Diabetes Research Working Group
Meeting of the Congressionally-Directed Diabetes Research Working Group (DRWG)
Natcher Building
National Institutes of Health (NIH) Campus
Bethesda, Maryland
July 9, 1998
Report by
Alastair T. Gordon
Table of Contents
This report is not intended to be a comprehensive summary of all the material covered at the Diabetes Research Working Group (DRWG), July 9, 1998. Because our primary interest is in research leading to the cure and prevention of type 1 diabetes, sections relevant to that particular endeavor are emphasized, and some conclusions are drawn. Not covered in any significant detail are sections dealing with type 2 diabetes, microvascular complications, macrovascular complications, diabetes and pregnancy, and disease management. In addition, many of the observations and conclusions are those of the author, and do not necessarily have the endorsement of others within the DRWG
The report was compiled from notes and recollections of the authors and discussions with other participants. However, there are likely errors and omissions that readers will observe. Please feel free to send an email detailing any such deficiencies, as the report can be easily amended.
The concept for the Diabetes Research Working Group (DRWG) was first introduced as a Bill (H.R. 1315) to the U.S. House of Representatives in April, 1997 by Representatives George Nethercutt (WA) and Elizabeth Furse (OR), both parents of children with type I diabetes. The essentials of H.R. 1315 were condensed and rolled into the Health & Human Services Appropriations Bill for 1998, which was signed into legislation by Congress and President Clinton in the Fall of 1997. The goal of the Working Group is “to develop a comprehensive plan for all NIH-funded diabetes research efforts”, as well as recommending “future diabetes research initiatives and directions.” This plan will be submitted to Congress by late August, 1998.
The Bill calls for the NIH Director to appoint a non-NIH member of the Working Group as its chairman (Dr. Ronald Kahn of the Joslin Diabetes Center). Members of the Working Group include high-level representatives from the NIH Institutes that have substantial diabetes research portfolios; leading diabetes researchers (not employees of the NIH); representatives from industry; and leaders of organizations that represent people with diabetes. It is unclear how and by whom the non-NIH members of the group were selected. The Director of the NIDDK (Dr. Phillip Gorden) and the Diabetes Mellitus Interagency Coordinating Committee will work with the DRWG in the development and implementation of the diabetes research plan.
On January 28, 1998, the first meeting of the DRWG was held. The three major agenda items included inventorying current assets and projects within NIH; reviewing the findings of the September workshop (Diabetes Mellitus: Challenges and Opportunities); and deciding how to develop the research plan for Congress. With selection of subgroup assignments by Working Group members, the framework was set for progression into the events of the April 29th meeting. Robin Harrison and I had an opportunity to be part part of the DRWG meeting on April 29, 1998 and to generate a report on the April 29 session highlights as they related to type 1 diabetes.
On July 9, 1998, I had an opportunity to attend another DRWG meeting,
and to speak to the gathering of NIH scientists, non-NIH scientists, JDF,
ADA, concerned citizens, and others.
Our Representatives at DRWG
As you may recall, our report from the April 29 DRWG meeting commented that:
Conspicuous by its absence was direct representation of people with diabetes, people who have no agenda other than eradication and prevention of the disease, and who have no vested interest in any research initiative, other than the ones most likely to cure and prevent diabetes.The need for direct involvement in the research funding process by people actually affected by disease was a major theme of the Institute of Medicine report entitled Improving Priority Setting and Public Input at the National Institutes of Health (report summarized in The Washington Post (July 9, 1998) and IOM press release). I am happy to report that at this session of the DRWG there was representation from people whose families have been affected by diabetes, and several of them had an opportunity to speak (presentation summaries below).
Representatives from the Juvenile Diabetes Foundation (JDF) were again out in force, including Dr. S. Robert Levine (Director of Government Relations), Dr. Robert Goldstein (VP Research), Emily Spitzer (former Chair of Research), Bill Schmidt (Director of Public Affairs), Larry Soler (Senior Legislative Counsel), and Jane Adams (Associate Director of Public Affairs). Most of the JDF participants either have diabetes themselves or in their families, and so have a strong vested interest in assuring that the best research receives NIH support and that more funding dollars are provided. JDF has had a long history with the DRWG, actually originating the idea of a Working Group and working closely with Congressman Nethercutt's office in the development of legislation.
