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DEPARTMENTS OF LABOR, HEALTH AND HUMAN SERVICES, EDUCATION, AND RELATED AGENCIES APPROPRIATIONS FOR 1999
Wednesday, January 28, 1998.
TESTIMONY OF MEMBERS OF CONGRESS AND OTHER INTERESTED INDIVIDUALS AND ORGANIZATIONS
WITNESS
JEROME PAULSON, M.D., AMERICAN ACADEMY OF PEDIATRICS
Mr. PORTER. This hearing begins the subcommittee's work on fiscal year 1999 funding for the departments and agencies within our jurisdiction.
Today we begin by hearing from public witnesses. We will then proceed to each of the departments and agencies, and then finally to Members of Congress. We will also receive testimony from the General Accounting Office. We have 29 days of hearings scheduled, covering just over 3 months, and we expect to have our final hearing right at the end of April.
Congress, as all of you know, is a busy place, and Members have many conflicting hearings, meetings, and other official responsibilities. However, let me assure you that while Members may not be present, your testimony is reviewed by them and their staffs and your views provide valuable information as we review the President's budget requests.
In order to accommodate as many members of the public as possible, we have scheduled over 20 witnesses for each session and are still not able to hear from all who wanted to testify. Overall, we will hear from over 200 witnesses in this segment alone. As a result, I have to enforce the rule limiting testimony to 5 minutes very strictly. My staff has acquired a new countdown device that they are going to use, and I would ask that, as you testify, you keep this limitation in mind in consideration of other witnesses that must follow you.
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I would also remind witnesses of two provisions of rules of the House. In addition to their written statement, nongovernmental witnesses must submit a curriculum vitae and a statement of Federal grant or contract funds they or the entity they represent have received.
If you have any questions concerning the applicability of this provision or questions as to how to comply, please contact this subcommittee staff.
We begin with Jerome Paulson, M.D., Associate Professor, Division of Pediatrics, Department of Health Care Sciences, George Washington University School of Medicine and Health Sciences, representing the American Academy of Pediatrics.
Dr. Paulson, we welcome you this morning.
Dr. PAULSON. Good morning, Mr. Porter. I am Jerome Paulson, M.D., a member of the American Academy of Pediatrics. I am a practicing pediatrician and an associate professor at George Washington University. On behalf of the Academy and our pediatric colleagues, I would like to thank the subcommittee for the opportunity to present this statement, and I want to thank you for putting children first on your agenda this morning.
This year, as we celebrate the bicentennial of the U.S. Public Health Service, we applaud how much has been accomplished. We have conquered many diseases and disabling conditions, especially for children; for example, smallpox, and we are close to the eradication of polio.
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To continue meeting the challenges ahead of new and emerging infectious diseases, food safety concerns, and costly chronic diseases, we must continue to invest in a continuum of public health activities that includes the full spectrum of biomedical, behavioral, and health services research, invests in disease prevention and health promotion, targets health care services for vulnerable populations, and educates a primary care and public health work force.
As pediatricians, we are on the front line and we see firsthand the impact of poverty and violence on the health of our children and adolescents, and we know that the future of our work force depends on the decisions we make today.
I will focus my oral remarks on a few observations from my own practice and research. The pediatric community has strived for decades to curb children and adolescents' access to and use of tobacco. Each day 3,000 children nationally begin to use tobacco. Of those people who will ever smoke, 90 percent begin before age 19. Young smokers suffer from respiratory problems and asthma, and among teens who are regular smokers, 1 in 3 will die from smoking.
As pediatricians, we counsel our patients about the addictiveness of nicotine and its detrimental health effects. In addition, we discuss with parents the impact of secondhand smoke on their children. These facts alone confirm that tobacco is truly a pediatric disease. As pediatricians, we strongly recommend adequate support for a wide array of tobacco prevention and cessation programs, including the CDC's office on smoking and health. These programs have a proven record of success in reducing smoking rates.
Let us not forget the environmental effects of tobacco smoke. Asthma is the most common chronic disease of childhood and is frequently exacerbated by tobacco. It is a disease that is on the increase, and disproportionately affects African-American and Latino children.
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According to the CDC, asthma-related costs to the health care system are continuing to grow because of increased morbidity and mortality. The current CDC projections for the year 2000 are $12.4 billion. Added to these health care costs are days lost from school for children and days away from work for their parents. However, with increased public health education, cost-effective environmental interventions and research, such as the NIH-, NIEHS- and NIAI-sponsored inner-city asthma study, asthma-related illnesses and costs can be dramatically reduced.
Injury is the leading cause of death and disability among children and young adults 1 to 44 years of age. Every day 60 children die from injuries and countless others are injured and disabled. Injury is costly on multiple levels: in the emotional toll it takes on victims and their families; in direct medical expenses, both acute and chronic; and in long-term economic costs due to the years of potential life and productivity lost, especially with respect to children.
In direct medical costs alone, injury costs the Federal Government $12.6 billion annually, and an additional $18.4 billion each year in disability and death benefits. Therefore, measures to prevent injury and reduce its severity are extremely cost-effective. The National Center for Injury Prevention and Control fulfills a unique function in this undertaking.
The Center's work addresses many types of injuries, both intentional, such as youth-perpetrated violence, and unintentional. Additional resources would enable the Center to continue its important leadership in the Safe America Program through which the NCIPC has brought together diverse public and private sector entities to develop and disseminate injury-prevention information and interventions.
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The initial focus of the effort is to reduce injury among children and adolescents. Additional resources would also enable the center to expand efforts to reduce physical and sexual violence among children, produce a comprehensive youth violence prevention program, and ensure that every U.S. resident has access to the lifesaving and cost-effective services of a poison control center through national and State-specific toll-free numbers.
As scientists and investigators, pediatricians encourage research decisions to continue to remain in the hands of scientists at the CDC. But what happens to children or adolescents when he or she is injured and requires emergency services? As a participant in a project funded by the EMSC program, I know how important those dollars are in enabling us to develop the information that will improve the system of care for children and save lives.
Thank you for this opportunity to provide you with our recommendations for the coming fiscal year. We hope that we can continue our dialogue with this committee following the release of the President's fiscal year 1999 budget. There are many important opportunities for pediatric behavioral and health sciences research. We know that you will not forget America's children.
Mr. PORTER. Thank you. Thank you for your fine statement, and thank you for being reasonably close to your time limit.
Mr. PAULSON. You are welcome, sir.
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[The prepared statement of Jerome Paulson, M.D., follows:]
"The Official Committee record contains additional material here."
Wednesday, January 28, 1998.
WITNESS
MIKE MILLER, M.D., FDA–NIH COUNCIL
Mr. PORTER. Mike Miller, M.D., Director of Federal Relations, Incorporated, testifying on behalf of the FDA Research Council.
Dr. MILLER. Thank you, Mr. Chairman.
On behalf of the FDA Research Council, I want to thank you for the opportunity to submit testimony concerning the importance of a sustainable, predictable funding base for the NIH. Our comments in full will be included for the record.
We emphasize the importance of the NIH in improving the quality of health care for all. In past years this committee has been vitally important in addressing the funding needs of the NIH and the Council, and the entire research community is grateful for your support.
Based on the Council's assessment of challenges and opportunities the FDA is presented within the next several years, the Council supports doubling the NIH budget for the next 5 years, starting with a 15 percent increase for fiscal year 1999.
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Why does the Council and NIAMS support such an increase for the NIH's budget? The answer to this lies, I believe, in understanding the goal of the entire process. It may seem obvious, but the goal of the entire biomedical research innovation and discovery process is to improve the quality of people's lives. In our detailed budgetary discussions that we had in Washington it is easy to forget that basic fact, but it is also sometimes not easy to see that the ultimate goal is a simple cure for a disease.
What everybody really wants is to be able to go to the physician, take a pill, and be cured for whatever ails them. Getting to that goal is not easy or simple. It takes a robust biomedical research system. Worldwide the U.S. is the leader in such a system, and the NIH is at its foundation.
What I would like to briefly discuss is the historical context of our current biomedical research system, because by understanding how we came to our current level of knowledge and the treatments from that knowledge, we can better appreciate the landscape ahead and better plan how we want to proceed in the future.
Most people who support the NIH understand intuitively what potentially lies ahead, but by understanding what seems intuitive, it can help to clarify its importance and relevance to our lives, our actions, and our future.
What has been the progress of medicine for the past 20, 50, or 100 years? For example, 100 years ago, biomedical research was largely a process of observation and classification. Medical practice was largely based upon simple chemicals and even herbal extracts which have been found by trial and error over many years to have some beneficial effects.
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In fact, in 1910, the AMA surveyed physicians and asked them what they thought were the most important therapies available to them. Number one on the list was ether, which enabled anesthetized surgery. Number two was morphine, a painkiller but certainly not curative. Number three was digitalis for heart ailments. Number nine was alcohol. Number 10 was mercury.
The AMA did a similar survey in 1945 and found significant advances. Number one was penicillin and the sulfa antibiotics, ether had dropped to number four, and digitalis to number five. Insulin had been discovered and was added to the list as number eight, and number ten was vitamins.
The question is: What would such a list look like if physicians were surveyed today. Insulin might still be on the list because it is very important for people with diabetes, but its form of purity has changed. Today human insulin is available through the common DNA technology. Other innovative medicines treat a variety of ailments which were not even understood in 1945, such as cancers, autoimmune disorders, AIDS, and neurological conditions. The list goes on.
One only needs to talk to friends or their children to find out what kind of medicines are being taken that are available today that were not available 5 or even 10 years ago. What has changed in the past 20 to 50 years? Some outstanding biomedical breakthroughs have taken place to lead to these new medicines. Heart disease, for example, is based on—new treatments for heart disease are based on a better understanding of specific receptors or enzymes, how they block or activate those enzymes or receptors. Each of these advanced treatments is only possible once it is understood how the receptor enzyme controls some aspect of health or disease. This is the essential basic research which NIH supports. Without this knowledge, new medicine would only be found through serendipity, not through rational science.
