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Summary of the Report and Recommendations of the Congressionally-Established Diabetes Research Working Group

A Strategic Plan for the 21st Century

Transmittal to the Congress

In accordance with directives from the House and Senate Appropriations Committees, I am pleased to transmit to the Congress the summary of the Strategic Plan for NIH-funded diabetes research developed by the congressionally-established Diabetes Research Working Group.* As specified by the Congress, this Research Plan includes recommendations for future diabetes research directions and corresponding overall budget estimates for implementing the proposed new research initiatives.

The Diabetes Research Working Group is an independent panel composed of twelve scientific experts in diabetes and four representatives from the lay diabetes community. In developing its Strategic Research Plan, the Working Group held plenary meetings and subcommittee discussions, analyzed the existing NIH diabetes research portfolio, sought the expertise of ad hoc scientists, and enlisted public commentary. The Diabetes Research Working Group believes that the culmination of this year-long, in-depth planning process is a set of recommendations that will be of value to the Congress and to the NIH.

Clearly, the congressional action calling for the establishment of the Diabetes Research Working Group and the development of its Research Plan reflects the strong and continuing commitment of the Congress to conquering diabetes. The implementation of the research initiatives recommended in this Strategic Research Plan is the next vital step toward attaining that objective.

C. Ronald Kahn, M.D.
Chairman
Diabetes Research Working Group

*Note: The congressional directives calling for the establishment of the Diabetes Research Working Group and the development of its Strategic Research Plan can be found in Senate Report 105-58 (1998, p. 76, p. 110), House Report 105-205 (1998 , p. 69, p. 98) and House Report 105-635 (1999, p. 69). The charge to the Diabetes Research Working Group called for the development of a comprehensive plan for all NIH-funded diabetes research efforts, including the recommendation of future diabetes research initiatives and directives. Congressional language specifically asked the Working Group to include overall cost estimates to accomplish its recommendations in the final research plan.

This Strategic Research Plan for diabetes research funded by the National Institutes of Health has been developed by an independent, congressionally-established Diabetes Research Working Group, with input from diabetes investigators, diabetes patients, and other members of the broad diabetes research community. The Working Group is composed of scientific and lay experts external to the National Institutes of Health, as well as leaders of major diabetes voluntary organizations. The views, conclusions, and recommendations expressed in this document are solely those of the members of the Diabetes Research Working Group and do not necessarily reflect the positions or judgments of the National Institutes of Health, the Department of Health and Human Services, or the Administration, which must weigh the competing requirements of multiple programs and activities. (NIH Publication No. 99-4398)

Art/Photo Credits
The photographs in this document are not available for republication and may not be reproduced in either print or electronic format without the express written consent and permission of the photographer.
Cover: Robert Reichert (Center), Mark Tuschman (Top right), American Diabetes Association, Honolulu (Lower left), Bristol-Myers Squibb (Lower right)
Page 4: Peter F. Kador, National Eye Institute

The Challenge

Sixteen million people in the United States have diabetes mellitus. In both human and economic terms, it is one of our nation’s most costly diseases. Diabetes is the leading cause of kidney failure, blindness in adults, and amputations. It is a major risk factor for heart disease, stroke, and birth defects, shortens average life expectancy by up to 15 years, and costs the nation in excess of $100 billion annually in health-related expenditures. At present, more than one of every ten health-care dollars and about one of every four Medicare dollars are spent on people with diabetes. Over the next decade these numbers will grow as the number of people afflicted by diabetes continues to increase at an accelerating rate. At present, there is no method to prevent or cure diabetes, and available treatments have only limited success in controlling its devastating consequences. This problem is made more complex by the fact that diabetes mellitus is not a single disease, but occurs in several forms, and has complications that affect virtually every system of the body. The most common forms are Type 1 (insulin-dependent) diabetes, which usually starts in childhood or adolescence, and Type 2 (non-insulin dependent) diabetes, which typically affects adults and increases dramatically with age and obesity.

Congress has clearly recognized the gravity of diabetes through the establishment of a bipartisan Diabetes Caucus and has concluded that the only way to reduce the tremendous burden of this disease is through intensified biomedical research. Over the past three years, Congress has emphasized diabetes research in funding increases provided to the NIH and through other special initiatives. Realizing the critical need to build upon these important steps, the Congress directed the establishment of the Diabetes Research Working Group (DRWG) and charged it with developing a comprehensive plan for diabetes research. This plan is intended to help increase the effectiveness of NIH-funded diabetes research and find solutions to the extremely serious problems posed by this disease. During 1998, the DRWG and its subcommittees held a series of meetings, consulted with a wide range of experts in the field, and heard public commentary. It evaluated all aspects of the diabetes problem in an effort to develop a comprehensive plan for submission to the Congress. This document summarizes the Strategic Research Plan of the Diabetes Research Working Group.

