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reached can be applied to develop more advanced disease management strategies for our patients.

APPROPRIATE MANAGEMENT OF FEDERAL DOLLARS AND INCREASED SUPPORT FOR

ARTHRITIS RESEARCH ARE CONSISTENT GOALS The ACR recognizes that all those who come before the appropriations committees to advocate funding levels above the Administration's proposed budget for specific programs should expect to provide reasons why their programs deserve to be singled out for favorable treatment. More than ever these reasons must be “airtight. As physicians and scientists engaged in research on arthritis and related diseases, we openly welcome Congress' justified attention to federal "cost-cutting," because the reason we present for increased federal funding for arthritis research funding is such a good one: arthritis research is cost-effective.

Although arthritis costs this country over $25 billion each year in medical costs, lost income and reduced productivity, research can reduce these costs. Arthritis research is just beginning to yield significant improvements in treatments for patients and better management strategies for their diseases. Yet, much more remains to be done. With an increased federal commitment, existing management strategies can be built-upon to generate improved treatments and therapies applicable to a larger portion of patients. As the transfer of technology takes place, cost-savings will result as a smaller portion of people with arthritis require custodial care, fewer disability cases will require insurance payments, and less disability relating displacement and related insurance costs will be incurred.

One recent study estimated that a 30 percent reduction in morbidity associated with osteoarthritis, ant reduction of between 25 and 50 percent in morbidity associated with rheumatoid arthritis, could be expected by the year 2015, if scientific research continues "on course.” With this, a reduction of between $160 and $179 billion in costs associated with these diseases could be gained over the 25-year period from the present through the year 2015. However, these predicted savings cannot be realized unless federal funding for arthritis research continues to be made a high priority. For fiscal year 1992, the ACR supports an increase of $47 million over the President's proposed budget for the NIAMS. There Is no question that this recommended increase is modest compared to the substantial health care cost-saving we have the potential to achieve.

For research funding for arthritis and related diseases, we have reached a "critical mass” as we assess “expenditures versus “results.” If we do not continue at our current pace, we risk losing our forward momentum, and face the prospect of losing out on many important opportunities in arthritis research a advances in patient care which are within our grasp. Moreover, we turn our backs on mtential costs savings; something that, in these economic times especially, we cannot afford to do.

NIAMS' RESEARCH AND FUNDING ISSUES Last year represented a turning point for the NIAMS, as Congress provided the institute with a significant funding increase in order to enable it to fund more of the research within its broad mandate. Collectively the disease areas within the NIAMS purview are the leading cause of limited mobility and the second-leading cause of restricted activity in the United States. These diseases affect a range of individuals from children to the elderly. As a result of favorable treatment by the Congress in the fiscal year 1991 funding bill, several important research achievements have been made. Only with continuation of funding increases can these advances be translated into improved disease management strategies. The following examples illustrate what has been accomplished so far, as well as what remains to be done.

Over 16 million Americans suffer pain, stiffness and loss of mobility as a result of osteoarthritis (OA). OA causes the protective cartilage of the joints to fray, wear and ulcerate, and in extreme cases, to disintegrate entirely. OA is also the major reason for the more than 150,000 total joint replacement operations that are now performed each year in the U.S. For the first time, a gene has been found that causes one form of osteoarthritis, and perhaps can cause other hereditary forms of the disease, including the common types affecting the hands. From a patient care perspective this finding is important in terms of being able to recognize those individuals who are at risk for osteoarthritis. The second step, only possible with additional funding, will be to develop intervention measures aimed at preventing joint breakdown, thereby gaining the ability to halt the disease process.

