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2013 FDLI Annual Conference

Remarks by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
2013 FDLI Annual Conference
Washington, DC
April 23, 2013

It is a pleasure to join you once again this year, to kick off the annual conference of the Food and Drug Law Institute.  There is a special relationship between our organizations, firmly anchored in our shared commitment to the open discussion and examination of important issues in public policy, law and regulation.

And we have a long history of supporting this annual conference:  we always bring a contingent of our senior officials and experts to engage in dialogue; and sometimes we do even more – we provide you with leaders, like your current President and CEO, Susan Winckler.

Today marks my fourth keynote address to your annual conference.  This is striking in part because it scarcely seems possible that I’m about to complete my fourth year as FDA Commissioner.  But more important, there are very few organizations that I make a point of meeting with each year.  That in itself is testimony to the importance of our relationship.

Looking back as I approach my four-year mark at the FDA, it has been an extraordinary time.  Each of those years has given me a clearer perspective on FDA’s critical and unique role, whether it involved establishing a brand new center from the ground up devoted to tobacco products; dealing with foodborne outbreaks or the current crisis concerning the meningitis outbreak and compounding pharmacies more broadly; championing regulatory science; or experiencing the process that led to last year’s passage of the Food and Drug Administration Safety and Innovation Act or FDASIA.

Over the years, I have frequently characterized the FDA as a science-based regulatory agency with a public health mission.

Time and again, my experience has underscored the importance and magnitude of FDA’s work to protect and promote the health of Americans.  And our responsibilities continue to grow.

Science and technology are advancing at breakneck speed, and the world’s economy is increasingly global.  So is our agency, which is engaged in a race to keep up with scientific breakthroughs while assuming more responsibilities both at home and far from our shores.

These are challenging times, even under the best circumstances, but they are made all the more difficult as we seek to fulfill our mission with the added pressure of the “new austerity.”

We all recognize that budgets will be tight. But on top of that sequestration means that FDA will lose about 209 million dollars this fiscal year – $126 million in budget authority and $83 million in user fees. Although we will continue to collect these fees, they will remain on deposit in the U.S. Treasury. They cannot be used to support critical tasks such as issuing  regulations and guidances, keeping up with inspections, and delivering on performance commitments to speed the pathway to approval for many of the products Americans depend on.

As close observers of the agency, you know that we have long lived with limited resources. We take pride in being a lean agency that supports smart, risk-based regulation. And certainly, in these austere times, we’ll continue that approach. Fortunately, we expect to make it through this fiscal year without any furloughs, and we intend to absorb our cuts in the ways that will have the least impact on vital public health programs and services. 

The President’s recent funding request for FDA’s fiscal year 2014 – a 21 per cent increase over fiscal year 2012 – is encouraging, and in these difficult times, reflects a recognition of the importance of FDA’s work. But I also have to note that the majority of the budget reflects enhanced user fees-not budget authority-and includes some user fee programs that don’t yet exist.

However you look at it, there is no question that having adequate resources for our public health mission remains a constant concern. And despite having received much welcomed plus-ups in our budget over these past four years, I remain enormously troubled that FDA’s responsibilities continue to outstrip available resources. This should worry us all…and it is something that I believe we must all continue to work on together.

But let me turn now to several of the arenas where we have received essential new tools—and in some cases resources—to do our job…and to do it in ways that best serve the health, safety and well-being of the American public.

Of course I’m referring to the Family Smoking Prevention and Tobacco Control Act of 2009, the Food Safety Modernization Act (FSMA) that Congress passed late in 2010 and was signed by the President in early 2011, and FDASIA passed last summer.  Each of these new laws has given FDA new responsibilities and authority—and they are reshaping many of our programs and policies.

This morning, I want to review briefly, some of the progress we have made in implementing these landmark pieces of legislation, and why they matter so much.

I also want to address two major public health challenges currently facing our nation, and the renewed focus that I believe we all need, on quality.

First, looking at the legislation…

Perhaps the biggest change of all was required by the Family Smoking Prevention and Tobacco Control Act. The law required FDA to create its first new product center in 25 years and gave us the mandate to bring science-based regulation to the manufacturing, marketing and distribution of tobacco products. For the first time,  the FDA now regulates cigarettes, cigarette tobacco, roll-your-own tobacco, and smokeless tobacco.

