FDA’s Special Agents: On the Job to Protect the Public

By: John Roth

As noted in my previous three posts, FDA’s Office of Criminal Investigations (OCI) is an integral part of FDA’s mission to protect the public’s health. Our top-flight special agents –who have investigative authority similar to other federal law enforcement agencies – give the FDA unique fact-finding tools and provide for strong, industry-wide deterrence. Their work is different from, but enhances, the regulatory inspectors and investigators that make up the bulk of FDA’s field operations. 

Who are these special agents? They are federal law enforcement officers and they have experience: our average agent has been in federal law enforcement for over ten years, a necessary requirement given the sophistication required to work the wide range of OCI cases. 

Each special agent undergoes specialized training to be effective in their job, including firearms and personal safety training, advanced Special Agent training, and training in FDA law.  Throughout their career, OCI agents will keep up to date on the latest trends by participating in what is called “in-service training.”  Additionally, agents will take specialized training in other areas of federal law enforcement, including cybercrime investigations, computer forensics, financial tracing/asset forfeiture investigation, polygraphy, leadership and management, and advanced law enforcement techniques.

As you can see from this training regimen, we demand a lot from our agents – these skill areas are exceedingly complex. Moreover, agents must also learn such intangible skills as being able to work well with others, remain alert and focused, and hone that age-old requirement for any law enforcement officer: good instincts and a devotion to old fashioned shoe-leather doggedness. 

Our agents are located in more than 37 offices nationwide, from Hawaii to Puerto Rico, and work with law enforcement counterparts in many countries, as well as international organizations like Interpol, Europol, and the Permanent Forum on International Pharmaceutical Crime. 

No agent can go it alone, of course, and we rely on an outstanding supporting cast to help us: our “can-do” support staff, who make running a complex nationwide law enforcement program look effortless, the unmatched scientific and public health experts in the FDA centers and the civil-side inspectors and compliance officers in the field, who we rely on for help with complex, scientific cases, and finally prosecutors in the Department of Justice, who have embraced the FDA public health mission. 

My three previous FDA Voice posts highlighted several of the more significant, colorful, and sometimes tragic cases with which OCI has been involved. The first post looked at how we use our top-flight federal agents to work undercover in investigating shadowy overseas drug counterfeiters. The second post looked at high-profile instances of non-compliance right here in the United States – with our work resulting in a $1.4 billion fine. The third post looked at the callous and utter disregard for life caused by corporate actors, which necessitated a criminal response. Yes, these cases involved detailed, complex organization and a range of professional skills. 

But let me emphasize: OCI is doing this work every day. In fact, about every 30 hours, throughout the year, we are gaining a conviction, in crimes ranging from street level pharmaceutical diversion schemes to corporate fraud. In Fiscal Year 2012, OCI agents were responsible for cases that yielded over $4.9 billion in fines and restitution – monies that are paid directly to the U.S. Government or to specific victims of the criminal acts we investigate – an average of over $22 million per special agent. 

FDA’s Office of Criminal Investigation is the only federal law enforcement agency whose mission is exclusively directed at protecting the public’s health. And, criminal enforcement is a critical part of FDA’s enforcement and compliance strategy, a strategy designed to protect people from dangerous products, fraudulent schemes, and unscrupulous criminals. 

Through these four FDA Voice posts, I hope that we have made our OCI team better known to our FDA colleagues and to the public. The vast majority of the FDA-regulated entities respond to FDA’s ordinary regulatory tools. However, OCI stands ready and on watch to locate, investigate, and bring to justice those individuals and entities who disregard and break our public health laws. 

John Roth is Director of FDA’s Office of Criminal Investigations

Spirit of Cooperation Informs Guidance for Egg Producers

By: Michael R. Taylor, J.D. 

To make sure that the eggs you serve your family for breakfast are safe to eat, FDA went directly to the source: the farm. 

What we learned in visits to farms across the country gave us a real sense of some egg producers’ practical needs and the challenges they face. 

