FDA is Working Closely with Manufacturers of Meningitis B Vaccines

By: Karen Midthun, M.D.

Meningitis has been in the news recently because of outbreaks of a specific strain (called “serogroup B” or “MenB”) on college campuses. Infections caused by MenB are uncommon in the U.S, but can be very serious. According to the Centers for Disease Control and Prevention (CDC), 160 of the 500 cases of meningococcal disease in the U.S. in 2012 were caused by MenB. There are vaccines licensed (approved) in the U.S. to prevent meningitis, but none include this strain.

Dr. Karen MidthunTo address this critical public health need, FDA worked closely with CDC, in order for CDC to make an unapproved MenB vaccine available as quickly as possible to the universities where CDC determined outbreaks had occurred. This was accomplished under FDA’s expanded access program for investigational (or unapproved) products. The program allows the use of unapproved drugs or vaccines to treat or prevent serious or immediately life-threatening conditions when other options are not available.

FDA has been working closely with manufacturers pursuing the development and approval of MenB vaccines for the U.S. The approval of any vaccine in the U.S. is an extensive process that requires submission of a Biologics License Application (or BLA) by a manufacturer. FDA medical and scientific staff then perform a detailed review of data supporting the safety and effectiveness of the vaccine, and FDA staff inspect the quality of the manufacturing process. Because the potential usefulness of a preventative vaccine must be weighed against any unintended side effects, the evaluation of each submission includes a careful assessment of the benefits and risks to public health.

The agency has a variety of regulatory tools – breakthrough therapy designation, accelerated approval, the fast-track program, and priority review – that have enabled FDA to help make innovative and effective new treatment options available to patients more rapidly for serious conditions such as MenB. More information about these programs is available on FDA’s web site.

Although the law generally prohibits FDA from disclosing the existence of pending applications, Novartis has given the agency permission to disclose that the firm plans to submit a BLA for Bexsero (serogroup B meningococcal vaccine) for review in the second quarter of 2014. Pfizer, which also is developing a serogroup meningococcal B vaccine, issued a statement on March 20, 2014, in which it acknowledged receiving breakthrough designation from the FDA for its vaccine, and that it intends to submit an application for review by mid-2014.

FDA is committed to working with manufacturers to bring important medical products to patients as quickly as possible. The health and well-being of patients is our top priority.

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

FDA Wants Your Perspective on Clinical Trial Demographic Data

By: Jonca Bull, M.D.

When designing clinical trials, it is essential to test the safety and effectiveness of medical products in the people they are meant to treat. Although FDA’s policies, guidances, and regulations reflect decades of agency efforts to foster the participation of diverse patient populations in clinical trials, more work is required.

Jonca Bull (2488 x 3738)FDA is seeking your comments on this important public health issue. On Tuesday, April 1, 2014, we’re holding a public hearing on the challenges of collecting and analyzing information on demographic subgroups—including sex, race, ethnicity and age—in clinical trials for FDA-regulated medical products.

We’re looking for ideas and viewpoints from our stakeholders—from clinical researchers, academia, industry, health care professionals and patient advocates. As director of FDA’s Office of Minority Health, I’m inviting you to attend this hearing in person or online, or to submit your comments before or after the hearing on issues that are vital to you.

Your perspectives will be critical as we develop our FDA action plan for improving public health across all demographic groups. The action plan will include recommendations on ways to enhance the collection and analysis of information about the sex, race, ethnicity, and age of clinical trial participants in applications that medical product developers submit for FDA review and approval. We are also seeking ideas and views about how to improve the communication of crucial information on medical products to patients, health care professionals and researchers.

Recently, in Section 907 of the Food and Drug Administration Safety and Innovation Act of 2012, Congress asked FDA to produce a report on this topic and to follow it up with an action plan. In the development of the report, FDA carefully examined 72 product applications approved in 2011.

We determined that the statutes, regulations and policies we have in place generally give drug developers a sound framework for providing information in their applications on the inclusion and analysis of these demographic groups. We also found that medical product developers generally are describing the demographic profiles of their clinical trial participants, and most applications submitted to FDA include analyses of these demographics.

