Soon after becoming NIH Director in May 2002, Dr. Zerhouni convened a series of meetings to chart a "roadmap" for medical research in the 21st century - a process that identified major opportunities and gaps in biomedical research that no single institute at NIH could tackle alone, but that the agency still needed to address.
"NIH must lead the way in instituting the changes in medical research necessary to improve the health of all Americans," HHS Secretary Tommy G. Thompson said. "It is clear that NIH has a compelling vision for the future of medical research and what will have the most profound impact on research and most importantly the health of all Americans."
"There has been a scientific revolution in the last few years. The opportunities for discoveries have never been greater, but the complexity of biology remains a daunting challenge. With this new strategy for medical research, NIH is uniquely positioned to spark the changes that must be made to transform scientific knowledge into tangible benefits for people," said Dr. Zerhouni.
"We have made remarkable progress in medical research in recent decades, and NIH-led research has changed the landscape of many diseases. However, very real - and very urgent - needs remain," Dr. Zerhouni said. "NIH is now drawing all fields of science together in a concerted effort to meet these challenges head-on."
Developed with input from more than 300 nationally recognized leaders in academia, industry, government and the public, the NIH Roadmap provides a framework of the strategic investments that NIH needs to make to optimize its entire research portfolio. The NIH Roadmap builds on the tremendous progress in medical research achieved, in part, through the recent doubling of the NIH budget. In setting forth an ambitious vision for a more efficient and productive system of medical research, the NIH Roadmap focuses on the most compelling opportunities in three main areas: new pathways to discovery, research teams of the future and re-engineering the clinical research enterprise.
To be part of the NIH Roadmap, scientific initiatives had to be deemed of high potential impact, had to enhance the disease and mission-specific activities of all of NIH's 27 institutes and centers, and had to respond to the needs and concerns of the public. NIH will begin to implement all of the initiatives in fiscal year 2004. Some initiatives that build upon existing research efforts are expected to achieve their goals rapidly, while other newer or more complex endeavors are expected to take several years to come to fruition.
"Through these new initiatives, we hope to remove some of the biggest roadblocks that are keeping research findings from reaching the public as swiftly as possible," Dr. Zerhouni added. "These efforts cover a broad spectrum of points between the lab and the clinic - from basic biological research, such as determining protein structure, to the front lines of clinical research, such as improving the training of the physicians and nurses who run clinical trials."
After an intense process of discussion and scientific review, the directors of NIH's 27 institutes and centers approved an NIH Roadmap strategy that features 28 initiatives to be carried out by nine implementation groups arranged under three main themes.
New Pathways to Discovery
With this theme, New Pathways to Discovery, the NIH Roadmap addresses the need to understand complex biological systems. Future progress in medicine will require quantitative knowledge about the many interconnected networks of molecules that comprise cells and tissues, along with improved insights into how these networks are regulated and interact with each other. Researchers predict that more precise knowledge of the combination of molecular events that lead to health or disease will help to revolutionize the practice of medicine in the 21st century.
New Pathways to Discovery also sets out to build a better "toolbox" for today's biomedical researchers. To fully capitalize on the recent sequencing of the human genome and many new discoveries in molecular and cell biology, the research community needs wide access to technologies, databases and other scientific resources that are more sensitive, more robust and more easily adaptable to researchers' individual needs. Among the resources to be produced are libraries of chemical molecules that can provide probes of biological networks, innovative tools for capturing real-time images of molecular and cellular events, improved computational infrastructure for biomedical research and tiny, nanotechnology devices capable of viewing and interacting with basic life processes.
These initiatives will provide a solid scientific foundation for new strategies for diagnosing, treating and preventing disease. Implementation groups in this area are:
- Molecular Libraries and Molecular Imaging
- Bioinformatics and Computational Biology
- Structural Biology
- Building Blocks and Pathways
"There is no doubt that after the successful completion of the Human Genome Project, our next frontier is to understand all of the myriad elements of cells and organs that are encoded by DNA. Even more importantly, we need to determine how this enormously complex machinery functions in health and disease. The NIH Roadmap process has really helped all of us zero in on key initiatives that will empower biomedical researchers to understand and treat disease," said National Human Genome Research Institute Director Francis S. Collins, M.D., Ph.D., who co-led two of the working groups that developed initiatives in this area.