This knowledge helped us craft a draft guidance that represents FDA’s latest action related to the “Egg Safety Rule”, a multi-phase 2009 regulation enacted to help keep eggs safe from the bacterium Salmonella Enteritidis (SE), the most common cause of foodborne illness outbreaks tied to consumption of shell eggs. 

The new guidance is specifically intended to help those egg producers who provide their poultry with outdoor access comply with the rule’s various requirements such as biosecurity, rodent and pest control, cleaning and disinfection and refrigeration. 

Egg producers who allow outdoor access face different environmental realities from facilities that keep their hens inside. The new guidance provides suggestions on how egg producers with 3,000 or more laying hens can protect their poultry from predators, pests, wild birds and other animals and comply with the new egg regulation, yet still provide hens outdoor access. (Egg producers with fewer than 3,000 laying hens and egg producers who sell all of their eggs directly to consumers are exempt from the Egg Safety Rule.) 

Before issuing the draft guidance document, FDA did its homework, talking to producers and regulators at state, regional and local meetings to explain our rules and to get first-hand information. In addition, recognizing that there are a wide variety of poultry house styles designed to provide hens with outdoor access, we visited eight poultry farms in California, Texas, Arkansas, Washington, Wisconsin, Indiana, Michigan and Massachusetts in August and September 2012. The farms we visited included operations producing eggs which are certified organic by the U.S. Department of Agriculture’s National Organic Program (NOP). The farms utilized many of the house styles producers are using. Prior to these visits, FDA worked very closely with colleagues at USDA’s NOP in order to understand NOP standards and jointly visited several organic egg farms in Pennsylvania. 

In effect, we were on a fact-finding mission to see for ourselves how these farms operate and to better understand the unique challenges these producers face. We gained a better understanding of these challenges and used this knowledge to develop a draft guidance document specifically addressing the challenges and concerns we observed. 

We strive not just to be regulators, but to work cooperatively with industry as fellow problem solvers. In this instance, producers were concerned about their ability to meet the requirements of the Egg Safety Rule, were eager to ask discerning questions that made us rethink some of our initial thoughts on how to approach the guidance, and were reassured when they learned that we intended to focus on practical, reasonable advice. 

FDA is committed to working hand-in-hand with the people who produce our foods. This approach helps ensure that we understand production processes, and put forth the best advice we can to protect consumers, whose health is our first consideration. 

We firmly believe that producers can provide hens with outdoor access and still be in compliance with the Egg Safety Rule. 

Michael R. Taylor, J.D., is Deputy Commissioner for Foods and Veterinary Medicine

FDA Uses Web Tool to Better the Odds for Food Safety

By: Ted Elkin

When most people hear the words, “Monte Carlo,” they may think about high-stakes gambling.

We, however, think about reducing the risk in food safety through the use of FDA-iRISK, an innovative Web-based food safety modeling tool developed by the Food and Drug Administration and our partners.

Launched in October 2012, FDA-iRISK uses mathematical logic and Monte Carlo simulation (a computer program named for the gambling mecca) to integrate data and generate results that compare and rank risks of the contamination of foods by various hazards.  Unlike a traditional risk assessment of a single food and a single contaminant, FDA-iRISK allows users to compare multiple hazards – microbial or chemical – in multiple foods.

How does FDA-iRISK work?

Through extensive outreach to and collaboration with partners, we developed built-in templates and other features that allow the user to create real-world (or hypothetical) food safety scenarios.

The user provides the data for seven elements: the food(s), the hazard(s), the population of concern (for instance, elderly or immune-compromised), the production or processing system being used for the food, the consumption patterns, the dose response (what level of exposure will have a health impact), and how the health effects are to be calculated.

This allows the user flexibility, for instance, to look at the impact of potential interventions at various stages of the food production system as well as the populations affected.  And it’s easy to use.

Of particular benefit to the user is FDA-iRISK’s ability to generate reports that measure the health impact of an intervention in terms of the widely used public health metric, DALYs (“Disability-Adjusted Life Years,” meaning years of healthy life lost to illness or death).  This measure lets us know the “bang for the buck” of a particular intervention.

