The NIH roadmap: timing is everything
Authors: David A. Williams
Journal: Molecular Therapy (2005)

The Director of the National Institutes of Health (NIH), Dr. Elias Zerhouni, initiated planning for the NIH Roadmap in May 2002. The purpose of this effort was to identify major gaps in biomedical research that the NIH could impact so as to facilitate progress in medicine. The NIH invested approximately $128 million in this initiative in 2004, and forecasts cumulative funding of $2.2 billion between 2004 and 2009. During this same period, the total NIH budget is likely to exceed $90 billion. The vision is to focus these resources on three main areas: 1) The identification of new pathways to discovery; 2) the development of the research teams of the future; and 3) the re-engineering of the clinical research enterprise. Researchers focused on the development of molecular therapies could benefit from additional resources in all of these areas, but none more so than in clinical research.The NIH Roadmap (http://www:NIHRoadmap.nih.gov) evolved after a number of discussions aimed at providing a framework for the prioritization of the NIH research portfolio. These meetings included representatives from academia, government, industry and the public, and focused on the identification of ways that the NIH could most effectively impact progress in medical research. A major theme of the process was the identification of initiatives that could not be accomplished by a single institute, and thus warranted an “NIH-wide” approach. Institute Directors chaired working groups that defined timelines, milestones, and mechanisms for coordination.The timing of the NIH Roadmap could easily determine its success or failure. The recently completed doubling of the NIH budget, now totaling approximately $18 billion per year, and the accelerating pace of scientific discovery bring into focus a key question: How will scientific discoveries be used to advance medicine? The answer may lie partly in the past track record, which likely informed Roadmap planning. According to the Clinical Research Roundtable (CRR) of the Institute of Medicine, there is a significant disconnect between discovery research and improvements in health (Sung et al., JAMA, 2003). Myriad reasons are cited, most of which will be familiar to the readership of Molecular Therapy. Not the least of these are excessive regulatory burdens, shortage of both translational researchers and willing participants—made worse by lack of funding for innovative trials, and fragmented infrastructures and databases. An example of regulatory burden can be found in the current gene therapy trial for Fanconi anemia at Cincinnati Children's Hospital Medical Center. A fifteen-page consent form was required to meet the requirements of the local Institutional Review Board and the Food and Drug Administration. Imagine trying to open this trial in more than one place in the United States. Even more challenging, but necessary for rare diseases, would be to launch such complex studies as multi-institutional, international trials.The CRR concluded that significant resources must be deployed to develop mechanisms and infrastructure to accomplish translation and implementation of new knowledge to the patient. As currently envisioned, the NIH Roadmap could address many of these challenges. However, with the current and likely continuing constriction of the NIH budget throughout the planned implementation period of the NIH Roadmap (2004-2009), timing—as the saying goes—is everything. Will the implementation of the important programs outlined in the NIH Roadmap succumb to the falling paylines of R01s? Or will the NIH Roadmap provide the needed leverage through which the American taxpayers’ investment in the NIH—particularly the doubling of the NIH budget over the last five years—pays full dividends? In the end, the purpose of the research funded by the NIH and carried out at our nation's academic health centers is to improve the health of our population. We can only hope this goal remains our focus during the tough times to come. The Director of the National Institutes of Health (NIH), Dr. Elias Zerhouni, initiated planning for the NIH Roadmap in May 2002. The purpose of this effort was to identify major gaps in biomedical research that the NIH could impact so as to facilitate progress in medicine. The NIH invested approximately $128 million in this initiative in 2004, and forecasts cumulative funding of $2.2 billion between 2004 and 2009. During this same period, the total NIH budget is likely to exceed $90 billion. The vision is to focus these resources on three main areas: 1) The identification of new pathways to discovery; 2) the development of the research teams of the future; and 3) the re-engineering of the clinical research enterprise. Researchers focused on the development of molecular therapies could benefit from additional resources in all of these areas, but none more so than in clinical research. The NIH Roadmap (http://www:NIHRoadmap.nih.gov) evolved after a number of discussions aimed at providing a framework for the prioritization of the NIH research portfolio. These meetings included representatives from academia, government, industry and the public, and focused on the identification of ways that the NIH could most effectively impact progress in medical research. A major theme of the process was the identification of initiatives that could not be accomplished by a single institute, and thus warranted an “NIH-wide” approach. Institute Directors chaired working groups that defined timelines, milestones, and mechanisms for coordination. The timing of the NIH Roadmap could easily determine its success or failure. The recently completed doubling of the NIH budget, now totaling approximately $18 billion per year, and the accelerating pace of scientific discovery bring into focus a key question: How will scientific discoveries be used to advance medicine? The answer may lie partly in the past track record, which likely informed Roadmap planning. According to the Clinical Research Roundtable (CRR) of the Institute of Medicine, there is a significant disconnect between discovery research and improvements in health (Sung et al., JAMA, 2003). Myriad reasons are cited, most of which will be familiar to the readership of Molecular Therapy. Not the least of these are excessive regulatory burdens, shortage of both translational researchers and willing participants—made worse by lack of funding for innovative trials, and fragmented infrastructures and databases. An example of regulatory burden can be found in the current gene therapy trial for Fanconi anemia at Cincinnati Children's Hospital Medical Center. A fifteen-page consent form was required to meet the requirements of the local Institutional Review Board and the Food and Drug Administration. Imagine trying to open this trial in more than one place in the United States. Even more challenging, but necessary for rare diseases, would be to launch such complex studies as multi-institutional, international trials. The CRR concluded that significant resources must be deployed to develop mechanisms and infrastructure to accomplish translation and implementation of new knowledge to the patient. As currently envisioned, the NIH Roadmap could address many of these challenges. However, with the current and likely continuing constriction of the NIH budget throughout the planned implementation period of the NIH Roadmap (2004-2009), timing—as the saying goes—is everything. Will the implementation of the important programs outlined in the NIH Roadmap succumb to the falling paylines of R01s? Or will the NIH Roadmap provide the needed leverage through which the American taxpayers’ investment in the NIH—particularly the doubling of the NIH budget over the last five years—pays full dividends? In the end, the purpose of the research funded by the NIH and carried out at our nation's academic health centers is to improve the health of our population. We can only hope this goal remains our focus during the tough times to come.