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Remarks at the Prix Galien USA Awards Gala

Remarks at the Prix Galien USA Awards Gala
As Delivered by Margaret A. Hamburg, M.D., Commissioner of Food and Drugs
New York City
October 16, 2012
 
Thank you Dr. Vagelos. I have long been an admirer of your leadership and vision, so it is a great honor to be introduced by you. 

I also want to acknowledge Professor Elie Wiesel, who originally reached out to me about this event.  He reminds us of what it takes to make a difference and the qualities that make us most human -- courage, compassion, resilience and tolerance. 

As scientists we try to understand human biology and physiology.  But Elie Wiesel understands – and through his writings and activism reveals – the human spirit.
***
It is wonderful to be here together in this glorious setting. I have been here many times, but this evening is special.  I don’t often have a chance to talk under the watchful gaze of a 94 foot blue whale. In fact, in Washington I am more likely to feel surrounded by sharks!

Thank you, of course, to the Galien Foundation for hosting this gala – and for recognizing tonight some of the most recent advances in life sciences research, and the men and women whose commitment to excellence in scientific innovation are responsible for those advances.

The goal is patient-centered research…that not only wins awards – but through rigorous science, discovers winning therapies that treat disease, save lives, hold families together, and serve the cause of human health and dignity.

So to this year’s Prix Galien award recipients: I applaud your innovative thinking, the quality of your research, and the impact you will have on public health and medical care.
                                                            ***
The great southern writer, William Faulkner, on another awards occasion--his receipt of the Nobel Prize in Literature-- said “I believe man will not merely endure, he will prevail…”
And prevail because of “a spirit capable of compassion and sacrifice and endurance.”

As we address the many public health and medical care needs before us, I share his optimism that humanity –men and women-- can  and will prevail against new and persistent infectious disease threats; food borne illnesses; cancer; heart disease; chronic metabolic disorders; and degenerative brain disorders – to name just a few of the challenges before us.

We will prevail because the human spirit is indomitable, but also because those dedicated to improving health  can stand on the shoulders of so many giants, including Claude Galen—the spirit behind these awards and tonight’s gala evening.

Galen was a medical pioneer.  Born in 131 AD, he studied human physiology, learned how to make medicinal products, and was the first to recognize that good medicine is a combination of theory, observation, and experimentation.

Even today – Galen’s reliance on observation, deduction, and investigation – and his ethic that the purpose of medicine must be patient care – influences the way we think, the way we conduct clinical trials, our priorities for biomedical research, and the way we care for patients.

These same values influence – and inform – the work we do at the Food and Drug Administration. Thousands of our scientists, biomedical engineers, and health professionals dedicate their lives to the proposition that advances in public health can only be built on a foundation of science – science that combines the kind of insightful theory, careful observation, and rigorous experimentation that Galen practiced and taught.

We are equally dedicated to helping the pharmaceutical industry develop and manufacture products that are safe and effective, stable and reliable, and provide real clinical benefits for patients.

Yes, we are your regulators.  But we are also your partners and your supporters.   These roles are not mutually exclusive.  History shows that the more effective we become as regulators – the more innovative, profitable, and trusted by the public you become.

Just a few days ago, the FDA marked the 50th anniversary of the Kefauver-Harris Drug Amendments. Passage of this legislation was a notable event, which in many ways changed the face of modern therapeutics by dramatically raising the standard of evidence.
This Congressional action occurred in response to the Thalidomide tragedy.  As you no doubt recall, this drug was widely marketed in Europe as a popular therapy for morning sickness but proved to be highly teratogenic. Thousands of babies suffered devastating and disfiguring birth defects.
 
The United States was largely spared because FDA, in the person of Dr. Frances Kelsey, blocked  it from the market. But these events revealed  serious shortcomings in the existing Food, Drug and Cosmetic Act.

Congress acted to require that the new drugs had to be proven safe and effective for their intended use– in well controlled studies – before they could be approved and marketed.
Many opposed these amendments, concerned that this kind of drug testing would be too expensive, cumbersome and time consuming. But there were real benefits for patients and for industry. Now the best drugs, rather than the most aggressively marketed drugs, could rise to the top.

In fact, the pace of drug approvals quickened dramatically, and America’s pharmaceutical industry is now the most advanced, trusted, prosperous, and emulated in the world.

Nevertheless, difficult challenges to innovation remain. Despite unprecedented spending on research and development by government and industry alike, the pipeline of new products is distressingly low. The costs of bringing new drugs to market have skyrocketed—both because the science is more complex and development costs have increased.  Venture capital is harder to come by, and many companies are facing a patent cliff.  Moreover, Big Pharma is slashing R & D budgets, and government science agencies like the National Institutes of Health and FDA are also under the knife.
So how can we more fully realize all the extraordinary opportunities that this golden age of biomedical research has to offer? 

Yes, we are making remarkable progress in key areas like sequencing the human genome, combinatorial chemistry, high throughput screening, and synthetic biology.  And new electronics, nanotechnology, and materials science have the power to transform medical devices. 

But despite these remarkable advances in science and technology, patients are urgently waiting for new therapies and often not getting them.

The challenge of translating cutting-edge research into innovative products is real – and cannot be solved incrementally.  We need a comprehensive, integrated, and coordinated strategy that engages the full “ecosystem” to advance biomedical product innovation.

This kind of engagement demands strategic investments in science, new regulatory pathways that move products through the development and manufacturing pipeline in less time, and a true collaboration among health professionals, industry, government, academia, and our global health partners.

It also demands that we look at other key elements of this complex ecosystem, including intellectual property and patent issues, economic policies (including incentives and access to capital), and on the other end, reimbursement policies as well.