FDA-iRISK is quickly gaining acceptance and use in the food safety community.  As of the middle of May, almost 500 users had established accounts with FDA-iRISK and they came from every continent. Because it is web-based, FDA-iRISK is available to anyone in the world who sets up an account, and it is free to use.

Therefore, the knowledge and sharing power of FDA-iRISK is exponential.  As more users use it and generate reports that are then available to the other users, a more consistent, well documented, systematic, structured and quantitative picture of risk in the food supply will emerge, as well as scenarios for reducing risk.

“Information provided by iRisk can aid in developing global scientific exchanges aimed at maintaining and developing agricultural markets around the world,” according to Jamilah (Fagbene) Cassagnol, an international trade specialist at the U.S. Department of Agriculture.

FDA-iRisk is supported by an exceptional project team. FDA staff members Sherri Dennis, Yuhuan Chen, David Oryang, Regis Pouillot, Karin Hoelzer and Susan Cahill developed the tool in collaboration with Risk Sciences International, RTI International and the Institute of Food Technologists.

Ultimately, for food safety, Monte Carlo shouldn’t mean taking a gamble.  Rather, it’s all about using a quantified, standardized and transparent methodology to better understand what interventions and controls will reduce the risk and improve our public health.

Ted Elkin is Director, Office of Analytics and Outreach, at FDA’s Center for Food Safety and Applied Nutrition.

At the Center of Innovative Safe and Effective New Biologics

By: Karen Midthun, M.D. 

We’ve all read news reports of research achievements that promise exciting and powerful new treatments such as gene therapies to treat diabetes and cell therapies to rebuild failing hearts.  

These medical products are biologics and may seem like the stuff of science fiction. But they could transform the way doctors treat certain illnesses and conditions. Other, more traditional biologics, such as vaccines and blood products, may play an important role in protecting against bioterrorism

As the part of FDA that has regulatory authority over many of these products, the Center for Biologics Evaluation and Research (CBER) plays a critical role in their development through its regulatory oversight and research. CBER comprises a variety of offices, including three product offices responsible for 1) gene, cell, and tissue therapies, 2) vaccines and allergy products and 3) blood and blood products. 

Biologics differ from chemically manufactured drugs; they are usually derived from living sources (humans, animals, microorganisms). Work in this field is demanding because of the unique challenges biologics pose to manufacturers as well as to the reviewers at CBER. For example, the sensitive makeup of some biologics (e.g., DNA and cells) means they can’t be sterilized, so great care is needed in manufacturing them. In addition, biologics are complex and often based on new scientific knowledge that CBER reviewers must be familiar with. 

One way CBER meets the challenges of reviewing these products is by conducting its own research, which creates new knowledge and helps advance development of innovative medical products. CBER shares this knowledge by publishing results in peer-reviewed journals, drafting guidance documents to assist in product development, and sharing information through global collaboration and conferences with colleagues in research institutes, government agencies, industry, and stakeholders. Scientists at CBER can bring this added knowledge to their review of new products. CBER research  enhances CBER reviewers’ ability to evaluate a product’s safety and effectiveness—and to spot problems in product development that might not be recognized by manufacturers. 

Thanks to all of this work, CBER has approved important new products over the past couple of years, including 1) the first over-the-counter test that enables consumers to determine their own HIV (the AIDS virus) status in about 30 minutes—in private, 2) five stem cell products derived from cord blood (placenta-umbilical cord) for use in rebuilding the populations of blood forming cells and the immune system; 3) three influenza vaccines that protect against four strains of influenza virus rather than the previous limit of three strains, and 4) two other influenza vaccines, which are produced using mammalian cells or insect cells, rather than with eggs, as is customary. These two new production techniques for influenza vaccine offer the potential for faster startup of the manufacturing process than egg-based manufacturing, as well as the ability to rapidly address an unexpected, spreading epidemic. These vaccines also offer an alternative for individuals who are allergic to egg products. 

Importantly, CBER’s product safety efforts don’t stop after a product goes to market. The Office of Biostatistics and Epidemiology works with collaborators such as Medicare and the FDA Sentinel program on projects that can help evaluate whether certain adverse reactions are caused by approved treatments. 

