Opportunities and Challenges at NIDDK in Digestive Diseases Research

Abstract

Diseases of the digestive system are responsible for substantial morbidity, mortality, and direct and indirect health care costs in the United States, affecting a majority of Americans at some point during their lifetime. The United States benefits from the presence of the world’s largest and most diverse research enterprise focused on digestive diseases. Long-standing support of the US Congress for the National Institutes of Health (NIH), which reported expenditures of >$1.2 billion for digestive diseases research in fiscal 2005 (www.nih.gov/news/fundingresearchareas.htm), has yielded important advances in basic, translational, and clinical research at an ever-increasing pace. Among notable advances is the continued decline in the death rate from colon and rectal cancer, the leading cause of GI-related death. Despite many successes, the high burden that digestive diseases impose on the population necessitates a continuing, vigorous long-term research effort to improve the lives of individuals who have or are at risk for digestive diseases. Of the 27 institutes and centers at the NIH, many of which support digestive diseases research relevant to their missions and priorities, the National Institute of Diabetes and Digestive and Kidney Disease (NIDDK) accounted for approximately 30% of the reported expenditures for digestive diseases in fiscal 2005. Although expenditures of digestive disease research increased substantially during the NIH doubling period (from fiscal 1999 to fiscal 2003), expenditures since then have been relatively constant, and have actually declined in inflation-adjusted dollars.The NIDDK supports research in basic aspects of the digestive system and disease-oriented research of the luminal gastrointestinal tract, liver, biliary system, and pancreas and related areas of research in diabetes, metabolic and rare diseases, obesity, and nutrition. The majority of digestive diseases research in the NIDDK is supported by the Division of Digestive Diseases and Nutrition, which includes programs in the previously named areas, including support for epidemiologic research and clinical trials. Specific details about research areas of interest to the NIDDK are included in the research funding area of the Institute’s website (www2.niddk.nih.gov/Funding/). Although these sources of information can provide helpful guidance, investigators interested in applying for research support are strongly encouraged to directly contact extramural program staff to discuss their specific research interests and receive individualized advice (see www2.niddk.nih.gov/Research/ScientificAreas/DigestiveDiseases/).There is considerable debate, particularly in times of budgetary limitations, about the appropriate balance among the many different types of research support, including investigator-initiated research project grants, solicited research (Request for Applications) and Requests for Proposals for new NIDDK-originated initiatives, research resources and infrastructure, clinical trials, research career and research training awards, and support for new investigators. In the NIDDK, the investigator-initiated research project, most typically supported through the R01 grant mechanism, has been the central core of the research portfolio. The investigator-initiated R01 grant and the independent peer review system of evaluating the scientific merit, technical feasibility, and program relevance of applications are widely considered to be the backbone of NIDDK-supported research. Support for the R01 grant mechanism is generally thought to have ensured support for innovative and highly productive research over many decades. In recent years, the number of applications for R01 grants has continued to increase steadily. However, with relatively fixed budgets, success rates (the proportion of grant applications that receive funding) of necessity have declined to <20%. Moreover, the paylines (percentile score below which an institute will usually provide funding) have declined substantially over the last 3 years. Low success rates and paylines are widely regarded as very problematic for established investigators who have long-term commitments to research projects and require continuity of support for highly specialized research staff. In addition, low success rates and paylines are likely to be a significant negative factor in research career choices of young investigators. The NIDDK has taken a variety of steps to attempt to partially ameliorate the effects of budget limitations. In an attempt to maintain as high a level as possible of support for investigator-initiated research, the NIDDK has delayed or sharply diminished commitments to new, institute-solicited research projects other than those of the very highest priority, such as essential clinical trials or critical core resources. In addition, the NIDDK has implemented approaches to ensure that new investigators are given special consideration for funding of their applications. In the NIDDK, a 2-percentile advantage in the payline is provided for new investigators and new investigators are given special consideration for funding with funds set aside for applications that fall over the institute’s payline. The NIDDK has strongly supported research career and training programs (http://grants.nih.gov/training/index.htm) that have been successful in promoting earlier stages of career development, and NIDDK continues to provide the same level of support for these programs. The NIH Loan Repayment Program has been an additional opportunity to encourage and support the research careers of young investigators (www.lrp.nih.gov/about/5lrps.htm).The current budgetary landscape at the NIDDK has accentuated the need for careful planning of future research initiatives. Such planning efforts must identify not only promising opportunities for new research investments, but also potential new sources of funding, potential partnerships with different stakeholders in the research process, and opportunities for increased economy and efficiencies by maximizing use of resources. The National Commission on Digestive Diseases, under the leadership of the NIDDK, is charged with responsibility of developing a plan for NIH research in digestive diseases over the next 10 years (www.niddk.nih.gov/federal/ncdd/). These and many other important planning efforts will help to guide new research programs, but will by no means supplant investigator-initiated research projects that are inspired by new knowledge being generated at an ever-increasing rate. The NIDDK has invested its resources carefully in the past, with budget variability that has ranged from the expansionary period of the 5-year budget doubling to years of relatively steady-state budgets. Undoubtedly, the NIDDK will meet current budget challenges while pursuing its compelling mission to improve the health of the nation and to