We Moved Forward on Many Fronts This Year

By: Margaret A. Hamburg, M.D.

At the FDA, the agency that I’ve had the privilege to lead for the past five years, I am gratified to report that we have a lot to be proud of this year. In fact, this past year’s accomplishments on behalf of public health have been as substantial as any in FDA’s recent history.

Margaret Hamburg, M.D.We moved significantly forward, for example, in creating a system that will reduce foodborne illness, approving novel medical products in cutting-edge areas of science, and continuing to develop our new tobacco control program. We worked successfully with Congress and with regulated industry to reach agreement on a number of difficult issues, while continuing to use the law to the full extent possible to protect consumers and advance public health.

While there were many significant actions and events to recognize, below are some of the highlights of 2013.

In the foods area, there were many new actions this year that will have a long-standing impact on improving our food supply for consumers. Throughout the year we have been proposing new rules to reach the goals set forth by the FDA Food Safety Modernization Act (FSMA). These science-based standards will help ensure the safety of all foods produced for our market, whether they come from the U.S. or from other countries.

We also took important steps towards reducing artery-clogging trans fat in processed foods, and understanding the health impact of arsenic in rice. With a final rule that defines when baked goods, pastas and other foods can be considered free of gluten, people with celiac disease can have confidence in foods labeled “gluten free.” And we are studying whether adding caffeine to foods may have an effect on the health of young people and others.

There have likewise been many accomplishments in advancing the safety and effectiveness of medical products. We worked closely with Congress on the recently enacted Drug Quality and Security Act, which contains important provisions relating to the oversight of human drug compounding. The law also has provisions to help secure the drug supply chain so that we can better help protect consumers from the dangers of counterfeit, stolen, contaminated, or otherwise harmful drugs.

Using tools provided by last year’s landmark Food and Drug Administration Safety and Innovation Act (FDASIA), we are continuing to improve the speed and efficiency of medical product reviews, including those involving low-cost, high quality generic drugs and innovative new medical devices. The average number of days it takes for pre-market review of a new medical device has been reduced by about one-third since 2010. The percentage of pre-market approval applications that we approve has increased since then, after steadily decreasing each year since 2004.

We launched a powerful new tool to accelerate the development and review of “breakthrough therapies,” allowing FDA to expedite development of a drug or biologic (such as a vaccine) if preliminary clinical evidence indicates that it may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. This offers real opportunities to get promising drugs more quickly to patients who need them. In fact, using this new approach, FDA recently approved two advanced treatments for rare types of cancer and one for hepatitis C. We have also strengthened efforts to ensure product quality, increased protection of the drug supply chain, and reduced drug shortages.

We confronted the growing misuse of powerful opioid pain relievers by advising manufacturers on how to make these drugs harder to abuse with formulations that are more difficult to crush for inhalation or dissolve for injection. And we recommended that hydrocodone combination products be subject to stricter controls to help prevent abuse. 

We took an important step towards fighting the development of antibiotic-resistant bacteria by implementing a voluntary plan to phase out the use of antibiotics to enhance the growth of food-producing animals, and to move any remaining therapeutic uses of these drugs under the oversight of a licensed veterinarian. So-called “production” use is considered a contributing factor in the development of bacteria that are resistant to the antibiotics used in human medical treatment.

In many areas of our work we are supporting the emerging field of personalized medicine. Advances in sequencing the human genome and greater understanding of the underlying mechanisms of disease, combined with increasingly powerful computers and other technologies, are making it possible to tailor medical treatments to the specific characteristics, needs, and preferences of individual patients.

Many cancer drugs today are increasingly used with companion diagnostic tests that can help determine whether a patient will respond to the drug based on the genetic characteristics of the patient’s tumor. In May, FDA approved two drugs and companion diagnostic testing for the treatment of certain melanoma patients with particular genetic mutations.

Advances in science and technology are also seen in the creation of new medical devices. For example, 3-D printing - the making of a three-dimensional solid object from a digital model – was once considered the wave of the future. But in February, FDA cleared for marketing a device created by 3-D printing – a plate used in a surgical repair of the skull that is built specifically for the individual patient.

While we have worked hard to get therapies to patients, we are at the same time using the tools available to us to remove unsafe and dangerous products from the market. In November, we used new enforcement tools provided by the food-safety law to act quickly in the face of a potential danger to public health presented by certain OxyElite Pro products. These supplements had been linked to dozens of cases of acute liver failure and hepatitis. After FDA took action, the manufacturer agreed to recall and destroy the supplements.

Finally, we made significant progress in implementing the letter and spirit of the Family Smoking Prevention and Tobacco Control Act. We have signed contracts with numerous state and local authorities to enforce the ban on the sale of tobacco to children and teens; conducted close to 240,000 inspections; and written more than 12,100 warning letters to retailers. And, in the first quarter of 2014 we will launch a public education campaign aimed at reducing the number of young people who use tobacco products.