However, we recognize that more can be done. So, as part of the process of developing FDA’s action plan, we’re holding this public hearing to get your views on these and related issues. We can’t do this without your help, so we hope you’ll join us at the hearing in person or online on Tuesday, April 1!

Jonca Bull, M.D., is Director of FDA’s Office of Minority Health

Sentinel: Harnessing the Power of Databases to Evaluate Medical Products

By: Michael D. Nguyen, MD

Clinical trials are designed to evaluate the safety and effectiveness of medications. But the number of participants in clinical trials represents only a fraction of the number of people who will ultimately use the product after FDA approves it. Occasionally, rare and unexpected health problems (which we call “adverse events”) become apparent only after a medical product is on the market and many more people use it. That’s why it’s so important for FDA to continue to monitor the safety of medical products, a practice called surveillance.

Michael NguyenMost safety surveillance systems are passive: They rely on health care professionals and consumers to notice and report adverse events. Although these passive systems remain essential, they have certain limitations. For example, health care professionals and consumers might not recognize that the product is the cause of an adverse effect and not report it to FDA. Or, they might report a suspected adverse event that’s not truly the cause of a problem the consumer experiences.

Now imagine if we could actively search more than 100 million health insurance records to uncover possible adverse events, rather than relying on doctors and patients to report them. Such a system would enable us to get continuous feedback on the use of medicines under real-world conditions – feedback that might help us to discover unexpected patient reactions or unexpected drug interactions.

FDA scientists have partnered with the Harvard Pilgrim Healthcare Institute to create such a surveillance system, called Sentinel. Within Sentinel, FDA has supported the development of software that analyzes information from health insurance and health record databases to search for evidence that certain products are linked to specific adverse effects. Although these data are protected behind tight firewalls and remain under the control of the original health insurance plans that created them, the software makes it possible to analyze the information without disclosing identifying information in order to strictly maintain patient privacy.

FDA and Harvard Pilgrim Healthcare Institute are using this surveillance system to determine whether a certain type of immune therapy is associated with heart attacks or strokes, and to better define the true rate of acute lung injury after transfusions of certain blood components.

More recently, FDA completed its first study using the Sentinel system, which evaluated the safety of the two current vaccines (RotaTeq and Rotarix) that prevent rotavirus infection (the leading cause of severe diarrhea and dehydration in infants). The new study revealed that these rotavirus vaccines slightly raise the risk of a rare bowel problem (intussusception) that previously caused a prior rotavirus vaccine (Rotashield ) to be voluntarily withdrawn from the market by its manufacturer. But the Sentinel study showed that the newer vaccines have a much lower rate of this bowel problem and are safer, with the benefits outweighing the risks, including the risk of intussusception, associated with vaccination.

The Sentinel rotavirus vaccine and immune therapy studies are examples of how FDA scientists are harnessing the power of big electronic databases to ensure the safety of the medical products we use every day.

Michael D. Nguyen, MD, is the Acting Director of the Division of Epidemiology in FDA’s Center for Biologics Evaluation and Research

Vaccines: A Critical Tool in Protecting and Promoting Public Health

By: Karen Midthun, M.D.

Vaccines are a critical tool in protecting and promoting the public health in the U.S. and around the world. The risks from childhood diseases like measles, mumps and polio have been greatly reduced, or in the case of polio, eliminated in the U.S. Vaccines are one of the great public health success stories of the 20th century.

Dr. Karen MidthunFDA is responsible for ensuring the safety and effectiveness of vaccines available for use in the U.S. Our lifecycle evaluation begins during the development stages and continues through approval and after the vaccines are on the market. As part of the Department of Health and Human Services, FDA is but one agency that plays an important role in developing and implementing the Department’s National Vaccine Plan. The plan outlines five goals:

  • Develop new and improved vaccines;
  • Enhance the vaccine safety system;
  • Support communications to enhance informed vaccine decision-making;
  • Ensure a stable supply of, access to, and better use of recommended vaccines in the United States;
  • Increase global prevention of death and disease through safe and effective vaccination.