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I may have given the impression with my oversimplified account simply that innovation proceeds in a linear, stepwise fashion. It is actually sinuous and weblike, with many blind paths and interlinking discoveries. With NIH in the lead identifying promising paths, the other partners in the process will be able to support the intellectual discoveries made possible by NIH funding, and to work with NIH and academic researchers to take these discoveries out of the test tube and into a form where they can provide significant benefits to millions of Americans.
To accomplish this, support for the NIH must be across its range of programs, including those which provide means so everyone in the community can take advantage of scientific opportunities. One other exciting avenue which is just beginning to be explored involves the race to develop and validate diagnostic measures and tests to diagnose or to determine the status of primary diseases before the symptoms will develop. Such steps will attempt to prevent disease progression in individuals and help researchers to determine whether an experimental treatment is or is not effective, and thus either make it more widely available, or attempt the investigation in order to pursue other opportunities or paths.
These are some of the exciting opportunities and challenges, Mr. Chairman. The Council appreciates your support for the NIH, and urges you to consider our request for doubling over 5 years and a 15 percent increase. We realize you have a tough job and many priorities, but thank you for this opportunity. We are willing to take any questions.
[The prepared statement of Mike Miller, M.D., follows:]
"The Official Committee record contains additional material here."
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Mr. PORTER. Dr. Miller, thank you, sir, for your excellent statement. Let me say that the American people do intuitively understand the importance of biomedical research, but all of us, all of you, have to somehow get the message out as to what progress has been made, why it is important to human beings. It is not only important to improve lives, it saves health care costs tremendously. It improves the quality of life in many different ways.
Somehow we need to get that message out to every American person, not just the Members of Congress but the whole population at large. All of us have to put our shoulder to that wheel if we are going to get the kinds of increases that we think are necessary for NIH.
I would say one other thing. We can only work with what we are given to work with. It is the budget process that allocates the funds. If we sit and wait until the subcommittee marks up its bill and says, oh, fine, you know, and we don't do anything up to that point, we could well have lost the opportunity to make the kinds of commitments to biomedical research that we need to make.
So the fight is right now with the budget process, and getting the kinds of allocations that allow us to make the kinds of increases that both of us and everyone in this room, I suspect, believe are our goal, and what we think will really help advance the cause of biomedical science and improve the lives of every person on the planet. Thank you, Dr. Miller.
Dr. MILLER. Thank you. I couldn't agree with both your points any more strongly. Thank you.
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Wednesday, January 28, 1998.
WITNESS
JOHN M. CRAWFORD, D.D.S., Ph.D., AMERICAN ASSOCIATION FOR DENTAL RESEARCH
Mr. PORTER. John M. Crawford, D.D.S., Ph.D., Professor of Periodontics, the University of Illinois at Chicago, College of Dentistry, representing the American Association for Dental Research.
The Chair welcomes a fellow Chicagoan.
Dr. CRAWFORD. Thank you very much. Thank you for this opportunity to address the committee.
As you indicated, I am here to represent the American Association for Dental Research and its 5,300 members. I would like to discuss the 1999 budget allocation to the National Institute for Dental Research and the Agency for Health Care Policy and Research.
When you deal with the justification of allocations, you have to look back and you have to look forward. You look back to document the successful and the productive and wise use of funds, and the consequent increase in the health of the American people. I think that the NIDR has very good evidence that it has done this in a very successful way. This evidence we will present in our written testimony, and the Director of NIDR will present more evidence for this in his oral testimony.
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However, you have to look forward, and you have to present evidence that there are problems yet to be solved. These problems can be solved with the manpower and technology available. It is this point I would like to discuss briefly today.
I would like to present a scenario for oral health management in the 21st century. I think this will focus on identification of people at risk for oral disease, and there is a lot of evidence that this is not distributed evenly through the population of the U.S. There are people at risk and people relatively immune.
We have already saliva-based tests to predict dental decay activity, and there is interesting recent work which begins to show us that we can predict the activity of gum disease as well. Having identified people at risk, we will then be able to offer them lifetime immunity from these diseases, and again, the National Institute for Dental Research has supported research which clearly shows the ability to produce a safe and effective vaccine for dental caries, and there is interesting work in the animal models for certain diseases, with mice and monkeys, that shows we may be able to offer a safe and effective vaccine for gum disease as well. So there is a scenario for cost-effective health research focusing on at-risk populations.
I would like to take a moment to describe the situation in 1997, using myself as an example. My dentist tells me to clean my teeth 2 minutes with a toothbrush at night, and added to that, 2 minutes with dental floss. That adds up to about 36 1/2 hours every year, and if I live to the age of 76, my actuarial limit, I will have spent 6 months cleaning my teeth. Life is too short.
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I would like to also indicate that if you are susceptible to gum disease, you have a lifetime of struggle combatting this disease, slowing it down, and trying to arrest it. This is a chronic, disabling disease. The treatment is very sophisticated, but it is also time-consuming, very costly, and leads to lost productivity hours.
I would like to point out that teeth are the only organs that we frequently discard before the end of our lives. And I would like to point out that periodontal disease is a major health problem in this country. Recent NIDR-sponsored research has shown that 35.7 million Americans have at least mild bone loss. But more surprisingly and more seriously, 13.1 million Americans have moderate to severe bone loss. So this is a public health problem of pandemic proportions.
I would like to envisage in the near future a longer, healthier life, but at the end of that life, we will have our healthy teeth in our mouths.
Mr. Chairman, the American Association for Dental Research supports the ad hoc group's overall recommendation for NIH of $15,695,000,000. More specifically, the American Association for Dental Research supports the request for $240,822,000 for the National Institute for Dental Research.
I would also like to take this opportunity to support the work of the Agency for Health Policy and Research, because we strongly feel that research into health care policy is essential for the oral health of the American people.
I would be happy to attempt to answer any questions that you may have.
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[The prepared statement of John Crawford follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Dr. Crawford, did you say 15.95 billion?
Dr. CRAWFORD. The figure that I quoted was $15,695,000,000.
Mr. PORTER. What does that represent?
Dr. CRAWFORD. That represents a 15 percent increase, I believe.
Mr. PORTER. Whose figure is that?
Dr. CRAWFORD. The ad hoc group's overall recommendation for NIH.
Mr. PORTER. I have already given my sermonette about how we get there, but let's all work together to achieve that.
Dr. CRAWFORD. Yes, indeed.
Mr. PORTER. Thank you very much for your good testimony.
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Wednesday, January 28, 1998.
WITNESS
DAVID RECKER, M.D., AMERICAN COLLEGE OF RHEUMATOLOGY
Mr. PORTER. David Recker, M.D., Member, ACR Government Affairs Committee, representing the American College of Rheumatology.
Dr. RECKER. Good morning, Mr. Porter. I am David Recker, M.D. I am a rheumatologist from Auburn, Pennsylvania, and I am here today to testify for the American College of Rheumatology in support of funding for the National Institutes of Arthritis and Musculoskeletal and Skin Diseases and for the NIH as a whole.
The ACR is a professional organization of rheumatologists. It includes practicing physicians, research scientists, nurses, physical and occupational therapists, and other allied health professionals. We are dedicated to understanding, treating, and hopefully one day curing the more than 100 types of arthritis and related disorders that involve joints, skin, bones, and connective tissue.
These conditions often result in severe disability and sometimes even in death. In just a few years we face the coming of a new millennium. Amid the hoopla and celebration of this event, there is a certain amount of uncertainty and speculation; but future events await us, our children, our children's children. What challenges does our Nation face in defining its existence? Will John Elway, one of the last baby-boomers, continue to be able to play into the 21st century?
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What we do know is that in the year 2000, some 20 million American baby-boomers will turn age 50. We also know that this Nation's aging population will be stricken with an ever-growing burden of arthritis unless something is done to halt the progression of these debilitating conditions. A study from the Journal of the American Medical Association revealed that nearly half of all Americans age 60 and above suffer from some form of arthritis.
Studies have shown that no condition impairs the overall quality of life in more older Americans and to a greater extent than does arthritis. Swollen and disfigured joints not only hurt, they significantly hinder effective individuals in their ability to perform even the most basic of daily functions such as bathing, dressing, and eating. I can tell you from my personal clinical experience how vibrant, happy, productive individuals have become dependent, pain-ridden, despondent, and even depressed as these afflictions rob them of their functional capacity and steal from society their irreplaceable productivity.
As I have already mentioned, our ever-aging population means more and more individuals will face the potential ravages of arthritis. Providing care for this growing number of Americans means that the government, private insurers, and indeed society as a whole will bear an ever-increasing economic burden.
While arthritis typically is encountered in older populations, in reality no age is spared from this group of diseases. Disorders such as juvenile rheumatoid arthritis, which can strike children at virtually any age, can lead to lifelong disability, disfigurement and pain. Younger adults are also subject to the pain and suffering imparted by rheumatic diseases. Systemic lupus erythematosus, for example, is typically a disease of young, otherwise healthy women.
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Through increased research into the basic pathology and origin of these diseases, as well as through increased investigation into better treatment and management strategies, we can potentially greatly mitigate the costs of these diseases in both personal and economic terms, and we can improve the overall quality of life of afflicted individuals.
I am here on behalf of the ACR to voice support for the appropriations for the NIH as a whole, and more specifically, the ACR joins with the other 200 other organizations who together form the Ad Hoc Group for Medical Research Funding to support a 15 percent NIH-wide increase in biomedical research and training, as indeed a first step to doubling the NIH budget in the next 5 years.
The ACR, along with other members of the Coalition of Professional and Voluntary Groups Concerned with the Programs of NIAMS, endorsed this increase, translated to this specific Institute's budget. This would result in funding NIAMS at approximately $316 million, $40 million more than the current fiscal year.
What can we do with this increased funding? Funded in part by NIAMS, researchers have recently discovered mutations in certain genes which may lead to an overgrowth of synovial tissue. The linings of joints in patients with arthritis have severely thickened synovia, and it is this thick synovia that is felt to be responsible for much of the disease's devastating problems. By further investigating how to control and limit the growth of synovia, we can potentially mitigate some of the ravaging effects of rheumatoid arthritis.
