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Remarks as Prepared for Delivery at the BIO Annual ConventionIt's a delight to be here in the Midwest. I'm a Westerner by birth, and always will be by nature. About 200 years ago, Illinois was Western frontier. It was both a developing promise in its own right and a gateway to the future. For, on the banks of the Wood River (located in southwest Illinois), a group of about 30 men were wintering, waiting to begin what would become a journey of astonishing discovery and undaunted courage. The journey of Captains Merriweather Lewis and William Clark would consume two and a half years and cross thousands of miles of unexplored wilderness. The captains did not know the way. None did. But they had a clear destination in mind, and a clear purpose in heart. The object of the journey, as President Thomas Jefferson wrote Captain Lewis, was "to explore the Missouri River ... by its course and communication with the waters of the Pacific Ocean." The footsteps of the Corps of Discovery opened a frontier that would not be closed for almost a century, one that continues to benefit countless people today. Progress always involves pioneering and pioneering starts with a mental picture of the destination. Today, I want to talk about my mental picture of a new frontier in medicine. The next ten years will be seen as a signal point of transition in healthcare. Medicine will be transformed from an instinctive art of alleviating symptoms to a science of personalized health care. The next decade will be viewed by future generations as the time when treatments became preventive, predictive, and personalized. This transformation will touch every aspect of care—from sensors that allow doctors to detect the first sign of a potentially fatal form of cancer to information networks that allow scientists to scan hundreds of thousands of records for the scent of a cure to a debilitating disease. A decade from now, we will have a health care delivery system in which doctors, pharmacists and other health care providers customize treatment and management plans for individual patients based on vast amounts of information that is readily accessible at clinics and hospital bedsides—information like medical history, genetic variability, and even patient preferences. I see a health care system in which doctors and their colleagues are able to predict treatment outcomes with increasing certainty, and in which they have more time to talk with their patients about their choices of treatment, and the implications that will follow. We are entering the era of patient-centered care:
Because we are gathering today at a BIO meeting, let me describe the next decade from the perspective of a patient, an entrepreneur, and a pharmacist. Let's take the patient first. She is a young woman who is not sick, but has reason to be concerned about her health. Her mother and her grandmother suffered unexpected strokes at young ages. In 2006, much of her care would focus on prevention. Clinicians would probably tell her to keep a close eye on her blood pressure, to watch her diet, and to take up some sort of exercise. If a crisis comes, she will be given medicines that limit the damage and help repair wounded tissues. But the true roots of her family's disease might never be uncovered. Things will be different in the future. She will have more information, more choices, and more control. For example, she may decide to register to be part of a national genetic profile registry that stores medical histories, genetic information, and family history data. The database would knit family medical histories together for many generations on both sides of the young woman's family. By running her genetic data against the entire database, the young woman's doctor will be able to rule out hundreds of different disorders that could have caused the death of her family members, and find the true cause of the disease—an abnormal protein that causes clots to form in the heart and brain. Imagine her sitting at her desk one afternoon and receiving an email from her doctor explaining that a new drug is now available for experimental testing against that abnormal protein. Imagine her interest upon learning that she is a great candidate for the study. "Will it take much time?" she asks. Her doctor explains that the test is done by implanting a sensor under her skin, which transmits testing information using a wireless signal to her desktop computer, which in turn updates her medical record and the clinician doing the research. That's a new era in patient-centered care. In today's world, that would likely not happen—too few patients have the protein to attract interest in that type of research. Financing a drug from discovery to market in 2006 requires a billion dollar investment and can take a dozen years. Today, few have the financial capacity to survive the clinical trials. So many drugs that can produce miracles are never pursued, or get cast aside when side effects are suffered by a few. Simple economics has forced a dwindling number of giant drug companies to focus on a handful of blockbuster drugs that have broad market segments while millions of people continued to suffer because it was not financially feasible to pursue a remedy to a rare disease. Our current system is remarkably inexact and frustratingly slow. We need better tools and the bio-entrepreneurs of the future will have them. Let's talk about the bio-entrepreneur of the future by imagining an entrepreneur named Thom who is the CEO of a small biotech firm. At some point, researchers will have access to information collected by a new segment of the biotech industry: health data firms. Through such an organization, our friend Thom has access to real-time health data and detailed histories from more than 35 million people all across the U.S., Canada, and Mexico. The data would be fully privacy-protected. That information allows researchers at Thom's lab to analyze data from 120,000 patients in their quest for a possible treatment for Alzheimer's. The researchers find that, of a large number of people who have developed an antibody to a common cold virus, a few have experienced an unusual reaction that has led to initiation of the Alzheimer's disease process. In a matter of few days, researchers at Thom's firm survey the medical histories and blood samples of the entire data set. The data shows that more than two-thirds of the known Alzheimer's disease population has a biomarker for the unusual reaction, and only a small percent of the unaffected population do. Thom knows it's a significant discovery, but he needs to find additional financing to continue its pursuit. In today's world, financing a project like this would be a serious problem for a small company. But, in the future, new financing mechanisms that allow participating drug discovery companies to sell shares in their pre-competitive compounds could be