A report on the work of FDA and its sister agencies in addressing these five goals – the State of the National Vaccine Plan – was recently issued by the Department’s National Vaccine Program Office. The report gives consumers, health care professionals and other stakeholders a look into the ongoing efforts of the various agencies, and how those efforts align with the plan’s vision of enhancing all aspects of vaccines and vaccination.

We at FDA are very proud of the work we do to ensure the safety, effectiveness, and availability of vaccines for the United States. The activities highlighted in the report offer a glimpse into this important work being done at FDA and across the Department – work that lies at the heart of our public health mission.

Dr. Karen Midthun is the director of FDA’s Center for Biologics Evaluation and Research

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.

Rare Diseases in Children Pose Unique Challenges

By: Gayatri R. Rao, M.D., J.D.

Rare diseases – those that affect fewer than 200,000 people in the United States but collectively affect 30 million Americans – are often chronic, progressive, debilitating, and life-threatening. Because most are genetic in origin, they disproportionately affect children. The agency is strongly committed to advancing safe and effective therapies for these young patients.

Gayatri R. Rao, M.D., J.D., is Director of FDA's Office of Orphan Products DevelopmentIn recognition of International Rare Disease Day on February 28, 2014, we are focusing on pediatric rare diseases.

While developing products for any rare disease poses challenges, in part because of the small patient populations, developing products for children raises unique considerations.  Historically, pediatric care has involved the use of off-label therapies that are unstudied or under-studied in children. For example, pediatric drug dosing often involved adjusting adult doses based on a child’s decreased weight, without considering potential age-based differences in drug metabolism and toxicities. Similarly, many medical devices used in children have been adapted – in homes, clinics, and operating rooms – to solve problems quickly, with little time to consider the long term consequences of a device’s effect on a growing child’s physiology and development.

And while incentives currently exist to promote the development of products for pediatric rare diseases, development in this area continues to lag due to the compounded challenges associated with studying both a rare and pediatric population.

In recognition of these challenges, Congress directed FDA to issue a report and strategic plan focused on accelerating and encouraging the development of therapies for pediatric rare diseases. In response, FDA convened a series of public meetings from January 6 – 8, 2014 to discuss the many challenges in developing treatments for rare diseases, specifically for children with rare diseases, and how to overcome those challenges.  These meetings generated a great deal of interest in the rare disease and pediatric communities. Hundreds of people attended either in person or via webcast and represented a wide swath of these overlapping communities, including patients, academicians, researchers, clinicians, industry, and governmental agencies, many of whom were noted experts in their respective fields.

There were frank, robust and productive conversations on a wide range of topics. A few common themes emerged, especially the important role that patient advocacy groups, including parents of pediatric patients, play in furthering drug development, such as participating in the development of robust patient registries and natural history studies, and providing their perspectives on the risks and benefits of specific treatments. Another common theme was the need for strong collaborations between patients, researchers, industry, and government.

My office, the Office of Orphan Products Development (OOPD), is now coordinating a cross-agency effort with Center for Drugs Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), the Center for Devices and Radiological Health (CDRH), and the Office of Pediatric Therapeutics (OPT) to develop a report and strategic plan to encourage and accelerate the development of therapies for pediatric rare diseases. The goal of this report is to incorporate the valuable insights gained from these public meetings to inform and tailor ongoing and future agency initiatives to more effectively advance the development of such therapies.

OOPD, in collaboration with CDER, is also launching a web-based educational tool for rare disease patients, advocacy groups, researchers and industry on various FDA-related topics. Current topics include the essentials of interacting with FDA and expanded access to products under development. The goal is to continue to add to this educational resource over time. For the new educational tool, as well as additional information, visit the OOPD Educational Resources web page. In addition, FDA and the National Institutes of Health (NIH) will jointly celebrate Rare Disease Day with a one-day program at NIH Masur Auditorium highlighting various rare diseases programs, research activities, and initiatives. For more information about this event that is free and open to the public, and available via webcast, visit the OOPD web page.

