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Center for Health Transformation
Preparing the Country for the Alzheimer’s Epidemic:
A View from Science, Business, Government and Caregivers
National Press Club
Washington, DC
Andrew C. von Eschenbach
Acting Commissioner of Food and Drugs
This text contains Dr. von Eschenbach's prepared remarks. It should be used with the understanding that some material may have been added or deleted during actual delivery.
Thank you, Bob [Essner, CEO Chairman, Wyeth Pharmaceuticals], very much. Having listened to this morning's presentation, and particularly, having had the opportunity to view the video and experience the emotion you all are feeling, I realize the suffering, the pain and the devastation that's all around us. So, I'm going to speak to you today more from the heart than from prepared text. I'm going to talk to you about the importance of this conference, the importance of you being here, what you are getting involved in, and what you will be responsible for.
The video brought me back—in the context of listening to Dr. Gandy's [Samuel Gandy, M.D., Farber Institute for Neuroscience] scientific presentation—to my roots at M.D. Anderson, where I spent 26 years living with a dual reality. On the one hand, I was able to be a part of some of the most profound breakthroughs in biomedical research and in science and technology, yet, at the same time, every single day I was confronted with suffering and death and the ravages due to a disease like cancer. And I knew that those two realities needed to be, and could be, reconciled. All the progress—the kind that Dr. Gandy talked about this morning—could now lead us to a point where we no longer have to witness and tolerate that suffering and death, whether it is a disease like cancer or the ravages of Alzheimer's disease. That is within our grasp. That is our opportunity. That is why this meeting and your involvement and participation are so important.
Almost five years ago, I had the privilege of coming to Washington to lead the National Cancer Institute (NCI) with a vision, with a passion, and with a commitment. I set a goal that we would focus and commit our efforts to eliminating the suffering and death due to cancer by 2015. I was immediately criticized, perhaps appropriately, in terms of not having had the opportunity to explain the rationale and the reason behind that goal. But the one person who didn't criticize me as being overly ambitious, or perhaps being irrational, was Newt Gingrich, who reminded me that our challenge, and perhaps my challenge, was not only to lead us to a better way of dealing with cancer, but in fact, to help catalyze a transformation in health care.
I would present that same perspective to you this morning. As you are engaged passionately and appropriately in seeking and driving for a solution to the problem of Alzheimer's, you also are involved in a larger transformation: a transformation in health, in health care, and in fact, in our health care delivery system. What all of us together are involved in—what the challenge for this nation is with regard to ensuring this future—is that we are in the midst of the most profound transformation to occur in the entire history of medicine. That history has been based on the fact that for thousands of years we've practiced medicine based on what we have known about diseases using our five senses—what we could see, feel or touch, what we could hear, and even what we could smell. And 100 years ago or so we went from that very macroscopic perspective to a microscopic perspective. Technology provided us tools like the microscope and the X-ray machine and we could see better, we could see the cells that were responsible for a tumor under the microscope. We could see a lump, not just on the surface but deep inside the body. We could look at an autopsy and see the plaque in the brain that was responsible for what we had observed as Alzheimer’s disease.
We may think of that macroscopic and that microscopic perspective of disease as ancient history but when I began my career in oncology at M.D. Anderson—the world's largest cancer center—the only way I had of detecting prostate cancer was what I could feel with the tip of my finger on digital rectal examination. I was wrong 50 percent of the time. That has been, and up until very recently is, the history of medicine: a macroscopic and microscopic perspective. And it was discontinuous with being able to determine what we should do about disease once we observed it. Our diagnosis did not inform us with regard to therapy and so therapy was discontinuous, based on the very empiric “trial-and-error” model.
But, about midway through the 20th century, perhaps with the discovery of DNA, a transition occurred. Throughout the latter half of the 20th century, science and technology—the science that you listened to this morning so eloquently presented by Dr. Gandy—has led us to a transformation. About 10 years ago or so, we crossed a threshold, a threshold in which medicine and our understanding of diseases was no longer based on a macroscopic and microscopic perspective, but on a molecular perspective. For the first time, we can not only see but we can understand diseases at their molecular and genetic levels. We are beginning to understand the mechanisms that are responsible for disease processes that previously we could only observe. That understanding of those fundamental mechanisms is opening up the door to immediately understanding the interventions that will enable us to modulate those disease processes by preempting them from ever occurring in the first place, by being able to detect them earlier in a way that we can eliminate them, or by being able to modulate or control them.
Some of the things that you heard from Bob Essner with regard to the vision of Wyeth, around the concept of disease modulation, is for the first time bringing to us those tools, that new armamentarium, that only 10 to15 years ago we could not begin to imagine or understand. It leads us to new challenges to recognize that we are in the midst of a change process that is so profound that it is not a linear extrapolation of the past, but it is so profound a transformation that it is a metamorphosis—a metamorphosis in the sense that we will be looking at a future in health and health care that is no more like the past than a butterfly is like a caterpillar. It is that profound and that significant. It is a change process that will affect not just one part or piece of this equation, but every single component. It is a change process that we are all immersed in because it transcends the full continuum from our very notions and concepts of understanding health to what those interventions are that we will be able to deliver and use in our health care system. The interventions will be available to every single person in need—these new concepts and new contexts that will emerge out of this molecular metamorphosis.
