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艾意凯咨询:2023提高对肿瘤相关终点的理解度、接受度和使用度白皮书(英文版)(27页).pdf

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1、Improving the understanding,acceptance and use of oncologyrelevant endpoints in HTA body/payer decision-makingWHITE PAPERPROMISQLQ-BR23OSProgression-free survivalMinimal/measurable residual diseaseComplete responseDuration of responseTIME TO METASTASISTime to next treatmentMETASTASIS-FREE SURVIVALRe

2、lapse-free survivalEvent-free survivalDISEASE FREE SURVIVALTIME TO PROGRESSIONDISEASE-SPECIFIC BIOMARKERSClinical benefit rateDisease control ratePathological complete responseOVERALL RESPONSE RATEPartial responseMD Anderson Symptom InventorySF-36FACT-GEQ-5DQLQ-C30NSCLC-SAQQLQ-LC13FACT-CQLQ-CR2PDQSE

3、PTEMBER 20232|Improving the understanding,acceptance and use of oncologyrelevant endpoints in HTA body/payer decision-makingSEPTEMBER 2023Improving the understanding,acceptance and use of oncologyrelevant endpoints in HTA body/payer decision-makingThis thought piece has been developed as part of an

4、EFPIA project to drive awareness of the use of oncology-relevant endpoints in HTA body/payer decision-making.Oncology-relevant endpoints refer to all endpoints used in oncology clinical trials to measure outcomes relevant to patients.These can include,in addition to overall survival,patient-reported

5、 outcomes as well as progression-free survival in some metastatic settings,pathological complete response or event-free survival in early oncology settings.The thought piece was written with the support of L.E.K.Consulting and was informed by a literature review as well as 13 qualitative interviews

6、with clinicians,patient advocates and former HTA bodies/payers to better understand the value of oncology-relevant endpoints and the challenges facing their adoption.The findings were discussed and refined at three roundtables with the projects sounding board.CONTENTSCONTENTS3|Improving the understa

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本文主要讨论了在肿瘤药物的监管和卫生技术评估(HTA)/支付者决策中,总体生存期(OS)和其他与肿瘤相关的终点的使用。文章指出,OS作为衡量药物临床益处的指标,具有客观性和适用性,但过度依赖OS数据存在三个主要局限性:1)OS数据无法捕捉除生存期之外对患者重要的结果,特别是那些反映健康相关生活质量(HRQoL)的结果;2)随着癌症预后的改善和多线治疗,获取成熟的OS数据所需时间越来越长;3)OS数据在决策中可能导致延迟患者对新药的获取。文章强调,除了OS之外,其他与肿瘤相关的终点(如无进展生存期、无病生存期、病理完全缓解和患者报告的结果)可以帮助解决OS在评估新药时的一些挑战,并可作为OS或其他目标结果的替代指标,提供药物疗效的早期测量。文章建议采取一系列行动,包括在治疗设置中为每种癌症类型和阶段选择适当的其他肿瘤相关终点,确保数据收集的一致性,以及生成和传播适当的证据,以提高这些终点的认可度。
肿瘤相关终点的价值是什么? 肿瘤相关终点的使用面临哪些挑战? 如何提高肿瘤相关终点的接受度?
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