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Keynote Address

Keynote Address, International Conference on Emerging Infectious Diseases
As delivered by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
Atlanta, Georgia
March 11, 2012

Thank you, Dr. Frieden, for that warm and generous introduction.  It is an honor to be here today.

This is a very distinguished and expert assemblage.  It is more than a little humbling to be kicking off this meeting, as there is very little I can tell you that you do not already know...but I am delighted to be here – among many friends and colleagues – to offer some perspective on current challenges and new opportunities in controlling infectious disease from my various roles and perches, including my current position as FDA Commissioner. 

There are so many things I could talk about, but I am going to focus mainly on medical product development. 

This is a terrific event to be part of for many reasons.  First, the nature of this event:  held every other year, this conference attracts the best and brightest in infectious disease from around the world.  I am certain that your interactions over the next three days will help bring new knowledge and insights to the issues before us, and help chart a course of action for the next two years, and beyond.

And we certainly don’t lack for challenges.  As you all know, we live in an increasingly complex world, a world in which microbial threats to health continue to emerge, resurge, and persist.

These include:  well-recognized diseases emerging in new numbers; in new places as a result of expansions in travel, trade and commerce, and in new forms, through the development of resistance, mutation, and adaptation. 

Also truly new  – or at least newly recognized – pathogens and diseases that can emerge and take hold for many reasons, such as changes in patterns of living, agricultural practices, climate change and more.

And, sadly, bioterrorism, the deliberate use of biological agents to do harm.

At the same time, the opportunities in science, technology, medicine and public health to make a meaningful difference have never been greater. 

Like so many of you, I am here because of the urgency to work together – across disciplines, sectors and across nations – to try to make that difference.

But there is another, more personal, reason that I’m glad to be here today.  That is because it gives me the chance to take credit for giving Tom his first real job in what has been – and will continue to be – a brilliant career in public health.

Back in 1991, when I was appointed New York City Health Commissioner, Tom was working there as a CDC Epidemic Intelligence Service (EIS) officer.

He was finishing up his training, and I quickly hired him to direct the city’s tuberculosis control program.  Strikingly, before he took it on, the position was part-time...just one reflection of the fact that TB was then viewed as a declining public health problem.

In fact, throughout the 1980’s, New York City was busy dismantling the infrastructure it had created to deal with the disease.  By 1988, staff at the city’s TB control bureau had been decimated, two-thirds of New York’s chest clinics had been disbanded, and 1000 hospital beds designated for TB patients had been eliminated. 

During the very same time period, though, in fact the numbers were on the rise, and resistant strains of tuberculosis were gaining a foothold in the city’s hospitals, prisons, homeless shelters and beyond.

Certain areas of the city were most profoundly impacted.  In central Harlem, for example, the rate of TB was some 20 times the national average and higher than in most developing nations.

And as Tom documented in a pivotal study he did as an EIS Officer, the rates of drug resistance were astoundingly high.  We discovered that the treatment regimens we were routinely using were increasingly ineffective, and in fact were likely making things worse.

I’ll never forget the New York Post newspaper’s headline about "Killer TB" on the subway.  I wasn’t thrilled at the time – it seemed a bit sensational – but in some ways it was our ticket to action.

Looking back, I think it’s fair to say that when Tom accepted my job offer, he probably didn’t fully appreciate what he was getting himself into.  That full-time job turned into a round-the-clock battle against a rising epidemic.

It took his energy, dedication and vision, along with a well-designed, data-driven strategy with a comprehensive approach, adequate resources, and real political backing from the Mayor on down...to address this growing problem.  But we managed after only a few years to see the number of TB cases in the city plunge, in fact, more quickly and dramatically than we had ever actually imagined. 

The mistaken assumptions that led to the TB resurgence and the lessons we learned in developing and implementing a comprehensive treatment and control plan, remain – for better or worse – just as relevant today as they were 20 years ago. 

Of course those efforts represent only a small piece of a much larger puzzle – but it convinced me that we can make a difference, and secured my commitment to continuing efforts to help find solutions for other diseases...and for other people.

And now I find myself at the FDA...and I want to turn to its role – as a regulatory and a public health agency – in supporting the innovations and practical strategies needed for meaningful and enduring improvements in health. 

But before I do, I also have to note another historical event...it is so timely that I can’t resist.  Seventy years ago today, a woman named Anne Sheafe Miller was near death at New Haven Hospital, suffering from a virulent streptococcal infection. 

She had been hospitalized for more than a month, her temperature spiking wildly.  Her doctors had tried everything they had – sulfa drugs, blood transfusions and surgery, to no avail.  As she began to fail, her doctors injected her with an experimental drug called penicillin.

Overnight, her temperature dropped, and by the next day Ms. Miller began to regain her health.  The hospital chart that documents this event – the very first life saved by penicillin – is now preserved in the Smithsonian Museum in Washington, D.C.

In the decades since, the miraculous became the mundane; we came to expect that antibiotics would not only save lives and prevent death, but that they would cure practically every sneeze and sniffle.  But we are now grappling with a reality that was not yet clear in those earlier days – that inherent in every attempt to kill a microbe is the counterbalance of the microbe’s ability to adapt and survive.  