exploit abundant research opportunities. Diseases of the digestive system are responsible for substantial morbidity, mortality, and direct and indirect health care costs in the United States, affecting a majority of Americans at some point during their lifetime. The United States benefits from the presence of the world’s largest and most diverse research enterprise focused on digestive diseases. Long-standing support of the US Congress for the National Institutes of Health (NIH), which reported expenditures of >$1.2 billion for digestive diseases research in fiscal 2005 (www.nih.gov/news/fundingresearchareas.htm), has yielded important advances in basic, translational, and clinical research at an ever-increasing pace. Among notable advances is the continued decline in the death rate from colon and rectal cancer, the leading cause of GI-related death. Despite many successes, the high burden that digestive diseases impose on the population necessitates a continuing, vigorous long-term research effort to improve the lives of individuals who have or are at risk for digestive diseases. Of the 27 institutes and centers at the NIH, many of which support digestive diseases research relevant to their missions and priorities, the National Institute of Diabetes and Digestive and Kidney Disease (NIDDK) accounted for approximately 30% of the reported expenditures for digestive diseases in fiscal 2005. Although expenditures of digestive disease research increased substantially during the NIH doubling period (from fiscal 1999 to fiscal 2003), expenditures since then have been relatively constant, and have actually declined in inflation-adjusted dollars. The NIDDK supports research in basic aspects of the digestive system and disease-oriented research of the luminal gastrointestinal tract, liver, biliary system, and pancreas and related areas of research in diabetes, metabolic and rare diseases, obesity, and nutrition. The majority of digestive diseases research in the NIDDK is supported by the Division of Digestive Diseases and Nutrition, which includes programs in the previously named areas, including support for epidemiologic research and clinical trials. Specific details about research areas of interest to the NIDDK are included in the research funding area of the Institute’s website (www2.niddk.nih.gov/Funding/). Although these sources of information can provide helpful guidance, investigators interested in applying for research support are strongly encouraged to directly contact extramural program staff to discuss their specific research interests and receive individualized advice (see www2.niddk.nih.gov/Research/ScientificAreas/DigestiveDiseases/). There is considerable debate, particularly in times of budgetary limitations, about the appropriate balance among the many different types of research support, including investigator-initiated research project grants, solicited research (Request for Applications) and Requests for Proposals for new NIDDK-originated initiatives, research resources and infrastructure, clinical trials, research career and research training awards, and support for new investigators. In the NIDDK, the investigator-initiated research project, most typically supported through the R01 grant mechanism, has been the central core of the research portfolio. The investigator-initiated R01 grant and the independent peer review system of evaluating the scientific merit, technical feasibility, and program relevance of applications are widely considered to be the backbone of NIDDK-supported research. Support for the R01 grant mechanism is generally thought to have ensured support for innovative and highly productive research over many decades. In recent years, the number of applications for R01 grants has continued to increase steadily. However, with relatively fixed budgets, success rates (the proportion of grant applications that receive funding) of necessity have declined to <20%. Moreover, the paylines (percentile score below which an institute will usually provide funding) have declined substantially over the last 3 years. Low success rates and paylines are widely regarded as very problematic for established investigators who have long-term commitments to research projects and require continuity of support for highly specialized research staff. In addition, low success rates and paylines are likely to be a significant negative factor in research career choices of young investigators. The NIDDK has taken a variety of steps to attempt to partially ameliorate the effects of budget limitations. In an attempt to maintain as high a level as possible of support for investigator-initiated research, the NIDDK has delayed or sharply diminished commitments to new, institute-solicited research projects other than those of the very highest priority, such as essential clinical trials or critical core resources. In addition, the NIDDK has implemented approaches to ensure that new investigators are given special consideration for funding of their applications. In the NIDDK, a 2-percentile advantage in the payline is provided for new investigators and new investigators are given special consideration for funding with funds set aside for applications that fall over the institute’s payline. The NIDDK has strongly supported research career and training programs (http://grants.nih.gov/training/index.htm) that have been successful in promoting earlier stages of career development, and NIDDK continues to provide the same level of support for these programs. The NIH Loan Repayment Program has been an additional opportunity to encourage and support the research careers of young investigators (www.lrp.nih.gov/about/5lrps.htm). The current budgetary landscape at the NIDDK has accentuated the need for careful planning of future research initiatives. Such planning efforts must identify not only promising opportunities for new research investments, but also potential new sources of funding, potential partnerships with different stakeholders in the research process, and opportunities for increased economy and efficiencies by maximizing use of resources. The National Commission on Digestive Diseases, under the leadership of the NIDDK, is charged with responsibility of developing a plan for NIH research in digestive diseases over the next 10 years (www.niddk.nih.gov/federal/ncdd/). These and many other important planning efforts will help to guide new research programs, but will by no means supplant investigator-initiated research projects that are inspired by new knowledge being generated at an ever-increasing rate. The NIDDK has invested its resources carefully in the past, with budget variability that has ranged from the expansionary period of the 5-year budget doubling to years of relatively steady-state budgets. Undoubtedly, the NIDDK will meet current budget challenges while pursuing its compelling mission to improve the health of the nation and to exploit abundant research opportunities.