established. Once financed, "the race is on..." Thom and his colleagues download more than 3,000 computer-designed compounds for testing that could block the action of the antibody in question. Then, as now, the trick will still be finding the right key for the right lock. But testing will be much faster and much more economical. Thom's team winnows the 3,000 compounds to 20 possibilities that can then be used in a computer-assisted clinical trial. Thom then goes to see the FDA. Thom remembers his first trip to the FDA. Compounds had to go through a long series of slow empirical trials, which rarely gave the exact information about desired efficacy. The costs of testing meant that many compounds went undeveloped. But, in the future, the painstaking plodding of trial and error that characterized clinical trials will have been transformed into a six-month sprint from the breakthrough discovery of a compound to clinical testing. That transformation has happened because researchers have much more information and many more sophisticated tools at their fingertips, such as:
Researchers also have access to real-time feedback about adverse events, thanks to online patient reporting. Safety testing of Thom's compound proves successful in a matter of months, not years. I said I would describe the future through three sets of eyes—a patient, entrepreneur, and a pharmacist. I am fully aware of the impact on physicians, but in the interest of time, I will skip right to the pharmacist. Today, significant periods can pass in between the introduction of new medicines. Medications often offer little opportunity to tailor their application. Many medicines treat symptoms, but fail to touch the roots of disorders. In the future, I can see new medications coming to the market on a far more frequent basis. Many of them will target specific disease processes or will be aimed at preempting diseases that have been detected by genetic tests. Drug delivery systems will change, too. In addition to the pills they provide today, pharmacists of the future might also offer tiny encapsulated particles, which have the ability to deliver up to ten different medicines at the same time. Pharmacists may also be able to provide their patients with devices that allow them to overcome losses of vision and hearing. For instance, implanted computer chips might fill in blind spots created by macular degeneration. Pharmacists may provide computer support for those devices, too. The pharmacists of the future will be partners with their patients in managing medications and making informed choices about their health. One of the most pressing questions for the transition to personalized medicine is privacy. People properly worry that confidential personal information will be misused. The reality is, if they are not fully confident of the security of their data, people will not participate. It is as simple as that. It will not be the technology or the science that limits progress. It will be sociology. These are profound changes. The era of patient-centered medicine with its preventative, personalized, and preemptive medicine, will unleash forces of change within the biological and pharmaceutical industry that will resemble what the Internet did to telecoms. Things will happen fast, and change is disruptive. It is disruptive to patients, providers, payers, and processes. Our regulatory structure is not yet prepared to deal with it. We have to reinvent the regulatory process so we are enabling, not inhibiting, progress and we will. We have to. Biomedical research funding has increased from $37.1 billion in 1994 to $94.3 billion in 2003, a doubling in terms of inflation, but costs have also increased, and approvals have fallen. That must be reversed, and we have constructed an agenda to get there. The FDA's Critical Path Initiative is an important step. The goal is to find better ways to rapidly develop and approve safer treatments, at lower cost, and with a greater degree of success. Last month, we laid out a list of more than 70 potential opportunities—science projects—aimed at creating new and better tools to modernize the drug development process by 2010. Critical Path is just one part of an aggressive agenda to bring about this new era of patient centered medicine. Another part of the agenda is the NIH Roadmap project. It identifies major opportunities and gaps in biomedical research that no single institute can tackle. Roadmaps were developed with the input of more than 300 nationally recognized leaders. It lays out a vision for more efficient and productive systems of medical research in new pathways to discovery, research teams of the future, and re-engineering the clinical research enterprise. Another important centerpiece to the puzzle is the trans-institute project at NIH, the Genes and Environment Initiative. It represents an aggressive crusade to pinpoint the actual causes of cancers, and to open new ways to treat them. The American Health Information Community is a third critical piece. Health IT is at the heart of all this. The Community is building the health information technology infrastructure of the future. All three of these efforts have one thing in common: each is a large-scale collaboration utilizing a networked approach to involve the medical and scientific community. I am committed to use the resources of the Department of Health and Human Services to serve as a rallying point toward the common goal of healthcare that is personalized and patient-centered. Over the next one thousand days, this will be my focus. I am organizing a team in the Office of the Secretary. Every day we're going to wake up thinking about patient-centered healthcare. The glimmerings of the possibilities are already there. The cost of scanning genomes continues to fall. This past June, the Food and Drug Administration approved a drug—Bidil—that is more effective at treating heart failure in African Americans than Caucasians, giving suffering individuals another treatment choice. And earlier this week, researchers supported by the NIH announced that they created a new scan that can detect oral tumors with a beam of fluorescent light, giving a glimpse of the promise of molecular medicine. Today, as Lewis and Clark did over 200 years ago, we again stand at the edge of a new frontier. The distance again seems daunting. Some of the obstacles seem insurmountable. And challenges await that will test our resources and try our resolve. But again we stand, with a clear direction in mind, and a clear purpose in heart. I am persuaded that we have the will to find the way, the power to discover the path to the golden shores of patient-centered medicine. And we'll experience the excitement and the joy that William Clark did as the destination of the Corps of Discovery finally grew near, exclaiming, "Ocean in view! O! the joy." Thank you. Last revised: April 14, 2006 |
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