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STRATEGIES FOR FUTURE RESEARCH ON DISEASE MANAGEMENT

The breakout group on disease management also elected not to prioritize their assigned set of recommendations (recommendations #s 8–13, 17) because all were viewed as worthy objectives, according to Dr. Henry Claman, the group's rapporteur. He added that “it is probably a logical mistake to prioritize groups of recommendations that are so heterogeneous.”

Therapeutics

The breakout group felt that the recommendation (#8) for protection and repair of neural cells, including stem cells, was a “good area for collaborative and interdisciplinary research among various people in neurobiology, including partnerships with the pharmaceutical industry,” said Dr. Claman. Despite controversy over stem cells in federally supported research, the group noted that the National MS Society has an explicit policy permitting their use in research.

The breakout group noted that improvement and validation of therapeutic approaches to symptom management has received relatively scant scientific attention (#9). As background, Committee member Dr. Patricia Coyle pointed out that, while better symptomatic treatments were being developed, “there is much room for improvement” because symptoms of fatigue, depression, spasticity and pain, among others, have “tremendous impact for every patient on their quality of life” and that clinicians are not managing these symptoms with available treatments.

Dr. Claman relayed that the breakout group found symptom management to be a “good candidate for collaborative research.” If the National MS Society is interested in promoting this area, the group felt the Society should take active steps to raise awareness within the scientific community and to ask leaders in the field what directions should be taken. Dr. Claman noted that symptom management could be addressed within ongoing discussions, spearheaded by the National MS Society, on the needs of patients grouped according to gender and age.

Regarding the recommendation (#10) for integrated approaches for delivery of currently available therapies, Dr. Claman relayed the group's support for the recommendation and a role for the pharmaceutical industry, but the group did not devote much discussion to implementation.

The recommendation (#11) for better strategies to extract maximum scientific value from MS clinical trials was, according to Dr. Coyle, an outgrowth of the following concerns: the limited number of MS patients; growing ethical problems with placebo-controlled trials; the need for standardized protocols and assessment methods; the economic constraints on pharmaceutical companies to minimize the length of treatment trials; and the breadth of unanswered treatment questions, including which patients should be treated with disease-modifying therapies for their first attack of MS. The breakout group did not discuss this recommendation except to note that the International MS Trials Research and Resource Center is now being set up to provide a database of existing clinical trials.



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Page 6 STRATEGIES FOR FUTURE RESEARCH ON DISEASE MANAGEMENT The breakout group on disease management also elected not to prioritize their assigned set of recommendations (recommendations #s 8–13, 17) because all were viewed as worthy objectives, according to Dr. Henry Claman, the group's rapporteur. He added that “it is probably a logical mistake to prioritize groups of recommendations that are so heterogeneous.” Therapeutics The breakout group felt that the recommendation (#8) for protection and repair of neural cells, including stem cells, was a “good area for collaborative and interdisciplinary research among various people in neurobiology, including partnerships with the pharmaceutical industry,” said Dr. Claman. Despite controversy over stem cells in federally supported research, the group noted that the National MS Society has an explicit policy permitting their use in research. The breakout group noted that improvement and validation of therapeutic approaches to symptom management has received relatively scant scientific attention (#9). As background, Committee member Dr. Patricia Coyle pointed out that, while better symptomatic treatments were being developed, “there is much room for improvement” because symptoms of fatigue, depression, spasticity and pain, among others, have “tremendous impact for every patient on their quality of life” and that clinicians are not managing these symptoms with available treatments. Dr. Claman relayed that the breakout group found symptom management to be a “good candidate for collaborative research.” If the National MS Society is interested in promoting this area, the group felt the Society should take active steps to raise awareness within the scientific community and to ask leaders in the field what directions should be taken. Dr. Claman noted that symptom management could be addressed within ongoing discussions, spearheaded by the National MS Society, on the needs of patients grouped according to gender and age. Regarding the recommendation (#10) for integrated approaches for delivery of currently available therapies, Dr. Claman relayed the group's support for the recommendation and a role for the pharmaceutical industry, but the group did not devote much discussion to implementation. The recommendation (#11) for better strategies to extract maximum scientific value from MS clinical trials was, according to Dr. Coyle, an outgrowth of the following concerns: the limited number of MS patients; growing ethical problems with placebo-controlled trials; the need for standardized protocols and assessment methods; the economic constraints on pharmaceutical companies to minimize the length of treatment trials; and the breadth of unanswered treatment questions, including which patients should be treated with disease-modifying therapies for their first attack of MS. The breakout group did not discuss this recommendation except to note that the International MS Trials Research and Resource Center is now being set up to provide a database of existing clinical trials.