Based on its extensive review and deliberations, the DRWG recognizes both great urgency and unprecedented opportunities in diabetes research. The seriousness of the disease and the widespread problems associated with it demand accelerated and expanded research programs, not only to discover the means to prevent and cure diabetes, but also to develop better and more effective treatment strategies. Meeting these challenges requires a well-thought-out and continuously updated research plan; a cadre of talented researchers and physician-scientists; a supportive infrastructure; and appropriate budgetary resources. The DRWG is convinced that taking action now to increase significantly NIH support of diabetes research will save many thousands of men, women and children from the severe consequences of a dangerous, often disabling and potentially even fatal illness, and will also save the nation many billions of dollars in medical care and lost productivity. From both human and scientific perspectives, now is the time for the United States to move swiftly and decisively to begin to ensure a future for America without diabetes.

The Federal Investment in Diabetes Research

Reducing the tremendous health and human burden of diabetes and its enormous economic toll depends upon identifying the factors responsible for the disease and developing new methods for treatment and prevention. These advances can only occur through increased biomedical research. Although Federal support for diabetes research has produced a number of major advances in the past two decades, many scientific opportunities are not being pursued due to insufficient funding, lack of appropriate mechanisms, and a shortage of trained researchers. Improvements in technology and the general growth in scientific knowledge offer unprecedented opportunities for advances that might lead to better treatments, prevention and possibly cure. Unfortunately, the current funding, level of effort, and scope of diabetes research fall far short of what is needed to capitalize on these opportunities.

The U.S. Government, through the National Institutes of Health (NIH), will spend an estimated $443 million in FY 1999 on diabetes-related research. While this amount has steadily increased since 1981, there is unanimous agreement in the DRWG that this funding level is far short of what is required to make progress on this complex and difficult problem. In fact, the current federal budget for diabetes research represents less than one-half of one percent (0.5 percent) of the annual cost of diabetes to the U.S. economy. When compared with the 5 to 15 percent budgets for research and development in other high-technology sectors, the investment in diabetes research is trivial.

Meeting the challenges posed by diabetes requires investment of additional resources to conduct the needed research and a well-conceived, comprehensive research plan for its effective use. This Plan by the DRWG is the first step in this direction.

The Research Plan

The Diabetes Research Working Group is convinced that a significant investment in research today will greatly speed progress in understanding and conquering this disease and its complications. The Strategic Research Plan set forth has two overarching goals:

  1. Understand the causes and define approaches to prevent the development of Type 1 and Type 2 diabetes and their complications.
  2. Develop methods for optimal management, treatment and ultimate cure of diabetes and its complications.

The DRWG has divided this Research Plan into the following three major components, and provided specific recommendations concerning the types of efforts, budgetary requirements and program mechanisms that should be pursued to realize compelling research goals:

Extraordinary Opportunities

Exciting and rapid research advances in recent years have opened the door to a new understanding of diabetes. The next decade offers important research opportunities that, if seized now, can vastly improve the lives of people with or at risk for diabetes. The Diabetes Research Working Group has identified five areas that offer extraordinary opportunities for making genuine and significant progress toward understanding, more effectively treating, and ultimately preventing and curing diabetes. They are: the genetics of diabetes and its complications; autoimmunity and the beta cell; cell signaling and cell regulation; obesity; and clinical research and clinical trials of critical importance.

Genetics of Diabetes

Because Type 1 and Type 2 diabetes and their complications have strong genetic determinants, defining the specific genes involved is essential to prevention and could lead to new and better therapies. Defining the genes for diabetes and its complications will also help isolate the environmental factors involved in the disease and may identify genetic factors that contribute to variations in response to medications. Thus, a major goal for the coming decade must be to identify these predisposing genes.

Although most of the basic tools for genetic studies are in place and much progress has been made, current approaches are inadequate to tackle the vital genetics questions in a reasonable time frame. Three major impediments are inadequate resources; the lack of an appropriate mechanism to bring together the groups of researchers and patient samples to conduct the necessary studies; and fragmented genetic repositories.