All of us take great pride in the skill and vigor with which we overcame the year’s challenges and new demands. And so, as the year draws to a close, I extend my gratitude to the employees at the FDA who work tirelessly on behalf of the American public year in and year out. To all of our stakeholders, my heartfelt wishes for a joyous holiday season and a safe and healthy 2014.

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

Driving Innovation Is a Key Part of Our Mission

By: Margaret A. Hamburg, M.D. 

Ensuring the safety of the food supply and the safety and effectiveness of drugs, devices and biologics has always been at the core of FDA’s responsibility to protect the public health – and always will be.

Margaret Hamburg, M.D.But what is often lost or neglected in discussions about FDA’s vigilance on behalf of consumer and patient safety is how this kind of oversight and regulation, when done right, can be a key driver of innovation throughout society. Whether it involves the development of some remarkable new drug or oversight of a particular product, the addition of rigorous regulatory science from FDA helps to safeguard the safety and effectiveness of innovative new products; ensures that these scientific achievements quickly reach their full potential; and builds a pathway for ongoing innovation. That’s why we continue to adapt and change regulatory policies in response to – and in anticipation of – scientific opportunities so that they can best be harnessed to improve health and medical care.

There are many recent examples of this thoughtful approach to innovative products. For instance, we have put in place a new breakthrough pathway to market for promising drugs that may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. Using this new approach, we have already approved two treatments for rare types of cancer and one for hepatitis C. And there may be considerably more in the weeks and months ahead because many companies have expressed interest. So far we have received 119 requests for designation and granted 35. 

In a very different realm, another example is mobile medical apps. We took a measured and sensible approach that promotes innovation when we finalized guidance related to these devices earlier this year. Although many mobile apps pertain to health, CDRH intends to focus its oversight on a very small subset of those mobile apps that are medical devices. We have called that subset “mobile medical apps” and we are approaching their regulatory oversight within a risk-based framework. We believe this approach will promote innovation while protecting patient safety by focusing on those mobile apps that pose greater risk to patients. 

As explained in the final mobile medical application guidance, our regulation of software as a medical device – and a mobile app is software – is based on risk and functionality, and that functionality should be treated the same regardless of the platform on which it is used. For example, an electrocardiography device – an ECG machine – that measures heart rhythms to help doctors diagnose patients is still an ECG machine regardless of whether it is the size of a bread box or the size of a smartphone. The risks it poses to patients and the importance of assuring for practitioners and patients that it is safe and effective are essentially the same. 

And, just a few weeks ago, we authorized for marketing four innovative gene-sequencing devices. Two of these products comprise a system that allows laboratories to develop clinical tests that can look at a person’s genetic makeup and detect abnormalities that could be responsible for illness. FDA realized the innumerable uses of these systems from the outset, and rather than focusing on specific diseases or areas of the genome, we took a tool-based regulatory approach. We assessed whether the devices overall measure what they are intended to measure accurately, reliably and precisely so that there can be greater confidence in the test results. These types of genome sequencers represent a significant step forward in the ability to generate genomic information that may ultimately improve patient care.  

Each of the new medical products we approve and usher to the market involves a balancing of risks and benefits, which is based on study and evaluation of hard data and the best available science. It is a huge responsibility that FDA is charged with, nowhere more so than when dealing with unfolding technologies that offer enormous potential – but that also may present real risks for people and their health.

We know there will be new and continuing challenges that arise with additional technological developments and advancements in science and medicine. This is why FDA’s regulatory role in these emerging areas continues to develop as well, sometimes even as the technologies themselves are taking shape 

But our goal remains constant – to protect the public health through smart regulation that helps to enhance innovation and ensure that new medical technologies have real value to the people who will use them, and that they are used effectively and safely to address their needs.

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

Your Input is Bringing Change to Food Safety Rules

By: Michael R. Taylor

Michael R. TaylorYou spoke. We heard you.

We began 2013 with the proposal in January of two rules required by the FDA Food Safety Modernization Act: the Produce Safety Rule and Preventive Controls for Human Food. The former would set science-based standards for the produce industry while the latter would set safety requirements for food facilities.

We were determined from the beginning to be transparent in our processes and to engage all stakeholders in the work of crafting final regulations that would work across the broad spectrum of food-producing operations. An unparalleled outreach effort followed the proposal of these rules. My team and I traveled across the country and around the world to discuss these food-safety requirements with the people who would be most affected, including farms of varying types and sizes.