Another exciting breakthrough was achieved in the area of research on spondyloarthropathies, those forms of arthritis that affect the spine (as well as other joints). For some time, we have known that a specific genetic marker is found in

eases.

the vast majority of patients with spondyloarthropathies. Using an animal model, researchers have now demonstrated that the marker may also be a major cause of the disorders. This work constitutes the first step In efforts that will allow physicians to more effectively diagnose these diseases in humans, and with additional research, to find possible means of prevention or cures for this class of disabling dis

The NIAMS' research grant program has benefited substantially from the $24.7 million increase Congress asforded the institute in fiscal year 1991. The increase will allow this relatively small institute to support approximately 221 competing research grants in fiscal year 1991, compared to 149 funded in fiscal year 1990. The ACR believes that funding additional grants the institute's single-most important responsibility in its efforts to speed scientific knowledge toward applications relevant to patient care. For fiscal year 1992 funding, we urge the Congress to be cognizant of the importance of maintaining stability in research support, especially in the case of the NIAMS, where a large number of high quality research grant proposals in many diverse fields are available to be conducted as soon as additional federal funds become available.

The institute's clinical trial program is another crucial mechanism which works to translate science into patient care advances and health care cost-savings. Because the NIAMS has a relatively small clinical trial budget (less than 4 percent of the institute total) funding limitations have compelled the institute to extend the planning horizon for some clinical studies, so that it will be conducting them over a greater number of years than would otherwise be the case. When this happens, less research data is generated over time, and a delay results in obtaining information about the efficacy of the treatment strategies under study. Under the Administration's request for fiscal year 1992, the NIAMS will be unable to start any new clinical trials, due to inadequate funds. The ACR has identified at least 9 clinical studies which should be conducted, and are awaiting susficient funding to be carried out. One of particular interest to ACR, would focus on early and aggressive therapies for rheumatoid arthritis. There is growing conviction among rheumatologists that early and aggressive use of combinations of drugs should be evaluated for the treatment of RA. The rationale for combining drugs is that the disserent modes of action of the drugs can be exploited, when used together, to improve the efficacy of the drugs. Use of drug combinations may also mean that lower doses of drugs could be effective, and problems associated with toxicity thereby limited. With sufficient federal funding, a systematic approach to testing many possible drug combinations could be carried out, possibly modeled on the successful evaluations of cancer drug combination therapies. Support for long-term follow-up to examine the impact of new drug combinations as compared to current practices also would be an essential component of this research initiative.

If additional resources are provided in the institute's fiscal year 1992 budget, a clinical trial aimed at answering questions concerning treatment for the late manifestations of Lyme disease (such as chronic erosive arthritis and neurologic complications) should also be carried out. Lyme disease has emerged as the most common insect-borne disease in this country, in part due to an increased number of deer (on which disease-carrying ticks feed) especially in populated and recreational areas. Because of the increased distribution of this complex infectious disease, the NIAMS has assumed a leadership role in both research and outreach programs related to Lyme disease.

Several areas within-the broad research mandate of the NIAMS have elicited strong Congressional interest. Osteoporosis is one of the primary health interests of several Members. As the lead institute for the study of osteoporosis, the NIAMS organized a national scientific conference on research advances in osteoporosis in February, 1990. Recommendations for future research directions and opportunities resulting from that conference were incorporated in a report to Congress last year. In order to continue to stimulate the greatest possible effort in research on osteoporosis, the NIAMS has issued two requests for applications on osteoporosis in the current fiscal year. A coordinated effort to study the potential therapeutic uses of bone growth factors will be a priority. The NIAMS plans to hold a workshop to discuss current knowledge and identify research gaps and opportunities regarding these growth factors, and to begin to identify promising areas of study. A clinical trial to evaluate the long-term effects of exercise and diet on bone mass, focusing on teenage girls in school-based programs, is also among the studies that the Institute could initiate if additional funds are provided in fiscal year 1992.

Another area of high Congressional concern and interest is lupus, an autoimmune disease that disproportionately affects women of childbearing age, and affects black women more frequently than white women. The NIAMS has set up a task force on Lupus in high risk populations in response to findings that certain populations (young, black women specifically) experience not only an increased incidence of the disease, but also disproportionately higher mortality. The NIAMS is developing information kits which will help health professionals and community organizations conduct health promotion programs targeted to young, black women. Future efforts will be instituted to focus on other minorities who also suffer disproportionately from Lupus. In addition, an upcoming NIAMS-sponsored international workshop on lupus will assess current knowledge about Lupus and will lay the groundwork for development of future research directions, including those relating to the pathogenesis of the disease, environmental factors, reasons for the disproportionate prevalence in women and blacks, and possible therapeutic avenues.