Recommendations:

Autoimmunity and the Beta Cell

Type 1 diabetes is an "autoimmune" disease in which the body’s own defense system mistakenly attacks and destroys insulin-producing beta cells of the pancreas. Important discoveries and concepts have emerged during the past decade from research in basic immunology, cell biology, and autoimmune diseases, including Type 1 diabetes. Based on this solid research foundation, the DRWG believes that aggressive pursuit of three scientific areas over the next decade could lead to dramatic improvements in diabetes therapy and prevention.

Recommendations:

Cell Signaling and Cell Regulation

Intracellular and intercellular communication is the basic mechanism for the regulation of all cells. Disturbances in cell signaling are central to disturbances in insulin secretion and action, which lead to diabetes and to both micro- and macrovascular complications. Basic research in this area is not only essential to understanding diabetes, but is also critical to understanding many diabetes-related complications. Most importantly, this type of "discovery" research can identify important targets for new treatments. It would also complement the important new information about the genetic underpinnings of disease.

Recent progress in research on signaling systems and in the ability to use genetic methods to study these pathways has created an extraordinary opportunity to determine the exact mechanisms of signal communication and its alterations in diabetes. A parallel opportunity exists to identify the molecular events responsible for the insulin resistance characteristic of Type 2 diabetes.

The DRWG has identified five areas of opportunity in cell signaling and regulation that warrant increased research. These are: dissection of insulin and hormone signaling pathways; understanding and countering insulin resistance; defining mechanisms regulating beta cell function; metabolic staging of diabetes; and defining alterations in signaling pathways that lead to development of diabetes complications.

Recommendations:

Obesity–Critical in Diabetes and a Major Problem of Its Own

Obesity is a major risk factor for the development of Type 2 diabetes and insulin resistance, as well as a major cause of morbidity and mortality in the U.S. One of every two Americans is overweight, and the prevalence has increased 30 percent in the past decade alone. Obesity disproportionately affects minorities. Over 60 percent of African American, Mexican American, and Native American women meet the criteria of being overweight and between 33 and 37 percent are obese. Moreover, obesity in children and adolescents is increasing at alarming rates, leading to occurrence of Type 2 diabetes in these groups.

Obesity results from an imbalance between energy intake and energy expenditure. The recent discovery of the fat cell hormone, leptin, and other appetite-regulating hormones has demonstrated that certain types of obesity are not simply due to overeating, but are the result of misregulated pathways that control the balance between appetite and energy expenditure. These new discoveries have provided a revolutionary understanding of obesity at the molecular level, thus leading to extraordinary opportunities in biomedical and behavioral research.

Recommendations:

Clinical Research and Clinical Trials of Critical Importance

Translation of basic research into human therapies depends on an active and vigorous clinical research program. Studies in test tubes, cells and animals can answer questions of fundamental importance, and often provide the basis for development and initial testing of potential interventions. However, it is clinical studies in patients with diabetes that are essential for validating these observations and their relevance to human disease. In addition, clinical studies give key insights into the genetic, immune, hormonal, metabolic and environmental factors involved in the disease, and allow true testing of therapeutic strategies. Several prevailing forces, however, have significantly hampered clinical research and clinical trials in diabetes. Investigator-initiated clinical research is decreasing as a result of decreasing numbers of clinical investigators, limitations on funding of clinical research, the high cost of clinical research, and the complexity of clinical challenges. A major factor hampering clinical trials is the lack of infrastructure to organize and support them.

For diabetes, the long-term nature of the complications adds to the complexity of clinical trials. In most clinical studies, it is difficult to have adequate representation of high-risk minority groups due to the ad hoc nature of the organization of clinical trials. For robust and effective clinical research, additional well-trained clinical investigators and increased funding of meritorious clinical studies are required. Also needed are efficient systems for clinical research to provide the necessary numbers of patients and the stability of operations for long-term studies, and opportunities to include sufficient numbers of appropriate minority groups.

A comprehensive program for tackling a major public health problem such as diabetes requires a major investment, not only in basic research, but also in clinical research and clinical trials. The latter are particularly needed to document the safety and efficacy of various therapeutic strategies and generate the knowledge base for "evidence based medicine" that will lead to better treatment of diseases. There are two major needs to achieve these goals. The first is creation of an infrastructure to facilitate clinical trials—both improving efficiency and lowering cost. This need is especially apparent in diabetes where clinical trials to "hard endpoints" may take many years and even decades. The second need is a commitment to using clinical trials as a mechanism to develop the proper base of knowledge and to assure steady improvement in the care of people with diabetes.