The American Diabetes Association (ADA) were also present, including Stephen Smith (Chairman, Government Relations Committee), Gerry Lynam (Manager of Government Relations), and Richard Kahn, Ph.D. (Chief Scientific and Medical Officer).
Deb Butterfield, founder and director of the Insulin-Free World attended, and had an opportunity to speak to the group (see below).
And of course, yours truly from The Islet Foundation was there
to wave the flag.
The DRWG Meeting
The meeting was held on the National Institutes of Health (NIH) campus, in the Natcher Building in Bethesda, Maryland. The meeting was chaired by Dr. Ronald Kahn, Director of Research at the Joslin Diabetes Center, Harvard Medical School. Dr. Kahn was the driving force, and did a commendable job of maintaining the momentum and focus throughout the day. The DRWG is made up of NIH Members, NIH Support Staff, Non-NIH Members, and Ad Hoc Consultants.
Following is the agenda for the Meeting of the Congressionally-Directed
Diabetes
Research Working Group (DRWG) held in the Natcher Building at the National
Institutes of Health (NIH) Campus, Bethesda, Maryland on
July 9, 1998:
| 08:00-08:45 | Opening Remarks and Plans for the Day
General Discussion |
Dr. C Ronald Kahn |
| 08:45-09:15 | Review of JDF Research Portfolio | Dr. Robert Goldstein |
| 09:15-09:45 | Review of ADA Research Portfolio | Dr. Richard Kahn |
| 09:45-10:00 | Break | |
| 10:00-12:00 | Public Presentations: | Alastair Gordon, The Islet Foundation
Dr. Norma Kenyon, University of Miami Deborah Butterfield, Insulin-Free World Foundation Don Smith, Islet Transplant Recipient Celia Barash, Mother of Son with Diabetes Charles Rizzo, Father of Daughter with Diabetes John B. Engle, The Whittier Institute for Diabetes Dr. Alberto Hayek, University of California at San Diego Dr. Robert Genco, American Academy of Periodontology |
| 12:00-13:00 | Lunch | |
| 13:00-17:00 | Presentation and Discussion of Subgroup Workgroups | Type 1 Diabetes Subgroup, Hugh O. McDevitt, MD
Type 2 Diabetes Subgroup, Nancy Cox, PhD Microvascular Complications Subgroup, Robert Frank, MD Macrovascular Complications Subgroup, Peter Savage, MD Women, Children, and the DCCT, Boyd Metzger, MD Research Resources Subgroup, C. Ronald Kahn, MD Training Subgroup, Ronald Margolis, MD Industry/Academia Subgroup, Lester B. Salans, MD |
| 17:00-18:00 | Wrap-Up
Future Meetings and Plans |
Dr. C. Ronald Kahn |
As each of the 8 subgroups presented its draft for the final report to Congress, Dr. Kahn was consistent in encouraging each presenter to be bold, and to bring forward the research initiatives that could make a real difference to the health of people with diabetes. He encouraged each subgroup not to simply create a shopping list of interesting research, but to articulate ideas that could have a real impact on the devastation of diabetes, and to do so without regard to cost. Although Dr. Kahn made this request on many occasions, the reports of each group tended to feature incremental research challenges, rather than bold and captivating ideas and commitments. Researchers still do not appear comfortable signing on for missions in which there is a real risk of demonstrable failure, even though there is also the potential of glorious reward. I expect that between these interim reports and the final submission, some bold ideas will coalesce, ideas that will captivate Congress and give members a clear handle on what is possible if the right research is adequately supported.
Review of JDF Research Portfolio
The first presentation was a review of the Juvenile Diabetes Foundation research portfolio, presented by Dr. Robert Goldstein, VP of Research. I personally was very pleased with the research portfolio that JDF intends to support and pursue. The change in focus that has occurred over the past year is commendable, and a cause for renewed hope. In addition, JDF clearly demonstrated its efficiency in the high percentage of donors' funds that are directed to research activities, and the minimal overhead burden.
JDF's research portfolio was divided into three main areas: Prevention of Diabetes, Restoration of Normal Blood Glucose Levels, and Complications of Diabetes. Looking at each area in greater detail:
1- Prevention of Diabetes and its Recurrence:
The ultimate answer to the scourge of diabetes is to assure that it never occurs. Hence, prevention is an important area of research with the ultimate hope of developing a vaccine. This research may also have relevance to curing diabetes, as there is concern that the original autoimmune process may kill transplanted islets (although there is some evidence that autoimmune diabetes does not recognize porcine islets transplanted into mice). Hence a preventive technology may be part of an islet transplant to defeat a repeat autoimmune attack.
