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通过管理准入协议创造共赢局面?在机会之窗内优先考虑基因和细胞疗法。

Creating win-win-win situations with managed entry agreements? Prioritizing gene and cell therapies within the window of opportunity.

机构信息

Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, the Netherlands.

Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, the Netherlands; National Health Care Institute (ZIN), Diemen, the Netherlands.

出版信息

Drug Discov Today. 2024 Jul;29(7):104048. doi: 10.1016/j.drudis.2024.104048. Epub 2024 Jun 1.

Abstract

Outcome-based reimbursement models are gaining attention for managing the clinical uncertainties and financial impact of gene and cell therapies. Little guidance exists on how such models can create win-win-win situations, benefiting health-care payers, health-technology developers and patients. Our innovative approach prospectively prioritizes therapies for which a 'window of opportunity' might occur through the analysis of health-technology assessments and product characteristics. Within this window, one size does not fit all, and depending on the extent of clinical uncertainty and potential added benefit levels, different win-win-win situations exist in the United States, the United Kingdom and the Netherlands. Dutch Horizon scanning data prioritized etranacogene dezaparvovec (Hemgenix) and mozafancogene autotemcel for their potential to benefit from outcome-based reimbursement models. These insights extend beyond gene and cell therapies, and could help to provide sustainable health care and patient access to innovative therapies.

摘要

基于结果的报销模式正受到关注,以管理基因和细胞疗法的临床不确定性和财务影响。关于这些模式如何创造三赢局面,使医疗保健支付方、医疗技术开发商和患者受益,目前几乎没有指导。我们的创新方法前瞻性地优先考虑治疗方法,通过对卫生技术评估和产品特征的分析,这些治疗方法可能会出现“机会之窗”。在这个窗口期内,一刀切并不适用,根据临床不确定性的程度和潜在的附加效益水平,在美国、英国和荷兰存在不同的三赢局面。荷兰的前瞻性扫描数据优先考虑 etranacogene dezaparvovec(Hemgenix)和 mozafancogene autotemcel,因为它们有可能从基于结果的报销模式中受益。这些见解不仅限于基因和细胞疗法,还有助于提供可持续的医疗保健和患者获得创新疗法的机会。

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