansive experience to academia, ensuring that the new scientific approaches being developed at these institutions can be applied in a way that increases their usefulness for evaluating FDA-regulated products. And most important of all, patients and consumers will ultimately benefit from the investment.

Like those FDA previously established at the University of Maryland and Georgetown University, CERSIs are part of FDA’s effort to promote a vibrant, collaborative, regulatory science culture that enables us to tackle the scientific challenges presented by breakthroughs in medical product development and to improve food safety and quality.

As with the others, the joint UCSF-Stanford and the Johns Hopkins CERSIs will be managed by OCS’s Office of Regulatory Science and Innovation, together with teams of scientists from across FDA. Each new CERSI brings specific goals and unique strengths to enhancing FDA’s regulatory research and review.

The UCSF-Stanford CERSI will bring West Coast representation to the CERSI network and enable FDA to access UCSF’s powerhouse in quantitative sciences and pharmacology. Pre-eminent teams of scientists from both institutions and FDA scientists will be working together to develop and offer courses and workshops in drug development and regulatory science through UCSF’s American Course in Drug Development and Regulatory Sciences (ACDRS).

This CERSI will also offer scientific exchanges and training that target three of FDA’s regulatory science priority areas: transforming toxicology to improve product safety, improving clinical studies and evaluation, and harnessing diverse data through information sciences to improve health outcomes. In addition to FDA funding, the UCSF-Stanford CERSI is leveraging funds from the two academic institutions, through courses like the ACDRS, and from a recent Burroughs Wellcome Foundation Award in Innovation in Regulatory Sciences.

The Johns Hopkins CERSI will focus on three core FDA strategic priorities: clinical evaluations, social and behavioral science, and food safety. The university’s internationally recognized faculty in these areas and its geographic proximity to FDA will facilitate intellectual exchange among university faculty, FDA staff, and scientists. FDA staff can take advantage of workshops, symposia, courses, certificate programs, and a Master’s degree in Regulatory Science as well as others areas close to FDA’s strategic goals. Johns Hopkins is also known as a leader in innovative approaches to educational and life-long learning, including Internet-based courses that will be available to FDA scientists and staff worldwide.

Collaborating with our academic partners is crucial to our ability to expand the scientific foundation and infrastructure FDA needs to deliver on the promises of using 21st century science and technology to fulfill our regulatory mission.

Stephen M. Ostroff, M.D., is FDA’s Acting Chief Scientist

We’re Reinventing Ourselves to Keep Your Food Safe

By: Michael R. Taylor and Howard Sklamberg

Congress enacted the FDA Food Safety Modernization Act (FSMA) in response to dramatic changes over the last 25 years in the global food system. It grew out of an understanding that foodborne illness is both a significant public health problem and a threat to the economic well-being of the food system. And FSMA was embraced by a public whose confidence in the food system was being eroded by a series of foodborne outbreaks.

Michael TaylorThe law directs a comprehensive overhaul of our food safety system, using three broad themes:

  • Advancing Public Health – by focusing on prevention of food safety problems through broad, consistent industry implementation of modern preventive practices.
  • Leveraging and Collaborating – by working in close collaboration with other government agencies (federal, state, local, tribal and foreign), the food industry and other stakeholders to make the best use of all available food safety resources.
  • Strategic and Risk-Based Industry Oversight – including clear FDA guidance on standards; outreach and technical assistance to facilitate voluntary compliance; and the use of adaptable, risk-based inspection and compliance strategies that focus on public health outcomes and the effectiveness of overall systems of prevention.

Since January 2013, we have proposed seven new rules to establish the comprehensive framework of modern, prevention-oriented standards mandated by FSMA, covering the production and transportation of human and animal foods, whether produced in the U.S. or overseas. There is a lot of work to do to get these standards right, and we are very focused on that work.

Howard SklambergAt the same time, however, we must be laying the foundation for the next phase: effective and efficient implementation of the new standards. This requires fundamentally new approaches to collaboration and oversight to achieve high rates of compliance with FSMA’s prevention standards. And from a public health and public confidence standpoint, this is where the rubber meets the road.