On a more practical matter, again, researchers funded by NIAMS have assessed the medical and economic impact of joint replacement, currently an option for Americans enduring the pain of arthritis. Joint replacement is nevertheless expensive. It costs about $48,000 for a typical hip replacement. The cost for nonsurgical intervention, however, has been shown by these researchers to be $165,000, three times more expensive. This analysis does not even consider the impact of the quality of life.
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The cooperative efforts as well, of NIAMS-funded researchers, can investigate questions that will further the mission of the Institute. Through collaboration with the Aging Institute, with the National Institute of Child Health and Human Development, and with NEI and NIRD, we can again further the mission statement.
The ACR certainly commends the leadership demonstrated by this committee, and you particularly, Mr. Porter, in support of the Nation's biomedical research effort. We strongly urge this subcommittee to continue its effort and to support the 15 percent funding in programs of the National Institutes of Health.
Thank you.
[The prepared statement of David Recker, M.D. follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Dr. Recker, thank you very much.
I have to say, sometimes, often, there is a great deal of frustration for the chair, because there are lots of questions I would like to ask you about what genetic basis for the disease, if any, has been discovered, environmental factors, mental factors, and the like. But I don't have time to ask those kinds of questions and receive your answers at this point. It is true of almost all of our witnesses, and the difficulty is, we are limited by the time we have, so we have to do the best we can.
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But thank you very much for your testimony today. I very much appreciate it. Obviously, we are going to do our very best to meet the goals that you have described for NIH and for NIAMS.
Dr. RECKER. We will be happy to answer any questions.
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Wednesday, January 28, 1998.
WITNESS
ROBERT O. KELLEY, Ph.D., ASSOCIATION OF AMERICAN MEDICAL COLLEGES
Mr. PORTER. Robert O. Kelley, Ph.D., associate vice chancellor for research and executive associate dean of the Graduate College, University of Illinois College of Medicine, representing the Association of American Medical Colleges.
Dr. Kelley.
Dr. KELLEY. Thank you, Mr. Porter.
My name is Robert Kelley. The University of Illinois, especially the University of Illinois at Chicago, and the Association of American Medical Colleges thanks the subcommittee for their continued support of the National Institutes of Health and the other programs I will discuss.
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In particular, Mr. Chairman, we want to thank you for your leadership on behalf of medical and biomedical research. I will summarize my written statement with the following points.
The Federal investment in medical research, through the NIH, continues to yield a profusion of fundamental and applied knowledge transforming the practice of medicine. In addition to its enormous benefit to the health of the American people, NIH-sponsored research also continues to provide the basis for much of the sustained success of the biotechnology and pharmaceutical industries.
Still, America faces serious health problems, and new threats constantly appear. Congressional support for medical research has produced a wealth of scientific opportunities to answer these challenges. If we are to sustain this momentum and translate the promise of science into improved health for all Americans, we must redouble our national commitment to medical research.
The Association of American Medical Colleges endorses the recommendation of the Ad Hoc Group for Medical Research Funding for a 15 percent increase in the NIH budget as the first step toward the goal of doubling NIH funding over the next 5 years. Science is changing at a breathtaking pace, and we must invest in new technologies, new personnel, and new research infrastructure if we are to take full advantage of this science.
The Association of American Medical Colleges urges the subcommittee to pay particular attention to the needs of the National Center for Research Resources, which provides support for the general clinical research centers and other essential elements of a vigorous research environment.
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The transformation of the health care system to a market-driven, price-competitive structure poses a significant threat to the ability of medical schools and teaching hospitals to maintain an environment for research and innovation. The Association of American Medical Colleges strongly urges the Congress to provide for flexible but accountable institutional support by funding the Biomedical Research Support Grant program, BRSG.
The geographic and specialty maldistributions of physicians in the United States are critical issues facing both the Congress and the Nation. The Association of American Medical Colleges urges the subcommittee to provide additional funding for the National Health Service Corps and the health professions education programs authorized under Titles VII and VIII of the Public Health Service Act, which play a major role in addressing these problems.
The Association of American Medical Colleges thanks the subcommittee for sustaining funding in fiscal year 1997 for the health professions and nursing education programs, and the AAMC joins the Health Professions and Nursing Education Coalition in urging the subcommittee to provide at least $306 million for fiscal year 1999.
The drive to cut health care costs raises concerns about the quality and appropriateness of care and the choices available to consumers. The Agency for Health Care Policy and Research is charged with sponsoring health services research designed to improve the quality of health care, decrease health care costs, and provide access to additional health care services in a rapidly changing marketplace. The Association of American Medical Colleges believes strongly in the value of health services research as this Nation continues to strive to provide high-quality health care for all of its citizens.
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The Association of American Medical Colleges endorses the Friends of the Agency for Health Care Policy and Research in their recommendation of a fiscal year 1999 funding level of $175 million for the AHCPR. As you know, it is the major Federal program supporting the widely-recognized need to strengthen our knowledge of and commitment to evidence-based medicine.
Finally, we wish to emphasize the importance of research, training, and health professions programs targeted at the racial and ethnic groups that are underrepresented in medicine and research. Support for these programs is more crucial than ever.
The Association of American Medical Colleges appreciates the continued support of your subcommittee and the support that has given to all the programs cited in my written statement. We take very seriously the charge you just gave to others and look forward to working with you on behalf of those charges.
[The prepared statement of Robert Kelley follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Dr. Kelley, thank you very much.
Now that my colleague, Congressman Stokes, is here, so that both sides of the aisle are represented, I can ask this question. Does the AAMC have a reaction to what the President said regarding research in his State of the Union Address last night?
Dr. KELLEY. We are certainly supportive of biomedical research and the goals it brings, to the benefit of the American people. I think when we represent all of the people and all of our cities, all of our States, I think everything we can do as a people to support the goals of biomedical research we would certainly stand behind.
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Mr. PORTER. What I understood from the President's remarks, and, Lou, you can correct me if I am wrong, but he said a 50 percent increase in all research, all basic research, over the next 5 years, not the 100 percent increase that our witnesses here have been talking about.
Dr. KELLEY. I think the AAMC would certainly stand behind doing as much as we could. We would certainly support the ad hoc group's recommendations and I think the position that you are representing, trying to do as much as we possibly can for NIH and biomedical research.
Mr. PORTER. Tell the President and the Committee on the Budget.
Dr. KELLEY. We certainly shall. Thank you.
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Wednesday, January 28, 1998.
WITNESS
ERIC NEILSON, M.D., AD HOC GROUP FOR MEDICAL RESEARCH FUNDING
Mr. PORTER. Eric Neilson, M.D., the C. Mahlon Kline professor of medicine and pediatrics at the University of Pennsylvania School of Medicine, and founding president of the Association of Subspecialty Professors, representing the Ad Hoc Group for Medical Research Funding.
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Dr. NEILSON. Good morning, Mr. Porter. Thank you.
I am testifying on behalf of the Ad Hoc Group for Medical Research Funding, as the first coalition of nearly 200 organizations representing patient groups, medical scientific societies, academic and research organizations, and industry.
Mr. Chairman, we thank you and the members of the subcommittee for making the National Institutes of Health one of your very highest priorities.
I would like to make the following points to summarize my written statement. As a consequence of the Federal commitment to medical research, an ever expanding base of scientific knowledge about health and diseases is being developed that is revolutionizing both the conduct of the scientific inquiry and the practice of medicine. If we are to sustain this momentum and reap the full benefits of this investment, then the Nation must commit to its plan for significant and sustained funding for the NIH.
We commend the leadership demonstrated by the congressional sponsors of proposals to double the NIH budget over the next 5 years. For fiscal year 1999, the Ad Hoc Group supports a 15 percent increase in NIH funding as the first step towards the goal of doubling the NIH budget over the next 5 years. We believe this increase is justified both by the health needs and research capacity of the Nation. Such a long-term commitment will also stabilize sensible planning through use of those funds.
Congressional support for the NIH over the past 40 years has produced a wealth of opportunities in basic and clinical science that will ultimately alleviate and eliminate many of the health challenges we currently face. In addition, medical research plays an important role in the growth of biotechnology and pharmaceutical industries.
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I personally have been supported throughout my career by the NIH. I am now a principal investigator of five NIH grants. I serve on the National Advisory Council for the National Institutes on Diabetes, Digestive and Kidney Diseases.
Permit me to make the following observations on the needs for additional funding that we are recommending. Science is changing at a revolutionary pace. It requires investment in new technologies and a renewed research infrastructure, including people with appropriate skills to meet this new science.
As someone who has trained numerous physician scientists over the last 25 years, I can say that one of the most pressing needs we face in the area of training, particularly for clinical research, is for physician scientists. We need to expand clinical research training opportunities. Additional funds could be used to raise the stipends and to lengthen the training period for clinician scientists to at least 3 years. We have a crisis in the Nation in attracting and retaining new investigators in these areas. Direct attention to this budget item would be most important.
Additional funds could also be used to enhance clinical research infrastructure, including increased support for general clinical research centers. A reinvigorated GCRC program could serve as a focal point for clinical training as well as expand clinical research activities. This would happily avoid a new administrative entity and focus initial efforts towards institutions with established records of clinical research excellence.
At the same time, of course, we must also sustain and enhance our basic research, including increased support for current researchers, as well as promoting opportunities for new investigators in those areas of biomedical science that have been underfunded.
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We particularly need to enhance the institutional research capacity, including renovation of outdated facilities, creating new approaches to support animal facilities, providing state-of-the-art instrumentation and other research equipment, promoting informational and computer technology, and substantially increasing the funding for research support programs under the National Center for Research Resources.
Finally, continued progress in medical research depends on the advancement in related fields of science, including chemistry, physics, mathematics, and engineering. We urge the administration and the Congress to consider the breadth of this Nation's research efforts as interdependent and fund them accordingly.
In closing, we recognize the difficulty of achieving the goal we have articulated under the Center spending caps. A national commitment to doubling the NIH budget over the next 5 years will necessitate additional resources to sustain the growth of the coming years. The Ad Hoc Group urges the Congress to explore carefully all options to identify new resources. We feel that leveraging other entities and other opportunities is very important to this process in bringing other individuals into the discussion.
We stand ready to work with the supporters of medical research on Capitol Hill to achieve this goal, beginning with fiscal year 1999 and beyond, and thank you very much for your time and attention. I am happy to answer any questions relating to my comments.
