Objective: The aim of this study was to determine priorities for specific intractable disease from the viewpoint of countermeasures.
Methods: A significance evaluation to provide a priority framework for intractable disease was performed with a questionnaire distributed to the staff of preventive medicine and public health departments of medical schools in Japan. To determine the actual conditions of these intractable diseases question were directed at the chairmen of individual clinical study groups. The priority to be assigned countermeasures for such diseases was obtained from the two questionnaires.
Results: When the 4 factors "rare nature of diseases," "level of clarity of causes and pathology," "unestablished curative treatment", and "influence on daily life" were evaluated using 100 point as a full score, the mean scores were 14.5, 27.1, 28.5 and 29.9 points, respectively. In attaching importance to the various items, regarding the "rare nature of disease" the elements of "few patients nationwide" and "few specialist doctors nationwide" proved important; for the "level of clarity of causes and pathology," this was the case for the elements of "unclear triggering mechanism" and "diagnostic criteria not established." With the "unestablished curative treatment," the "no efficacious treatment available" and "low 5-year survival rate elements were important; and with the factor of "influence on daily life," the two most significant were "high proportion of patients needing assistance in daily life" and "high percentage of patients impeded from attending school or obtaining a job (playing a role in society)".
Conclusion: When the priority among the 118 intractable diseases was evaluated by combining the overall results of the questionnaire survey with preventive medicine and public health staffs, and actual condition data from survey investigation of the intractable diseases among chairmen of clinical study groups, it was suggested that some intractable diseases whose medical expenses are covered by the medical aid program have a low ranking.