Research Teams of the Future
The scale and complexity of today's biomedical research problems increasingly demand that scientists move beyond the confines of their own discipline and explore new organizational models for team science. For example, imaging research often requires radiologists, physicists, cell biologists and computer programmers to work together on integrated teams. Many scientists will still continue to pursue individual research projects, but they too will be encouraged to make changes in the way they approach the scientific enterprise.
NIH wants to stimulate new ways of combining skills and disciplines in both the physical and biological sciences. A new funding mechanism, the NIH Director's Innovator Award, will encourage investigators to take on creative, unexplored avenues of research that carry a relatively high potential for failure, but also possess a greater chance for truly groundbreaking discoveries. In addition, novel partnerships, such as those between the public and private sectors, will be encouraged to accelerate the movement of scientific discoveries from the bench to the bedside.
As part of its theme, Research Teams of the Future, the NIH Roadmap seeks to encourage scientists and scientific institutions to test alternative models for conducting research. Implementation groups in this area are:
- High-Risk Research-NIH Director's Innovator Award
- Interdisciplinary Research
- Public-Private Partnerships
"Biological, physical and information sciences have converged at an incredible pace. This demands that we break down barriers among disciplines, as well as among our own institutes and centers. We need to challenge ourselves to find even more innovative and effective ways of doing biomedical research and converting that into cures," said National Institute of Dental and Craniofacial Research Director Lawrence A. Tabak, D.D.S., Ph.D., who co-led one of the NIH Roadmap working groups that developed initiatives in this area.
Re-engineering Clinical Research
Ideally, basic research discoveries are quickly transformed into diagnostics, drugs, treatments or methods for prevention. Such translation lies at the very heart of the NIH's mission. Although biomedical research has succeeded in curing many diseases, and converting many others once considered uniformly lethal into more chronic, treatable conditions, it has become clear to the scientific community that the United States must recast its entire system of clinical research if such efforts are to remain as successful as they have been in the past. Over the years, clinical research has become more difficult to conduct. However, the exciting basic science discoveries currently being made demand that clinical research continue and even expand, while at the same time striving to improve efficiency and better inform basic science efforts. This is undoubtedly the most difficult but most important challenge identified by the NIH Roadmap process.
At the core of this vision is the concept that clinical research needs to develop new partnerships among organized patient communities, community-based physicians and academic researchers. In the past, all research for a clinical trial could be conducted in one academic center; that is unlikely to be true in the future. In these initiatives, NIH will promote the creation of better integrated networks of academic centers that work jointly on clinical trials and that include community-based physicians who care for sufficiently large groups of well-characterized patients. Implementing this vision will require new ways to organize the way clinical research information is recorded, new standards for clinical research protocols, modern information technology, new models of cooperation between NIH and patient advocacy alliances, and new strategies to re-energize the clinical research workforce.
"I am excited by the prospect of NIH exploring ways to truly reshape the national vision for clinical research. The era of the single-purpose isolated clinical trial with no standardization across trials is coming to a close. The development of a common infrastructure for clinical research must be explored aggressively as a way of expanding our knowledge of disease mechanisms and accelerating the therapies of the future," said National Institute of Arthritis and Musculoskeletal and Skin Diseases Director Stephen I. Katz, M.D., Ph.D., who co-led one of the working groups that developed initiatives in this area.
The NIH Roadmap's theme of Re-engineering the Clinical Research Enterprise is intended to address this crucial area by promoting better integration of existing clinical research networks, encouraging the development of technologies to improve the assessment of clinical outcomes, harmonizing regulatory processes and enhancing training for clinical researchers. Another major goal of this initiative is to more fully involve and empower the public in the research process. Implementation groups in this area are:
- Harmonization of Clinical Research Regulatory Requirements
- Integration of Clinical Research Networks
- Enhance Clinical Research Workforce Training
- Clinical Research Informatics: National Electronic Clinical Trials and Research Network (NECTAR)
- Translational Research Core Services
- Regional Translational Research Centers
- Enabling Technologies for Improved Assessment of Clinical Outcomes
Taken together, the components of the NIH Roadmap initiatives are an integral part of a well-thought out national portfolio of research to meet the health demands of the 21st century.
The NIH, which is comprised of 27 institutes and centers, is an agency of the Department of Health and Human Services. The Web site for the NIH Roadmap is located at: http://nihroadmap.