[The prepared statement of Eric Neilson, M.D., follows:]
"The Official Committee record contains additional material here."
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Mr. PORTER. Dr. Neilson and Dr. Kelley, I want to explain that I was encouraged by what the President had to say in his speech last night. He talked, Dr. Neilson, not only about biomedical research, but all basic research.
Dr. NEILSON. Yes.
Mr. PORTER. For the reasons you have stated, but also for the strategic political reason that I do not think we can set one type of research against another in our Federal budget, they have to go relatively hand in hand.
I think that the President's remarks were very encouraging, and all of you probably realize—at least this is the way I think about it—that when the President of the United States gives support for a figure for NIH that is an increase of about 8.2 or 3 percent, that becomes, to us, a floor from which we perhaps, if we get the resources, can work and even do better.
So I feel very encouraged by what the President had to say. I said that publicly, both to the electronic and print media, and I think it indicates a new commitment from the White House to put this at a very high priority.
Having said that, all of us have to realize that the President's budget has about $100 million of new revenues that very possibly could never materialize. There are many in the Congress who are very opposed to any kind of new revenues, even tobacco taxes, which I think are very justified, and whether we can get the revenue increases to support the spending increases is at this point, I think, very problematical.
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I was very encouraged by what the President had to say, and I have to say, Lou, I said that to everybody who asked and, in fact, some who didn't ask.
So thank you, Dr. Neilson, for your very good testimony. We appreciate it, and we are going to do our very best to work very closely with the Ad Hoc Committee and push very hard to put biomedical research at a high priority within all of the programs of the administration.
Thank you.
—————
Wednesday, January 28, 1998.
WITNESS
ROBERT R. RICH, M.D., THE AMERICAN ASSOCIATION OF IMMUNOLOGISTS
Mr. PORTER. Robert R. Rich, M.D., vice president and dean of research, Baylor College of Medicine, Houston, and chairman of the Committee on Public Affairs, representing the American Association of Immunologists.
Dr. Rich, welcome.
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Dr. RICH. Thank you very much, Mr. Chairman. It is a pleasure to have the opportunity to represent the 6,000 scientists of the American Association of Immunologists.
I would like to begin this morning by simply saying how much we are indebted to you and to this committee for the extraordinary support you have given to the biomedical research efforts over the past 3 years.
Indeed, at a time when you have been mandated to cut more than $50 billion from the allocations to this subcommittee, you have managed to provide important increases in the allocation to NIH in every year, and we are very, very grateful for that.
We are also very grateful to you and to Mr. Miller for your attention to an issue that we called to your attention several years ago relating to the regulatory burdens that we face as we try to make the research that we do with your appropriated dollars increasingly more efficient. We understand that this is going forward now, as you have called for a private sector study from NIH to look into this issue. We are anxious to work with that study in any way we can to be helpful.
Mr. PORTER. Dr. Rich, I want to say that Dan Miller has taken the lead on that and done, I think, a very, very fine job of putting that issue before us and pushing hard to get it resolved.
Dr. RICH. We are very grateful for that, and we very much appreciate that.
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What I would like to talk about today are two issues. The first is a problem and the second is an opportunity.
The problem we have already heard about from the AAMC. That is, with the increasing competitive managed care environment, we find that our academic medical centers really can no longer afford to support the research faculty which is so vital to the activities of NIH.
Interestingly, as they indicated to you, a solution already exists in the law; that is, the Biomedical Research Support Grant program, or BRSG. Indeed, in 1990, NIH said, this program recognizes the need to support new investigators, to explore new and unorthodox research ideas and techniques, to respond promptly to opportunities that develop in the course of active and diverse research programs, and to provide central shared resources.
For the 27 years of its funding, the BRSG program received a median of 2.4 percent of the research project grant budget that was given to the institutions. This year, that would equate to approximately $186 million. However, in 1992 funding for the BRSG program was discontinued over concerns regarding its accountability.
Mr. Chairman, I submit that that is a problem we can fix. Last year, I advocated that we reestablish the BRSG program with full peer review and accountability. The subcommittee agreed with this proposition, requesting that the NIH study the feasibility of reestablishing a revised Biomedical Research Support Grant program.
I come before you today requesting an appropriation for a new BRSG program on an experimental basis for a 3-year trial period. I would suggest an appropriation of $155 million in the first year, which would be about 2 percent of the research program grants budget.
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I would further suggest that such a program be established based on three fundamental principles: One, that the monies be used for currently defined purposes; second, that the funding be distributed according to a process of strict institutional peer review; and, third, that the NIH be charged to develop a mechanism for accountability. I provided details of each of these principles in my written testimony.
This brings me, then, in closing, to the opportunity. This year we celebrate the 20th anniversary of Edward Jenner's discovery of the principles of vaccination. Within the past 50 years, we have seen many of the historical scourges of mankind essentially disappear. Indeed, the target of Jenner's work, smallpox, has been eradicated from the Earth.
Yet, I need not remind the members of this subcommittee that we still face formidable threats. We all heard about the bird flu from Hong Kong in very recent weeks. And recognizing that the only way we are going to really confront the problem of HIV and defeat it is to develop an effective vaccine, the NIH last year established a vaccine research center on the campus in Bethesda. The AAI supports this initiative of vaccine development but would like to make some suggestions to you in terms of how it might be modified, because we believe that, first of all, the time is now to extend it beyond the NIH campus and to extend its focus beyond HIV.
Indeed, we recognize that there are many important scourges of an infectious nature that remain before us. Perhaps more excitingly, however, is the idea that the principles behind vaccination can now, with new scientific opportunities, be turned to diseases of a noninfectious nature. We are very excited, at AAI, about the possibility that the human immune system might be programmed to attack a variety of diseases before they develop.
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What kind of diseases am I thinking about? I am particularly attracted to the notion of autoimmunity and cancers. That is right, Mr. Chairman. As an immunologist, I can tell you that we can look to the day when we might be able to vaccinate our children against lupus, multiple sclerosis, diabetes, and rheumatoid arthritis, much as we vaccinate them against measles and polio today.
Thank you again very much for the subcommittee's support. We appreciate the opportunity to testify before you.
[The prepared statement of Robert Rich, M.D., follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Dr. Rich, thank you very much.
I have one quick question. Is the BRSG program going to appear in the President's budget? In other words, is he suggesting that we provide this funding, do you know?
Dr. RICH. We have not seen that. I would be surprised if we see a line item appropriation. I think that—but we have not seen the details of the President's budget.
Mr. PORTER. Thank you very much. We appreciate your testimony.
—————
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Wednesday, January 28, 1998.
WITNESSES
CLAUDIA E. MILLER, Ph.D., M.T., AMERICAN SOCIETY OF CLINICAL PATHOLOGISTS
SARA BROWMAN, JOHN EDWARD PORTER SCHOLAR
Mr. PORTER. Dr. Claudia Miller, director of Medical Technology Programs and chair of Allied Health at National Lewis University in Evanston, Illinois—that is my hometown—accompanied by Sara Browman, the Porter Scholar.
Hi, Sara.
Welcome, Dr. Miller.
Dr. MILLER. Good morning, Chairman Porter, Mr. Stokes.
I represent the American Society of Clinical Pathologists. I have served on their Educators Committee, and I have been a regional representative and recruiter.
The American Society of Clinical Pathologists, the ASCP, is a medical not-for-profit society which has as its goal scientific and educational purposes. It has 75,000 members, and they include not only board-certified pathologists but other physicians, Ph.D. scientists, and board-certified technologists and technicians.
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The allied health professions serve 60 percent of the health care work force today, and we serve in all different tiers, from hospitals to health care, extended health care, skilled nursing, and even physicians' offices.
The Allied Health Grant program, which is under section 767, Title VII, of the Public Health Service Act, has been very effective in dealing with allied health needs, but more resources are needed simply because we need more allied health professionals to serve the United States population. Shortages are evident by vacancy rates, and I will give you some examples of those.
In laboratory medicine, we have in histotechnology an 11.7 percent shortage. These are the people who prepare tissues for examination. In cytotechnology supervisors, the people who look at cells to assess the status of cancer malignancies, there is a 14.1 percent.
Some people argue that it is not the responsibility of the Federal Government to aid allied health programs. We agree. The private sector does have to take responsibility. The ASCP does this by offering 50 scholarships annually to medical technology, medical laboratory technicians, histotechnology technicians, and also cytotechnology students.
I have here beside me Sara Browman, who is the 1998 recipient of the scholarship named in your honor, Mr. Porter, because of your support to allied health.
But there is a difference. The ASCP gives monies to students like Sara, but the Allied Health Project Grant program gives money to schools so that students like Sara will have the opportunity to receive an education and be able to work in allied health.
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We have very many success stories. One is at the University of Nebraska Medical Technology Program, where, with the third $358,000 they received in 1992, they were able to start a world health program. Fifty-three medical technology students graduated, and 94 percent remained in the rural area to serve people in clinical laboratory medicine. They are now self-sufficient, and many of their students from the regular on-campus program are actually seeking employment in rural areas.
The University of Maryland, using a grant that they received in 1991, has addressed the problem of minority recruitment and retention. I am very happy to tell you that in 1996 the Medical Technology Program had 52 percent minority students, and they have a 95 percent retention rate. This is at a majority institution, and the retention rate is one of the highest in the Nation.
The monies from Allied Health Project Grants actually are used to help establish critical need programs. Project outcomes are tracked; information is shared with other programs so that they are able to go out and start programs that are needed using limited resources.
Keeping in mind that these programs have been so successful and that we so desperately need more allied health professionals, we urge you to consider funding the Allied Health Projects Grant program for fiscal year 1999 for $10 million and the Health Professions Training program for $306 million.
Thank you for your consideration.
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[The prepared statement of Claudia Miller follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Thank you very much, Dr. Miller.
Sara, your hometown is what?
Ms. BROWMAN. Rockford, Illinois.
Mr. PORTER. Rockford, Illinois. My wife's mother lives in Rockford, Illinois, so I am there frequently. We welcome you. I am very honored that you are the Porter scholar.
There are a number of scholarships that you have named for a number of different individuals at National Lewis; is that correct?
