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NATIONAL PLAN

Senator HARKIN. Thank you very much, Dr. Shulman.
How soon are we going to have this national plan, did you say?

I'm sorry.

Dr. SHULMAN. It is going to be before the next hearings.
Senator HARKIN. Good.

Well, I had a question on osteoporosis, but you covered that. And lyme disease you covered that quite well. And lupus.

Just a moment. [Pause.] My apologies. I have to run over and cast another vote right now. My apologies. (A brief recess was taken.)

QUESTIONS SUBMITTED BY THE SUBCOMMITTEE Senator HARKIN. Thank you very much. There will be some additional questions which will be submitted for your response in the record.

[The following questions were not asked at the hearing, but were submitted to the Institute for response subsequent to the hearing:)

QUESTIONS SUBMITTED BY THE SUBCOMMITTEE

OSTEOPOROSIS

Question. Doctor, last year an additional $6.0 million was provided to the Institute for scientific research on osteoporosis, its causes, treatment and prevention.

Please describe the progress you have made in this area.

Answer. We are pleased to have played a lead role in the preparation of the Department's Report on HHS-wide Research, Education, and Health Promotion in Osteoporosis, prepared with the help of the Arthritis and Musculoskeletal Diseases Interagency Coordinating Committee and delivered to the Senate Appropriations Committee. The Report provided information on the current status of osteoporosis research, specific research activities on osteoporosis by several agencies in the Department of Health and Human Services, and promising areas for future research.

In response to current congressional interest and support for osteoporosis, two. Requests for Applications (RFA) have been issued to solicit applications in research on osteoporosis. One invites additional basic research on fundamental causes. The other invites more grant applications on clinical and epidemiological research on osteoporosis.

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) expects a vigorous response from the research community to these solicitations. We anticipate funding up to 4 million dollars in outstanding new investigations in both the basic and clinical aspects of osteoporosis through this mechanism.

The NIAMS is also participating with NIA in encouraging new applications for studies of the pathophysiology and mechanisms associated with particular intervention strategies against osteoporosis in the elderly. The NIAMS and NIA will each contribute up to $600,000 to fund studies solicited through an RFA.

The NIAMS has participated for the last several years with the NHLBI and other institutes to fund the Postmenopausal Estrogen/Progestin Intervention (PEPI) Study. This is a multicenter, randomized, controlled clinical trial that will test the efficacy of various estrogen and progestin preparations in postmenopausal women. A variety of outcomes are being tested including bone mass and lipoprotein profiles.

Question. I am aware that the drug "Etidronate" is a promising new treatment for preventing bone loss in humans. Can you summarize what is known about the potential of this treatment? At what point might it be available to the general public?

Answer. The drug etidronate is an important new method for preventing bone loss in persons with osteoporosis. It does not promote formation of new bone, but it does retard further loss of bone. Etidronate has been available to the public for many years for other indications. It has been learned that giving the drug on a cyclic basis, to allow the bone to recover between treatments, prevents the impaired mineralization of bone that occurs when the

drug is given on a continuing basis. New drugs, of the same class as etidronate, are being developed that may prove to be effective while not impairing mineralization.

Question. What is the status of NIAMS's effort to educate the public and the health community about osteoporosis?

Answer. The NIAMS has led a variety of activities aimed at better informing and educating these audiences.

In terms of the public:

o Our information office and clearinghouse handle hundreds of

inquiries on osteoporosis each year. For this purpose, we have developed an information package that provides selected important articles on the topic and refers callers to other information sources, such as the National Osteoporosis Foundation.

o We have distributed over 125,000 copies of our brochure

"Osteoporosis: Cause, Treatment, Prevention." We are also developing a series of fact sheets on individual aspects of the disease that will enable us to provide thorough, up-to-date information as new findings are discovered.

o We have a new brochure on osteoporosis, based on a Medicine for the

Layman lecture, given by the Director, NIAMS.

o We are reporting research results for the press and public, with a

special mailing just before National Osteoporosis Prevention Week (starting May 8).

In terms of the health community:

o We continue to sponsor major scientific workshops on osteoporosis

to review progress and up-to-date Information, and suggest research directions. In 1990 we supported two such scientific workshops, one in February here in Washington and another in Copenhagen in October, an international consensus development conference that was recently published in the American Journal of Medicine.

o At the request of the Department of Health and Human Services, we

organized and coordinated with 16 other agencies a report to the Senate on osteoporosis research, education and health promotion activities in the Department, with recommendations for future activities. We will be printing this report in quantity this spring for both the research community, and health educators and program planners. We will utilize the report to encourage, lead and coordinate future work in both research and health education in this important disease.

o The NIAMS sponsors an information clearinghouse that has prepared

two in-depth bibliographies of educational materials, one for health professionals and the other for patients. The bibliographies list both printed and audiovisual materials. Through its database, the clearinghouse maintains an up-to-date listing of such materials for use by both health educators and health professionals.

o We are in the planning stages of developing a national resource

center on osteoporosis.