As medical innovation accelerates in the 21st Century, CBER will continue to be at the forefront of ensuring the development of innovative safe and effective biologics. 

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

Resolving Disputes Concerning FDA and Medical Devices

By: David S. Buckles, PhD, FACC, and Lawrence “Jake” Romanell 

Disagreements are inevitable in science, medicine – and even life. As part of a regulatory agency committed to public health, the FDA’s medical devices center occasionally confronts scientific and policy disagreements among our staff and with the various stakeholders we strive to serve. 

Disputes can occur at any stage of a particular interaction, from an initial inquiry or pre-submission review to a final regulatory decision on an application or submission.

David S. Buckles, PhD, FACC

As Ombudsmen for FDA’s medical devices center, our office investigates complaints from outside the FDA and facilitates the resolution of disputes between FDA’s medical devices center and the industry we regulate. Since part of our job is maintaining impartiality and neutrality, we are a good starting point if you have a complaint, question, or dispute of a scientific, regulatory, or procedural nature. Given the inevitably of disputes, what we find important is how we deal with those disagreements. 

Legislation passed last year by Congress introduced the term “significant decision” to our regulatory lexicon and included certain expectations of how we are to deal with disagreements over such decisions. FDA has proposed its interpretation of what constitutes a “significant decision” in a question and answer draft guidance document, entitled, “Center for Devices and Radiological Health Appeals Processes: Questions and Answers about 517A.” This draft guidance is available for public comment. We believe that when it comes to disputes, all stakeholders play an important role in resolution. Therefore, we strongly encourage interested parties to provide comments and suggestions to improve our appeals process to help us meet our goal of providing a fair, equitable, predictable and transparent means for seeking resolution of disputes. 

In our current experience, differences of opinion that arise before a final decision is reached can usually be resolved through discussion, or even, occasionally, mediation. If interactive discussion with a lead reviewer, team leader or Consumer Safety Officer does not move the ball forward, we have found that outreach by the stakeholder to engage Branch and Division management in the discussion usually has a good chance of success. We have never yet had a senior manager turn us down when we’ve asked them to take a look at a situation, and we strongly encourage stakeholders to make a good faith effort to connect with management at least through the Division level before considering more formal approaches. 

Lawrence "Jake" Romanell

When discussion and interaction through the Division level fails to resolve a dispute, stakeholders usually have several options. By far the most common approach is to request internal agency supervisory review as provided in the Code of Federal Regulations, at 21 CFR 10.75. When a request is made for internal agency review of a decision of an FDA employee, the decision is subject to review by the FDA employee’s supervisor. Generally, such a request for internal agency review is filed with the manager at the next organizational level above the level that either signed the document in dispute or was substantively involved in the decision. 

In the past several years FDA’s Center for Devices and Radiological Health has come a long way in standardizing the appeal process and setting consistent expectations, both internally and externally, to ensure that stakeholders with a legitimate dispute receive a fair and impartial hearing by senior management. Our updated guidance documents will make our process even more clear—and clarity helps make for smoother decisions. 

Wasn’t it Gandhi who said that honest disagreement is often a good sign of progress? 

David S. Buckles, PhD, FACC, and Lawrence “Jake” Romanell, are in FDA’s Center for Devices and Radiological Health, Office of the Ombudsman

Why FDA Proposes an ‘Action Level’ for Arsenic in Apple Juice

By: Michael R. Taylor, J.D. 

FDA has always been—and will always be—committed to making sure that the food you eat is safe for you and your family. It’s a challenging job in today’s complex, global marketplace. 

One of those challenges can be summed up in one word: arsenic. This chemical element is found in the Earth’s crust. It’s everywhere in the environment and can be found in water, air and soil, in both organic and inorganic forms. Human activities also can introduce arsenic into the environment. That means that it can also be found in some foods and beverages. 

Today, FDA is acting to help ensure that consumers do not come in contact with apple juice that has levels of inorganic arsenic that exceed 10 parts per billion. That’s the same level that the Environmental Protection Agency (EPA) has set for drinking water, which is consumed in much greater quantities. 