Dr. MILLER. We have a lot of scholarships, yes, especially Mr. Lewis, who gave us all the money for our university.
Mr. PORTER. Especially Mr. Lewis. That makes good sense.
Well, Sara, we appreciate your joining Dr. Miller this morning, and we wish you well in your career. We are honored that there is a scholarship in my name and that you are the scholar. Thank you both for being here.
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Mr. Stokes.
Mr. STOKES. Mr. Chairman, I would just like to take a moment and thank Dr. Miller for sharing with us the success of her minority recruitment program. This is welcome news, particularly at a time when, because of the assault upon affirmative action, we are finding now that throughout the Nation there has been a considerable drop in even the applications by minority students because of the fear and the actual threat of not being accepted, and so forth.
The fact that you are having this type of success with it is very encouraging, and I think it speaks loud and clear to other institutions, particularly majority institutions, as you have cited, of the absolute need to do something more in this area. I really appreciate your sharing this with us.
Dr. MILLER. Thank you, Mr. Stokes.
I think that the success of the program at the University of Maryland is because of early intervention. They start in elementary school, track them through middle school, and then continue on until they receive their bachelor's degree.
Mr. STOKES. I may want to get a chance to talk with you a little further about your program and get some information. Maybe we can help some of the other institutions in this respect.
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Dr. MILLER. That would be wonderful. Thank you very much.
Mr. STOKES. Thank you.
Mr. PORTER. Thank you, Dr. Miller and Ms. Browman.
Wednesday, January 28, 1998.
WITNESS
JOHN F. NEYLAN, M.D., AMERICAN SOCIETY OF TRANSPLANT PHYSICIANS
Mr. PORTER. Dr. John F. Neylan, President-Elect of the American Society of Transplant Physicians, testifying on behalf of the Society.
Dr. NEYLAN. Mr. Chairman and members of the subcommittee, thank you for the opportunity to present testimony on behalf of the American Society of Transplant Physicians. I am John Neylan, Medical Director of Kidney Transplantation at Emory University and President-Elect of the American Society of Transplant Physicians.
The ASTP, which receives no government support, has over 1,100 physicians, surgeons, and scientists actively engaged in the research and practice of transplantation medicine and immunobiology, and represents the largest and broadest number of transplant professionals in the United States.
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Over the past 30 years, transplantation of solid organs has moved from experimental to accepted therapy, with over 20,000 performed in 1997 in the U.S. The success of this procedure has improved greatly, and now almost all solid organ transplant recipients have an 83 to 97 percent survival rate at 1 year. Much of this success can be attributed to the basic and clinical research that has been funded by previous NIH appropriations. Our better understanding of the body's response to foreign proteins has led to countless other breakthroughs in all areas of medical science.
However, we still have a long road ahead. During the next hour, four new names will join those 56,793 individuals in this country waiting for a solid organ transplant. By the time I get home to Atlanta this evening, 10 individuals will have died because the wait for a transplant was just too long. For those fortunate individuals who received this gift of life, the long term still holds potential pitfalls and obstruction for poorly understood mechanisms of immune destruction as they exact a relentless attrition.
But, Mr. Chairman, with increased funding for research, there is hope. Research is central to all that occurs in the transplantation process. The ASTP believes that we are on the threshold of many important scientific breakthroughs in the areas of immunology and cell biology.
We are on the threshold of developing safer and more effective means of immunosuppression to combat the complex pathways of acute and chronic rejection. With further insights, we may see the dawn of conditioning regimens that may one day lead to indefinite and drug-free immunologic tolerance. In parallel, we may devise means to overcome the daunting immunologic hurdles of xenotransplantation, the transplantation of a potentially limitless supply of animal organs.
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Finally, advances in developmental cellular biology may one day lead to reconstruction of human tissues and organs damaged by disease, and restore these to health and normal function. None of these goals are possible without strong support for research.
Because of this, the ASTP strongly urges the subcommittee to continue its leadership in the area of biomedical research and provide a 15 percent increase in funding for the NIH for fiscal year 1999. By continuing to increase the level of funding, this subcommittee will achieve the goal of doubling the NIH budget in 5 years, a goal supported by the ASTP, and as you have just heard, by the societies who are members of the Ad Hoc Coalition for Biomedical Research.
With this level of support, this subcommittee and the Congress as a whole will have the personal satisfaction of knowing that they are responsible for expanding the general transplantation research authority at the NIH, and in turn will be providing new hope to the countless citizens of this country who may one day benefit by these efforts.
Thank you, Mr. Chairman and all the members of your committee for allowing me to present the views of the ASTP and the transplant community.
[The prepared statement of John Neylan, M.D., follows:]
Offset folios 132 to 139 insert here
Mr. PORTER. Dr. Neylan, thank you very much for your excellent statement. You kind of give us a hope for the future that—I remember talking to a physician about 10 years ago saying that we are on the edge of being able to replace worn-out or defective organs, and now you are telling me that that is increasingly coming true, and all we need is a stronger commitment to research to make it happen even more rapidly.
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So we appreciate very much your testimony this morning. Thank you for being with us.
[CLERK'S NOTE.—Information required pursuant to clause 2(g)(4) of Rule XI of the Rules of the House of Representatives was not received from this witness or from an entity represented by this witness]
—————
Wednesday, January 28, 1998.
WITNESS
DR. HARRY S. JACOB, THE AMERICAN SOCIETY OF HEMATOLOGY
Mr. PORTER. Dr. Harry S. Jacob, President-Elect of the American Society of Hematology, representing that society.
Dr. Jacob.
Dr. JACOB. Good morning.
Thank you, Mr. Chairman, for letting me meet with you today. I am a professor of medicine at the University of Minnesota and President-Elect of the American Society of Hematology. Our society has over 8,000 active members, and probably hosts approximately 15,000 attendees at our annual scientific meeting. Our membership includes physicians who treat blood diseases and scientists who study the causes of those diseases.
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I would like to begin by thanking you for your sustained support of biomedical research, and by endorsing your vision that this support should be broad and flexible.
I shall take the next few minutes to discuss how your additional support might help us in our mission to improve patient care. I shall in this forum use two relevant examples: bone marrow transplantation and gene therapy. I will provide other examples in my written testimony.
Bone marrow transplantation is an increasingly-utilized and lifesaving procedure that was invented by hematologists. In fact, a member of our society, Don Thomas, recently won the Nobel Prize for his pioneering work in this area. At present, the procedure is used to treat patients with various forms of leukemia, as well as bone marrow failure and rare immunodeficiency syndromes which affect particularly children.
Also, promising new uses of the technique have been reported in hematologic patients with sickle cell anemia and Cooley's anemia. There is also growing excitement about its ability to treat nonhematologic diseases like systemic lupus, rheumatoid arthritis and multiple sclerosis.
The major limitation in bone marrow transplantation is that the procedure works best when patients have a genetically compatible brother or sister. Unfortunately, this favorable situation occurs for only 1 in 4 transplant candidates. Others must settle for riskier transplantations from genetically matched but unrelated donors, or they may have to forego the procedure altogether. Research is needed to understand how to safely transplant partially matched marrow so that this lifesaving procedure may become available to many more patients.
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The second example I would like to discuss is in the area of gene therapy, an area of great interest to all biomedical scientists and physicians. The bone marrow is the usual source of cells for gene therapy. Sickle cell disease, many forms of immunodeficiency, and severe bleeding disorders, like hemophilia, are just a few examples that may be amenable to correction with gene therapy.
The application of this technology is impeded, however, as we are still unable to regularly transfer genes into cells at high enough levels and maintain these genes in active form for prolonged periods of time. Fundamental research is needed to improve our understanding of these processes so this technology may become more useful. This will require a larger commitment to basic research, but successful resolution will reap enormous rewards for diverse patients. I predict that the appropriate use of gene transfer techniques will soon astonish even the most cynical, and remarkably improve our Nation's health.
The American Society of Hematology is committed to research because our members constantly confront the limits of medical knowledge as they treat catastrophic diseases of the blood, like leukemias, lymphomas, and sickle cell anemia. Our members are also clear that what they are able to do for their patients is based almost entirely on the fruits of previous research.
One example of our commitment to research is that we allocate a major fraction of our limited resources to the funding of fellowships and grants to young investigators. Our members know, however, that our site, although well-intentioned, can merely seed and supplement what the Federal Government sustains through its funding of research.
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We ask that you continue your commitment to all high-priority research by supporting a 15 percent increase in NIH funding for fiscal year 1999, as recommended by the Ad Hoc Group for Medical Research Funding. We fervently hope that you can build on the administration's proposal and develop a method to double the NIH budget in the next 5 years.
On behalf of our society, thank you for your attention today, and for your very hard work and generous past support.
[The prepared statement of Dr. Harry Jacob follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Dr. Jacob, thank you for your testimony. I am sure you are aware that whenever the subject of bone marrow transplantation arises, there is always at the table in this subcommittee a champion of that cause, and that is Bill Young of Florida. So we put that at a high priority, not only because of its intrinsic need and value, but because Bill was always there telling us we should. So we appreciate very much your testimony this morning. Thank you for coming down to be with us.
Dr. JACOB. It is a pleasure to meet you finally.
Mr. PORTER. We have now completed 10 of the 20 scheduled witnesses this morning. We have done so in approximately an hour. We are going to stand in recess for about 3 minutes, and then attempt to allow the next 10 witnesses the remaining hour. We stand in recess briefly.
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[Recess.]
Wednesday, January 28, 1998.
WITNESS
DR. WADI N. SUKI, THE AMERIAN SOCIETY OF NEPHROLOGY
Mr. PORTER. The subcommittee will come to order. Our next witness is Dr. Wadi N. Suki, President of the American Society of Nephrology, representing that society.
Dr. SUKI. Thank you, Chairman Porter, for allowing me the opportunity to speak before your committee this morning.
I am Wadi Suki. I am a professor of medicine and Chief of Nephrology at Baylor College of Medicine in Houston. I am the current President of the American Society of Nephrology.
Our society is the largest professional society in nephrology. We have a membership of some 6,500 clinicians and scientists dealing with function and disorders of the kidney, and dedicated to curing kidney disease. We are here to speak in support of the increased funding of the National Institutes of Health in general, and the National Institute of Diabetes, Digestive and Kidney Diseases, in specific.
