Solà-Morales Oriol, Volmer Timm, Mantovani Lorenzo
Health Innovation Technology Transfer and International, University of Catalonia, Barcelona, Spain.
SmartStep Consulting GmbH, Hamburg, Germany.
J Mark Access Health Policy. 2019 Jan 22;7(1):1562861. doi: 10.1080/20016689.2018.1562861. eCollection 2019.
Reimbursement decisions on new oncology drugs are now often made while uncertainty remains about a drug's risk-benefit profile. One consequence of this is a delay in patient access to valuable new medicines. We share our perspectives on strategies to mitigate sources of uncertainty in the health technology assessment process. These include flexible approaches for evaluating the additional benefit, such as better use of surrogate endpoints and health-related quality of life data, and renewed research efforts to define the optimal target population and generate real-world evidence post-authorisation.
目前,在对新型肿瘤药物的风险效益状况仍存在不确定性的情况下,往往就会做出报销决定。这样做的一个后果是患者在获取有价值的新药方面出现延迟。我们就减轻卫生技术评估过程中不确定性来源的策略分享我们的观点。这些策略包括评估额外效益的灵活方法,如更好地利用替代终点和与健康相关的生活质量数据,以及重新开展研究工作,以确定最佳目标人群并在获批后生成真实世界证据。