The needs of rare disease patients and the pediatric population are complex and pose challenging issues. We are committed to working with the pediatric rare disease community to face those challenges head-on and to accelerate the development safe and effective therapies for these diseases.

Gayatri R. Rao, M.D., J.D., is Director of FDA’s Office of Orphan Products Development

Regulatory Science Supports FDA’s Regulatory Mission

By: Carolyn A. Wilson, Ph.D.

You might only think of FDA as a regulatory agency that oversees medical and food products. But FDA scientists, including those in the Center for Biologics Evaluation and Research (CBER), also perform research. In this first of two blog posts, I will describe how regulatory science, as it is called, helps to turn innovative medical research done at FDA and other places into life-saving or life-enhancing biological products.

Carolyn WilsonMost of the discoveries made at CBER support the development of new or improved vaccines, blood and blood products, and tissue, gene and cell therapies. This research also helps CBER make very informed decisions about new products and policies. That’s because many of the same CBER scientists whose research puts them at the cutting edge of science also review potential new products, inspect commercial facilities that make products, and help develop new policies and guidance documents for industry. In the past year, discoveries that CBER scientists have published in research journals have contributed significantly to public health by addressing issues that affect the safety and effectiveness of vaccines, gene therapy, and a treatment for a serious blood disorder.  

For example, scientists in the Office of Vaccines Research and Review (OVRR) took a big step in solving the mystery of why the rates of pertussis (whooping cough) in the United States have been increasing since the 1980s – despite widespread use of a pertussis vaccine. OVRR scientists showed in baboons that even though the vaccine can prevent symptoms of pertussis, animals receiving it still had the bacteria that cause the disease in their airways for up to six weeks.

These animals were then able to spread the bacteria to other animals. This suggests that while the vaccine protects children from getting pertussis symptoms, vaccinated children can still spread the bacteria through coughing to other children for several weeks – especially if those children aren’t vaccinated. This information is important because it can help scientists and public health officials design new vaccines and strategies to reduce the rate of pertussis in the US.

Statisticians and epidemiologists at CBER also make critical contributions to regulatory science. Serious adverse medical events sometimes occur in patients treated with licensed products (i.e., vaccines). When physicians or consumers report such events to the FDA, epidemiologists at the agency work to determine whether these events are actually caused by the licensed product or are just a coincidence. For example, epidemiologists and statisticians in the Office of Biostatistics and Epidemiology (OBE) studied whether getting the vaccine for 2009 H1N1 influenza (the so-called “swine flu”) several years ago increased the risk of developing a nerve disease called Guillain-Barré Syndrome (GBS). GBS can sometimes occur after infections or vaccinations, causing weakness in the arms and legs and reducing reflexes. The concern about the 2009 vaccine was based on the occurrence of GBS over 30 years ago among some people who received the vaccine against a related strain of H1N1 virus in 1976. CBER’s epidemiologists asked whether the more recent vaccine used to protect against the 2009 H1N1 virus also increases this risk. To answer this question, OBE researchers reviewed the medical records of 23 million individuals who received the 2009 H1N1 influenza vaccine during the 2009-2010 influenza outbreak. Their statistical analysis showed that the risk of death or hospitalization from H1N1 infection was about 500 times greater than the risk of developing GBS from the vaccine. 

Studies like these are very important because they help FDA regulators and public health officials to determine whether potential adverse effects are actually linked to the use of a particular product. In this case, confirming the safety of the vaccine was an important public health measure because it reassured the public that this vaccine was safe to take. 

In my next blog post I’ll be discussing important contributions CBER scientists recently made to gene therapy and the treatment of a blood disease called hemophilia. 