FDA tests hundreds of foods and beverages for all kinds of potentially harmful substances, and we have been monitoring the levels of arsenic in foods for decades. Of the two forms of arsenic, we worry about the inorganic kind because long-term exposure can be harmful. 

The agency has always found that the amount of arsenic in apple juice is generally low—much lower, in fact, than the levels allowed in drinking water. Consumer Reports did an important story highlighting its own testing. And in 2011, we substantially increased testing and analysis of apple juice to continue and enhance our monitoring efforts. 

We found that our original belief was correct, that the levels of inorganic arsenic in apple juice are too low to cause immediate or short-term health damage. Working with colleagues in EPA, the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC), we then looked at potential risk from long-term exposure. 

That risk assessment helped lead us to the “action level” of 10 parts per billion. We believe that this action level will keep any apple juice that may have more inorganic arsenic than that out of the marketplace.  

We will continue to remain vigilant, work with the food industry, and take regulatory action when appropriate to minimize as much as we can the presence of arsenic and other unwanted contaminants in our food supply. 

Michael R. Taylor, J.D., is Deputy Commissioner for Foods and Veterinary Medicine

Inspiring Young Voices on Type 1 Diabetes

By: Margaret A. Hamburg, M.D.

As FDA Commissioner I frequently learn about exciting and promising new developments in science and medicine to treat disease and improve lives. Some involve advances in science and technology and others relate to the development of improved ways of doing business that promote innovation. But the most inspirational moments relating to these developments are when I have the chance to meet with people who deal personally with the challenges of a particular disease or condition and to hear first-hand what is needed in terms of education and research and development, as well as the difference that FDA’s work can make in their lives.

Earlier this week, I had just such an opportunity, when I met with officials and representatives from the JDRF, formerly known as the Juvenile Diabetes Research Foundation, who were in town for their annual Children’s Congress. 

It was a delight to meet with the group’s President and CEO, Jeffrey Brewer and several other top officials of the organization, with whom FDA has a strong working relationship. But the highlight of the meeting was when we heard from three children with diabetes and their parents who accompanied the group’s leaders. Ten year old Willa Spalter from California, nine year old James Cravero from Iowa, and thirteen year old Fallon Blackbull from New Mexico were among 150 children who were in town to meet with government officials and share their stories of life with diabetes in order to stimulate efforts to develop policies to cure, treat and prevent the disease.

These eloquent and inspiring young people offered compelling personal stories about their struggles with the disease, which, shockingly, included having it properly diagnosed. In fact, the doctors failure to diagnose their disease until the symptoms became life-threatening highlights the very real need for more education, not simply of the public or Congress, but of the medical community itself.

As I listened to these young people I was pleased to learn how FDA’s work is making a difference in their lives and the lives of others with juvenile diabetes. Perhaps most exciting are the remarkable strides being made to develop a safe and effective “artificial pancreas.” This innovative device type automatically monitors blood glucose and provides appropriate insulin doses in people with diabetes who use insulin. It can be life-changing for individuals with insulin-dependent diabetes, helping them reduce dangerously high and low blood sugar levels and lowering the risk for future diabetes-related complications.

To advance the development of this revolutionary type of system, we have worked to foster discussions between government and private researchers and have sponsored public forums to promote rapid and efficient development of artificial pancreas devices.  Additionally, at the FDA we have prioritized the review of artificial pancreas research studies and have provided clear guidelines to industry on performance and safety standards for these systems — efforts that we hope will shorten overall study review time and ultimately get these devices into the hands of researchers and users more quickly. Most recently, we approved clinical studies for artificial pancreas devices to take place at diabetes camps this summer. This is a major milestone, as it will represent the first time in the U.S. that we have been able to study artificial pancreas devices in camp settings, where children participate in camp activities and wear the artificial pancreas during the day and night.

It was gratifying to hear how Willa, James and Fallon each participated in trials related to the artificial pancreas. They know better than anyone the benefits that come from pushing this agenda and making sure the best science is available to take on juvenile diabetes. 

I am so pleased to have had the opportunity to meet with these three courageous and inspiring young people. Hearing their stories and watching how they bravely and competently manage their diseases will provide me and my colleagues additional motivation in our work on behalf of these and other children.

