我国罕见病药品进口现状及挑战的调研分析

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中图分类号R95 文献标志码A 文章编号 1001-0408（2025）17-2097-05

DOI10.6039/j.issn.1001-0408.2025.17.03

Investigationand analysis of the current status and challnges in importing rare disease drugs in China WU Jingjing1，2，SU Qinning'，TAO Xueyi'，YANG Yufei12，MAO Ningying1，²（1.School of Intermational Medicine Business，China Pharmaceutical University，Nanjing ，China;2. Research Center of National Drug Policy & Pharmaceutical Industry Economics，China Pharmaceutical University，Nanjing ，China）

ABSTRACTOBJECTIVETo analyze thecurrent status and challnges in importing rare disease drugs in China，providing references foroptimizing theimport processandimprovingrelevant policies.METHODsQuestionnairesandinterviews were conductedwithstakeholdersinvolvedinrarediseasedrugimportation，includinggovernmentdepartments，multinational pharmaceuticalenterprises，healthcareinstitutions，andpatientorganizations.Thisexploredthecurrentsituationandchallenges encounteredbyeachpartyExpertopinionsweresynthesizedtoproposeimprovementsuggestions.RESULTSAquestionaire surveyofrepresentativesfrom25multinationalpharmaceticalcompaniesintherarediseasefieldrevealedthatthesecompanieshad astrong willingness to import rare disease drugs，with 58.33% ofthem practicing diverse import models.However，significant challenges hindered this process，including unclear regulations （54.17% ），complex approval procedures （ 45.83% ），and excessively long approval cycles （ 41.67% ），negatively impacting their motivation.Meanwhile，interviews with 13 experts from government departments，healthcareinstitutions，pharmaceuticalenterprises，andpatientorganizationsidentifieddeficiencies inpolicydesign， approval processes，sampling inspectioncosts，andcommunication eficiencywithregulators.Aditionaly，thedrugimportmodel inspecial medical zonesalsorequired improvement.CONCLUsIONsTheimportion ofrare disease drugs in China faces challengessuchasincompletepolicies，inflexibleregulatorymechanisms，andinsuffcientcommunicationchannels.Itis recommended toenhance therare diseasedefinition criteria，optimizeimport incentive policies，andrefineregulatory models，soas to further optimize the import process of rare disease drugs and improve relevant policies.

KEYWORDSrare disease；drug importation；policy recommendations；China

罕见病是指患病率很低、很少见的疾病，一般为慢性病或严重危及生命的疾病，尚无统一的界定标准。（剩余8892字）