We are thus pleased to be sharing with our partners and stakeholders a document that captures in broad, high-level terms our current thinking on strategy and guiding principles for implementing the produce safety rule, the preventive controls rules, and FSMA’s new import tool kit, after the final FSMA rules are issued in late 2015 and early 2016. We are making this available as the springboard for discussion with the entire food safety community.  And we know discussion is needed, because the strategy that will make FSMA a success requires significant change in how we at FDA do our work and how we work with our partners.

For example, FSMA calls for a national integrated food safety system that builds on FDA’s longstanding collaboration with state governments on food safety inspection and compliance, but we must take that collaboration to a new level, especially when it comes to the new and unique challenge of implementing the produce safety rule. We aspire to rely heavily on state agriculture departments and other state and tribal departments with on-farm food safety responsibility, taking advantage of their food safety commitment, their knowledge of local conditions and practices, and their local presence to deliver training, technical assistance and compliance oversight. But we have to work closely with our state partners to convert this aspiration to reality. That work includes finding the funding they will need to play an expanded role on produce safety and other areas of FSMA implementation.

FSMA is also helping drive internal governance change at FDA to be sure that all headquarters and field elements of our program are working seamlessly and efficiently to achieve our public health goals. You may have seen the memorandum that Commissioner Margaret Hamburg issued in February 2014 directing a more vertically integrated alignment of the program centers and the Office of Regulatory Affairs (ORA) working in particular areas, such as food safety and drug quality. This is aimed at increasing specialization across FDA, including the programs as well as frontline investigators and compliance officers. It is also intended to streamline interactions between ORA and Center experts so we can devise effective oversight plans, make well-informed judgments during inspections, and achieve timely corrective action when needed to protect consumers.

As the deputy commissioners for Foods and Veterinary Medicine (FVM) and Global Operations (GO), we share leadership responsibility, on the Commissioner’s behalf, for implementing these changes within FDA. To facilitate our efforts and share responsibility, we have created a new FVM Governance Board, which we co-chair and which includes as members: Michael  Landa, director of the Center for Food Safety and Applied Nutrition (CFSAN); Bernadette Dunham, director of the Center for Veterinary Medicine (CVM); and Melinda Plaisier, associate commissioner for Regulatory Affairs (ORA). This Board will help ensure that CFSAN, CVM, and ORA partner fully on major strategic decisions that affect successful implementation of FSMA’s new prevention paradigm.

This is the commissioner’s vision of an agency that works seamlessly across borders, both internal and external, when it comes to protecting public health.

Finally, the work of developing detailed plans for implementing the produce safety and preventive controls rules and the new import safety system is being done by teams of FDA employees overseen by the FSMA Operations Team Steering Committee. This steering committee is led by Roberta Wagner, CFSAN deputy director for regulatory affairs, and Joann Givens, ORA Central Region, acting regional food and drug director, and it will play a key role in the dialogue we will be seeking with our government partners and stakeholder community on our FSMA implementation plans. You’ll be learning about this team’s crucial leadership role in an upcoming FDA Voice blog.

We need your engagement in this important work. Together, we can build a modern food safety system that works well for the food system and for the consumers we all serve.

Michael R. Taylor is FDA’s Deputy Commissioner for Foods and Veterinary Medicine

Howard Sklamberg is FDA’s Deputy Commissioner for Global Regulatory Operations and Policy

The way forward on opioid abuse: A call to action for science-based, comprehensive strategies

By: Margaret A. Hamburg, M.D.

The recent attention paid by state policy makers around the serious public health problem of misuse, abuse, addiction, and overdose of prescription opioid painkillers is commendable. These efforts reflect the strong desire on the part of states where communities have been devastated by opioid addiction and overdose to prevent further tragic loss of life. However, it is important that such efforts comprehensively address the real root causes of the problem, are grounded in science, and will make a real and lasting difference.

Margaret Hamburg, M.D.The most recent state actions in Massachusetts and Vermont would require healthcare providers to take certain steps such as screening for abuse risk and documenting medical need before prescribing the opioid Zohydro ER. They would also require prescribers to participate in and regularly check state databases that track how often the drug is prescribed to a given patient – a measure that can help identify misuse.