In our travels, we saw first-hand how everyone is committed to food safety.  We especially spent a lot of time talking to farmers, both those who are smaller and work the land their family has owned for generations, and those who oversee large, diverse operations. We have heard concerns that certain provisions, as proposed, would not fully achieve our goal of implementing the law in a way that improves public health protections while minimizing undue burden on farmers and other food producers.

And because of the input we received from farmers and the concerns they expressed about the impact of these rules on their lives and livelihood, we realized that significant changes must be made, while ensuring that the proposed rules remain consistent with our food safety goals.

For that reason, we are planning to revise language in the proposed rules affecting farmers and plan to publish it in the Federal Register for public comment by early summer. These include changes to sections covering water quality standards and testing, standards for using raw manure and compost, certain provisions affecting mixed-use facilities (such as a farm that has a food-processing operation), and procedures used to withdraw the qualified exemption to these requirements for certain farms. As we consider the comments we’ve received, we may decide to include other changes for public comment. We recognize that completing these rules is essential to protecting the public health and are committed to completing them as quickly as possible.

We always knew that the rules governing farmers would be complex, in part because of the incredible diversity in the size and nature of farming operations. The standards we set must accommodate that diversity and be feasible to implement.

In our efforts to get first-hand information about how these rules would work in the real world, we visited nearly 20 states, Europe and Mexico; toured small and large farms and met with farmers across the country; met with the Amish, organic producers and other groups deeply involved in farming; collaborated with officials from other federal and state public health agencies; and held many public meetings. We also met with coalitions of consumer groups and other stakeholders. Our outreach work has been focused on ensuring that we never took our eyes off the ultimate goal: Keeping the food that you and your family eat safe.

We believe that this decision to change  these proposed rules—in response to the careful consideration of many people involved in supplying our food—is critical to fulfilling our commitment to getting them right. I urge you to review these changes when they are ready and let us know what you think.

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

FDA Confers with International Counterparts to Advance Regulatory Science

By: Margaret A. Hamburg, M.D.

I have recently returned from the 8th International Summit of Heads of Medicines Regulatory Agencies, which was hosted on December 3-6, 2013, by the Netherlands’ Medicines Evaluation Board in Amsterdam. These annual meetings are an important forum for the exchange of information, views and regulatory strategies among the chief executives of major and like-minded medicines regulatory agencies. I was particularly pleased to be able to contribute to these discussions as a speaker on a panel on regulatory science together with Dr. Tatsuya Konda, M.D., Ph.D., the chief executive of Japan’s Pharmaceuticals and Medical Devices Agency.

Margaret Hamburg, M.D.The theme of this year’s summit was “Changes in the Regulatory Landscape,” and my foreign colleagues and I had plenty to talk about. Overcoming the challenges and reaping the benefits of regulatory science is even more critical today, when the FDA and other regulatory agencies face new and growing tasks in the global marketplace. All of us have to contend with the huge changes in the size and nature of international trade caused by emerging markets, developing economies, and increased cross-border flows of goods, information and capital.

As regulators, my international counterparts and I have many issues in common. They include the increasing complexity of new drug products and drug development; growing geographic distribution of markets; greater demands for public accountability and transparency in our work; budgetary and political challenges to regulatory oversight; and, the overriding need to keep up with the rapid changes in science and technology. Given these shared concerns, building cross-border partnerships and finding common solutions is paramount.

I reiterated to the conference our goal to encourage and strengthen cooperation and collaboration among those nations that are actively working to advance regulatory science. Regulatory science endeavors to use current and emerging knowledge to create new tools, standards and approaches for reliable assessment of the safety, effectiveness, quality and performance of medical products. At its best, this process is based on findings, evaluations, discussions and collaboration by scientists throughout the world.  And it is meetings like the recent summit in Amsterdam that help enhance this cooperation and the development of strategies that promote and strengthen the understanding, acceptance and application of regulatory science.

As the FDA embraces its international role in today’s complex regulatory environment, we fully accept the need to think and act globally more than ever before. I look forward to working with other nations’ regulators, the academic community, non-governmental organizations and industry as we join forces to advance regulatory science, the road to even better protection and advancement of the public health.

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

20 Years Later: Returning to FDA to Regulate Tobacco

By: Mitch Zeller, J.D.

Since its emergence in colonial times, tobacco has been one of America’s most lucrative industries – and one of its least regulated. But four years after the Family Smoking Prevention and Tobacco Control Act became law and gave FDA the authority to start regulating tobacco, we can say that FDA isn’t only making policy, we’re making history.

Mitch Zeller, J.D., Director of FDA's Center for Tobacco Products

Mitch Zeller

This summer, FDA’s first decisions on substantial equivalence marked the very first time any tobacco maker was told they could either sell, or not sell, a tobacco product because of its potential public health implications. It was a historic moment for all of us at CTP and the entire FDA family, but it had particular resonance for me, personally.