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Mr. Chairman, this country has a very heavy burden in terms of patients with kidney disease. In our country today there are approximately 300,000 patients who suffer from end-stage renal disease. Their numbers increase by approximately 70,000 new patients every year.
The cost of taking care of these patients is staggering. The total cost approximates some $13 billion, and these patients are increasing, had been increasing at the rate of some 9 percent per year. Hypothetically, due to some of the advances in research, the rate of growth seems to be slowing down, but in the last 3 years has been on the order of about 7 percent per year. At this rate, we anticipate in the next 10 or 15 years there will be something between 600,000 and 900,000 patients with kidney failure in this country.
The major causes of kidney failure are diabetes and high blood pressure. These two disorders affect disproportionately some of the minority groups in our country, particularly Hispanics and African-Americans. The incidence of kidney failure in the African-American is 5 or 6 times that in the white American. The incidence of end stage kidney failure in the Hispanic American is 2 to 3 times that in the white American. So high blood pressure and diabetes represent major concerns for our society and our membership.
In spite of the huge cost to our Nation for treating patients with end stage renal disease, the expenditure on research on kidney disease is only about 2 percent of the total cost. We think that increased spending on research in this area will bear important fruit and hopefully increase the quality of life, save lives, and hopefully in the end save our country money as well.
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Now, major advances have been made in research in kidney disease in the past several years. Experiments in Dr. Brenner's laboratory in Boston in rats have led to innovations of treatment that now has been shown to reduce the development of kidney failure in diabetes by 50 percent. This is estimated to save this country something on the order of $2.6 billion in the next 5 years. This is a testimony to how good an investment in basic research is, in that it would eventually bear fruit at the clinical level.
There have been very many other advances in recent years. The gene for cystic kidney disease and a variety of other genetic and familiar diseases have been cloned and the defects identified. It is our hope that better understanding of the functions of all of these genes will culminate in cure and prevention.
There are a lot of other studies that demonstrate exciting new findings about arresting experimental nephritis in experimental animals. These advances are about to be tried in human beings, so there is a lot of promise for new breakthroughs and new advances.
There have been many advances. When we look back to when I started in nephrology back around 1965 and where we are today, some 30 years later, major advances have been made. Dialysis and transplantation were dreams. Now they are realities. Unfortunately, as you have heard from my colleague, Dr. John Neylan, neither treatment, dialysis, nor transplantation is currently perfect. Many more advances need to be made and much more research needs to be made.
[The prepared statement of Wadi Suki, M.D., follows:]
"The Official Committee record contains additional material here."
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Mr. PORTER. Dr. Suki, our time is up. Let me say two things. First, and I say this often, but I want to emphasize it; none of us up here consider these matters in a vacuum. My wife has diabetes, so I know the dangers of that disease and how it affects the population of our country and the whole world.
Secondly, I recently have been shown some home dialysis units that promise, they tell me, to vastly reduce the cost, which is a major cost in your field. Do you have anything to tell us about that technology and whether it is as promising as I have been led to believe?
Dr. SUKI. Yes. Indeed, dialysis in the home is now feasible and has been for some time. It is practiced in most large centers and in many smaller ones, as well. Somewhere between 12 and 15 percent of patients on dialysis actually do undergo dialysis in the home, and dialysis is being perfected, but I think the point that I have tried to make earlier is that dialysis does not replace the normal kidney, and therefore, our task should be to try to prevent kidney disease.
Mr. PORTER. Absolutely. My understanding from the people that I talked to is that if this could be very broadly adopted, that it would save a great deal in terms of health care costs and, in a sense, free up dollars that could be spent for research in finding a way to prevent or cure the disease.
Dr. SUKI. Very importantly, it could save a great deal in terms of personal hardship. Patients on dialysis have to travel to a dialysis facility 3 times a week, which is a considerable hardship and cost.
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Mr. PORTER. Thank you very much for your testimony, Dr. Suki.
Dr. SUKI. Thank you for allowing me to be here and for your support of biomedical research.
—————
Wednesday, January 28, 1998.
WITNESS
JOSHUA M. JAVITS, ALS ASSOCIATION
Mr. PORTER. Joshua M. Javits, a member of the board of trustees representing the ALS Association.
Mr. Javits, it is nice to see you again. Thank you for joining us.
Mr. JAVITS. Thank you very much.
Mr. Chairman and members of the committee, thank you for the opportunity to testify before the committee on behalf of the Amyotrophic Lateral Sclerosis Association and the 30,000 Americans afflicted with ALS, most commonly known as Lou Gehrig's disease.
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Speaking for myself and ALS, I would like to express my deep appreciation to you, Mr. Chairman, and the members of your subcommittee who over recent years have provided strong leadership and support for the research projects on ALS being undertaken by the NIH and the NINDS. I would also like to offer support for congressional efforts to double NIH's research funding over the next 5 years. As we all understand, a dollar spent on the disease today saves many lives and many dollars over the years to come.
We pledge to work with you, Mr. Chairman, and your colleagues on that crucial initiative. As you know, for the last 6 years the subcommittee has included language in the Labor-HHS appropriations bill that is specifically aimed at increasing the funding for ALS research at NIH within the existing NINDS budget. All of us associated with ALS are appreciative for these efforts and we look forward to working with the subcommittee this year and in the years to come until ALS is eliminated.
Mr. Chairman, I am a son of the late Jacob Javits who proudly served this country for 24 years as a Senator from the State of New York and as a Member of the House of Representatives for 8 years. I stress this point because my father died in 1986 after a valiant struggle with ALS, a degenerative and always fatal disease. While in Washington and later in private life, my father maintained an amazing schedule of practicing law and giving speeches and writing articles despite his diagnosis with ALS. As the disease progressed he required life support, including a respirator and a wheelchair, and eventually becoming virtually paralyzed.
ALS has also claimed the lives of other notables, and took the name of the baseball great, Lou Gehrig, as well as actor David Niven, jazz great Charles Mingus, boxer Ezra Charles, and former Vice President Henry Wallace, and the developer James Rouse.
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I serve as a member of the board of trustees of ALS. And ALS, to be specific, is a progressively degenerative neuromuscular disease that attacks the nerve cells and pathways in the brain and spinal cord. Motor neurons, among the largest of all nerve cells, reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When these motor neurons die, as with ALS, the ability of the brain to start and control muscle movement dies with them. The voluntary muscle action is affected and patients become immobilized, eventually leaving the person unable to speak or to eat or to breathe. Yet throughout the degeneration the mind remains completely intact.
ALS was first identified in 1869, but only recently have there been real discoveries that significantly advance the medical and scientific community's understanding of the disease, which underscores our belief that a cause and a cure can be found. To date there are no known causes, it is not known what the cause is, nor is there a cure for those afflicted with ALS. The majority of people diagnosed live only 2 to 5 years. Over 5,000 new cases are diagnosed each year.
ALS knows no boundaries and can strike anyone, regardless of age, race, color, or geographic origin. It is not a rare disease. It is projected that about 300,000 Americans alive and well today will ultimately die from ALS, more than die from Huntington's disease or multiple sclerosis.
Mr. Chairman, I want to mention something we are very proud of. Last year, with the inspired leadership of the late Congressman Walter Capps and Chairman Ben Gilman, H.R. 2009, the Amyotrophic Lateral Sclerosis Research and Treatment Act of 1997, was introduced in the House.
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This bill was the first bill Congressman Capps introduced while in the House and we are deeply honored by that fact. We are appreciative to Chairman Gilman for carrying forward the legislation in the wake of Congressman Capps' tragic death. The bill would allow Medicare to cover the cost of drugs used to treat ALS and would waive the 24-month waiting period for disabled persons to be covered by Medicare.
The last point is especially critical for ALS patients, who on average do not live more than 2 to 3 years after they become disabled. We believe that Congress, out of a sense of fairness and sound public policy, should look favorably on this legislation. To date 61 of your colleagues have cosponsored H.R. 2009 and we are hopeful that this number will continue to grow.
In the area of research, a major portion of ALS Association's annual budget is committed to fund ALS-specific research. The Association's grant program follows the format and rating procedures established by NIH. The Association, in the last 24 months, has funded over $4.7 million in funding. I would point out that the Association's research effort amounts to about 30 percent in addition to what NIH puts into ALS research.
Mr. Chairman and members of the committee, please accept our sincere appreciation for all the support you have provided for those afflicted with ALS and their families, the 30,000 people afflicted with ALS. We strongly urge Congress to continue the subcommittee's commitment to long-term medical research and to provide the funds necessary for NIH to carry out the mission. Thank you very much.
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[The prepared statement of Joshua Javits follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Mr. Javits, thank you for again appearing before our subcommittee. You have been a tireless advocate for preventing and controlling this terrible disease, and your father was a man who very clearly made a difference in this world. I think you are making a difference as well, and we very much appreciate your strong advocacy in bringing the dangers and scourge of this disease to our attention. Thank you for being with us.
Mr. JAVITS. Thank you for your very kind words, Mr. Chairman.
[CLERK'S NOTE.—Information required pursuant to clause 2(g)(4) of Rule XI of the Rules of the House of Representatives was not received from this witness or from an entity represented by this witness.]
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Wednesday, January 28, 1998.
WITNESS
EDWARD L. SNYDER, M.D., AMERICAN ASSOCIATION OF BLOOD BANKS
Mr. PORTER. Edward L. Snyder, M.D., Director of the Blood Bank at Yale Medical School and Yale-New Haven Hospital in New Haven, Connecticut, and President of the AABB, representing the American Association of Blood Banks.
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Dr. Snyder.
Dr. SNYDER. Thank you. Mr. Chairman and members of the subcommittee, my name is Ed Snyder. I am Director of the Blood Bank at Yale, and the Yale-New Haven Hospital in Connecticut. I am testifying today as President of the American Association of Blood Banks in support of increased funding for NIH and NHLBI.
The AABB is a professional society for 8,500 individuals involved in blood banking and transfusion. We represent 2,200 institutional members, including community and Red Cross blood centers, and hospital blood banks that collect, process, and transfuse blood. Our members are responsible for virtually collecting all of the blood in the United States, and more than 80 percent of the blood transfused.