The need to keep the momentum going in light of the potential we now have to achieve significant advances.

Especially in the last year, members of the House and Senate Appropriations Committees have become increasingly aware of the opportunities which exist in the field of arthritis research. In crafting the fiscal year 1991 bill, Members acted favorably on the request of the research and patient communities concerning NIAMS funding. In these times of limited federal dollars, we ask you to again understand the importance of arthritis research funding as it relates to savings in national health care costs; and that you act favorably on our request for a $47 million increase in funding for the NIAMS, for fiscal year 1992. There is no question that more than this amount of federal support is scientifically justified. However, the ACR wishes to go on record with a sense of realism and understanding of the budget constraints that Congress is faced with, just as you have acted with understanding of the arthritis community's needs.

The ACR looks forward to a continued strong relationship with Congressional decision-makers, not only in the short term, but in the long-term as well. For this reason, we ask for your continued willingness to assess the "big picture" of national hearth care costs, and that you take action on fiscal year 1992 arthritis research funding levels that is consistent with lowering those costs. The ACR would like to express appreciation to the Committee for your continued sensitivity to the critical need for federal support of research to end pain, disability and dependency which affects many of your constituents and others who have arthritis and related diseases. The future welfare of these people, and the future economic welfare of our country will both benefit from a stronger national commitment to arthritis research. If our federal investment in arthritis research continues with the same momentum begun last year, Members of Congress should feel confident that the dollar appropriated will increasingly finance preventive care, and will decreasingly have to go toward the support of "sick care." As our nation faces a long-term future that may include economic "highs” and “lows,” but will most likely include continued hurdles associated with health care costs, this is all the more important.

STATEMENT OF NORMAN H. BELL, FORMER PRESIDENT, AMERICAN

SOCIETY FOR BONE AND MINERAL RESEARCH Senator BURDICK. Next is Dr. Norman Bell of the American Society for Bone and Mineral Research. Welcome, Doctor.

Dr. BELL. Thank you, Senator Burdick, for the opportunity to testify before this committee. My name is Norman Bell. I am a professor of medicine and pharmacology at the Medical University of South Carolina in Charleston, and I represent the American Society for Bone and Mineral Research.

Some 27 million Americans, from infants to the elderly, suffer from a variety of debilitating, skeletal disorders, including osteoporosis, Paget's disease, and related disorders of bone and mineral metabolism.

In this country, there are some 270,000 hip fractures every year. The mortality ranges from 12 percent to 20 percent, and half of the patients eventually end up in nursing homes or require the care of the members of their family or the community. Annual cost for these diseases is estimated to be on the order of $10 billion.

Musculoskeletal diseases are the leading causes of debility in the country. The incidence of both osteoporosis and Paget's disease are increasing because of the aging population, the fastest-growing segment of our society.

The American Society for Bone and Mineral Research represents the majority of scientists that are carrying out research in the bone and mineral field, and research support is obtained from six of the institutes at NIH, the National Institute of Aging, the National Institute of Arthritis, Musculoskeletal and Skin Diseases, National Cancer Institute, National Institute of Child Health and Human Development, National Institute of Dental Research, and the National Institutes of Diabetes, and Digestive and Kidney Disorders.

The diseases that are undergoing investigation by members of the society are divergent and encompass such areas as the ireatment and means of diagnosis of osteoporosis in older men and women, Paget's disease, cause and treatment of kidney stones, cause and treatment of bone diseases associated with kidney disease, identification of mutations in the genes for type I collagen that cause brittle bone disease or osteogenesis imperfecta.

And you heard an eloquent testimony from a small patient with the disease. The fact is that modified growth in the development of the skeleton in normal and diseased states, and the cause and treatment of dental diseases.

In recent years, much progress has been made to identify the growth factors and cytokines that are produced by bone cells and regulate the processes of bone formation, bone remodeling, and skeletal repair, and the structure and function and the factors that influence their production. It is important to develop an understanding of these areas to understand the causes of osteoporosis and other disorders.