Such requirements are consistent with the essential tenets of numerous medical society guidelines on appropriate pain management and – simply put – are precisely what responsible physicians should be doing. As the entities with responsibility for overseeing the practice of medicine, the states have an important role to play in addressing a critical driver of opioid abuse – inappropriate prescribing practices.

However, we can’t just focus on one drug, Zohydro, alone. These requirements would not apply to the prescribing of any of the other opioids on the market that account for some 250 million prescriptions and 18 billion tablets each year. Unfortunately, to date considerable misinformation appears to be diverting attention from more comprehensive policy solutions that apply to all opioids.

Last October, FDA approved Zohydro as a new treatment option for the management of pain severe enough to require daily, around-the-clock, long-term treatment, and for which alternative treatments options have proved inadequate. We have heard from many people who must cope with often severe pain on a daily basis. These are people who need a variety of therapies to have any hope for a quality life. And, importantly, Zohydro does not include the liver toxin acetaminophen, as many hydrocodone products (e.g., Vicodin) do.

Despite claims to the contrary, the fact is that the top dose of Zohydro is no more potent than the highest strengths of other extended-release opioids like Oxycontin and extended-release morphine. And since Zohydro is a Schedule II controlled substance, it is already subject to Drug Enforcement Administration prescribing restrictions. These include requiring patients to have a written prescription from their doctor instead of one provided to the pharmacist over the phone, and the prohibition of refills.

Moreover, the approval of Zohydro is not likely to significantly change overall opioid prescribing and use by patients suffering from pain. Rather, the most likely patients for this medication are those who are currently taking an immediate release hydrocodone product chronically, or people taking another extended-release/long-acting (ER/LA) opioid. And this drug is unlikely to increase the number of people abusing opioids.

Some have asserted that this drug’s approval should have been delayed until it had an abuse-deterrent formulation. Unfortunately, this puts too much faith in the current state of abuse-deterrent technology, which is still in its infancy and has yet to be fully tested in actual market or use conditions. Even the abuse deterrent properties of Oxycontin, the only opioid with an abuse-deterrent claim in its labeling that is consistent with the approach described in FDA’s 2013 draft guidance on this topic, are limited. For example, while the formulation is designed to make abuse by injection more difficult, it doesn’t prevent it and there is nothing barring someone from taking more Oxycontin pills by mouth – the most common form of opioid analgesic abuse.

Addressing the opioid crisis by focusing on a single opioid drug will simply not be effective. Instead, we must focus our collective attention and energy on the key drivers of the problem, which include excessive prescribing, illegal activity by a small number of providers, improper disposal of unused medications, and insufficient prescriber and patient education.

FDA has a critical role to play in combatting misuse and abuse of all opioids. That includes our requiring pointed warnings in the labeling of every ER/LA opioid analgesic drug and revising them to incorporate evolving scientific knowledge of risks and benefits. That also includes FDA requiring all manufacturers of ER/LA opioid analgesics to make available prescriber training and to conduct post-market studies to assess the long-term abuse risks of these products. As an agency, we will be evaluating the success of the risk management steps required of manufacturers of ER/LA opioid analgesics, including Zohydro. And we intend to monitor the utilization of all opioids to identify any emerging abuse issues.

Also critical are the development of non-opioid pain medications, treatments for opioid addiction and products that reverse opioid overdose. Just in the past few weeks, for example, FDA approved a major new advance in the treatment of opioid overdose – an auto-injector formulation of naloxone, and we will continue to review new treatments for drug abuse.

Let me say, in no uncertain terms: the prevention of prescription opioid abuse is of the highest priority for the FDA. Nothing can erase the tragedy so many people have had to face as a result of abuse, addiction, or misuse of opioids. But we can make meaningful progress to reduce and prevent our nation’s prescription drug crisis. FDA is committed to reducing abuse of opioids and ensuring appropriate access to pain medicines for patients in need.