Recommendations:

Special Needs for Special Problems

Micro- and Macrovascular Complications
The different types of diabetes and the array of complications they present offer a wide range of specific research needs unique to each. The micro- and macrovascular complications of diabetes are responsible for most of the morbidity and mortality in both Type 1 and Type 2 diabetes. Their prevention and reversal will greatly reduce the burden of this disease on individuals and on the nation. Understanding and combating the complications of diabetes will require significantly expanded research in mechanisms involved in the development and progression of the complications of diabetes. Several promising research avenues must be pursued through intensified basic and clinical research. This will require an increased effort from the existing community of scientists working in diabetes, as well as important new input from scientists in immunology, genetics, neurology, atherosclerosis, obesity and maternal and child health.

Recommendations on Diabetic Kidney Disease:

Recommendations on Diabetic Eye Disease:

Recommendations on Diabetic Nerve Disease:

Recommendations on Macrovascular Complications:

Methods to Optimize Glucose Control

Despite the findings of clinical trials and other studies that have demonstrated the importance of tight glucose control to minimize the risk of long-term complications, many patients continue to have far less than optimal control. This is due in part to the risk of hypoglycemia, which increases with intensified therapy, and in part to the difficulty of obtaining optimal control in a general clinical setting. The Diabetes Research Working Group believes that identification of methods that promote implementation of these standards of treatment should be a high priority of current clinical research.

Recommendations:

Diabetes and the Environment

The environment appears to play important roles in Type 1 diabetes as a trigger for the autoimmune response and in Type 2 diabetes as a modifier of pre-existing genetic risk. While the latter influence is partly understood, but difficult to control, the former influence has been difficult to define in any specific way. Identification of these environmental factors would provide important information for any preventive strategies for either form of diabetes.

Recommendations:

Diabetes in Women, Children and the Elderly

Diabetes mellitus presents additional problems to women with its impact on reproductive health and vascular complications. Children and the elderly present special problems in management and may have additional physiological variables that must be addressed through specific research.

Recommendations:

Diabetes in Minority Populations

Minority populations, including African Americans, Hispanics, Native Americans, and Asians, have the highest incidence of diabetes and the highest rates of complications of the disease. Current research has only begun to address the reasons for this in a very limited way. These groups are rapidly growing segments of the population and specific research must address the reasons for the disproportionate impact of diabetes they bear.

Recommendations:

Genetic Engineering

The ability to modify the function of cells through genetic engineering opens up tremendous opportunities for new therapeutic approaches to diabetes and its complications. The Diabetes Research Working Group recommends that several applications of this technology be explored.

Recommendations:

Behavioral and Health Services Research

Lifestyle variables, such as dietary intake and physical activity, represent important risk factors for Type 2 diabetes. Type 1 diabetes management can also be influenced by behavioral patterns and can greatly influence personal, family and social dynamics.

Recommendations:

Magnitude of the Problem

The magnitude of the problem created by diabetes is clearly defined by a few simple facts:

The Federal Investment in Diabetes Research

Represents about 3 cents out of each dollar, that is about 3 percent of the NIH research budget. Although there is no accepted method for determining appropriate levels of research funding, this is clearly a small investment for a disease that affects 6 to 7 percent of the population and accounts for more than 10 percent of all health care dollars.

Summary of Budget Recommendations
(in millions of dollars)
Year 1
2000
Year 2
2001
Year 3
2002
Year 4
2004
Year 5
2004
Extraordinary Opportunities
Genetics of Diabetes40.572.085.099.0101.0
Autoimmunity and the Beta Cell30.045.058.066.079.0
Cell Signaling and Cell Regulation38.057.073.086.094.0
Obesity15.025.037.046.052.0
Clinical Research and Clinical Trials87.0139.0191.0252.0280.0
Sub-Total210.5338.0444.0549.0606.0
Special Needs for Special Problems
Microvascular Complications51.080.0106.5124.0129.5
Macrovascular Complications34.058.079.095.0102.0
Optimization of Glucose Control9.516.024.029.036.0
Diabetes and the Environment3.04.06.08.010.0
Special Needs in Women, Children and the Elderly20.040.060.080.080.0
Special Needs in Minority Populations9.015.022.530.032.0
Genetic Engineering8.015.022.028.035.0
Behavioral and Health Services Research8.013.520.027.040.0
Oral Complications of Diabetes1.01.52.02.53.0
Sub-Total143.5243.0342.0423.5467.5
Resource and Infrastructural Needs
Research Training and Manpower Development3.05.08.010.010.0
Diabetes Research Centers Program6.015.025.040.040.0
Technology Taskforce13.017.021.013.013.0
Regional Centers for Animal Models5.010.016.026.026.0
Human Material for Diabetes Research2.02.02.02.02.0
NIH-Pharmaceutical and Biotechnology Interactions0.50.50.50.50.5
Review of Intramural Programs of NIH0.50.50.50.50.5
Taskforce for Strategic Planning0.50.50.50.50.5
Sub-Total 30.550.573.592.592.5
Increment over FY99 Base to Implement
Recommendations in DRWG Strategic Research Plan
384.5631.5859.51065.01166.0
FY99 Base for Diabetes Research442.8442.8442.8442.8442.8
Grand Total for Diabetes Research827.31074.31302.31507.81608.8

