Strategic Vision for the Future
From Curative to Preemptive Medicine
NIH plays a significant role in the extension of life and the prevention and treatment of many diseases, transforming modern research and medicine in countless ways. For example, not long ago, acute, short-term and lethal conditions such as heart attacks, stroke, acute infections and cancers were the dominant causes of early mortality. Today, life expectancy has markedly increased due to progress made in reducing death from such acute conditions. However, these advances indirectly led to a major rise in the burden of chronic long-term conditions. It is estimated 75% of today’s healthcare expenditures relate to chronic diseases. The emergence and consequences of chronic conditions—like obesity, diabetes, or Alzheimer’s disease—are examples of the challenges we face. Healthcare costs are rising exponentially. We must continue our focus on not only how we best deliver healthcare, but more importantly, what healthcare we deliver.
A New Strategic Vision for Medicine
Given this dramatic shift from acute to chronic disease, the strategies for preventing and treating diseases are beginning to shift. Today, we intervene late, when the patient exhibits symptoms of disease. Our research is changing this approach, so that we may intervene much earlier in the natural cycle of diseases, years before they strike their victims. We must now develop a much more preemptive approach that manages disease over its entire life cycle, from identifying an individual’s susceptibility to a disease, to prevention, early diagnosis, reduction of complications, and smarter therapies.
This shift from a late curative paradigm to an early preemptive one is becoming increasingly possible, thanks to the avalanche of recent discoveries funded by NIH. For example, in 2002, when I became NIH Director, we knew of one important gene abnormality in type 2 diabetes. In the last year alone, researchers uncovered 7 new genes or genetic regions that provide new clues to how this disease may develop. Remarkably, I now receive about one report a week of a significant discovery in the field of genomics. Recent discoveries apply to a broad spectrum of chronic diseases, ranging from mental disorders to autism. We now can see a clear path to what we call "the 4 P’s of Medicine": medicine that will be more Predictive, Personalized, Preemptive, and Participatory.
To reach these key long-term goals, NIH is strategically investing in research to further our understanding of the fundamental causes of diseases at their earliest molecular stages. But individuals respond differently to environmental conditions, according to their genetic endowment and their own behavior. In the future, research will allow us to predict how, when, and in whom a disease will develop. We can envision a time when we will be able to precisely target treatment on a personalized basis to those who need it, avoiding treatment to those who do not. Ultimately, this individualized approach will allow us to preempt disease before it occurs, utilizing the participation of individuals, communities, and healthcare providers in a proactive fashion, as early as possible, and throughout the natural cycle of a disease process.
This prospective management approach to disease is vital to the transformation of medicine of tomorrow. Today’s discoveries are paving the way to make this future a reality. NIH continues its research efforts to search for cures to alleviate the suffering of the millions already affected by disease—and is greatly expanding the scope of research to discover entirely novel ways to stop disease in its tracks before it cripples us. This entails investing in completely new areas of investigation, while sustaining the level of our current efforts and supporting talented scientists using novel methodologies to explore new ideas and concepts that were impossible to envision only a few years ago.
Today’s Scientific Advances Are Tomorrow’s Medicine
Consider how more predictive and personalized treatments could improve the safety and effectiveness of medications. The same medication can help one patient and be ineffective for, or toxic to, another. With the emergence of a field of research called pharmacogenomics, we will increasingly know which patients will likely benefit from treatment and which will not benefit, or worse, be harmed. Cancer chemotherapy and the use of the anticoagulant Coumadin are good examples of how this might be applied.
Research on viruses is improving the lives of Americans and people around the world. NIH supported the early research that led to the discovery and development of antiretroviral therapies for HIV/AIDS. Today, antiretroviral therapies are benefitting millions of Americans as the most effective means of treating HIV infections. These therapies are also helping millions of people in Africa and the Caribbean through the President’s Emergency Plan for AIDS Relief.
Current HIV/AIDS therapies focus on the virus itself. Researchers are trying to understand how the virus enters the human cell and hijacks the cellular machinery, so it can replicate and spread. In a recent experiment, researchers made significant progress toward reaching this goal. Their new approach is based on a process called RNA interference discovered in 1998 and recognized with a Nobel Prize in 2006. Using RNA interference, the researchers suppressed the activity of every single gene in a type of human cell. They discovered more than 276 human proteins that seem essential to the replication of the HIV virus in human cells. This experiment, unthinkable a few years ago, can now be exploited to develop new ways of disabling this deadly virus.
Fundamental research can unexpectedly lead to revolutionary breakthroughs. Scientists at the National Cancer Institute, for example, developed a virus-like particle technology that formed the basis for new commercial vaccines that target specific cancers. In June 2006, the U.S. Food and Drug Administration approved the vaccine Gardasil, which is highly effective in preventing infections from the four types of human papilloma virus (HPV) that cause the majority of cervical cancers in women. Worldwide use of this vaccine could save the lives of 200,000 women each year. This is the first example of a truly preemptive strategy in cancer.
More often than not, it is the sustained combination of multiple approaches—from the most basic science to epidemiological and behavioral research—that makes advances in science effective. One important public health success story is the reduction in tobacco use and related diseases. In the last decade, overall cancer death rates dropped for the first time in a century, driven largely by the dramatic reduction in male smoking from 47% in the 1960s to less than 23% today. This reduction, along with more effective early screening tools like mammography and colonoscopy, is changing the landscape of cancer mortality. These successes reflect the outcome of significant research investments made by many NIH Institutes and Centers (ICs) and our sister agencies over the last 50 years.
FY 2009 Director's Budget Request Statement
U.S. Department of Health and Human Services
National Institutes of Health
Fiscal Year 2009 Budget Request
Witness appearing before the
House Subcommittee on Labor-HHS-Education Appropriations
Elias A. Zerhouni, M.D.
Director, National Institutes of Health
March 5, 2008