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March 21, 2006 - Scott Gottlieb, MD - Annual Biotechnology Meeting

 Speech before

Pacific Northwest Annual Biotechnology Meeting

Remarks by

Scott Gottlieb, MD
Deputy Commissioner for Medical and Scientific Affairs
Food and Drug Administration


March 21, 2006

This text contains Dr. Gottlieb's prepared remarks. It should be used with the understanding that some material may have been added or deleted during actual delivery.

I want to thank you for the opportunity to join you here today.

Tomorrow I will be attending an important event at FDA’s regional office here in the Pacific Northwest. As many of you know, FDA is celebrating the 100th Anniversary of the creation of the Food, Drug and Cosmetics Act, the legislation that gave rise to the modern FDA. Tomorrow’s event will commemorate that achievement, and the achievements of FDA over the past century as well as those we continue to make under the leadership of Acting FDA Commissioner Dr. Andrew von Eschenbach at enabling a lot of opportunities for consumers to improve their lives.

We will also be celebrating recent achievements, especially the triumph we have all made in recent years toward developing a base of science and technology that puts us on the threshold of a great change in healthcare and medical practice. In fact, when you look back at the last 100 years, when you read the original Food and Drug Act, it’s striking to realize all of the progress we have made in biomedicine and in medical practice.

It’s striking to see how much medical practice itself has changed as a result of technology and scientific innovation.

One hundred years ago we didn’t have antibiotics. We didn’t fully understand how germs caused disease. Didn’t know what viruses were let alone retroviruses, or prions. We didn’t know how to do what are today the most basic surgical operations. It’s hard to believe we are only fifty years removed from the central dogma of the double helix.

A remarkable transformation has occurred during the lifetime of those of us in this room, one that depends upon application of the laws of physics and the structural relationship of chemistry on the fundamentals of biology. And it’s impossible to divorce that scientific progress, made over the last century, from all of the gains in health and life expectancy and general wellbeing that we’ve achieved in our lifetime -- not just here in the U.S. but around the world.

And American companies and American research institutions – both public and private -- have led that scientific progress. It’s this progress that has enabled a global renaissance in health. And this achievement is perhaps our nation’s most important and enduring contribution to the world.

But this progress has not come without its own challenges, without sacrifice, and without its own costs. And it has not been a smooth ride along the way. In fact, even when you look back at that scientific progress and more generally, at the evolution of medicine and medical science since 100 years ago, or from any time in history, I think you see various inflection points, various points of transformation, where things plodded along, as we made small improvements, until medical practice and health and wellbeing more generally underwent major improvements owing to some important discovery that changed the way we approached life science.

This is true whether it was the basic discoveries about human anatomy that opened the door to approaches to surgery, or the advent of modern germ theory and the discovery of bacteria as the basis of disease -- or more recently, the firming of our understanding of the immune system and the advent of both active and passive immune therapies like monoclonal antibodies.

And it is surely true today, as this progress continues, and we stand on the brink of another inflection point – another transformation -- with our growing mastery of the genetic basis of disease through the science of genomics and proteomics.

The Pacific Northwest has been at the vanguard of much of that recent progress.

Washington State alone is home to more than 220 biotech companies; forty-two percent formed in the last five years. And this region is also home to one of the fastest growing public research centers in the world. This biotech industry is founded on and continues to thrive due to the region’s world-class research institutions such as the University of Washington, Washington State University, the Oregon Health Sciences University, the Fred Hutchinson Cancer Research Center, the Pacific Northwest Research Institute, the Virginia Mason Research Center, and many others.

Public-private partnership has been important to medical progress and it has been important to the Pacific Northwest. And it is important to our future. And it’s this future that I want to talk about today, the steps we need to take working together to enable it, and the steps FDA is taking right now.

While healthcare achievements in past decades have been impressive, I believe, owing to recent scientific advances, there is a lot of medical progress that we’re poised to realize in the next decade or two. And this progress may already be showing up at FDA, in the way of many more innovative molecules that we’re seeing filed with the agency as part of investigational new drug applications.

But with so many people suffering from diseases today that could be more treatable or even cured in the near future, these new molecules are still taking too long to be turned into practical solutions patients can safely use, and scientific breakthroughs aren’t resulting in medical benefits soon enough.