Within the field of prevention, JDF is emphasizing:
For most people who already have diabetes, the restoration of normal blood glucose levels would represent a "cure", although some argue otherwise. Within the field of restoring normoglycemia, JDF is emphasizing:
3 - Complications
Although the real goal of everybody is the prevention and cure of diabetes, there are no guarantees in medical research. Hence, we must continuously improve the management and treatment of diabetic complications. There are many people with diabetes who believe that restoration of normal blood sugar may not be far off, but who must have the absolute best treatment of complications until that day arrives. Within the area of complications, JDF is emphasizing:
Dr. Goldstein described some major new initiatives by JDF intended to create funding programs that are more likely to result in useful clinical advances. In addition to providing support for its ongoing research and training grants, JDF has set aside at least $20 million for research targeted to the following special emphasis areas:
JDF Research Funding for 1998
Speaking personally, I felt the JDF research budget for 1998 represents a realistic balance, with a strong emphasis on delivering measurable benefit to people with diabetes within a reasonable time frame. If the NIH, with its massive resources in people, infrastructure, and dollars, could parallel such a research portfolio, perhaps the devastation of diabetes could soon become part of history.
I. PRIMARY AND SECONDARY PREVENTION
| · Etiology
· Genetics · Immunology/Autoimmunity/Immune Mechanisms · Epidemiology · Tolerance Subtotal:
|
733,000
3,097,000
6,524,000
468,000
4,239,000
15,061,000
|
| · Beta Cell Growth, Preservation, Replication
· Pancreas and Islet Cell Studies · Metabolic Dysregulation · Transplantation · Gene Therapy, Gene Transfer, Engineering Beta Cells/Tissue · Glucose Sensing/Devices Subtotal:
|
4,336,000
1,546,000
4,265,000
8,730,000
1,685,000
830,000
21,392,000
|
| · Genetic Susceptibility
· Cell, Vascular & Endothelial Damage · Hypoglycemia · Retinopathy · Nephropathy · Neuropathy · Atherosclerosis Subtotal:
|
331,000
3,568,000
831,000
537,000
1,987,000
362,000
1,623,000
9,239,000
|
| · Other (Embryopathy, etc.) |
431,000
|
|
Total:
|
$ 46,123,000
|
As a fund raising organization, JDF deserves credit for its efficiency. During the fiscal year ending June 30, 1997, JDF’s total revenue was $75,083,582. JDF committed $31,491,974 (59%) to its research program; $11,504,082 (21%) for public diabetes education; $2,488,582 (5%) for management and general purposes, and $8,280,119 (15%) for fund raising. Since FY1997, we can expect to see an even greater percentage of donor contributions going to research, and the selection of research to be more strongly focused on usable results rather than interesting investigation.
Review of ADA Research Portfolio
Dr. Richard Kahn, Ph.D. (Chief Scientific and Medical Officer) of the American Diabetes Association presented the research portfolio of his organization. After the encouraging presentation from Dr. Goldstein of the JDF, I was disappointed by what I heard of ADA research funding. A donor to ADA who expected a significant percentage of his or her donation to be invested in research would be not be pleased by this message.
Looking at the efficiency of ADA in funding research in FY1997:
Dr Kahn stated the major areas in which ADA is encouraging research grant applications (It is important to remember that ADA is focused heavily on type 2 diabetes, a disease in which behavioral factors are much more significant in determining outcomes than is the case for type 1 diabetes):
I had the dubious honor of being the first to speak, and with the limited time available, decided to be as blunt and direct as possible. I felt that others would be taking a much more diplomatic approach in addressing the NIH, and that somebody needed to be more "on the fringe". I have found over the years that a direct voice, especially if presented clearly and rationally, will rarely ever be acknowledged by its intended target, but will have an effect in moving that target in the desired direction. Both the diplomatic and direct voices are needed to achieve our goal.
An interesting note is that my speech basically asked why all communiqués from NIH barely even mentioned islet transplantation without immunosuppression, possibly the best hope for restoring normoglycemia to people with diabetes. Of the nine presenters -- including scientists, advocates, and people with diabetes in their lives -- eight highlighted islet transplantation as the most promising and deserving research initiative. It is good to know that we are not the only ones who are calling for such a focused effort.