Innovation is very personal to me.  As a medical student, I remember watching in horror as a mysterious new illness emerged. No one knew what caused it.  No one knew how to care for it.  No one even knew what to call it.

The HIV/AIDS epidemic had begun.  And in those early days, for those who contracted HIV, we had no effective treatments – and no medicine on the horizon.  We could offer neither a cure nor hope.

But scientists and pharmaceutical companies creatively applied themselves to the task, patients mobilized, clinicians worked tirelessly, and the FDA responded by accelerating review and approvals.

When HIV/AIDS was recognized as a national health priority, leaders from across the medical, research and health care system responded by working together.  They produced tangible, meaningful, and lasting results.

Thankfully, HIV/AIDS is a no longer a death sentence.  It is a chronic, but manageable, disease.  This is one of the great scientific and technological achievements of the last 30 years, and it would not have happened without an all-out effort by the entire scientific, pharmaceutical, and health care community. 
This is what can happen when the entire innovation ecosystem trains its focus on a difficult challenge.
 
And that’s why more than a half century after Thalidomide, and more than a quarter century after the start of HIV/AIDS, innovation is a top priority at FDA – and one that we believe must be guided by some basic principles.
 
First and foremost, meaningful innovation in biomedical products must make a difference. It must benefit people where they live – and how they live. 

It must build on a deepening understanding of the underlying mechanisms of disease, pathophysiology, and human biology.  And it must be able to withstand scientific scrutiny both before the product enters the marketplace – and after.
 
And meaningful innovation must take into account that there will be failures along the way.  Even Galen – whose theories influenced western medicine for almost 2000 years – failed in some dramatic ways.

For example, he recognized that there was a difference between blood being carried in the veins and blood being carried in the arteries.  But he also thought that the circulatory system was two one-way systems, not one unified system with the heart acting as a pump.

While we must not be afraid of failure, it is part of FDA’s mission to help industry in order to lessen the chance of failure.

That means we want to work with you to build a more modern, streamlined regulatory process—grounded in science and committed to the standards for safety and efficacy which have served society so well—but also a pathway that will offer greater clarity, consistency and predictability in our important efforts to cross the translation bridge between opportunities in science and technology, and the delivery of innovative, real world products that really matter.
 
The best tool that FDA has to promote this forward progress is the advancement of regulatory science.

Regulatory science is the knowledge and tools needed to assess and evaluate a product’s safety, efficacy, quality, purity and performance. It involves the development of new methods, standards and models we can use to speed the development, review, approval and ongoing oversight of medical products.
To my way of thinking, it is an essential component of the scientific enterprise. Yet it has been under-appreciated, under-developed and under-funded for many years. It is the critical link between cutting-edge discoveries and real-world diagnostics, treatments, and cures. And at this time of enormous scientific opportunity but also increasing economic constraints, investment in regulatory science will fulfill many needs:

Regulatory science can help us recognize the potential of promising therapies that might otherwise be discarded in the early stages of development…and save critical time and dollars by making sure we have the tools we need to detect – at an early stage – unsafe or ineffective therapies.
Regulatory science can enable us to use our knowledge of biological pathways and gene variants to help identify promising new drug candidates and new potential targets for treatment…and can help usher in the era of personalized or precision medicine by linking advanced genetic data and biomarkers with targeted therapies

Regulatory science can help to develop and optimize innovative clinical trial designs and new analytics that require smaller patient populations, shorter timeframes, and lower costs. It can bring information technology and scientific computing to bear on drug development, as well as providing vital information, for example,  about a product’s safety and benefit profile after it enters the marketplace through post-market surveillance.

More broadly, with improved regulatory science, we can offer our health care system higher quality care, hopefully at a lower cost, by making treatments safer and more effective, and our medical products more innovative and targeted.
And, the overall American economy will benefit as well – as it always does when the U.S pharmaceutical and biotech industries are growing, prospering, and finding new markets.   
But a robust field of regulatory science—and the many benefits it can bring—depends on collaboration: among government, academia, industry, and the patients we serve.

The FDA is committed to building partnerships and research agendas that cut across disciplines, sectors – and with our global partners – time zones.
 
Also of note, earlier this year, Congress overwhelmingly passed the Food and Drug Administration Safety and Innovation Act, fondly known as FDASIA.

This bill will be a great catalyst for innovation and biomedical product advancement.  It assures stable funding for FDA in key areas of medical product development, and also identifies a set of important areas for collaboration in regulatory science, recognizes a new regulatory pathway for “breakthrough therapies” and clarifies others, and will engage stakeholders in assessing how best to balance risks and benefits.

There is much to be done.
  
***
Antoine de Saint-Exupery, the poet and World War II French aviator once wrote, “If you want to build a ship, don’t drum up people to collect wood and don’t assign them tasks and work.  Teach them to long for the endless immensity of the sea.”

Here in the Hall of Ocean Life, the mind is drawn to the many wonders of the sea…and with it to ponder the immense and far-reaching potential of innovation.

Innovation is not about assembling parts; it is about longing to discover, taking on previously unconquered challenges, and renewing the human spirit.

That is what the Prix Galien honors – the lifesaving drug or device at the end of the road, but also the journey of imagination, discovery, failure and success that takes us there. 

The evening’s honorees have made that journey and we, as a nation, are stronger, healthier, and more prosperous because they did.

And we will be stronger still if all of us who care about public health, care about unlocking the mysteries of human biology in the service of humanity, and care about the future of life sciences research – continue to work together as the founders of the Prix Galien envisioned.

That collaboration is what we celebrate today – and what will lead to the disease-conquering medicines that I know you will be creating tomorrow.   

Thank you – and enjoy this wonderful gala.