Throughout its 50-year history, the AABB's highest priority has been to maintain and enhance the safety of the blood supply. NHLBI is currently sponsoring several important transfusion medicine projects. There are, however, important opportunities in this field that require additional investigation to assure the safest possible blood supply.
I would like to emphasize five of these. First is detection of transfusion-transmitted disease. Despite the great progress that has been made in selection of donors who are at low risk for disease transmission and the use of and improvements to tests to eliminate infected donors, prevention of AIDS and other transfusion infections remains a top priority of transfusion researchers and all recipients of blood.
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Each improvement to testing has led to a decrease in the window period, the period of time between infection with a viral disease and the ability to detect the virus by a screening. The risk of acquiring viral disease through transfusion is lower than ever, yet worldwide travel and demographics could spread new viruses, bacteria, and parasites into the U.S. blood supply. I call your attention to the concern about the spread of CJK or mad cow disease into the blood supply in the United Kingdom. Prevention of transfusion-transmitted disease remains a priority of transfusion research.
Secondly is the role of biologic response modifiers in transfusion reactions. Studies have identified several substances released by human cells which play a role in changing the patient's response to transfusion. These adverse responses, known as transfusion reactions, range from fever and chills to severe allergic reaction, shock, and even death. Studies on the role of these chemicals and adverse reactions to transfusion and research into how to modify and control them is now necessary.
Third is the immunology of transfusion. Even though blood transfusion is a lifesaving therapy, transfused components are still recognized as a foreign substance by the human body. Blood transfusion can produce adverse changes in the body's immune system. This includes decreasing the natural defense of the patient for their fight against bacterial infection, or by decreasing their ability to prevent cancer recurrence. Lifesaving transfused blood may thus actually promote disease. Bone marrow transplant patients, cancer patients, and other immunosuppressed recipients are all at risk for immunologic complications. Fundamental basic research is needed to gain knowledge on how to combat these aspects of transfusion.
Fourth is stem cell research. The AABB is pleased that NHLBI is funding a 5-year study of transplanting stem cells collected from cord blood. Stem cells may become the ultimate vehicle for curing diseases through gene therapy. This initiative, however, poses new questions, and we support additional research in this area.
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Lastly, transfusion medicine, research training, and its clinical infrastructure needs further evaluation. Our infrastructure is currently nonexistent. We support development of linked centers of transfusion excellence for research and training. Such centers could provide a critical mass of resources needed to accomplish NHLBI-sponsored research in transfusion medicine.
The American Association of Blood Banks endorses a 15 percent increase in NIH funding for fiscal year 1999 as a first step towards doubling the goal of NIH—doubling the NIH budget over the next 5 years. This recommendation is consistent with congressional support for doubling the amount authorized for basic science and medical research for a number of research agencies, including NIH. This level of funding would also sustain the rate of growth NIH has experienced in the past decade.
Mr. Chairman and subcommittee members, on behalf of the many NHLBI-funded transfusion scientists, thousands of health care professionals, and millions of transfusion recipients, we appreciate the opportunity to discuss Federal support for research and transfusion medicine before the subcommittee. Thank you.
[The prepared statement of Edward Snyder, M.D., follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Dr. Snyder, can I ask a question? The best way to avoid the transmission of diseases through transfusion is for an individual to have their own blood preserved; is that not correct?
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Dr. SNYDER. The best way to avoid it is to avoid the transfusion.
Mr. PORTER. But if you have to have one, have your own blood supply?
Dr. SNYDER. You are absolutely right.
Mr. PORTER. How long does blood last if someone were to provide their own blood in sufficient quantity? How long could you keep it?
Dr. SNYDER. In the liquid state, it can be stored up to 42 days. Frozen, it can be stored up to 10 years.
Mr. PORTER. Ten years?
Dr. SNYDER. Ten years, frozen.
Mr. PORTER. In the frozen state, is it usable in most cases of transfusion?
Dr. SNYDER. Yes. The red cells you can freeze. It is a fairly complex issue. If you need platelets which help blood clotting, that cannot be frozen in most cases. It can in some special situations. Plasma can be frozen for up to a year. Red cells can be frozen for 10 years.
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Mr. PORTER. Are they the most significant in transfusions?
Dr. SNYDER. It depends on the illness. If someone has cancer, they may need platelets more than red cells. There is no easy answer.
Mr. PORTER. If you could, by law, require people to put aside at a certain age so much of their own blood, that might help a great deal in providing blood for transfusions if they need them, and also to prevent the transmission of diseases through transfusions; correct?
Dr. SNYDER. The Association certainly recommends that people donate their own blood for their own use wherever possible, and also those who are healthy enough and pass the required screening tests, donate for the general blood supply. We don't have enough blood donors to keep the Nation's blood needs kept going.
Mr. PORTER. Sometimes I think that people just need to be encouraged to do things that they would naturally want to do but don't have enough push to do it. I have also thought if we could simply tell people that when they reach Medicare eligibility, they have to make a decision. They don't have to do it, but they have to make a decision whether they want to leave a living will or not. They have to make a conscious choice as to whether they want heroic measures applied if they become what a doctor would determine as terminally ill.
I think most people would do that without any problem. I don't think they would have any problem with it. Some would decide they don't want to make a living will and would want the procedures, but most I think would say, no, not at that point. We will trust medical judgment and allow them to make the decision for us.
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It seems to me that maybe perhaps if at the same time we said, wouldn't it be a good idea to put aside some of your own blood if it is needed, we might accomplish a great deal in a very practical way, without any kind of intrusion on people's rights.
Dr. SNYDER. Certainly using such blood for that same individual would be reasonable. The concerns that are shared by the members of the community and the FDA would be if people were asked to donate blood for other people, with a similar sense of urgency, because of the concern over the accurate receipt of the blood donors' screening, that people are not donating for reasons other than altruism, because of the studies that have shown that when people are asked to donate and are urged to donate perhaps a little too much, that they may not be 100 percent truthful in the donor history.
It is a very complicated issue. But for autologous, as you appropriately point out, it would be reasonable to encourage people to donate their own blood for surgery that is planned.
Mr. PORTER. Thank you, Dr. Snyder. I have taken much more time than I should, but I am interested, obviously, in the subject.
Dr. SNYDER. Thank you very much.
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Wednesday, January 28, 1998.
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WITNESS
ALAN G. KRAUT, Ph.D., AMERICAN PSYCHOLOGICAL SOCIETY
Mr. PORTER. Allen G. Kraut Ph.D., Executive Director of the American Psychological Society.
Mr. KRAUT. Mr. Chairman, I am delighted to be here on behalf of the members of the American Psychological Society, many of whom receive NIH grants for such topics as brain behavior, addiction research, human development, aging, mental illness, hearing and vision research and chronic pain, to name just a few.
I ask that my written statement be entered into the record and I will just summarize here.
Let me begin by expressing our sincere thanks for the increase you have already given NIH, and to say that we will work hard with you, including visiting the Committee on the Budget, to double the NIH budget over the next 5 years.
As part of the Ad Hoc Group for Medical Research Funding, we are asking for a 15 percent increase in NIH as a first step towards that goal. But can NIH absorb this increase so quickly? I know that behavioral research stands ready. We are poised, both in terms of the role behavior plays in serious health problems and in the field's capacity to proceed in many important directions.
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Last fall, Mr. Chairman, during your hearings on the mind, body, and health, we were all encouraged to hear you talk about finding ways to open the door to increase NIH's receptiveness to behavioral science research. Let me suggest that one way would be to make this open door theme a standard part of your conversations with Institute directors as they appear here in front of you. We think that even a single consistent question from you, such as can you tell me how you might be encouraging behavioral research in your Institute, would send this important message.
Increasing NIH's receptiveness is my main concern today. Some of our Nation's most critical health questions involve behavior: What goes on in the thinking of young people that leads them to start drinking, smoking, taking drugs? When do we acquire patterns of behavior that may be with us for the rest of our lives? What about connections between stress and health? How does genetics interact with behavior? What can we do to help memory as we age?
There are many others, but NIH resistance to the science that addresses these kinds of questions continues, despite recommendations from you, the National Academy of Sciences, even from its own Institutes. One example is how NIH responded, or rather didn't respond to recommendations from the National Academy of Sciences to increase training for behavioral scientists specifically through national research service awards.
In a congressionally mandated assessment of training needs, the National Academy said that the size of NIH stipends should be increased and that the number of awards for behavioral science and for several other areas also should be increased. Since 1995, this committee and the Senate committee have been asking NIH to implement those recommendations, and after a 3-year delay in responding, the NIH now says the amount of money awarded in the NIH stipend will be increased but not the number of awards in behavioral science or in other areas. NIH cites budget concerns for this selective implementation, saying that neither National Academy members nor Congress ''fully appreciated the costs.''
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What are the costs? The National Academy's behavioral science recommendation to add fewer than 400 trainees would add about $4 million to the NIH budget, spread over 3 years and across all the Institutes. The cost clearly is not the concern here. So once again we ask the committee to encourage NIH to increase the number of their research service awards.
I should note that several Institutes have their own recognized needs for more behavioral researchers. With encouragement from this committee and from the Senate they have developed small grants called BStart that are awarded to new Ph.Ds in behavioral science. We ask you to continue to encourage the use of BStart throughout NIH.
NIH now has an Office of Behavioral and Social Sciences Research. That is the logical place to take a lead on these training objectives and on cross-cutting behavioral research priorities. But OBSSR's budget is tiny, only $2.6 million. We ask that that budget be increased, even to approach $20 million. We are in an era of exceptional promise in behavioral science, but we need a more encouraging Federal environment in order to realize its potential.
In my written statement, I have asked for your support for the behavioral science initiatives that are taking place in several individual Institutes. But despite this activity, NIH still needs to recognize that behavioral science is a core element in its mission of research and public health.
This committee has shown extraordinary largesse in the annual NIH budget and we are very grateful for that support. Now we are asking you to encourage NIH leadership to take meaningful steps to ensure that the health of the Nation receives the full benefit. Thank you.