The plain truth is that despite all of our scientific progress in recent decades, the chances that a new molecule will be successfully developed into a safe and effective medicine hasn’t changed one bit, and by many measures, it has slipped, even while the cost of developing a single new medicine has nearly tripled in just the last decade. And this has happened even while our investments in research have grown enormously as well, on the order of $120 billion in R&D spending, once you factor in the money spent by the National Institutes of Health as well as private companies and public institutions.

As health care costs continue to rise, it’s increasingly important to make sure we’re taking steps to address concerns that new medical innovations that can help end suffering and reduce overall healthcare costs are not themselves prices out of development. The plain truth is that we’ve seen a lot of discoveries made in laboratories in recent years but we’re not seeing enough of these discoveries turned into practical benefits for patients. Right now, the number of novel drugs approved by FDA is near an all time low. We’re simply not seeing all of the products many people expected from recent scientific advances in genomics and proteomics entering the late stages of development, and the drug development process itself may be one reason why that’s so.

So it’s clear that we stand at an important moment when it comes to drug discovery. And we have a lot of hard decisions to make together. On the one hand, it’s clear we are on the cusp of perhaps another great transformation in medicine. But on the other hand, we have some hard policy decisions that we need to make, and we have to decide whether we are going to have the will and the commitment to make sure that this new science results in practical benefits for patients and isn’t unrealized because we made the wrong policy decisions that squandered the vast research industry we have grown up in modern times.

In fact, some suggest right now that the problem rests with the drug industry itself, and that more of the research and development into new drugs should be handled by public institutions like the NIH and academic centers. Now, I don’t think basic science institutions would argue that they are equipped to do this kind of translational work, to actually develop drugs, and I don’t think it’s an either or proposition. The private drug industry has surely benefited from the basic discoveries that are made by public institutions. Now, I think it’s fair to say that the growth of a public research base and the NIH in particular has been one of the most successful endeavors our government ever undertook. We set out to establish a base of scientific expertise in order to grow up a modern life sciences sector in this country, and to the benefit of literally tens of millions of people, we have done just that. Because the result is that, seizing on this science base, for profit companies, fueled by the investment capital of millions of individual investors and the incentives offered by our well-developed equity markets, lead the world in chasing the hardest and most expensive medical solutions -- whether it’s a new drug based on a completely novel mechanism or a very intricate medical device infused with modern drugs and tissues or something completely novel that we have yet to fully envision.

But if we’re going to continue to make progress, and capitalize on a lot of this new scientific work and develop all of the practical medical treatments we need and expect, than I think the distinct roles that the private sector and public research institutions play are going to become even more important, and their unique endeavors and expertise even more discrete and more necessary.

That’s because the problem, I believe, is that we’ve hit a wall when it comes to the science of actually developing new drugs. All of that terrific, highly advanced science that we’ve developed in the last few decades is resulting in highly innovative new molecules. But once those molecules leave their sophisticated laboratories, they enter a development process where the scientific tools have often been stuck in time for decades or more.

I believe this is a big reason why we’re not seeing all the progress we expected from the enormous investments we’ve made in basic science. And what’s happening in drug development is the exact opposite of what has happened in other high technology fields in recent years. In other fields, the tools for developing finished products have undergone dramatic advances, resulting in better products being turned out faster and at a lower cost. Look what has happened to the design or airplanes or even microchips, where advances in the development process itself are largely responsible for the dramatic advances in the finished products that are produced.

But none of this is true when it comes to drug discovery. In drug discovery, the best ideas still bump up against some of the oldest development tools. In drug discovery, all of the work and investment and innovation have gone into the front end, the discovery side of science. Little or no attention has been paid to the middle part of the process, the development part, when drugs are tested and manufactured and turned into medicines.

And the result is that we’re not seeing many more medicines. The result is all of that good science on the front end is not coming out the other side. It’s getting stuck in the middle. The result is that when it comes to drug development, in too many cases, we’re uses tools to evaluate new drugs that were literally developed decades ago – sometimes 50 years ago or more, to test and evaluate new medicines.  The same kinds of scientific and process improvements that expand medical practice, making it more precise, more effective and safer can have the same benefits on the way we develop medicines.