But it is critical that we all play a role in developing and implementing smart policies that will result in real change. We urge those states with active prescription drug monitoring programs, as well as insurers and pharmacy benefit managers, to help identify and halt inappropriate prescribing. And we urge all states to consider requiring common sense, responsible pain management prescribing practices for all opioids.

Our nation’s front-line health care professionals, especially physicians and other prescribers, play a key role and have a responsibility to ensure that they are treating patients based on their individual needs. We need to be sure they receive adequate proper training and education and that they practice responsible opioid prescribing in order to improve pain management and minimize prescription drug misuse and abuse.

At the end of the day, the complex public health challenge of opioid abuse requires a comprehensive and science-based approach involving federal and state governments, public health experts, opioid prescribers, addiction experts, patient groups and industry. Then, and only then, will we truly solve this problem and protect the public health.

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

The Commissioner’s Fellowship Program: A Win-Win for FDA and Public Health

By: Dr. Stephen M. Ostroff

As part of my FDA Voice blog series on the important work going on in FDA’s Office of the Chief Scientist (OCS), I’d like to highlight an FDA program that is giving top-tier, early career health care professionals, scientists, and engineers the chance to gain broad exposure to FDA regulatory science and scientific review opportunities. Led by OCS’s Office of Scientific Professional Development, the Commissioner’s Fellowship Program (CFP) is accepting applications from April 16 to May 26, 2014. Those who are accepted into the CFP will be joining FDA’s 7th class of Fellows.

Stephen OstroffDuring the two-year program, Fellows complete rigorous graduate-level coursework and conduct cutting-edge research on targeted scientific, policy, or regulatory issues under the mentorship of an FDA senior-scientist preceptor.

In the CFP, a Fellow is able to gain real experience in an FDA biology, physics, or engineering lab, work with a clinical review team, or work at a regional field laboratory or office. The coursework provides a common core understanding of the science behind regulatory review, encompassing activities across all FDA-regulated product areas.

Specific Fellow projects may focus on FDA review of sponsor applications for new products, monitoring product quality and safety, or other scientific or engineering topics. Fellows work closely with FDA scientists to create better research and evaluation tools and approaches, like assays for chemical or pathogen detection, or methods to assess clinical or health care data. Other science and policy areas of focus may involve foods or medical products in disciplines ranging from laboratory sciences to engineering, law, and ethics.

FDA launched the Fellowship Program in 2008 to achieve three critical goals:

1)      Attract to FDA top-tier scientists who can help tackle targeted regulatory science areas;

2)      Provide regulatory science training to expand the pool of experts;

3)      Recruit top scientific talent — scientists who may not have considered FDA in planning their career.

Since the program started, FDA has graduated 164 Commissioner’s Fellows, 75% of whom continued to work at FDA after completing the program. Our graduates have produced 175 publications based on their Fellowship work, represented FDA with 211 regulatory science presentations, authored or co-authored 917 reviews – ranging from original applications to supplements – and 26 Fellows have been the proud recipients of FDA Honor Awards.

The Fellows have brought an infusion of innovative ideas, new talents, and skills to FDA to help build the strong scientific foundation we need in our research and review activities. In turn, the CFP has enabled Fellows to develop their regulatory expertise and work confidently in the FDA environment.

Those Fellows who pursue careers outside FDA bring a deeper understanding of regulatory science and of FDA to their organizations. They enrich the regulatory science enterprise, whether by improving the quality of applications to FDA or by applying the knowledge and tools they’ve acquired through the CFP to develop practical solutions to an important public health challenge.

Stephen M. Ostroff, M.D., is FDA’s Acting Chief Scientist

For more information on eligibility criteria for the FDA Commissioner’s Fellowship Program and to apply for the upcoming class, please visit this Web link:

FDA Commissioner’s Fellowship Program Application Checklist

Collaboration and Medical Countermeasures: Furthering Regulatory Science

By: Dr. Stephen M. Ostroff

“I hope no one ever needs these products,” isn’t something you hear too often, particularly from those developing drugs, biologics, or devices. That point of view—and a universal desire to protect the American public—is what sets biodefense professionals apart.

