我國(guó)基于臨床緩解率替代終點(diǎn)上市抗癌新藥的現(xiàn)狀及特征研究

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【中圖分類號(hào)】 R979.1【文獻(xiàn)標(biāo)識(shí)碼】A DOI：10.12114/j.issn.1007-9572.2024.0475

【Abstract】BackgroundCancerisaleading causeof death among residents in China，accounting for23.1%of all disease-relateddeaths.Toaddresstheurgentneedsof patientswithseverelylife-threateningdiseases，theNationalMedical Products Administration（NMPA）allowstheuseofclinicalresponserates（RRs）asasurogateendpoint tosupportconditional approvalofnewdrugs.ObjectiveToanalye thecurrntsituationandcharacteristicsofnewanticancerdrugsapprovedinChina basedonclinicalRRsasasurrogateendpoint.MethodsThisstudysystematicallycollcteddataonanticancerdrugsapproved bytheNMPAfromOl7to2O23，focusingonanticancerdrugsandtheirindicationsthatwereapprovedbasedonRRsassurrogate endpoint.Thestudyanalyzedtheinitialapproval typesof thesedrugs（includingregularappovalandconditionalapproval）， thetypesof eficacyendpointsusedforsubsequentconversiontoregularappoval，clinicaltreatmentlines，trialdesignsfor initialdrugapprovalandpost-marketingconfirmation，drug mechanismclassfications，andcancertypes.Aditionally，the studycompared thediferences inRRsamonganticancerdrugswithdiferentNMPAapprovaltypes.ResultsFrom2017to 2023，NMPAapproved95indicationsfor68newanticancerdrugsbasedonRRsassurrogate endpointforadvancedormetastatic cancers.Among these，21（ 22.1% ）were granted regular approval，while 74（ 77.9% ）were conditionally approved. Of the 74 conditionally approved indications，13（ 17.6% ）were subsequently converted to regular approval. The median RRs for all 95 indications was 59.0% （ 35.8% ， 75.8% ）.Specifically，47（ 49.5% ）indications had RRs higher than or equal to 60% ，while 16 （ 16.8% ）hadRRs lower than 30 % [with15（ 93.8% ）of these being for second-line or higher treatment].There was no statisticallysignificant diference inRRsbetweenconditionallyapprovedandregularlyapprovedanticancerdrugs （ P =0.076） . ConclusionThe standardsforconditional approval of novelanticancer drugs in China basedonRRsare strictand show no significantdifferencecompared toregular aproval.TheRRsof the majorityof novelanticancerdrugs exced internationally recommended standards.

【Key words】 Antitumor drugs； Indications；Clinical response rate；Complete response rate；Conditional approval

癌癥是我國(guó)居民的主要死因「」。（剩余10148字）