Now, despite considerable effort and some progress, there is still no accepted means of treatment of osteoporosis except estrogen that will increase bone mass and prevent fractures of the hip. More infurmation is needed to determine why loss of bone mass occurs with aging, and the cause of bone loss in patients with osteoporosis.

Despite success in investigations in these and other areas, funding for research is constrained at a number of the Institutes, and we ask that consideration be given to increase the budgetary budgets of the six institutes. Only with increased funding will NIH be able to fund more grants, train needed young investigators, support badly needed clinical trials, fund additional research centers, and increase intramural research programs.

We wish to thank the Members of the Congress and of this subcommittee for strong and continuing support of intramural and extramural research programs at the NIH, and particularly the increases that were given to the National Institutes of Aging last year, over 36 percent; NIAMS, which was 14.6 percent; the dental institute, which was over 10 percent.

PREPARED STATEMENT

We also appreciate the opportunity to appear before this committee and to represent the members of the American Society for Bone and Mineral Research, and the many Americans who have diseases of bone and mineral metabolism in this country. Thank you very much.

[The statement follows:]

STATEMENT OF NORMAN H. BELL, M.D., ON BEHALF OF THE AMERICAN SOCIETY FOR

BONE AND MINERAL RESEARCH The American Society for Bone and Mineral Research is grateful for the opportunity to appear before the subcommittee.

Some 27 million Americans from infants to elderly subjects suffer from a variety of debilitating skeletal disorders including osteoporosis, Paget's disease and related disorders of bone and mineral metabolism. Some 270,000 hip fractures occur every year in the United States. The mortality ranges from 12 to 20 percent and half of the patients will eventually require assistance either from members of their family or admission to nursing homes. The annual cost to society is estimated to be as much as $10 billion. Musculoskeletal diseases are the leading cause of disability in this country. The incidence of both osteoporosis and Paget's Disease is increasing because of the aging population, the fastest growing segment of our society. The American Society for Bone and Mineral Research represents the majority of scientists who carry out research in the bone and mineral field. Research by members of the Society is Supported by six of the institutes of the National Institutes of Health:

National Institute of Aging (NIA), National Institute of Arthritis Musculoskeletal and Skin Diseases (NIAMS), National Cancer Institute (NCI), National Institute of Child Health and Human Development (NICHD), National Institute of Dental Research (NIDK) and National Institute of Diabetes and Digestive and Kidney Disorders (NIDDK).

Diseases that are undergoing investigation by members of the Society are divergent and encompass: the causes, treatment and means of diagnosis of osteoporosis in older men and women, Paget's Disease, cancer-related diseases that cause hypercalcemia and its complications, the cause and treatment of kidney stones, the cause and treatment of bone diseases associated with kidney disease, identification of mutations of the gene of the receptor for vitamin D that cause vitamin D-depend. ent rickets, identification of mutations of the gene for type I collagen that cause brittle bone disease, the factors that modify growth and development of the skeleton in normal and disease states, and the cause and treatment of dental diseases.

In recent years, much progress has been made in identifying growth factors and cytokines that are produced by bone cells and regulate the processes of bone formation, bone remodeling and skeletal repair, their structure and function and the factors that influence their production. Knowledge in this area is important to develop an understanding of the causes of osteoporosis and other bone disorders.

Despite considerable effort and some progress, there is still no accepted means of treatment of osteoporosis except estrogen that will increase bone mass and prevent fractures of the hip. More information is needed to determine why loss of bone mass occurs with aging and the cause of bone loss in patients with osteoporosis.

Despite success in investigations in these and other areas, funding for research is constrained at a number of institutes. We ask therefore that consideration be given to supporting the following budgetary increases:

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Only with increased funding support by Congress will NIH be able to fund more grants, train needed young investigators, support badly needed clinical trials, fund additional research centers and increase intramural research programs.

We wish to thank the Congress for the strong and continuing support of intramural and extramural research programs at the National Institutes of Health.

We also appreciate the opportunity to appear before this committee and to represent the members of the American Society for Bone and Mineral Research and the many Americans who have diseases of bone and mineral metabolism in our country.

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