离散选择实验在罕见病用药价值评估及偏好测量中的应用OA

OBJECTIVE To systematically review the current application of discrete choice experiment(DCE)in the value assessment and preference measurement of orphan medicinal product(OMP),and to provide a reference for the standardized use of this methodology in China.METHODS The systematic search was conducted across Chinese and English databases including CNKI,Wanfang Data,VIP,CBM,PubMed,Web of Science,Medline,and Embase.Original studies that employed DCE to evaluate the value or preferences related to OMP were included.The methodological quality and reporting completeness of the included studies were assessed using the ISPOR Conjoint Analysis Checklist and the DIRECT Checklist,respectively.Respondent populations,attribute setting,and the relative importance of attributes were summarized and analyzed.RESULTS Eight eligible studies were included;all studies demonstrated high-quality reporting and methodological rigor.Respondents comprised the general public,patients/caregivers,policymakers,and other stakeholders.The number of DCE attributes ranged from 4 to 13(median=7.5).Through thematic synthesis,these attributes were categorized into three dimensions,namely"disease-related""treatment-related"and"economic/financial-related"along with 14 secondary criteria.The most frequently included secondary criteria were treatment efficacy(13 occurrences),disease severity(9 occurrences),safety(7 occurrences),unmet medical need(6 occurrences),and treatment cost(5 occurrences).Rankings of relative importance identified treatment efficacy as the most valued criterion across most studies,followed by health insurance financing.CONCLUSIONS DCE applications in the value assessment of OMP have begun to converge on a relatively consistent core attribute framework and selection preference.Future research should further promote the use of DCE to inform attribute and criterion selection in multi-criteria decision analysis frameworks for OMP.