Now it’s true that modernizing our approach to drug development has not been one of our goals. But that needs to change. And it’s going to require a lot of collaboration across public and private entities, because the scope of the scientific work that we need to do is just too large and cross-cutting.

Too often, in the past, we settled for the tried and true ways of doing things, even if better scientific tools were available -- drug developers and FDA preferred familiar approaches. That worked for a while when most of the medicines we were testing worked in similar ways, and were being aimed at similar ailments. But more and more, the new drugs that are being developed are based on completely new scientific approaches such as genomics and proteomics. More and more, new drugs are being targeted to very novel medical indications and they are increasingly bumping up against a scientific development process that isn’t sufficiently advanced to evaluate them and move them to the bedside. So the entire process that drug developers often follow is not only too slow and too inefficient, but it’s also needlessly expensive.

We need to fix that if we’re going to make sure that all of the discoveries that are being made in laboratories, all of the things you read about in the science section of the newspaper, are going to be made available to patients in the form of safe and effective new treatments. And FDA needs to be a part of that, because we’re often the ones who set the standards for how that development process unfolds. At FDA, we need to make sure that process is rigorous, safe, and well validated. But also that we’re incorporating the most modern scientific tools and approaches, even if it means breaking with something tried and true if we have clearly identified a new scientific approach that is better. With so many people suffering right now from diseases that may be treatable or even cured in the near future, we can’t wait many more years, and spend millions and even billions of dollars to develop each and every medicine, simply because our process for doing so isn’t incorporating the most modern scientific tools and approaches and therefore takes too long and costs too much and is too prone to failure.

The good news is that I think there are more opportunities than ever to improve the development process. With the reauthorization of the prescription drug user fee act this year, we have the opportunity to continue to bring modern management tools and approaches to the process for evaluating new molecules to make sure they are safe and effective. And with FDA’s new critical path initiative, we have the opportunity to modernize the science of drug development itself -- the science of how we actually go about evaluating new molecules.

And so at FDA, we have set out on two simultaneous efforts to confront these challenges. The first effort is to modernize the scientific tools we use to evaluate new drugs. And the second effort is to continue to make improvements in the process for regulating new medicines to make sure that we’re incorporating the best management approaches, the best information technology, and the best quality systems and review processes to perform our daily mission.

To tackle the first challenge, under the leadership of Dr. Janet Woodcock, we have undertaken a new initiative to modernize the science of drug regulation as part of our Critical Path initiative. Critical path is a broad initiative at FDA, involving all of our medical centers. The effort is aimed at catalyzing the creation of a new generation of scientific tools to enable product sponsors to better predict and evaluate the safety and effectiveness of candidate products; to make product development less risky and more efficient; and to enable individualization of therapy to improve effectiveness and avoid side effects.

Our goal is to translate the science developed under this initiative into FDA guidance to scientists and product manufacturers in order to clarify the regulatory path for modernizing approaches to bringing products to market. Under Critical Path, we are working on long-term science development as well as near term improvements and modernizations in our own regulatory procedures to accommodate new scientific tools and approaches.

For example, we are launching a clinical trial collaboration to validate the use of positron emission tomography imaging as a surrogate endpoint for cancer therapies, in partnership with the National Cancer Institute, the Centers for Medicaid and Medicare Services and other partners.  We hope to expand this collaboration to include additional imaging technologies.  We also recently launched a new biomarker qualification program in collaboration with the National Institutes of Health. And we announced other new initiatives aimed at improvement the scientific process for evaluating new drugs, such as a safe framework for conducting early studies with micro doses, as well as a safe harbor for the submission of pharmacogenomics data, which we hope to soon extend to other kinds of data submissions such as proteomic data.

At the same time, we are developing regulatory guidance for incorporating these new scientific tools into the development process. One such guidance, which should be out in draft form this year, addresses how product developers can seek approval for a new drug and a diagnostic test for guiding the targeted use of that therapy, all within the same registration trial.