The text of my speech is below:
First my thanks to the NIH and the DRWG for this opportunity. In the limited time available, I feel it is necessary to be blunt about the direction of NIDDK in its selection of funding priorities for diabetes research. Statements from NIH and NIDDK over the past year have met with almost universal disappointment from people whose lives have actually been touched by diabetes. On several occasions, we had hoped that our voices were being heard, but recent communiqués from NIDDK clearly indicate that the diabetic consumer is the justification for NIDDK's budget and existence, but not its beneficiary.
In announcing the National Diabetes Education Program (a massively funded joint effort by NIH and CDC), Dr. Phillip Gorden, Director of the NIDDK, said, "People with diabetes need to step forward and take control of their diabetes.'' Only someone whose life has never been touched by juvenile diabetes could make such a statement. How can any rational person expect a child to behave like a pancreas 24 hours a day? There is a profound lack of scientific rigor in failing to distinguish between type one and type two diabetes in such a context, and in failing to acknowledge the risk of brain damage and death from hypoglycemia, inevitable adverse effects of tight gylcemic control. The lives of children and adults with type one diabetes will suffer as NIDDK spreads this false and cruel gospel.
NIDDK's original strategic plan for diabetes research, entitled "Diabetes Mellitus: Challenges and Opportunities - Final Report and Recommendations", fails to include the word "cure" anywhere in the entire report. Instead, it brims with lofty initiatives such as
Not once does it mention the only technology to date that has shown any efficacy, however limited, in restoring normoglycemia -- namely, islet transplantation without immunosuppression. What is the reason for this tragic omission?"Develop and evaluate strategies that address social and cultural barriers to adherence"; "Study interventions to decrease psychiatric and social comorbidities in individuals with disease"; and "Mechanical approaches to metabolic control".
While we all hoped that NIDDK was hearing our voices, last month Dr. Gorden sent a form letter to a concerned mother of a diabetic child, outlining the direction of research within his institute. He presented the major initiatives at NIDDK as:
NIH has a history of favoring massive behavioral trials, starting with the DCCT, a $150-million, 10-year undertaking that proved the obvious. These outcomes could have been determined simply by analyzing existing medical records and correlating quality of control (through glycated hemoglobin tests) with frequency of complications. A massive and expensive prospective trial was not justified, and is held in contempt by many knowledgeable diabetics. And now we have the National Diabetes Education Program, the “Son-of-DCCT”, whose faulty premise and sloppy targeting will create a public perception that type one diabetics are largely to blame for their own misfortune.
NIH prides itself on funding researcher-initiated investigation, with only minimal direction coming via its RFA program. This model works when we seek to expand our general understanding of basic science, but it would never have worked in the focused context of a Manhattan Project or Space Program. Perhaps the government needs to create a Manhattan Project or Moon Shot whose 5-year goal is the restoration of normoglycemia to people with diabetes. Every research initiative within the program would be measured against this one clear goal. Maybe the war on disease now calls for a highly directed, military style of research funding. For people with diabetes and for taxpayers supporting this $100-billion per year burden, the old model is not working. Perhaps we need to consider something new.
There have been forums whose alleged purpose was consumer input into the funding decisions of NIDDK. It now appears that NIDDK has charted its course and these superficially democratic forums have changed nothing.
There is a strong perception that NIDDK is not listening -- not listening to individual people whose families have been devastated by diabetes, not listening to the Juvenile Diabetes Foundation, not listening to the Diabetes Research Working Group, not listening to the Institute of Medicine, not listening to Congress. Who is NIDDK listening to?
Presentation by
Norma Kenyon, PhD, University
of Miami
Dr. Norma Kenyon of the University of Miami is not only an accomplished researcher, but also has a daughter with diabetes. Her presentation focused on the potential of islet transplantation as a treatment for diabetes. Dr. Kenyon stated very clearly that we may now have the tools to cure diabetes in the near future, but funding for pre-clinical and clinical trials is essential. This funding should come from the NIH.
Dr. Kenyon stated that islet cell transplantation does work, and has been shown to:
Deb Butterfield has had diabetes for 24 years, and is now insulin-free as a result of a pancreas/kidney transplant. Deb's message to the NIH was to consider the impact of diabetes on individual people, not just the statistics of its broad effect on society at large. Therefore, each research program should be considered for its potential to improve the lives of individuals with diabetes, and should be judged by how soon its positive impact will be felt.
