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Andrew C. von Eschenbach, M.D. - Chemical Heritage Foundation

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.

Speech before
The Chemical Heritage Foundation

Philadelphia, PA

Remarks by
Andrew C. von Eschenbach, M.D.

Acting Commissioner of Food and Drugs

 

May 16, 2006

As has been emphasized by other authors in this volume, the FDA, the industries it regulates, and consumers’ lives are in the midst of an enormous transition in health care. For thousands of years, medicine was practiced based on a very macroscopic perspective of disease. Our understandings of disease processes – and even our definitions of them – were derived from what we could determine with our five senses. Around the time that FDA was founded, we transitioned from that macroscopic perspective to a microscopic one. For the first time, we could see the cells that composed a tumor, the microbes responsible for an infection, and for tainted foods. While that transition was indeed profound, it was to be eclipsed by the most profound change to occur in the history of medicine. In the middle of the 20th century DNA, the genetic code of life, was discovered, sending biomedical science on a journey of innovation that today is unraveling the most basic mechanisms of life and the root causes of disease processes.

Based on such progress, about10 years ago we crossed a threshold from macro/microscopic medicine, and entered the molecular era, where we now stand on the precipice of the genomic and proteomic revolution. We are beginning to more fully appreciate the incredible opportunities, as well as implications, that are emerging from scientists’ attention to questions about the fundamental nature of life. This movement from the macroscopic and microscopic to the molecular is more than a transformation. It is a metamorphosis, provoking us to envision a future that no more resembles the past than a butterfly looks like a caterpillar.

As it embraces this dramatic change, FDA must remain focused on its core value of placing the health and safety of patients and the public at the center of everything that it does. FDA’s practice of decision-making based on science underpins its role in setting standards for assuring the safety and efficacy of a range of products used by Americans and millions more people worldwide in the 20th century. FDA’s grounding in science has made it possible for Americans to not worry about the food that they eat, to use medical devices without questioning their safety and effectiveness, and to be able wake in the middle of the night and confidently take medication or, even more importantly, to give that medication to their children or grandchildren.

But as we move further into the 21st century of molecular medicine the world around the FDA is changing, and for the agency to maintain its status as the gold standard, FDA itself must also change. Even in an era of declining budgets, FDA has recognized the importance of modernizing and transforming operations to address these emerging needs and opportunities. We must align, adapt, and recognize the importance of our leadership role and our responsibility in the metamorphosis that will define our biomolecular era.
More specifically, FDA must accept and commit to the rapid transformation of health and of healthcare in this country by establishing itself as the bridge that supports new molecular-based interventions as they move across the discovery, development, and delivery continuum. Those interventions and solutions will not only include drugs, biologics, and devices, but also food, which as a molecular nutrient is integral to health. Several authors in this volume pointed to the role that nutrition education will play in empowering consumers to make choices that will help preserve health and prevent illness. FDA will support that form of consumer empowerment as a part of maintaining our commitment to promote and protect the public.

We are engaged in an ongoing process to strategically redeploy resources to address high-risk public health challenges and as a science-led regulatory agency FDA has initiated the Critical Path to Personalized Medicine, a program designed to modernize and ensure more efficient development and clinical use of medical products. FDA considers the Critical Path Initiative to be its top scientific policy initiative for at least the next five years and expects its implementation will bring modern science and technology to models of 21st century medicine.

The projects affiliated with the Critical Path Initiative, discussed in many of the preceding chapters, are intended to help provide FDA with the tools necessary for effective leadership in the modern “-omics” era of genes, proteins and metabolites. Under the Critical Path Initiative, we anticipate being able to dramatically increase the success rate in providing patients with innovative solutions that strike an optimal balance of high benefit and low risk because they are “personalized”.

Several specific projects that promise to help us transform the way we bring new solutions to patients are already underway. The Oncology Biomarker Qualification Initiative, which represents collaboration between the primary research, evaluation, approval, and medical treatment delivery and reimbursement divisions of HHS – including FDA, the National Cancer Institute (NCI), and the Center for Medicare and Medicaid Services (CMS) – offers one example of a key Critical Path project. It is intended to facilitate the identification of biomarkers, which are measurable characteristics that reflect physiological or disease processes. Biomarkers can be used to predict or monitor responses to therapy. Without clinically proven biomarkers and innovative trial designs, we cannot modernize medical product development and realize the potential of a personalized system of medicine. Indeed, the capabilities promised by advances in genomics and proteomics indicate that the healthcare system of the future will not only be personalized, but also predictive, preemptive, and more participatory.

This healthcare system of the future will be personalized by developing a sophisticated understanding of not only the disease process, but also of the individual patient. In understanding both the disease and the person at the molecular level, physicians will be able to provide treatment options uniquely suited to a patient’s particular needs. Instead of empirically prescribing a pill and hoping it works, patients will receive treatments designed specifically for them. We will use the new tools of molecular medicine to develop new individualized interventions and in the very process of molecularly monitored delivery to patients our understanding of the biology of disease in individual humans will be elucidated. In this way, delivery will become, in itself, a platform of discovery.

Product development is at the core of the innovation process. The fruits of the tremendous explosion in innovation that has been occurring in biomedical research will only have been reaped when they are translated into actual interventions that can be applied to patients. Looking to the future, then, we must envision an FDA that will be not only a science-based regulatory agency, but an FDA that will be a science-led facilitating agency; one which helps new products travel across the bridge of development more rapidly and efficiently while assuring their highest quality.