More detailed budgetary information presented in terms of the specific scientific recommendations and the corresponding additional funds proposed for individual Institutes and Centers of the National Institutes of Health will be found in the full Strategic Plan of the Diabetes Research Working Group.

Diabetes Research Working Group

C. Ronald Kahn, M.D., Chairman
Director, Joslin Diabetes Center
Mary K. Iacocca Professor of Medicine
Harvard Medical School

Jose Caro, M.D.
Lilly Research Laboratories

Nancy Cox, Ph.D.
University of Chicago

Lee Ducat
National Disease Research Interchange/
Human Biological Data Interchange

Joyce C. Dugan
Eastern Band of Cherokee Indians

Robert N. Frank, M.D.
Wayne State University School of Medicine

  James R. Gavin III, M.D., Ph.D.
Howard Hughes Medical Institute

Willa Ann Hsueh, M.D.
University of California, Los Angeles

Hugh O. McDevitt, M.D.
Stanford University

Douglas Melton, Ph.D.
Harvard University

Christopher B. Newgard, Ph.D.
University of Texas SW Medical Center

Daniel Porte, Jr., M.D.
University of Washington and
Seattle Veterans Affairs Medical Center

  Stephen Smith
American Diabetes Association

Emily Spitzer
Juvenile Diabetes Foundation International

Michael P. Stern, M.D.
University of Texas Health Science Center at San Antonio

Rena Wing, Ph.D.
University of Pittsburgh School of Medicine and
Brown University

Lester B. Salans, M.D.
LBS Associates, New York, served as Senior Medical Advisor.

Ad Hoc Consultants to the Congressionally-Established Diabetes Research Working Group

Kelly J. Acton, M.D., M.P.H., F.A.C.P.
Indian Health Service

Michael A. Brownlee, M.D.
Albert Einstein College of Medicine

Thomas A. Buchanan, M.D.
University of Southern California School of Medicine

Patrick Concannon, Ph.D.
University of Washington

Donald Coustan, M.D.
Brown University School of Medicine

George S. Eisenbarth, M.D., Ph.D.
University of Colorado Health Sciences Center

Edwin Fisher, Ph.D.
Washington University

Michael S. German, M.D.
University of California - San Francisco

Marvin C. Gershengorn, M.D.
Cornell University Medical College

Daryl K. Granner, M.D.
Vanderbilt University School of Medicine

  Thomas C. Hohman, Ph.D.
Wyeth-Ayerst Research

George L. King, M.D.
Harvard University

John Kitzmiller, M.D.
Good Samaritan Hospital

S. Robert Levine, M.D.
Juvenile Diabetes Foundation International

Michael Mauer, M.D.
University of Minnesota Medical School

Boyd E. Metzger, M.D.
Northwestern University

Richard Nesto, M.D.
New England Deaconess Medical Center

Jerrold M. Olefsky, M.D.
University of California, San Diego

Jeffrey E. Pessin, Ph.D.
University of Iowa

  Kenneth S. Polonsky, M.D.
The University of Chicago

E. Albert Reece, M.D.
Temple University School of Medicine

Douglas L. Rothman, M.D., Ph.D.
Yale University School of Medicine

Christopher D. Saudek, M.D.
Johns Hopkins University

Gerald I. Shulman, M.D., Ph.D.
Yale University School of Medicine

Anders A.F. Sima, M.D., Ph.D.
Wayne State University School of Medicine

Lorraine Valdez
Indian Health Service Diabetes Program

Additional copies of this document may be obtained from:

National Diabetes Information Clearinghouse
NDIC–DRWG
1 Information Way
Bethesda, MD 20892-3560
Phone: 301-654-3327
Fax: 301-907-8906

Posted 28 February 1999



                 
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