Moreover, we now recognize that a complex array of factors – relating to society, the environment, and our increasingly global interconnectedness – all enhance the likelihood of disease emergence and spread.

While dramatic advances in science and technology have enabled us to make extraordinary strides forward in medical care and public health, we have no room for complacency. 

In order to combat the drug-resistant strains of bacteria on the rise today – such as Methicillin-resistant Staphylococcus aureus (or MRSA) —and to ensure that we have the vaccines we will need to immunize people against a future flu strain, we need robust medical product development that reflects both changing needs and the opportunities that exist in science and technology today.

Yet I think we all recognize that there is, in fact, a dearth of new products in the pipeline, and especially ones that are truly innovative.  The number of newly approved anti-infectives – not just new formulations of previously existing drugs – has fallen steadily since the 1980s. 

And the range of new antibiotics is disturbingly limited in terms of the types or classes of antibiotics available and the diseases they can treat.

This is a major concern...and there are many complex reasons that underlie this worrisome trend, some that are common across all medical product development, and some that are unique to the realm of infectious diseases.

Overall, the time and costs of drug development are a significant barrier.

This becomes an increasing challenge for antibiotics, used for short-course treatment as opposed to chronic illness, which means that the market opportunity may be less attractive for investment by drug companies.  There may be additional restrictions to market returns because of medically important efforts to safeguard and limit antibiotic use. 

Layered on top of these concerns are regulatory and scientific uncertainties and challenges.  FDA must be focused – and proactive – in addressing these concerns.

I came to the FDA convinced by decades of public health work that the agency could be more of a catalyst for action in delivering on the unmet promise of biomedical research.

And believe me, I also hear it from the patients who need new therapies and from their health care providers who are desperate to have more medical options to offer.  I hear it from many involved in biomedical research and product development, and I understand the frustration with regulation that isn’t as sound or as streamlined as it should be. 

And while there will never be a guarantee that every product will be approved, we can help make the system more transparent, consistent, and predictable.  At the FDA, we are acutely aware of our obligation to help make sure that advances in biomedical research are translated into therapies that matter to patients. 

This translation requires regulatory flexibility in light of continually changing needs and circumstances.  But it also demands that we advance regulatory science:  the knowledge and tools necessary for the meaningful and timely development of new products and their review for safety, efficacy, quality and performance. 

Regulatory science is an essential part of the overall scientific enterprise, yet it has been under-appreciated, under-funded, and under-developed.  Because of this, we have been unable to apply the best possible science and technology to the tasks before us.

Without advances in regulatory science, promising therapies may be discarded during development because we lack the tools to recognize their potential or because outdated, inefficient review methods unnecessarily delay the approval of critical treatments.  On the other hand, both significant dollars and many years maybe wasted assessing a novel therapy that with better tools might be shown to be unsafe or ineffective at an earlier stage. 

I should emphasize that regulatory science cuts across an array of disciplines and approaches, and the knowledge generated from the studies informs a while body of innovation rather than a single product.

In recent years, we have been working hard to advance regulatory science, and devoting time and resources to the effort to identify areas ripe for strategic, targeted investments.

This includes, of course, strengthening the science base at the FDA.  But we are also focusing on building the partnerships – across government, and with academia, industry, patient groups and others – that will drive the development of innovative medical products and the delivery of better, safer products to the people who need them.

But what does all of this really mean, especially for emerging infectious diseases?  Let me give you a few examples.

For one thing, we need to develop much more focused research and development efforts that harness cutting-edge science, for instance, to develop antibiotics that act via well-defined mechanisms that reflect our best understanding of potential drug targets, disease pathogenesis, and human immune response.

And we need better, more advanced strategies for evaluating the safety and effectiveness of new antibiotics once they are in development.

For example, biomarkers are proving their benefit for predictive toxicology – if a drug is going to fail, best that it fail early.  And we know that the identification of appropriate biomarkers or surrogate endpoints can help support faster paths to assess the efficacy of various therapeutic interventions and support efforts to optimize clinical trial design.

Currently at the FDA, we are putting a lot of effort into developing new, more adaptable approaches to clinical trials that reflect the need for rigorous scientific answers but are realistic about constraints, such as the number of patients, the size and length of studies, and the cost of the trials.

We are also exploring with colleagues and policymakers whether a new approval mechanism might be of value for “special limited use.”  In the context of antibacterial drugs, one can easily imagine the situation where it might be appropriate to approve a new drug for use in a population of patients with a serious drug-resistant infection, but limit its use in the broader population who have less severe disease and whose infections are no resistant to other antibacterial agents.

Under current requirements FDA cannot be assured that use of a product will be limited, so drug development plans must often evaluate the risks in a broader populations resulting in larger, lengthier trials.

Such an approach – which would require working with Congress for the creation of a new drug approval pathway, could permit a more appropriate risk-benefit evaluation for serious infections and enable potentially lifesaving medicines to be made available to patients in a more timely way.

In addition, it is essential to think more creatively about the types of products or therapies that are really needed.  For example, as we know from experience with HIV and TB and other diseases, effective interventions may require attacking the disease process at several points of vulnerability.