There are now important new tools designed to reduce the access and appeal of tobacco products to young people, as well as to encourage current smokers to stop. And the law gives FDA the responsibility to review new tobacco products before they come on the market.

This law has no counterpart: no other regulatory authority anywhere in the world is required to evaluate new tobacco products before they are marketed.

Over these past three-and-a-half years, FDA has been transforming a legal statute into a thriving national tobacco product regulation program designed to reduce the impact of tobacco use on the nation’s health, especially among youth.

We’re creating the science base required for effective tobacco product regulation today and into the future – embarking upon the first-of-its-kind PATH Study (Population Assessment of Tobacco and Health – a 60,000 person prospective cohort study of tobacco use in the US), supporting innovative research on the impact of how altering nicotine levels in tobacco products could affect the way people use tobacco products and become addicted, and establishing the new Tobacco Centers for Regulatory Science.

We’ve implemented important restrictions on tobacco manufacturing and marketing to protect our nation’s children.

Working together with 45 state and territorial partners, we have conducted more than 158,000 inspections of tobacco retailers to ensure that they are not selling cigarettes or smokeless tobacco products to minors.

There is much more to do but I look forward to putting regulation into place that will ensure a healthier life for everyone. You’ll hear more about this from our new Center Director, Mitch Zeller.

Another major undertaking for the agency is the Food Safety Modernization Act, or FSMA. This new law replaced FDA’s traditional focus on identifying and mitigating foodborne outbreaks with the far more desirable, but also more complex task of preventing food contamination before it occurs.

Also, FSMA specifically addresses food safety issues arising out of the increasingly global origin of much of our food supply.  The law lays out strategies for strengthening FDA’s oversight of imported food:  it heightens the imperative need for working in partnership with the global food safety community, and it explicitly encourages arrangements with foreign governments to leverage resources, provide for mutual recognition of standards and strengthen capacity for regulatory action.

To implement this most sweeping reform of our food safety laws in some 70 years, we so far have proposed two rules.  One rule is focused on safe growing and handling practices for both domestically-produced and imported produce; the other proposal describes prevention practices in facilities that process, handle, and store food.

The proposed rules are the result of extensive outreach with our stakeholders as well as the research community. They build on existing voluntary industry guidelines for food safety, which many producers, growers and others currently follow.

Together with three more rules we’re planning to propose shortly, the principles and ideas set forth in these five documents should help prevent the large-scale food-borne outbreaks of recent years.

I want to emphasize that we are taking care to draft rules that are not only risk-based, but also flexible and responsive to the concerns of small farmers and small food businesses.  That’s why our produce rule would give small farms extra time to come into compliance, and exempt low-risk products – such as potatoes, which are rarely, if ever, consumed raw – and canned vegetables, whose processing kills microorganisms.

The third and most recent law of great import for our mission is FDASIA, a wide-ranging statute that strikes many important themes and affects numerous public health issues on FDA’s agenda.

In passing FDASIA, Congress granted FDA important new authorities, reauthorized human drug and medical device user fees, and authorized new user fees for generic drugs and biosimilars.  These authorities and fees are intended to increase the speed and predictability of medical product reviews; better protect the drug supply chain and combat counterfeits; reduce drug shortages; enhance regulatory science; and speed the review of more affordable versions of drugs that are essential in holding down health care costs.

Renewal of drug user fees will allow us to continue a state-of-the-art drug approval process that brings important new drugs to Americans as quickly and safely as possible. In 2012, FDA approved 39 novel drugs—the most in more than a decade--and the great majority were approved in the U.S. before any other country in the world. Increased medical device user fees should help us further reduce review times and shrink the backlogs that until a couple of years ago had been growing.

FDASIA gives a significant new role to patients by requiring FDA to develop strategies to consider patient views and outcome measures during the medical product development process, which we expect to facilitate a more systematic and expansive approach to obtaining the patient perspective on disease severity and unmet medical needs. 

To encourage and support such patient involvement, FDA is launching – this morning – the FDA Patient Network Website to educate patients on how their medications and medical devices progress from idea to the shelf. We hope this interactive tool will encourage patients to share ideas and concerns about the medical product development process.

FDASIA is truly a landmark law and it will keep us very busy.  Its many provisions require FDA to publish more than 30 draft and final regulations and guidances–many of them by July 9, 2014, the two-year anniversary of the act. 