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

FDA and Health Canada: Working Together for an Efficient Pathway for Drug Applications

By:  Robert Yetter, PhD 

At FDA, we work closely with national regulatory agencies around the world on issues relating to the safety, efficacy and availability of medical products. An exciting example of such collaborative efforts is the Common Electronic Submissions Gateway (or CESG), an outcome of the US-Canada Regulatory Cooperation Council (RCC). Through a cooperative research and development agreement, FDA worked with our counterparts in Health Canada, to share technology that will make it more efficient for industry to submit applications to both the U.S. and Canada for the approval of pharmaceutical and biological products. A common infrastructure would enable industry to submit to both countries using the same electronic format for technical documents. 

Robert YetterThe RCC Initiative was announced in February 2011 by President Barack Obama and Prime Minister Stephen Harper. Its goals are to promote economic growth, job creation and benefits to consumers and businesses through increased regulatory transparency and coordination. The electronic submissions gateway is one such project designed to meet those goals. 

So just what is this gateway? It’s an electronic “post office” that uses secure Internet connections to receive electronic versions of medical product applications and related documents from industry sponsors seeking regulatory approval. The technology was developed under contract, and implementation at FDA was led by the Center for Biologics Evaluation and Research.  FDA’s Electronic Submissions Gateway (ESG) has been in operation since 2006. It has now been modified to accommodate submissions from both Canada and the U.S. using the same interface and technology, and subsequently sending those submission transmissions to one or both regulatory authorities. 

The collaboration on the Common Electronic Submissions Gateway has the potential to yield long-term positive outcomes for both FDA and Health Canada. The collaboration continues the work between the two regulatory partners to streamline both agencies’ submission requirements while maintaining consistency in regulatory requirements. It could also lead to cost reductions for regulated industry, which would not have to follow separate technical requirements for submission to the two countries. 

We’re very proud of our work with Health Canada to make this technology accessible in a relatively short amount of time, going from concept to delivery in 26 months. This is yet another example of the steps FDA is taking as part of our Global Initiative, which envisions enhanced collaboration with our regulatory partners. 

Robert Yetter, PhD, is the Associate Director for Review Management in FDA’s Center for Biologics Evaluation and Research

New Law Enhances Safety of Compounded Drugs and Protection of the Drug Supply Chain

By: Margaret A. Hamburg, M.D.

Since last year’s tragic meningitis outbreak and subsequent events involving compounded drugs, Congress has been hard at work to pass new legislation to provide FDA with the appropriate authorities for regulating compounded drugs to help make these products safe for the American public.

Margaret Hamburg, M.D.Over a much longer period of time, efforts have been made in Congress to enhance the security of the drug supply chain and protect consumers from exposure to counterfeit, stolen, contaminated or otherwise harmful drugs.

I am pleased that the Drug Quality and Security Act can help FDA protect public health in both of these critical areas.

One part of the new law offers a step forward in FDA’s oversight of certain entities that prepare compounded drugs. The new law will enable these compounders to register with the FDA to become “outsourcing facilities,” making them subject to certain other requirements including Federal quality standards, known as current good manufacturing practice. These facilities will also be subject to inspection by FDA on a risk-based schedule. If compounders register with FDA as outsourcers, hospitals and other health care providers will be able to provide their patients with drugs that were compounded in facilities that are subject to FDA oversight and federal requirements for current good manufacturing practice, among others. To that end, we will be encouraging healthcare providers and health networks to consider purchasing compounded products from facilities that are registered with FDA and subject to risk based inspections.

Drugs produced by compounders that are not registered as outsourcing facilities must meet certain other conditions described in the law, or they will be regulated by FDA as conventional drug manufacturers.

Generally, the state boards of pharmacy will continue to have primary responsibility for the day-to-day oversight of state licensed pharmacies, including traditional pharmacy compounding. And FDA will continue to cooperate with state authorities to address pharmacy compounding activities that may be in violation of the Federal Food Drug and Cosmetic Act.