Specifically, Deb called for more funding of clinical trials, without
which there can be no translation of NIH research into clinical products.
Deb emphasized that there should be representatives on any NIH review committee
with experience in islet transplantation, stressing that there are capable
people who understand both diabetes and islet transplantation.
Presentation by
Celia Barash, Mother
of Jason who has Diabetes
Celia Barash spoke eloquently of her personal experience as Jason's mother. Jason is 13-1/2 and has had diabetes since he was 4 years old. Celia has worked with both ADA and JDF over the years, and has been very active in the diabetes community.
Celia said that the time for islet transplantation is now! She expressed her hope that NIH would get behind this promising procedure and support clinical trials. Celia's father was a medical researcher, and so she has an excellent understanding of what can realistically be expected.
Celia said that JDF should educate people as to the promise of islet transplantation in all its forms, and she is confident that families with diabetes would be eager to donate to such a promising cause. She expressed her frustration over the funds invested in non-invasive glucose sensors, mechanical pancreas, and nasal insulin. In the 9-1/2 years that Jason has had diabetes, she has seen nothing new for helping people with diabetes. Celia was also concerned about the failure to distinguish between type 1 and type 2 diabetes, and the resultant tendency to equate complications with personal failure.
We saw a videotape of Jason telling us first-hand what it's like to
be a kid with diabetes, and asking how much different his life and his
future would be if diabetes were cured.
Presentation by
Charles Rizzo, Parent
of Daughter with Diabetes
Charles and Marie Rizzo came to the DRWG meeting to tell of their decades of involvement with diabetes research. Their daughter is now 28 years old, and has developed retinopathy. Charles eloquently expressed the terror of parents who worry that the slippery slope of complications may be starting.
Charles spoke of his involvement as a board member of the Juvenile Diabetes Foundation, the Diabetes Research Institute, and the Rogossin Institute. The Rizzos met Dr. Paul Lacy in 1982 when he was starting his work on islet transplantation. For many years, the Rizzos prodded JDF and others to support clinical trials of islet transplantation, as the research had reached the stage where it needed to be tested in people.
Charles went on to say that NIH has done almost nothing to support islet transplantation, and must now recognize its potential to cure diabetes. Smaller research groups have had some early successes that must be advanced, including some collaborative effort between DRI and Rogossin. Rogossin have had some promising results with diabetic dogs and monkeys treated with macroencapsulated pig islets.
Charles described how these researchers were unable to raise any funding to advance their early results, while much less promising work was being supported. One of these research groups has raised funding itself, and is now in process with the FDA for clinical trials of islet xenotransplantation. NIH's unwillingness to support practical, applied research has delayed this potentially valuable work.
Charles had specific suggestions for NIH. He said that NIH must recognize the success of labs around the world, and let it be known that they will support clinical trials. He said that if the NIH is paralyzed by its own bureaucracy, then it should outsource the management of these trials to qualified third parties. NIH must no longer be an agent of delay in bringing this most promising research to real people.
Charles commented on the peer review process, calling it "incestuous" and "unimaginative". He said that unless the fresh views of islet transplant scientists are heard on the review committees, radical new ideas will always be passed over in favor of more incremental and familiar proposals, and the current stagnation will continue.
On the subject of scientific publishing, Charles said that actual published information is stale and sanitized. The real magic may lie in lab notes or in a researcher's memory. He asked if there was a way that NIH could be a conduit to assure that all this valuable information is freely exchanged among researchers by channels that are more productive and immediate than formal publishing.
Charles ended his talk by saying, "Islet transplantation is the only
hope for our daughter. NIH is the 800-pound gorilla with the power to cure
this disease, but the current conformity, fear, and lack of persistence
will never yield the cure."
Presentation by
John B. Engle, The Whittier
Institute for Diabetes
John Engle is the President and CEO of the Whittier Institute for Diabetes in La Jolla, California. He emphasized how the DRWG report can have a positive effect on the ability of the Whittier Institute to raise funds for diabetes research.
He made the point that people may be willing to donate $100,000 to Whittier
Institute for islet transplantation, but will ask, "Will my donation make
a difference?" The truth is that it will only make a difference if
NIH will support clinical trials of the fruits of that research. Without
clinical trials, research is no more than an interesting activity for researchers,
and will never benefit people with diabetes.