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

FDA, Small Businesses, and the Common Goal of Advancing Public Health

By: Margaret A. Hamburg, M.D.

When federal agencies celebrated “Small Business Week” last month, FDA had special reason to pay tribute. It is well known that the U.S. biomedical industry plays an essential role not only in advancing the health of individuals, but also the health of the overall economy. Less well appreciated is that small businesses account for much of this activity.  A new FDA report issued to Congress this week describes the multitude of ways we work with small businesses to support their innovative ability to craft new treatments, medicines, and devices that improve the health of all Americans. 

Margaret Hamburg, M.D.The outreach efforts described in this report are vital, because small businesses not only have a unique role but also unique needs in their involvement with a regulatory body like the FDA. That’s why we’re working on a number of fronts to strengthen the ability of small businesses to engage and to help ensure that they are not disadvantaged by their size.

One way we do this is by reducing or even waiving user fees for small businesses that meet certain criteria. Sometimes a startup company might have a groundbreaking product, but lacks the financial resources to cover the full cost of user fees, which are paid to the FDA to help cover the cost of product reviews. Encouraging this kind of small business innovation is the reason FDA participates in the Small Business Innovation Research (SBIR) Program, which funds research and development projects that have potential for commercialization and public benefit. Since 2008, FDA has awarded 36 SBIR grants with the average grant being just over $170,000. Small businesses are also eligible to apply for more broadly available FDA grants, such as Orphan Product Grants, which address rare diseases and disorders, and are tailored to meet the focus and needs of small firms.

Perhaps even more important to small businesses than funding is information. FDA works hard to maintain a variety of communications with small businesses. Seminars, webinars, and workshops open to, and often specifically designed for, small businesses are offered throughout the year free of charge. Links to these event listings can be found in Appendix D of the report. FDA’s product centers also have dedicated small business offices that give companies direct points of contact, which are identified in our new report. These offices provide technical support and education to small companies, hold meetings to hear the views and perspectives of small businesses, develop informational materials, and provide an accessible channel through which small businesses can acquire information from FDA. I hope small businesses will take advantage of these resources and reach out to FDA’s small business contacts.

Small businesses also benefit from early communication with FDA during the product review process. This early communication is especially valuable in FDA’s Rare Disease Program in which most product sponsors are small firms and the product evaluations can be particularly complex for companies with limited resources. Our centers have found that early communication between FDA and product sponsors gets safe and effective products to consumers faster. 

I encourage you to read the report for more information on how FDA promotes innovative research by small businesses, protects small businesses from unreasonable regulatory barriers, and thereby allows American ingenuity to thrive.

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

Looking Back and Looking Ahead: FDASIA’s One Year Anniversary

By: Margaret A. Hamburg, M.D. 

One year ago today President Obama signed into law the Food and Drug Administration Safety and Innovation Act, bipartisan legislation reauthorizing user fee programs for innovator drugs and medical devices and establishing two new user fee programs for generic drugs and biosimilar biological products. 

Margaret Hamburg, M.D.Coming at a time of continuing budget restraints, this steady and reliable source of funding is essential to support and maintain FDA’s staff of experts who review the thousands of product submissions we receive every year, and do so in a timely and thoughtful manner. Over the years, our user fee programs have ensured a predictable, consistent, and streamlined premarket program for industry and helped speed patient access to new safe and effective products. 

One of our major undertakings since last July has been putting in place the infrastructure for a new generic drug user fee program that will expedite the availability of low-cost, high quality generic drugs. The program has already achieved several significant milestones, including reducing the backlog of generic drug applications, enhancing review efficiencies, and streamlining hiring. Likewise, reauthorization of the medical device user fee program has helped to expedite the availability of innovative new products to market, and the program has already seen a decrease in the application backlog for device submissions. 

But user fees are by no means the only focus of the 140-page law. Additionally, FDASIA includes provisions to strengthen the drug supply chain, enhance engagement with FDA stakeholders, address the problem of drug shortages, and promote innovation. 