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Page 7 Quality of Life The quality of life recommendations by the IOM committee featured the need for better health status assessment methods (#12), better integration of disciplines studying quality of life (#17), and the need for more quality of life research with input from patients in setting priorities (#13). Improving the quality of life for MS patients was discussed by two separate breakout groups—those on disease management and research infrastructure. Committee member Dr. Lisa Iezzoni pointed out that there are numerous functional status and disability measures, yet no consensus on which were best applied to MS patients. The exception is the Expanded Disability Status Scale (EDSS), yet this scale is seen by researchers as outdated, limited, and placing too much emphasis on physical dysfunction. Other problems are the dearth of health services researchers interested in MS, of longitudinal studies, and of communication across disciplinary lines (nursing vs. health services research) and across chronic and disabling diseases. The breakout group on disease management agreed with the importance of better health status assessment scales, according to Dr. Claman. The group felt that a single new scale is likely to be insufficient and that more than one scale is needed depending on the study question. To simplify research, the group suggested developing a core data scale for all patients, with additional modules for assessment of different patient sub-groups, for example, patients with relapsing-remitting disease versus secondary progressive disease. The group pointed out that the National MS Society, NIH, and health insurance organizations would need to agree on the advisability and details of different scales before their validation. Several participants pointed to the difficulty of arriving at an internationally acceptable scale. In addition to problems with translation to different languages, cultures vary with respect to patient willingness to disclose their functional performance, particularly in relation to cognitive dysfunction, depression, and sexuality. The National MS Society's Dr. Nicholas LaRocca noted that the Society is updating earlier scholarly reviews of MS assessments and, to increase awareness in the scientific community, is exploring the possibility of making assessment information available on the World Wide Web. He also reported that the North American Research Committee on MS has an ongoing project examining standardization of data collection methods. Extended discussions concentrated on the availability of funds for rehabilitation and disability research. Committee member Sharon Juliano spotlighted the apparent “disconnect” between health services researchers' frustration at being turned down for funding and reports by funding agencies of unused funds. Several members of the IOM Committee remarked upon their surprise in learning of untapped pockets of grant funding. Representatives of several funding agencies, including the National MS Society, the Veterans' Administration (VA), and the NIH's National Center for Medical Rehabilitation Research (NCMRR) said they were at a loss to explain the discrepancy. They reported receiving few applications for funding MS rehabilitation and health services research in spite of their efforts to announce availability of funds. Dr. Reingold of the National MS Society

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Page 8noted that his Society's efforts to recruit applicants actively tried to reach beyond the MS field when publicizing the availability of funding. Yet he too conveyed his frustration that the few applications his Society received were of poor quality, even though the Society worked with researchers to help shape their applications. Dr. Iezzoni of the IOM Committee urged more “beating of the bushes” by funding agencies after she recounted that even though she is an established health services researcher, she had only learned of funding opportunities at the MS Society second-hand through her colleagues and not directly through any of the channels used by the Society. She encouraged funding agencies to be more proactive in recruiting health services researchers from outside the MS field who are studying symptoms (e.g., incontinence) of relevance to MS patients. She also suggested that funding agencies could act as brokers to link up practicing neurologists with health services researchers at nearby universities, to which Dr. Michael Weinrich of the NIH, National Center for Medical Rehabilitation replied that “I think certainly we would be very happy to coordinate with the MS Society or other agencies,” to promote awareness of funding. The discussion was summarized by Dr. Richard Johnston, who observed that current approaches by funding agencies are not working. The recommendation to encourage more quality of life research (#17) was also discussed by the third breakout group, which described the need for research as “an absolute must.” That breakout group suggested workshops to facilitate the transfer of ideas among biomedical researchers and allied health professionals at international meetings such the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) and the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS). Patients and Priority Setting Participants also debated the role of patients in the research priority-setting process. The breakout group agreed with the report's recommendation that patients and caregivers must have a voice in identifying quality of life research needs through various venues and forums. Dr. Reingold raised the broader question of whether patients should participate more generally in priority-setting across all types of MS research. Several participants described their experience in other health fields where patients successfully contributed to prioritizing research, in part, by lending urgency and direction to the discussion. Others pointed to the pitfalls. As both researcher and patient, Dr. Iezzoni observed that patients are heterogeneous and do not speak with one voice. Some emphasize research on a cure, while others are more concerned about their quality of life, often depending on age and severity of symptoms. A recent study of patients, conducted in Denmark and soon to be published, has borne this out, according to Dr. Clausen of the Danish MS Society. Some workshop participants were concerned about the hazards of having patients advise on scientific priorities in which they lack expertise. Finally, Dr. Claman commented on the unfairness of asking patients to prioritize when experts themselves are similarly divided.