But to do these things, we’re going to need your help. As I said, the biggest challenge is that a lot of the scientific work we’re trying to do under our critical path is too broad to be done by any one institution or company. And so its going to require a lot of partnerships across both private and public entities and a lot of it is going to require the help of leading research institutions like those here in the Pacific Northwest. We’re only going to be able to get these things done, and advance the science around drug development, if we all work together.

While we take these steps to modernize the scientific tools that are used during drug development, we are also mindful to continue to take steps to modernize our own processes and approaches so that we are adopting modern management approaches and so that new scientific tools can become effectively integrated into the registration process. The original vision behind the PDUFA legislation was that it would enable FDA to incorporate modern management approaches into how it went about its scientific work. The legislation has been a significant success in achieving that goal, but there is still more we want to do and more we can do. And so while the new reauthorization of PDUFA could provide another important opportunity to bring focus and resources on making additional improvements in the drug review process, there are many things we are doing right now under the leadership of Dr. Steven Galson, the head of our Center for Drugs, Dr. Jesse Goodman, the head of our Center for Biologics, and many others.

One way we can do this, for example, is to promulgate guidance on how scientists can use these new tools as part of the drug development process. We also want to continue to take steps to modernize our own internal approaches to evaluating the information we receive, and setting regulatory standards, whether it is through process improvements in our own work, through quality systems we adopt, or through technological improvements such as the incorporation of information technology to help us better evaluate the information we receive.

To these ends, we recently announced a new commitment by the FDA to improve the process for collecting medical information as part of the so-called phase four trials or “post marketing commitments” drug developers often undertake after drugs are approved. We will also soon be announcing a quality system around how we conduct meetings with sponsors and how we generate meeting minutes. The meetings we take with sponsors, especially early in the development process, have become an invaluable part of creating good development programs for new molecules.

A recent report that we commissioned by the consulting firm Booz Allen, to examine the root cause of why many molecules fail in development or need to undergo multiple cycles of review before they are eventually approved, found that sponsors that have early communications with FDA, seeking advice about their development programs, and sponsors that follow the advice they receive, stand a higher chance of success with their molecules. In fact, all things considered, early and effective consultation was one of the single best predictors of success in the review process. So we plan to take additional steps to make sure these consultations are as effective as possible. We will be finalizing soon a guidance document that deals with best practices for how these meetings are conducted, and how we can make sure that we are making the most of these opportunities to give feedback to sponsors on their development programs. We plan to issue this guidance in June.

We will also be undertaking an internal initiative to create process and procedural improvements in the way we generate written records from these meetings.

Something like minutes from a meeting sounds like the kind of work that goes on every day in ordinary business enterprises, but at FDA these minutes can sometimes be very detailed and intricate. We know that these meeting minutes – done right -- can be an important tool to help sponsors get good guidance on how to design appropriate trials to demonstrate safety and effectiveness. So we want to make sure we’re maximizing the value of this important information tool, to improve the consistency and the transparency of the review process.

The initiative we’re undertaking, with the help of outside experts, will evaluate current practices for ensuring accurate, quality meeting minutes content and format used in the drug and biologics centers, and it will recommend best practices for how to write clear, concise meeting minutes that accurately reflect meeting outcomes. We will also be providing in-depth training for staff within both of our centers on developing meeting minutes with good content and format. The professional staff at FDA devotes significant resources of time and talent to these meetings and continues to work to make sure they are a valuable part of the scientific exchange that takes place during the development process.

These are just a few examples, I spoke about today, of how we want to use modern science and modern management tools to make sure we’re taking the most effective approach to determining the safety and effectiveness of new medical products, and that we’re doing all of these things in an efficient way. Taking new ideas from the notebook to the market requires huge investments in research and development. This is a risky process, and risk is the enemy of investment. To continue to make progress, we have to do all we can to make sure that process is as modern, and efficient, and as effective as science allows.

The bottom line is this: There are many opportunities to improve our current process for drug development – improvements that could provide greater efficiencies to the development process, and even more important, improve our ability to determine the safety and effectiveness of a new medicine much earlier, and perhaps with less cost. Working together, by harnessing modern scientific tools and smarter approaches to our work, we can improve the development process, making it more efficient and more effective, so that innovative new medicines can get to the patients who need them. Thank you.