Stephen OstroffWithin FDA, our Medical Countermeasures initiative (MCMi) does things a little differently as well. Managed by the Office of Counterterrorism and Emerging Threats, in the Office of the Chief Scientist, MCMi thrives on collaborations. We work proactively with industry, academic and federal research centers, FDA product centers and offices, and other government entities in our mission to help protect the U.S. from chemical, biological, radiological, nuclear, and emerging infectious disease threats.

Collaborating is particularly important to advancing regulatory science: developing the tools, standards, and approaches needed to evaluate FDA-regulated products—in this case, medical countermeasures—for safety, efficacy, quality, and performance. When it comes to developing the necessary data for regulatory decisions, medical countermeasures often present unique and complex challenges since the diseases they target rarely occur naturally.

To support regulatory decision-making for medical countermeasures, FDA manages a robust research portfolio under the MCMi Regulatory Science Program. Working with other federal agencies to ensure alignment with U.S. priorities, we fund both intramural and extramural research programs to drive innovation in regulatory science.

Ongoing research projects include:

  • Developing models of radiation damage in lung, gut, and bone marrow organs-on-chips, and using these models to test candidate medical countermeasures to treat such damage;
  • Mapping immune responses to certain biothreat agents and medical countermeasures in humans and animal models to create species-specific immune function maps;
  • Assessing the feasibility of using electronic health record systems to conduct near real-time monitoring of health outcomes, including serious or unexpected adverse events associated with medical countermeasures used during public health emergencies.

For those interested in these and other medical countermeasure-related regulatory science developments, FDA will be hosting our 2014 MCMi Regulatory Science Symposium at FDA headquarters in Silver Spring, Maryland, June 2-3, 2014. The meeting is free and open to the public (pre-registration required).

We welcome poster abstract submissions until April 27, 2014, in topic areas including: animal models, biomarkers and correlates of protection, devices, diagnostics, emergency communication, new technologies, product quality, and surveillance.

I also encourage you to read more about MCMi in action in our Fiscal Year 2013 Program Update.

Stephen M. Ostroff, M.D., is FDA’s Acting Chief Scientist

FDA’s New Acting Chief Scientist Talks about His Office’s Vital Role

By: Stephen Ostroff, M.D.

This is a very exciting time to be stepping into the position of Acting Chief Scientist at FDA. A relative newcomer to the agency, I joined FDA’s Center for Food Safety and Applied Nutrition seven months ago as chief medical officer and senior public health advisor for the Office of Foods and Veterinary Medicine.

Stephen OstroffBut as the former deputy director of the National Center for Infectious Diseases at the Centers for Disease Control and Prevention and director of the Bureau of Epidemiology at the Pennsylvania Department of Health, I often worked with colleagues at FDA and have a solid appreciation for FDA’s public health mission and the pivotal role science plays in everything FDA does. So, I’m very enthusiastic about continuing the development of FDA’s scientific enterprise and positioning the Office of the Chief Scientist (OCS) to best support the agency’s scientific programs.

I’d like to take the opportunity over a series of blogs to discuss the important role that OCS plays in keeping our foods safe and nutritious and in getting essential therapies to the people who need them.

FDA has grown from a lone chemist in the U.S. Department of Agriculture in 1862 to an agency with a staff of 14,600 employees. More than 65% of them are scientific and technical staff, representing disciplines such as biologists, nurses, pharmacists, physicians, veterinarians, behavioral scientists, statisticians, epidemiologists, economists and engineers.

FDA requires this breadth and depth of expertise to ensure that science informs the decisions we make about the safety and effectiveness of drugs, biologics, and medical devices, the safety of foods and cosmetics, and the regulation of tobacco products, particularly as those products become increasingly complex. Only then can the public be confident that these products are rigorously reviewed and assessed before and after they go to market.

Transformative changes in society and technology over the past several decades have created numerous opportunities to improve public health. They’ve also created challenges affecting FDA-regulated products and the way FDA conducts its operations.