We have had to scramble to figure out how to do it all, but we’ve nearly completed an implementation plan and you can look for it soon.

There is a lot to be done, but it is really gratifying to recognize the importance of this work. Each of these three ground-breaking statutes was designed to help build strong, science-based systems.  And these three new laws also underscore the importance of prevention. Prevention is a fundamental principle of public health, and it must be a fundamental component of our work at FDA.

And, of course, apart from these three landmark bills, FDA has done much more to advance the public health in the past four years. As I mentioned, we have helped launch life-saving, cutting edge new medical products, including targeted therapies that signal the potential for personalized medicine, and novel treatments for previously untreatable diseases. We are making great progress on novel methods of producing vaccines and on extraordinary, ground-breaking devices, even ones that may help replace failing organs-- restoring vision -- or raise the prospect of an artificial pancreas to help type 1 diabetics to reach a whole new level of management and control.

We have published guidance to help ensure the judicious use of medically-important antibiotics in food producing animals.  We have launched the innovation initiative for medical devices.  We have spearheaded innovative clinical trial designs and new analytic approaches to help reduce the size, time and costs of clinical trials while still providing the robust scientific answers we need.

We continue to strengthen our ability to monitor safety throughout a product’s lifecycle, importantly including our strengthened post-marketing surveillance systems for drugs and devices. And we have made great strides in emergency preparedness and medical countermeasures.

These and many other achievements testify to the dedication that our FDA employees bring to the agency’s public health mission. This dedication is essential for everything we do. It is what enables us to serve the public and to earn the public’s trust and confidence.

When we talk about our mission, we often talk in terms of helping to ensure food safety, and the safety and efficacy of our medical products.  But what often gets lost is the third pillar:  quality.  Without quality, frankly, everything else is called into question.

Quality includes the careful practices used to make products, and the integrity of the supply chain that delivers these products to their users. Quality depends on the farmers who grow the food, the companies that manufacture the products, the suppliers who furnish their many components and ingredients, and the distributors who bring these products our stores, our clinics and our homes. 

The American gurus of quality, Joseph Juran and W. Edwards Deming, are famous for observing that quality has to be built into products.  In fact, the preamble to FDA’s first regulations covering Good Manufacturing Practices for Medical Devices, published in 1978, specifically cited Juran’s Quality Control Handbook and further stated that   “Quality assurance . . . .  will maximize the probability that only safe and effective devices will reach the marketplace.”

Quality that’s built into a product prevents problems. Building quality is how a company builds its reputation and trust in its products.  And it is axiomatic that the fundamental responsibility for quality lies with the companies making the products that we regulate.

Unfortunately, we’ve seen some serious quality lapses in recent years that have presented serious public health challenges, most notably those involving drug shortages and the compounding of shockingly unsafe drugs.

Over the past decade, drug shortages have become an increasing  threat to public health and the care of patients.  Since 2006 these shortages have multiplied nearly five-fold, and far too many of them have involved critical, medically necessary, generic injectable products.

Although the causes for these shortages can differ, the predominant cause has been problems with product quality, identified in nearly two out of every three drug shortage cases. 

We have made considerable progress correcting and heading off drug shortages, especially since the President’s Executive Order on this issue in October 2011.  That is when we stepped up our efforts to encourage drug and biological product manufacturers to report any circumstances that could lead to a drug shortage, including temporary interruptions in manufacturing or impending disruptions in supply.

The expansion of the early notification requirements as part of FDASIA efforts has also been enormously beneficial. In fact, the number of shortages was cut in half last year, down from 251 in 2011 to 117 in 2012. And many more shortages are now being averted. We prevented 282 drug shortages in 2012, 87 more than in 2011.

Despite this progress, the underlying root cause of the problems – a lack of attention to quality – continues.  And it remains a cause for concern.

The importance of quality is no more apparent than in last year’s deadly outbreak of fungal meningitis associated with a compounded medication.  Sadly, this outbreak has claimed 53 lives and sickened over 730 people in 20 states

This terrible tragedy must be a wake-up call to our nation. We cannot let such a crisis happen again. This is a shared responsibility. FDA is committed to doing everything that it can.