Technological innovations will also make this healthcare system predictive and, therefore, preemptive. Long before physicians ever prescribe a treatment, we will be able to use biomarkers to predict whether that intervention will achieve the desired outcome. Reliance on such molecular tools, rather than our current, limited phenotypic understanding of disease, will help us to preempt illness, fostering the shift from therapeutic models of treatment to a model more focused on prevention.

Finally, I expect that this healthcare system will be much more participatory. Increased utilization of information technologies and improved communication strategies will allow patients to assume a substantially more active role in their healthcare. It will be important for us at FDA to consider how we can work together with doctors and patients to make sure that they have the greatest opportunity to maximize the benefits of new medical products in a way that preserves their ability to make individualized choices free from restrictive burdens, and that the data derived from health care can be used to further enhance the safety and effectiveness of these products.

We can already see these trends of personalization, prediction, and prevention in how we think about food and nutrition. Much as people have become able to use their knowledge about food and nutrition to determine the most appropriate dietary practices for their own bodies and lifestyles, the decisions people will make about healthcare will be based on a similar kind of individualized knowledge.

Improving the efficiency and reliability of clinical trials in a period of rapid advances in science is another important priority for the agency. While it is not a member, FDA is serving in an important oversight role for the Predictive Safety Testing Consortia, which includes representatives from Bristol-Myers Squibb, GlaxoSmithKline, Johnson & Johnson, Merck, Novartis, Pfizer, and Schering-Plough. Members of the consortium are interested in sharing their knowledge and databases related to toxicity and biomarker utilization. These biomarkers will enable us to predict risks before treatments are even applied and also will enable us to detect warning signs, predictors, and indicators of toxicity or adverse events at the earliest stages of medical intervention. Using the modern tools of both the biological and information sciences, we will be able to streamline and improve our approval process and enhance our post-marketing surveillance to protect the health and welfare of the patients we serve.

Despite recent innovations, many serious and life-threatening diseases still lack effective treatments. The path from cutting-edge medical discovery to the delivery of safe and effective treatments is long, arduous, and uncertain. Products fail before they reach the market either because clinical trials do not adequately demonstrate safety or efficacy or because they cannot be manufactured at a consistently high quality. However, even well-designed trials often do not yield extensive information about product performance.

The Predictive Safety Testing Consortia thus represents a major step towards transforming the future of health and healthcare in this country. Industry collaboration under FDA leadership provides an opportunity to move away from redundant testing and wasteful procedures toward new approaches. In this effort, as in many others, FDA has an enormous opportunity and leadership responsibility. In taking these steps, FDA will retain all the rigor, discipline, and precision of regulation, but its efforts will be geared towards accelerating the rate at which new, beneficial innovations can be approved to the benefit of the public.

Our common purpose is to serve the public, who look to FDA not simply for the discovery of new genes, not simply for the development of a particular new drug, but for solutions to their health problems. Solutions in the molecular era must be integrated, but FDA alone cannot coordinate this broad range of activity. FDA’s leadership will come in defining how partnerships should work, evaluating results, and developing guidelines on the use of new approaches that researchers find to be effective. To facilitate completion of projects in a timely manner, the FDA will continue to bring together partnerships and consortia. Coordination in leadership is essential to illuminate the pathway to an era of personalized medicine, when doctors will be able to eliminate – or even prevent – dozens of today’s most devastating diseases.

FDA is committed to creating a world where the right patient will receive the right intervention for the right reason, delivered at the right time, and to the right location to attain the predictable right outcome that we will measure in real time. The no choice about the metamorphosis, which is already underway; however, unlike the caterpillar, we do have a choice about what we will become. Whereas it is easy to envision what we might potentially achieve, it remains up to us to decide to make it happen and bring it about.

Molecular science is already presenting us with an enormous amount of data as well as with the challenge of turning that raw information into useful knowledge. Therefore, it is essential that FDA immediately move to the rapid implementation of effective information technology infrastructures that will facilitate and enable the agency’s work. Science and medicine are intimately interconnected. The importance of sharing of data and information needs to be coordinated and integrated into a much more seamless network and effort. Through integration and collaboration, we can influence the outcome of our change process.

For as long as I have the privilege of leading the FDA, I look forward to collaborating and cooperating with all the other components engaged in this metamorphosis. Collectively, we can fulfill our first and our foremost responsibility: to serve the public, whose lives, welfare, and health are dependent upon us. The challenges facing FDA range across nutrition, drugs, devices, and biologics – for ourselves as well as the animals and pets for which we are responsible. We must be good stewards of the resources that we have at our disposal and innovate in order to meet the demands of this incredibly diverse and complex portfolio. I believe that technology will help us do so, even as it continues to provide great challenges. I look forward to continued dialogue and discussions – not just about our end and our purpose, but rather about how to most rapidly and effectively achieve that end.

The perspectives brought together in this volume show that regulators and industry have already embarked on the transformation of medicine and healthcare. In 2056, when we mark the 150th anniversary of FDA, the regulation of food, drugs, medical devices, cosmetics, nutritional supplements and other consumer goods will rely on different testing and monitoring technologies; will involve deeper collaborations with manufacturers, providers, and consumers; and yet will have retained the same core goal of ensuring individual and public health and wellness.