For example, globalization is bringing an increasing volume of foods and drugs to our shores, often produced in countries that may not have our high standards of regulatory oversight. New areas of science and rapidly evolving technologies are showing real promise in our ability to prevent and cure some of today’s biggest killers, such as diabetes, cancer, and Alzheimer’s.

To meet these challenges, we are “advancing regulatory science.” In other words, we are developing the new tools, methods and approaches that will be needed for a globalized regulatory environment and for translating new discoveries into innovative medical treatments. But advancing regulatory science research and training requires multidisciplinary and interdisciplinary collaboration within FDA and with our partners in academia, industry, other government agencies and with patient advocates. OCS works to meet these goals by strengthening FDA’s scientific infrastructure, forging a common vision, and working with our stakeholders to identify critical regulatory science and innovation needs.

In my future blogs, I will discuss examples of the exceptional work underway in the different OCS components—the National Center for Toxicological Research (NCTR), the Office of Counterterrorism and Emerging Threats, the Office of Minority Health, the Office of Regulatory Science and Innovation, the Office of Scientific Integrity, the Office of Scientific Professional Development, and the Office of Women’s Health. I can think of no more exciting place to be than at the core of FDA’s pioneering regulatory science culture.

Stephen Ostroff, M.D., is FDA’s Acting Chief Scientist

Supporting Innovative Research Through Regulatory Science

By: Carolyn A. Wilson, Ph.D.

In my last blog post I discussed aspects of regulatory science, that is, how scientists in FDA’s Center for Biologics Evaluation and Research (CBER) help to turn innovative medical research into life-saving or life-enhancing biological products. I also described how FDA scientists help determine if potential health problems are linked to the use of a particular medical product. In this post, I’ll discuss two more studies that made important contributions to public health.

Carolyn WilsonSometimes CBER research changes the way scientists look at a problem so their research is more efficient. For example, in the field of gene therapy, a strain of the common cold virus called an adenovirus, is used as a vector – delivering therapeutic genes to treat both inherited and non-inherited conditions. However, success of this therapeutic approach has been hampered in part by the finding that an immune response to the adenovirus may prevent efficient delivery of the therapeutic genes to their targets, such as cancer cells. The problem appeared to be that once inside the body, the adenovirus attaches a blood clotting protein called FX to itself and binds to liver cells. As a result the vector doesn’t reach the desired target cells where it would deliver the therapeutic gene.

Some scientists thought that altering the virus so it couldn’t bind FX would let it avoid the liver, making it a more efficient vector. However, scientists in the Office of Cellular, Tissue and Gene Therapies (OCTGT) discovered that adenovirus commandeers the FX protein to use as a shield to evade attack by the immune system. So removing it would likely enable the immune system to attack and disable the adenovirus and block treatment. This new knowledge that the adenovirus needs FX to disguise itself from the immune system will help guide researchers to design gene therapy vectors that survive in the bloodstream and reach their desired target cells.

Another group of scientists, in the Office of Blood Research and Review (OBRR), has contributed to our understanding of why African Americans are significantly more likely than whites to produce antibodies against a drug used to treat hemophilia A. People with hemophilia A carry a mutation in the gene for the protein Factor VIII (FVIII) – a protein that plays an essential role in clotting and preventing blood loss. This mutation either eliminates or greatly reduces the amount of Factor VIII in the blood. Fortunately, there is a therapeutic form of FVIII made through biotechnology that is used to replace faulty or missing, natural FVIII. But unfortunately, some African Americans with hemophilia A make antibodies against therapeutic FVIII. These antibodies attack it and disrupt treatment. The FDA scientists discovered certain genetic variations in the gene for Factor VIII made by these individuals that appear to be responsible for this immune system attack. The discovery is an important step in developing ways to predict which patients will develop antibodies against this complication. And that is an important step toward developing a personalized-medicine approach to hemophilia A by custom-designing medical responses to this life-threatening disease.

The examples I’ve given of CBER research here and in my previous blog are just a small sample of the important knowledge our scientists are creating that supports efforts of medical researchers striving to develop products that improve public health nationally and globally.  In 2013, CBER scientists published their research findings in over 200 journals and books.

I’ll be back to update you on more exciting research from CBER during 2014.

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