Traditionally, FDA has required drugs for combination therapy to be developed, reviewed and approved in a serial manner, but while scientifically more difficult, the health of the public requires another approach.  We have encouraged sponsors to look to developing drugs in combination, and to support his effort, we recently published a guidance to help clarify strategies to co-develop two or more novel drugs to be used in combination, and to provide advice on addressing the scientific and regulatory issues that may arise.

These are all examples of how we at the FDA are trying to facilitate the development of innovative products, while always maintaining our commitment to safety and effectiveness.

In this context, I want to mention another area of critical importance – to FDA and to all of us here today – and that is public health preparedness.   For a range of potentially catastrophic biological threats, both natural and deliberate, the world remains ill-prepared. 

We are without many of the medical countermeasures – the drugs, vaccines, and diagnostics – needed to combat known threats – and we lack the capacity to rapidly develop new countermeasures in the face of new threats.

This is unacceptable.  We need a far more flexible, nimble system to respond rapidly in the face of any attack or threat.  As part of a broader effort in the Department of Health and Human Services to strengthen the Public Health Emergency Medical Countermeasures Enterprise, FDA has launched an initiative to support the development and availability of medical countermeasures.

It is certainly an area where there is a compelling and urgent need to translate advances in science and technology into new and better products . . . but it is also an area of enormous challenge to resolve outstanding scientific issues and develop the tools and regulatory pathways necessary to support the development of new medical countermeasures, especially where study populations are limited or non-exisitent and market incentives are lacking or unpredictable.

The needs and benefits of work will be far reaching.

The recent controversy over lab-created strains of H5N1 avian flu influenza virus that can spread in mammals has reawakened attention and concern about the potential for viruses to cause devastating pandemics, and the potential, for scientific knowledge, in the wrong hands to be intentionally used for destructive purposes. 

These are very real issues, but in the heated discussions about whether we should or should not be undertaking such research – and if, how and where it should be published – let’s not lose sight of the important and related issue that we are not prepared for a severe pandemic of any type, and we are not doing all that we can to use available and emerging science to make us better prepared. 

This is a time to redouble our efforts.  I am sure that I do not need to remind this group that in 2009, a vaccine only became available considerably after the H1N1 pandemic began – and event then there was only enough supply for about 20% of the world’s population.

We have talked for years about the need for better and faster vaccine technologies – including universal flu vaccines and new vaccine platforms.  And some progress has been made...but much more needs to be done, and not only in the realm of vaccines.

So as the controversy swirls, perhaps we can harness some of the energy and attention back into the needs for public health preparedness, and replace any complacency with a renewed sense of urgency and commitment. 

That’s why FDA will support enhanced review and novel manufacturing approaches.  We will work interactively with developers and government partners from very early in the development process to define viable regulatory pathways – speeding progress toward product approval by helping to anticipate and resolve bottlenecks, and to identify and address scientific problems that may emerge.

In closing, let me share with you a few quick examples where regulatory science at FDA was successfully leveraged to encourage effective product development and to speed product release.

In 2010, a meningococcal A conjugate vaccine developed through the Meningitis Vaccine Project was pre-qualified by the World Health Organization to ensure it met international standards of quality, safety, and efficacy.

Since the vaccine – which was manufactured by the Serum Institute of India – was introduced, vaccination campaigns have been held in Burkina Faso, Mali, and Niger...and nearly 20 million people were vaccinated in just six months.

According to the WHO, and as Tom mentioned earlier, the vaccine is expected to eliminate the primary cause of epidemic meningitis in Africa if introduced in all 25 countries of what has become known as the “meningitis belt” and to save as many as 150,000 lives by 2015.

I’m proud that FDA scientists, specifically those in our Center for Biologics Evaluation and Research (or CBER), were instrumental in facilitating the development of this vaccine.  They developed a method called conjugation technology that can increase the immunogenicity and duration of protection afforded by the vaccine. 

We then established a Cooperative Research and Development Agreement with the Serum Institute of India, and CBER scientists transferred the technology to the Indian scientists and provided them with technical support as they scaled up to produce the vaccine for millions of people.

We’ve also used regulatory science approaches to avoid unneeded clinical trials and determine drug dosing in infants and children.

For example, pediatric dosing recommendations for use of intravenous Peramivir, under Emergency Use Authorization to treat the 2009 H1N1 influenza, were derived from a model-based analysis of data available from health adults and acute uncomplicated influenza infected patients.  Also, FDA’s work on dosing the antiviral Tamiflu in children under one was a model adopted by countries around the world.  

These are meaningful success stories – and they’re also great examples of finding new and better ways of doing things.  We must engage in innovative partnerships – across sectors...across disciplines...across national borders.  We must think in new ways and act accordingly.

We have the power to prevent the next pandemic, and to defeat emerging infectious diseases – but only if we all step up the fight together.

Seventy years ago, prospects may have looked great to win the war against infectious disease with the extraordinary recovery of Ms. Miller.  But we all know that the struggle continues...and I am afraid that we have a lot of work to do together.

Thank you and best wishes for a great conference.