We are focused, of course, on immediate and present concerns like Alzheimer's, cancer, AIDS, diabetes, obesity, aging in general, acute diseases, pandemics, and avian influenza. But one of the most important considerations that we must be focused on is the challenges. We must see them in a larger context. And so it is not that we be Alzheimer’s-centric, but that opportunities can be Alzheimer’s-led. We are together, collectively and cooperatively in the midst of being able to change the entire future of health care by embracing and fully developing across the continuum of discovery, development and delivery the new molecular reality and molecular opportunity. And it holds the promise for being able to radically conquer diseases like Alzheimer's. Not only is the magnitude of change that significant, but the pace of change is equally significant such that we no longer need to think of time horizons in decades and centuries away, as we did in the past. We must see this as, not evolution but revolution in medicine.
I have had the privilege of moving from the NCI, where we had the opportunity to drive the agenda of our understanding of molecular mechanisms of a disease process like cancer, to thinking about that disease not as an event but as a process in which those genetic and molecular and cellular events occur over a period of time. As a result, they will offer us ample targets for intervention that could preempt its outcome of suffering and death. When listening to this morning's presentations, we can recognize that that is exactly the same paradigm for Alzheimer's. It is a disease process that occurs over time, and as we understand the fundamental mechanisms as outlined by Dr. Gandy, we can begin to develop interventions as presented by Bob Essner that could prevent or preempt or modulate that disease process in a way that we eliminate the outcome—that tragic, horrible outcome—that we witnessed on the video.
The FDA is positioned as the bridge that needs to be responsible for making certain that all the fruits of that discovery and that development come to be applied to patients who are in need. It is the FDA's commitment to be that bridge—to be that bridge, not of the past but of the future. And for that, like each of you and like every other part of this equation, FDA must change. It has a proud record over the last 100 years of being the world's gold standard, but the FDA of the past is not adequately equipped for this new reality. It must change not in isolation, but in context and in collaboration and integration with all the other parts and pieces of the equation. And so, we've embarked upon an opportunity to look internally about what those transformations are that must occur within the agency itself. And what those opportunities are to collaborate and integrate both on the discovery and the delivery end of the continuum, to bring that process about.
I don't have the opportunity this morning, with the amount of time given, to present to you all of those initiatives and opportunities that we will be embracing and embarking upon, but you are familiar with some of the preliminary outlines: Critical Path and the need to fully implement many of the strategic initiatives so that we've braved a new science that is making possible discovery and development into the regulatory process; the use of biomarkers instead of simply waiting for the kinds of outcomes that we've alluded to earlier having to do with autopsy findings; the ability to completely revamp our clinical trials process and begin to look at different adaptive trial designs and models that are to be adapted to the new realities; to begin to bring tools of modern information technology and bioinformatics into the regulatory process; to collaborate and cooperate with the industry to assure that we are effectively, proactively facilitating the development of those new interventions in ways that ensure not just their efficacy but their safety; to be able to stay invested not only on the front end of their development but also to continue to monitor and modulate their behavior once they reach much larger populations.
The FDA is looking forward to continuing to work with you in providing this insight and opportunity for the kind of changes that we will need to embrace.
I know the efforts of your organization are extremely important, having met with Harry Johns and others. We're looking forward to that continued dialogue. One of the things that we have done is look at ways in which we can bring the advocacy groups more actively into the process. The patient-consultant program will include the ability to bring advocate participation into FDA's regulation and development of new treatments for serious neurological diseases. The patient-representative program will welcome your participation in advisory committees. We have created an FDA interagency neurology working group that will enable us to integrate across the entire portfolio of the FDA our opportunities to look at our use of neurological diseases like Alzheimer's, as a model—just like we can look at cancer as a model to the activities that we have around the interagency oncology task force to drive this integrative and collaborative process.
Bob commented about globalization of this problem this week. For the very first time I had the privilege of hosting in Washington 24 of my peers—regulators—from around the world—China, India, all of Europe, and South America—to talk about how we as regulators can collaborate and cooperate to embrace this new era and these new challenges in molecular medicine and avoid fragmentations, since we are in fact dealing with global problems in a global industry.
There is much for us to do and much for us to change. But it is also a truism that the only human organism that likes change is a 6-month-old with a dirty diaper. So, change for FDA will be difficult. Change for you will be difficult. And clearly, change for our nation will be difficult. But change we must. We, like the caterpillar, are in the midst of a metamorphosis and we have no choice about that change. That metamorphosis is already occurring. Unlike the caterpillar, who also has no choice about what it will be—it is destined to be a butterfly—we do have a choice about what we will be. We are creating a new reality for which there is no description. We must come together collaboratively and cooperatively in a design-build mode to find that way forward to create that new reality in which no one need suffer or die from a disease like Alzheimer's or cancer, AIDS or diabetes, or even infections like avian flu. The tools and the opportunities and the knowledge are emerging. That butterfly is coming out of that cocoon. We must seize and capture this opportunity. This conference typifies what we need. We need knowledge coming from scientists, we need commitment coming from developers of interventions, we need vision coming from public leaders, like the Center for Health Transformation, and we need leadership and advocacy and passion, coming from you. And collectively, cooperatively together, we will create a new world free of suffering, not just for patients of Alzheimer's and cancer, but for everyone.
You have the opportunity to help make that happen, and it's a privilege for me to work with you. And after listening to you this morning, I am reminded of why I am here.
Thank you.
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