Presentation by
Dr. Alberto Hayek, University
of California - San Diego
Dr. Alberto Hayek is Professor of Pediatrics at UCSD. He has many connections to diabetes, including a number of relatives with the disease, extensive research activities, and pediatric patients with diabetes.
Dr. Hayek pointed out that islet allotransplantation is not the answer to curing diabetes because of the limited supply of donor tissue. However, human islet studies must continue in order to advance the techniques that will be required to transplant islets from other sources. He commented on the importance of research into expanding human islets, both fetal and human. Despite the controversy surrounding the use of fetal islets, Dr. Hayek said it would be criminal to waste fetal islet tissue when it can do so much good in advancing our understanding of islet expansion and transplantation.
As with the other speakers, Dr. Hayek said that NIH must support clinical
trials of islet transplantation.
Presentation by
Don Smith, Islet Transplant
Recipient
Don Smith has had diabetes since 1963 and has been insulin-free for the past 5 years thanks to an islet transplant performed by Dr. Paul Lacy. Don's islet transplant was undertaken in three stages, two of which were in conjunction with a kidney transplant. Because of the kidney transplant, Don is on conventional immunosuppression.
Don spoke eloquently of how his life has improved since receiving islets. He had suffered most of the complications of diabetes, and has seen those complications stabilize and even reverse since normalizing his blood glucose. Don stressed how important it is for him to be a healthy and productive member of society, a role that diabetes was stealing from him.
Don's only visible health problem was a very sore knee as a result of
failing to recognize his age while playing baseball with his son!
Presentation by
Dr. Robert Genco, American
Academy of Periodontology
Dr. Robert Genco, DDS, PhD, spoke of the connection between diabetes, periodontal infection, and cardiovascular disease. Epidemiological studies have shown an increased incidence of periodontal infection in people with diabetes, and a worsening of glycemic control in the presence of periodontal infection. Furthermore, periodontal infection has been connected to heart disease. Therefore, this axis of diabetes / oral infection / heart disease may be an underlying mechanism that connects many of the classic complications of diabetes.
Many of these studies were conducted among the Pima population, a group
with a very low incidence of smokers. Hence, the effects observed were
tightly coupled to the presence of diabetes alone.
Report of the Type 1 Diabetes
Subgroup
Although each of the subgroups presented its part of the overall report that will be presented to Congress in the fall, this summary will concentrate on the report generated by the Type 1 Diabetes Subgroup.
Hugh O. McDevitt, MD, Professor of Microbiology, Immunology and Medicine at Stanford University, presented the report for the Type 1 Diabetes Subgroup to the DRWG. The highlights are as follow:
The major research goals of the Type 1 Diabetes Subgroup are:
If NIH implements the goals and and strategy of this plan without dilution
or vacillation, we will all have cause to rejoice.
Comments and Questions
There were many excellent comments and questions, and I would like to feature a subset below:
Dr. Gilman Grave: Islet transplantation is a most compelling approach for curing diabetes. We have heard 8 out of 9 speakers state that very clearly. Can we conclude that this will be the main focus of NIH in funding diabetes research? Dr. Chris Newgard answered that in the short term transplanted islets are the best solution, but that longer term other sources will offer advantages, and the type 1 subgroup report will relflect that evolution.
Dr. Chris Newgard: Will funding of diabetes research be affected in any way by the loss of revenue from the recently defeated tobacco settlement? Dr. Phillip Gorden said that this event will have no direct effect on diabetes funding, and that funding proposals should not be drafted with this factor in mind.
Dr. Phillip Gorden: The debate on research priorities should focus less on number of dollars allocated, and more on what science NIH should be supporting. You need to tell us how NIH can be most effective.
Dr. Ronald Kahn: Dr. Kahn pointed out some very interesting statistics on the amount of federal funding directed to diabetes research as compared to other diseases. Funding of HIV/AIDS research amounts to 14.1% of the annual disease cost. Funding of diabetes research amounts to 0.3% of disease cost. This differential represents a 47 times (4,700 %) greater relative funding of AIDS over diabetes. Why?
Marie Rizzo: My daughter has diabetes and retinopathy. We
are all talking about research priorities. Can I walk away and believe
that NIH, JDF and ADA are a hundred percent behind islet transplantation,
and that it is their top priority?
Washington Post: Institute of
Medicine agrees with us!
