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
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中图分类号R956 文献标志码A 文章编号 1001-0408（2026）07-0835-07
DOI10.6039/j.issn.1001-0408.2026.07.02
Application of discrete choice experiment in value assessment and preference measurement for orphan medicinal product
ZHI Teng，TANG Xian，LUO Yanzhou，HU Ming（West China School of Pharmacy，Sichuan University，Chengdu ，China）
ABSTRACTOBJECTIVETosystematicallyreview the current aplicationof discrete choice experiment （DCE）inthevalue assessment and preference measurementoforphan medicinal product（OMP），and to provideareference forthestandardizeduseof thismethodology in China.METHODsThesystematicsearch wasconductedacross Chineseand English databasesincluding CNKI，Wanfang Data，VIP，CBM，PubMed，Webof Science，Medline，andEmbase.Original studies thatemployed DCE to evaluate thevalueorpreferencesrelatedtoOMPwere included.Themethodological qualityandreportingcompletenesof the included studies were assessd usingthe ISPOR Conjoint Analysis ChecklistandtheDIRECTChecklist，respectivelyRespondent populations，atributeseting，andtherelativeimportanceofatributesweresummarizedandanalyzed.RESULTSEighteligible studieswereincluded;allstudiesdemonstratedhigqualityreportingandmethodologicalrigor.Respondentscomprisedthegeneral public，patients/caregivers，policymakers，andotherstakeholders.ThenumberofDCEatributesrangedfrom4tol3（median 7.5）.Throughthematicsynthesis，theseatributeswerecategorizedintothreedimensions，namely“disease-related”“treatmentrelated”and“economic/financial-related”alongwith14secondarycriteria.Themostfrequentlyincludedsecondarycriteriawere treatmenteffiacy（13occurrences），diseaseseverity（9occurrences），safety（7occurrences），unmet medicalneed（6 occurrences），andtreatmentcost（5occurrences）Rankingsofrelativeimportanceidentifiedtreatmenteffcacyasthemostalued criterionacrossmost studies，followedbyhealth insurance financing.CONCLUsIONsDCEapplications inthevalueassessment ofOMPhavebegun toconvergeonarelativelyconsistentcoreatributeframeworkandselectionpreferene.Futureresearchshould further promote theuseofDCEto informatributeandcriterionselection inmulti-criteria decisionanalysis frameworksforOMP.
KEYWORDSorphanmedicinal product；discretechoiceexperiment；valueasessment；societal preference；systematicreview; multi-criteria decision analysis
罕见病又称为孤儿病，是一系列在普通人群中发病率极低的疾病的总称。（剩余12686字）