In order to better understand the nature and scope of the concerns associated with the higher risk compounding pharmacies and identify any acute or serious potential public health threats, we recently completed a series of inspections of compounding pharmacies—more than 25 “for cause’ in response to directed complaints, and another 31 proactive inspections of compounding pharmacies that are known to have produced sterile drugs in the past.

Though most of these pharmacies are not required by current law to register with us or report adverse events, we identified them using a risk-based model that included factors such as past serious adverse events or product quality reports, historical inspection data, referrals from State regulators or sometimes public information/media reports.

These inspections specifically focused on production of sterile drugs to identify firms that may pose a higher risk of producing contaminated sterile products.

During the course of our many inspections over the past several months, we observed problems at some of the firms concerning sterility practices, inappropriate conditions for sterile processing, and other practices that create the risk of contamination.

Some of our observations during the inspections were truly startling:  unidentified black particles floating in vials of supposedly sterile medicine, rust and mold in “clean rooms” where sterile injectable medications were produced; technicians handling supposedly sterile products with bare hands, and employees wearing non-sterile lab coats.

But we were surprised that even in the light of recent events, our inspectors ran into resistance when they attempted to fully inspect some of the facilities, including questions as to our authority to hold them to certain inspection standards, access records and take samples.

In two cases our inspectors had to secure administrative warrants to be able to do their work. These obstacles are not new; they have hampered our oversight of compounding facilities for many years, and they are part of the reason why I have asked Congress for additional authorities.

We believe there should be a distinction between two categories of compounding: traditional and non-traditional.

Traditional compounding would include the combining, mixing, or altering of ingredients to create a customized medication for an individual patient with an individualized medical need, in response to a valid patient-specific prescription.

Non-traditional compounding would include sterile compounding that is performed without a prescription or in anticipation of receiving one, and where the product is distributed out of the state in which it’s compounded. 

Under FDA’s proposed framework, such non-traditional compounding would be subject to Federal standards adequate to ensure that the compounding could be performed without putting patients at undue risk, and FDA could inspect against and enforce these Federal standards. 

These non-traditional compounding pharmacies would be required to report all serious adverse reactions to the FDA, and there would be clear FDA authority to examine records.

In thinking about the debate over pharmacy compounding, I recall my discussion of a “public health approach” to the law in my speech at this meeting three years ago.

I emphasized then that the law should facilitate, not inhibit, the public health mission of the FDA, and “should ensure that, for unusual cases . . . or for emerging new concerns . . . we have the option of saying that we in fact need additional legal authority to reach the right public health outcome.”

In the case of compounding, we have reached that point.  Our authorities are limited and lack clarity in critical ways. They are also not the right fit for FDA to provide appropriate and efficient oversight of this growing industry.

A new breed of specialty pharmacy compounding has evolved, with so-called “outsourcers” producing high volumes of drugs that require sterility, often for hospitals that rely on them to meet critical patient care needs.

The magnitude and complexity of these operations have clearly outpaced the current patchwork of state compounding pharmacy laws that differ in prescription requirements and quality control rules.

But if FDA treats them like conventional manufacturers, under current authorities, they will be subject to a much broader set of requirements and it will almost certainly cause these firms – the good and the bad – to shut-down, resulting in possible disruptions in the supply of drugs to hospitals and other health care providers.

New legislation is needed that will enable us to aggressively address the now-established safety concerns, and that will offer more tailored authorities appropriate for this type of compounding pharmacy.

As the history of the FDA powerfully reveals, all too often it is the intersection of a public health crisis with the legislative response that has allowed us to evolve in ways that better protect and promote the health of the public. Since our very creation with passage of the Food and Drugs Act in 1906, this agency’s mission—and our ability to fulfill it—has reflected this cycle in important and powerful ways.

The FDA and our responsibilities have undergone huge transformations since those early days, and so has the science that undergirds the products we regulate; the health needs we serve; the social, cultural, economic and political environment that shapes the context for our work; and, of course, the laws that govern us. Yet our core mission remains largely the same.

As I have learned over these past four years at FDA, there is little time for rest. There is simply too much to be done. We will press on, working with all of you and all of our many stakeholders…to meet every challenge, surmount every obstacle, and explore every idea for advancing the cause that has been entrusted to us – to enlist both science and the law to protect and promote the health of the public. 

Thank you.