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[The prepared statment of Alan Kraut follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Mr. Kraut, thank you for your excellent statement. I have to say that obviously I agree with where you want to go. The process by which the Congress interacts with NIH and attempts to put its opinion into their thinking is a very complex and subtle. We do not feel that it is up to us to tell NIH what to do. They are scientists and we are not.
We do have matters that we are very concerned with personally, and that we encourage them to address. The hearings or briefings that we had on the mind and its role in health and healing was meant to convey a message to them that this is an area that we think has been neglected in a broad sense, and to encourage them to be more responsive. So we will continue to press on that process without the heavy hand of direction. We will have to see what comes of that, but it is something that is part of a much broader process.
We realize also that NIH doesn't exist in a political vacuum within the scientific community either, and that scientists are at all times attempting to influence the directions of NIH and the priorities that NIH undertakes. So it becomes an even broader process where we all kind of impact this great institution that we all admire and respect and try to move it a little bit in the direction that we think it ought to go. We appreciate very much your testimony, again, and thank you for being with us. We will do our best to be responsive.
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Wednesday, January 28, 1998.
WITNESS
KAREN MURRAY, COALITION FOR HERITABLE DISORDERS OF CONNECTIVE TISSUE
Mr. PORTER. Karen Murray, member, National Marfan Foundation and Chair, National Marfan Foundation's Have-A-Heart Campaign, testifying in behalf of the Coalition for Heritable Disorders of Connective Tissue.
Mrs. MURRAY. Good morning, Mr. Porter. I am Karen Murray, a member of the National Marfan Foundation, member organization of the Coalition, which represents more than one-half million Americans affected by heritable disorders of the connective tissue. Marfan's syndrome is one of 200 such disorders which include names most of us have never heard of unless or until a family member is diagnosed with one.
My son Michael was born August 13, 1991, 6 years ago, in one of the top hospitals in New York City. He was born with a dislocated hip, long fingers bent backwards at the knuckle, and an indented chest bone. I overheard the physicians discussing among themselves, but not directly with me, the possibility of Marfan syndrome. They sent Michael and me for echocardiograms, which are scans of the heart, pronounced us fine, and released us from the hospital.
The same doctors continued to follow Michael for the next 5 years, during which time he grew faster, longer, taller, and more awkwardly than all of his peers, and his indented chest became more pronounced. I went to all kinds of specialists, all but a cardiologist. I would constantly describe Michael's symptoms: Why is he so tall, thin, awkward, loose ligamented, with an indented chest, long fingers, arms, and legs? I was told to let it go, he is a beautiful, healthy child. But I knew there was something wrong.
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For Michael's fifth birthday, I bought him an Apple computer which came with a free CD ROM called the Family Doctor. Late one night, when he was sleeping, I clicked into it. As I read the first paragraph under Marfan syndrome, I recognized the word Marfan syndrome; children with Marfan syndrome grow taller than their peers. They have indented or protruded chest bones. Fingers are disproportionately long, excessive joint mobility, muscle weakness, and so on.
The very next morning, after not sleeping, I brought him back to the same prominent New York City hospital where he was born and 5 hours later, after demanding an echocardiogram, my diagnosis was confirmed. Michael's aorta was indeed dilated and he did indeed have Marfan's syndrome.
Marfan's syndrome is a genetic disorder that affects the connective tissue throughout the body. In almost all cases it affects the heart and the aorta. Over 500,000 Americans are affected by Marfan's syndrome and related connective tissue disorders. At the time of Michael's diagnosis his aorta was already dilated. Severe dilation can lead to dissection, which is what we know as an aneurysm.
If medical personnel can't recognize the signs, the outcome is usually fatal. My point is this. Doctors at the best hospital in New York City, extremely aware of Marfan's syndrome and who suspected Marfan's syndrome at birth, failed to make the diagnosis. In the hands of the best medical personnel in New York City, my son Michael slipped away. Had I not heard the word Marfan or bought him a computer, Michael would have been another statistic in his teens.
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But it doesn't have to be that way. Although Marfan's syndrome is incurable and a progressive disorder—which is difficult for me to say those words—it is diagnoseable and treatable. So I have hope. As a parent of a 6-year-old Marfan child, I am nervous but optimistic that Michael will be okay. I now know to have his heart monitored every 3 months. I have him on medication in the hope that his aorta will be less stressed.
I live daily with the fear of when or how often will Michael's aorta dissect. Marfan's syndrome also affects all tissue throughout the body and presents orthopedic problems such as loose joints, scoliosis, elongated, uneven bones in the ribs, chest wall, spine, hips and legs. I cannot express as a mother the concern and the amount of questions I have.
There is still so much I don't know, and I am often told, I am sorry, we haven't researched that yet, especially not in children. Most children who go undiagnosed and untreated die at a young age. Today, tens of thousands of children go undiagnosed. Before Michael was born I never heard the word Marfan. I never thought about connective tissue.
Now there is not a night that I don't fall asleep without wondering or worrying about the fact that I am giving my son calcium channel blockers every single day in the hope it is helping his aorta, but what are the side effects after years of calcium channel blockers? No one can tell me. I keep asking if calcium channel blockers are okay for children. They don't know. There is no research.
Can and should Michael exercise at all? Can he take a regular gym class? They don't know what to tell me. What is more effective, surgery or a brace for his scoliosis? Do children with Marfan syndrome have weak tissues in their respiratory system? Is that why my son also has asthma, since it is not in my family? Why do Marfans' lungs collapse? Since Michael has so many orthopedic issues, what can I do to help him live a life of less chronic pain? I am scared to have another baby, but I would love to. Are there tests I can take to determine the health of the fetus?
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Research is needed in all of these areas. It is essential. Undiagnosed Marfan patients end up in the emergency room. The emergency room staff does not know what to look for and most Marfan patients die. I am fortunate that I at least know that he is diagnosed. The key is to build awareness and educate the medical community, from OB–GYNs to pediatricians to emergency room doctors, so they can recognize and diagnose Marfan's syndrome. Early diagnosis and careful daily management is critical in order to preserve and enhance the life of a Marfan patient.
Similar challenges are faced by other heritable disorders of connective issues. The advances in genetic research today bring hope to the one-half million individuals affected with these disorders. Yet more dollars are needed. The Coalition supports the proposal of the Ad Hoc Group for Medical Research Funding, which calls for a 15 percent increase in funding for the NIH in 1999 with a view towards doubling the budget over the next 5 years. Please help fund these important programs so we can learn more about how to care for Michael and so the medical community is more aware of Marfan syndrome and heritable disorders of connective tissues, so that lives can be saved. Thank you.
[The prepared statement of Karen Murray follows:]
"The Official Committee record contains additional material here."
Mr. PORTER. Mrs. Murray, I can't think of a more compelling case for providing resources for both research and helping the medical community understand and diagnose this disease. We very much appreciate your testimony.
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I think Marfan's only came to the attention of the American people recently, in respect to Abraham Lincoln. There was some thought that perhaps he was a victim of that disease. I think they even were going to—and I can't remember the outcome—look at the bone, the DNA.
Ms. MURRAY. They still are. It is suspected that he had Marfan's syndrome, along with Chris Patton and lots of other famous people. They suspect John Larsen, the director and writer of Rent, and lots of other famous people.
Mr. PORTER. The fact that famous people may have such a disease very frankly often helps the American public to understand the disease better. It gets their attention. I often tell—we have a lot of celebrities come and testify before this subcommittee regarding various diseases, and I think that helps a great deal for people to understand the disease, and gets them focused on the fact that even famous people suffer from these diseases, and that I think helps build us a support base for the research that we need to address them.
Ms. MURRAY. And more awareness.
Mr. PORTER. Thank you for coming to testify. I really appreciate that.
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Wednesday, January 28, 1998.
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WITNESS
DEBORAH KLEIN WALKER, ASSOCIATION OF MATERNAL AND CHILD HEALTH PROGRAMS
Mr. PORTER. Deborah Klein Walker, Ed.D, President, the Association of Maternal and Child Health Programs, testifying on behalf of the association.
Dr. WALKER. Thank you. I am Deborah Walker, President of the Association of Maternal and Child Health Programs. In Massachusetts, I serve as the Assistant Commissioner of Family and Community Health within the State Health Department. Thank you very much for the opportunity for us to appear before you today.
On behalf of the Association, I appreciate the subcommittee's support of the maternal and child health services block grant. The block grant forms the essential framework on which States have built and maintain their systems of care for women, children and youth.
For over 60 years the MCH programs have helped to reduce maternal and infant mortality, improve the health of newborns by preventing life-threatening diseases, improve the health status of school-aged children, prevent adolescent pregnancy and other at-risk adolescent behaviors, and assist children with special health care needs.
State MCH programs carry out core public health prevention activities, as well as provide direct services for women and children who lack the necessary health care. Investments in public health, including MCH services, is cost-effective and results in improved health outcomes.
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For example, newborn screening prevents chronic diseases and disabilities through early detection, diagnosis, and treatment of disorders. In Illinois, 99 percent of all newborns are screened for at least 6 disorders, including sickle cell anemia. Tobacco use, exposure to environmental tobacco smoke, poses exceptional and immediate risk to pregnant women and children. In 1995, the estimated costs of birth complications attributed to smoking were conservatively estimated at $1 billion. State MCH programs have worked in partnership with community-based groups and the private sector to prevent and end nicotine addiction.
I would like to also add, you have heard a lot about a very important research, but it takes an infrastructure to get the results of this research to the public at the lay level in the community. The MCH block grant is such a vehicle. MCH funds have supported major campaigns like the ''Back to Sleep Campaign,'' which has drastically brought down deaths due to SIDS, and is also funding campaigns on the importance of folic acid for pregnant women, so that spina bifida will be prevented.
In addition to these public health prevention strategies, the MCH block grant directly serves over 17 million women and children, including almost 1 million children with special health care needs. The health of our Nation's pregnant women and children has improved dramatically over the 50 years.
Through advancements in medical technology and improved access to care, we have seen significant reduction in many adverse health outcomes. However, many things still fall short of where we want to be, especially in our underserved and minority populations. For example, in the last 15 years, infant mortality for both whites and blacks has decreased cons