On many occasions we have felt as if our voices were being ignored,
and the NIH research agenda was being driven almost entirely by political
correctness and the wishes of scientists and bureaucrats. People actually
suffering from real-life diseases seemed to have no say in the research
funding process. What a relief to see our very concerns expressed so clearly
by the Institute of Medicine in its analysis of how the NIH sets
research funding priorities. The story below from the Washington Post
should be of great comfort to us all.
Political Pressure to Combat 'Pet' Diseases Cited as Influence on Funding Decisions
By Rick Weiss
Washington Post Staff Writer
Thursday, July 9, 1998; Page A17
The National Institutes of Health needs to be more scientifically rigorous
in
how it chooses spending priorities for its rapidly growing research budget,
according to an independent report critical of the recent politicization
of the
agency's appropriations process.
The report, released yesterday by the Institute of Medicine (IOM), an arm
of
the National Academy of Sciences, also recommends that NIH develop a
formal mechanism for giving patient advocates and other nonscientists more
direct influence over how much money gets spent on various diseases.
An organized system of advocacy would serve the public better than the
current one, in which various groups compete in a desperate effort to get
Congress to earmark spending for "pet" diseases, the report said.
The 119-page report, "Scientific Opportunities and Public Needs," does
not cite
specific instances of inappropriate spending by the $13.6 billion agency
-- the
nation's largest single funder of biomedical research. But it warns that
NIH
stands to lose its historically high credibility if it does not do a better
job of
justifying its spending decisions.
"There are some celebrated examples where very effective and forceful
lobbying and political pressure have affected resource allocations," said
Leon
Rosenberg, the Princeton University biology professor who chaired the IOM
advisory committee that authored the report. "Breast cancer and AIDS are
two
very clear examples."
On paper, at least, NIH has a commendable system for assigning spending
priorities, the report says. That system calls for an emphasis on diseases
that
have the greatest public health impact (as measured, for example, by the
number who die from that disease or the total cost of caring for patients);
the
potential for progress in a given field; the scientific quality of the
proposed
research; the proposed work's contribution toward the maintenance of a
"diversified research portfolio" at NIH; and the availability of scientists,
equipment and facilities appropriate to that line of research.
But, the report concludes, NIH does not hew to those rules. Critics in
Congress have estimated, for example, that NIH spends $110 a year on AIDS
for every death from that disease, but only $10 in cancer research per
death
from cancer and $2 in stroke research per death.
Concerns have mounted as advocacy groups have perfected their lobbying
acumen. The NIH budget has increased by 80 percent since 1990 -- compared
with a 48 percent increase in the rest of the nondefense discretionary
budget --
yet earmarked increases for specific diseases have in some years outpaced
the
agency's budget growth, forcing real losses in other areas of inquiry.
In one effective campaign last fall, a coalition persuaded the Senate to
approve
a Parkinson's disease package for 1998 that will require NIH to spend $100
million on research centers, training grants and other activities to help
people
with that disease, which affects perhaps 1 million Americans. After the
spending dust settled, Sens. Dan Coats (R-Ind.) and Bill Frist (R-Tenn.)
called
for an IOM assessment of NIH's budget process.
The report does not conclude that NIH spending should simply be pegged
to
cold indicators such as numbers of people affected. Rather it suggests
that
NIH consider data on the total burden that each disease inflicts upon the
nation
-- medically, economically, psychologically and otherwise -- as a major
factor.
Rosenberg said NIH leaders in the past balked at that prospect, saying
there is a
shortage of such data. In fact, he said, that information is available,
though
imperfect, and its use would "enhance the legitimacy" of NIH's priority-setting
process.
The report does not dispute Congress's authority to intervene in NIH's
ranking
of priorities, but it suggests the agency would find itself less prone
to such
machinations if it offered better documentation for its decisions.
The report also calls for creation of advisory committees within each of
NIH's
21 institutes and centers -- including the office of NIH Director Harold
Varmus, which is cited as being especially inaccessible -- through which
patient advocates could make their wishes known.
Varmus promised in a statement that he will review the report "in detail"
over
the next several months. He repeatedly has emphasized that basic research
in
one field often spurs advances in others.
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(Total Economic Impact) |
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| Heart disease |
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| Cancer |
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| Stroke |
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| Chronic lung disease |
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| Pneumonia, influenza |
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| Diabetes |
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| AIDS / HIV |
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| Chronic liver disease |
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| Kidney disease |
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| Septicemia |
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© Copyright 1998 The Washington Post Company
DRWG Participants