Another part of the new law enables certain prescription drugs to be traced as they move through the U.S. drug supply chain. The goal is to protect the public from exposure to counterfeit, stolen, or otherwise harmful drugs. This will require manufacturers, repackagers, wholesale drug distributors, and dispensers (other than most licensed health care practitioners) to provide product and transaction information with each sale and notify the FDA and other stakeholders of illegitimate products, which will result in improved detection and removal of potentially dangerous drugs from the supply chain.

Starting four years after enactment of the law, manufacturers, followed by repackagers, will be required to affix a unique product identifier to each drug package that contains the drug’s national drug code (NDC), serial number, lot number, and expiration date. Starting six years after enactment of the law, wholesale drug distributors, followed by dispensers, may only trade products that  are encoded with product identifiers and will be able to verify the product identifier if they determine that they have  suspect product. Ten years after enactment, supply chain stakeholders and FDA will benefit from an electronic, interoperable system which will facilitate the efficient exchange of product and transaction information for prescription drugs at the individual package level. The system, when fully implemented, will enable verification of the legitimacy of the drug product identifier down to the package level, enhanced detection and notification of illegitimate product, and improved efficiency of recalls.

The Drug Quality and Security Act is a significant step toward having new and stronger drug quality and safety laws. While the law does not provide FDA with all the additional authorities sought, these provisions are a sign of progress.

We are committed and prepared to implement the new law that will help us to further protect public health.

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

Personalized Medicine: The Future is Now

By Margaret A. Hamburg, M.D.

Margaret Hamburg, M.D.The difference between science and science fiction is a line that seems ever harder to distinguish, thanks in part to a host of astonishing advances in medical science that are helping to create a new age of promise and possibility for patients.

Today cancer drugs are increasingly twinned with a diagnostic device that can determine whether a patient will respond to the drug based on their tumor’s genetic characteristics; medical imaging can be used to identify the best implantable device to treat a specific patient with clogged coronary arteries; and progress in regenerative medicine and stem cell therapy using a patient’s own cells could lead to the replacement or regeneration of their missing or damaged tissues. Given these trends, the future of medicine is rapidly approaching the promising level of care and cure once imagined by Hollywood in futuristic dramas like Star Trek.

But these examples are not science fiction. They are very real achievements that demonstrate the era of “personalized medicine” where advances in the science of drug development, the study of genes and their functions, the availability of increasingly powerful computers and other technologies, combined with our greater understanding of the complexity of disease, makes it possible to tailor treatments to the needs of an individual patient. We now know that patients with similar symptoms may have different diseases with different causes. Individual patients who may appear to have the same disease may respond differently (or not at all) to treatments of that disease.

FDA has been playing a critical role in the growth of this new era for a number of years. Even before I became FDA Commissioner the agency was creating the organizational infrastructure and putting in place the regulatory processes and policies needed to meet the challenges of regulating these complex products and coordinating their review and oversight. It has been my pleasure to serve at FDA during this next exciting period and to help ensure that the agency continues to prioritize this evolution by anticipating, responding to, and encouraging scientific advancements.

I am very pleased to be able to present a new report by FDA as part of our ongoing efforts in this field. Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development describes many of the exciting developments and looming advances in personalized medicine, lays out the historical progress in this field, and examines FDA’s regulatory role: from ensuring the availability of safe and effective diagnostic devices, to addressing the challenges of aligning a drug with a diagnostic device, to post-market surveillance.

Outside collaboration and information sharing is essential for this field to flourish. On Tuesday, the American Association for Cancer Research and AdvaMedDX held a fruitful daylong conversation on personalized medicine to treat cancer. I was one of the speakers, participating in a conversation with Dr. Francis Collins, the head of the National Institutes of Health. Our discussion focused in part on current status of drug and diagnostic co-development and the challenges and potential of whole genome sequencing, where data can be collected on a patient’s entire genetic makeup at a reasonable cost in a reasonable amount of time.

FDA is committed to fostering these cooperative efforts, as it will require the full force of government, private industry, academia and other concerned stakeholders to maximize our efforts and fully realize the promise of personalized medicine. Our new report outlines that commitment, and helps chart the way forward so that more people can live long and prosper.

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