Twenty years ago, I joined the staff of then-FDA Commissioner Dr. David Kessler, and was soon given the assignment to examine the practices of the tobacco industry – an industry not regulated by FDA. That transforming experience led to my serving as associate commissioner and director of FDA’s first Office of Tobacco Programs. I oversaw FDA’s investigation of the tobacco industry from 1994 to 1996, and helped craft FDA’s 1996 inaugural tobacco regulation. In 2000, the Supreme Court overturned the agency’s assertion of jurisdiction over tobacco.

As we all know, in 2009 Congress provided FDA with explicit authority to regulate tobacco through the Family Smoking Prevention and Tobacco Control Act. By then I had left FDA to work within the public health community on behalf of tobacco control, but standing in the White House Rose Garden when President Obama signed the new law, I immediately knew that FDA would be on the leading edge of protecting Americans from the dangers of tobacco use. That’s why I didn’t hesitate for a moment when Commissioner Hamburg asked me earlier this year to take the helm as the director for the Center of Tobacco Products.

Much of what’s made my experience at CTP so gratifying is that I’ve had the opportunity to build on the strong foundation left to me by CTP’s first director, Dr. Lawrence Deyton. From that foundation, we will continue to move forward, using all the tools available to us in the Tobacco Control Act to transform tobacco for a healthier tomorrow. This is particularly important – especially since each year, more than 300,000 kids under 18 become regular smokers. But we can, and must, change that.

It’s true that tobacco use remains the leading cause of preventable death and disease in the U.S., resulting in more than 443,000 deaths every single year. But FDA’s ability to enact science-based regulation has true potential to reduce the huge death and disease toll from tobacco use.

I am thrilled to be back at FDA, working with such talented and dedicated colleagues who have joined forces to help reduce tobacco-related disease and death for all Americans.

Mitchell Zeller, J.D., is FDA’s Director, Center for Tobacco Products  

A ‘Vision’ Worth Honoring

By: Heidi Marchand, PharmD

My office serves as a bridge between FDA and a wide range of health professional, patient, consumer, tribal and industry audiences. Among our top priorities is collaborating with public-health minded groups and individuals to help us better understand and respond to the needs of stakeholders.

Dr. Helene Clayton-Jeter accepts award

Dr. Helene Clayton-Jeter, front center, accepts the 2013 AOA Leadership in Public Health Award alongside her colleagues, Beth Fritsch - OHCA Deputy Director, at front left, Heidi Marchand - OHCA Assistant Commissioner, at front right, Dr. Michael Duenas - AOA Chief Public Health Officer, at back left, Matt Willette - AOA Assistant Director, Federal Government Relations, at back center, and Jon Hymes, AOA Director, at back right.

One such example is the work we’re doing with the American Optometric Association (AOA) and the Entertainment Industry Council (EIC) on the Decorative Contact Lens Campaign Project. The goal of this important work is to inform young people about the potential risks of using decorative contact lenses improperly.

These lenses change the appearance of the eyes to give them a “cool” look – like making them look like vampire or cat eyes. They could change the eye color. But they can cause serious eye damage if they’re not fitted by an optometrist or ophthalmologist. Failure to properly clean and care for the lenses could cause vision-threatening infections.

Recently, Helene Clayton-Jeter, O.D., Health Programs Coordinator, who leads the project for my office, was honored by AOA for her superlative work on this project. Dr. Clayton-Jeter, who for years was a practicing optometrist, was instrumental in providing valuable insights that helped shape this public health message.

Dr. Clayton-Jeter is the first recipient of the Leadership in Public Health Award, which AOA plans to give annually hereafter to a member optometrist who has provided innovative and far-reaching solutions to public health challenges, augmented the optometrist’s role in public health, and advanced the provision of health care services by optometrists to Americans.

The project is set to be launched next spring, just in time for spring breaks and end-of-school-year celebrations. This is a time when students are more likely to purchase and use decorative contact lenses. By reinforcing the importance of using these lenses only under the supervision of a health care professional, we hope the project will protect the health and preserve the vision of the many young people who are otherwise apt to use decorative lenses unsafely. Working closely with Dr. Clayton-Jeter on this project were the FDA’s Office of Communications and Center for Devices and Radiological Health, which regulates contact lenses.

While making the formal presentation, Michael Dueñas, O.D., AOA’s Chief Public Health Officer, said this award is a testimony to Dr. Clayton-Jeter’s personal commitment and dedicated work toward the advancement of innovative and effective solutions in safeguarding the health of the American people.

I am very proud of Dr. Clayton-Jeter’s achievements and the integrity that she brings to her work every day at FDA.

Heidi Marchand, PharmD, is FDA’s Assistant Commissioner in FDA’s Office of Health and Constituent Affairs

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