Since last July, FDA continues to meet its FDASIA milestones, and is on track to implement more provisions very soon. Consider some of our more significant accomplishments. In the area of innovation, we launched the new breakthrough therapy designation for drugs that may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases and published guidance on the use of this and all of our expedited programs. In the area of engagement, we initiated the Patient-Focused Drug Development Program. The objective of this five-year effort is to more systematically obtain the patient’s perspective on a disease and its impact on patients’ daily lives, the types of treatment benefit that matter most to patients, and the adequacy of the available therapies for the disease. We have already held patient meetings on three major diseases and another is scheduled in September. 

Also, FDASIA is helping FDA take important steps to address the challenges posed by an increasingly global drug supply chain in which nearly 40 percent of finished drugs are imported and nearly 80 percent of active ingredients come from overseas sources. FDA has been able to halt food and devices from distribution if an inspector believes they are adulterated or misbranded, but the agency lacked this authority for drugs. FDASIA has extended the agency’s administrative detention authority to include drugs as well, and the agency is taking steps to implement this authority. In addition, earlier this year the agency pushed for higher penalties for counterfeiting and intentionally adulterating drugs before the federal sentencing commission – and succeeded. These are the first of several provisions that we must implement under Title VII, the section of FDASIA that strengthens FDA’s authorities over the drug supply chain. Later this week I hope many of you will join me at a public meeting to discuss how we might implement some of the other portions of this important section. 

To help the public keep track of our progress on these and other provisions, we’ve established a FDASIA web portal that includes a link to our three year implementation plan, which we intend to update on a monthly basis. 

Implementing FDASIA is a massive undertaking, requiring detailed planning to integrate these tasks with the rest of our workload. FDA is committed to implementing the requirements of FDASIA in a way that provides lasting improvements to public health, and we will meet these objectives as quickly as resources allow. 

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

Working together for new solutions to cancer in children

By: Margaret A. Hamburg, M.D. 

One of the greatest pleasures I have as FDA commissioner is the opportunity to meet with so many who are making a real difference in the world of health care, including researchers, doctors, drug industry leaders, foundations, and advocates for patients and families, including most recently the Alexandria Summit for Oncology. 

The group’s two-day meeting in New York City addressed a range of topics related to the challenges and advances in treatments for cancer and featured many of the leading scientists and researchers in the field, including my colleague Dr. Richard Pazdur, the director of FDA’s Office of Hematology and Oncology Products. 

Last night I had the chance to speak to the group about developments in treating pediatric cancer. I was joined by Nancy Goodman, a passionate and effective advocate, who is the founder of a patient advocacy group called Kids and Cancer. Nancy is helping to influence discussion on this topic, and her inspiring work gives enduring meaning to the life of her son Jacob, who died of cancer in 2009. 

Pediatric medicine is an area that has long faced historic obstacles, the result of the tension between our eagerness to respond as quickly as possible to treating and developing treatments for diseases in children, and our desire to protect children from potentially dangerous side effects, particularly in the early stages of research when effectiveness is not fully known. 

Today, we are in the midst of some exciting advances in the development of improved and better treatments for cancer that we expect can and will translate into new treatments for children with cancer. Scientific progress isn’t the only reason for these advances.  Regulators, researchers and industry are also thinking and acting in new ways that are allowing us to accelerate our investigation and approval process.  

Over the past 20 years we have evolved from a view that we must protect children from research, to a view that we must protect children through research, in order to assure their access to new and effective medications. Research studies are the only way to truly determine the safety and efficacy of medication in children and to avoid possible harm when children are given drugs approved only for adults. 

Recent laws have created new mechanisms to prioritize drug research and development for children, resulting in a dramatic increase in pediatric drug trials. We are already seeing progress, with more drug companies hiring pediatric experts, and some large pharmaceutical companies developing pediatric centers of excellence. 

FDA is working to promote earlier consideration and approval of treatments for children. But increased emphasis is also needed on discovery and development, which must come from the industry and the academic research community. The potential offered by our science has never been greater. We want to turn this potential into expanded treatments, new drug availability, and hopefully new cures. That’s why the enthusiasm and creativity this Summit generates is so promising. 

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