LYME DISEASE

Question. I was pleased to learn that the Institute will be sponsoring a conference in late March to educate physicians concerning proper diagnosis and treatment of Lyme disease.

The Committee has heard varying reports on the success of laboratory tests used to diagnose this chronic disease. comment on the effectiveness of the tests that are currently available?

Can you

Answer. Laboratory tests currently available to aid in the diagnosis of Lyme disease consist largely of tests to identify antibodies to the causative bacteria in the blood. with recent infection, it often takes a few weeks for the antibody test to become positive. Once they develop, these antibodies tend to remain present for months or years thereafter. They may, therefore, indicate previous exposure but not necessarily current disease. Thus, there are many misdiagnoses based on both negative and positive tests. The doctor's clinical judgement is, therefore, important in assessing both the clinical manifestations and the value of the tests in a given patient.

Last Fall, the Centers for Disease Control reported on an evaluation of seven selected commercially available Lyme disease serological test kits at the First National Conference on Lyme Disease Testing in Dearborn, Michigan. The evaluation was conducted at four state public health laboratories under contract with the Association of State and Territorial Public Health Laboratories Directors. The data presented at the conference suggest that the test kits evaluated do not meet sensitivity and specificity needs, and are not well standardized. The conference recognized the need for well-characterized, culture-confirmed sera from patients with and without Lyme disease, and the importance of establishing a spirochete repository for antigens to be used, as national standards, in test kit development and evaluation.

The NIAMS and the NIAID convened the first annual meeting of the 14 principal investigators who successfully competed for research grants under the joint NIAMS/NIAID Request for Applications to study the pathogenesis of Lyme borreliosis. Each investigator had the opportunity to present their research plans and, in some cases, preliminary results. Many of these research projects are addressing research questions that are directly relevant to the development of newer and better methods for diagnosing Lyme disease. These include some of the newer approaches that have been made possible by advances in molecular biology, such as the polymerase chain reaction technique for amplifying genetic material from specimens containing small amounts of genetic material from the causative spirochete.

The NIAMS and NIAID are also co-sponsoring a workshop in late March on the diagnosis and treatment of Lyme disease. National experts on these issues will discuss and assess both what is known and what is not known about the diagnosis and treatment of Lyme disease.

LUPUS

Question. Doctor, I have heard the statistic that women suffer from systemic lupus disease nine times more frequently than men.

What research is being done to locate the factors that "trigger" systemic lupus disease and address the high incidence of the disease among females, especially among minority women?

Answer. The reasons that women suffer lupus nine times more frequently than do men and the reasons that black women suffer three times more lupus than do white women are currently unknown. The NIAMS sponsors research grants that address these important questions. In addition, the NIAMS is developing new initiatives on minority and gender issues in lupus. These will include program announcements requesting applications to study the topic. The NIAMS is investigating high risk target populations and participating in a workshop in Barbados to look at the epidemiology of lupus in the special populations available in the Caribbean. The NIAMS recently convened a group of investigators interested in the genetics of SLE to plan for initiatives that would also involve these target populations.

CONNECTIVE TISSUE DISORDERS

Question. Doctor, I was delighted to learn that the Epidermolysis Bullosa (EB) registry is now in its second generation and has been quite effective. Are there any plans to develop similar registries for other connective tissue diseases?

Answer. The National Epidermolysis Registry has been particularly useful as a way to collect information about large numbers of patients for application in epidemiologic and treatment studies. Such data have already shown that EB is composed of a number of different conditions. While the EB registry has been beneficial, it needs to be employed within a framework of established investigators who can efficiently make use of the information produced. In addition, re cies consume a relatively large amount of resources. The NIAMS is actively determining the most appropriate manner to facilitate research for several different connective tissue diseases. Decisions concerning registries for other connective tissue diseases will take into account the base of existing knowledge about the disease, investigators who could utilize such a facility, the research objectives to be achieved by a registry, other mechanisms to achieve these objectives, and the availability of funds.

Question. What initiatives are being undertaken to develop specialized centers for basic research and treatment of EB?

Answer. Epidermolysis Bullosa (EB) is a group of rare diseases. Very few, if any, single research institutions have a sufficiently large patient population necessary to assure the high quality clinical research component required in a SCOR. The clinical sites of the National Epidermolysis Bullosa Registry (NEBR) together have identified the majority of EB patients alive and diagnosed in the United States. Funds for clinical studies, including clinical trials, have been incorporated into the request for proposals for the

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