Pignatti Francesco, Josephson Filip, Demolis Pierre, Tenhunen Olli, Péan Elias, Postmus Douwe
European Medicines Agency, Amsterdam, The Netherlands.
Swedish Medical Products Agency (Läkemedelsverket), Uppsala, Sweden.
Clin Pharmacol Ther. 2025 Sep;118(3):642-648. doi: 10.1002/cpt.3708. Epub 2025 May 13.
The purpose of this study was to describe the perspectives and trade-offs of EU regulators about expedited approvals for treatments of patients with advanced cancer. The study consisted of a cross-sectional survey with assessors and experts from the network of the European Medicines Agency (EMA). First, general attitudes were collected using a 5-point Likert scale. Second, a conjoint analysis was used to assess the compatibility of "conditional approval" for different scenarios of response rate, response duration, and toxicity from a single-arm trial, and timeliness of confirmatory data. The survey was completed by 60 participants out of 351 invited participants. The majority agreed that outstanding activity is indicative of efficacy, that toxicity is a concern, and that confirmatory trials should be ongoing at the time of approval. The majority also agreed that conditional approval is not well understood by patients and doctors. Opinions varied on the usefulness of external comparator studies. Higher antitumour activity could outweigh higher toxicity or longer time to confirmation. Based on confidence with expedited pathways and evidence generation, participants could be categorized as "supportive," "pragmatic," and "cautious." The importance of toxicity and timeliness of confirmatory studies was similar between groups but varied for antitumour activity. This study highlighted different attitudes when balancing speed of access with incomplete evidence in situations of high unmet medical needs. Confidence in expedited pathways and early evidence varied. There is an opportunity for improving communication and better alignment on evidentiary standards for expedited approvals.
本研究的目的是描述欧盟监管机构对于晚期癌症患者治疗加速批准的观点和权衡。该研究包括对来自欧洲药品管理局（EMA）网络的评估人员和专家进行的横断面调查。首先，使用5点李克特量表收集总体态度。其次，采用联合分析来评估单臂试验中不同缓解率、缓解持续时间和毒性情况下“有条件批准”的兼容性，以及确证性数据的及时性。在351名受邀参与者中，有60名完成了调查。大多数人同意显著活性表明疗效，毒性是一个关注点，并且在批准时确证性试验应该正在进行。大多数人还同意患者和医生对有条件批准的理解并不充分。对于外部对照研究的有用性，意见存在分歧。更高的抗肿瘤活性可能超过更高的毒性或更长的确证时间。基于对加速途径和证据生成的信心，参与者可分为“支持型”、“务实型”和“谨慎型”。各组之间毒性和确证性研究及时性的重要性相似，但抗肿瘤活性的重要性有所不同。本研究强调了在医疗需求未得到满足的情况下，在获取速度与不完整证据之间进行权衡时的不同态度。对加速途径和早期证据的信心各不相同。在加速批准的证据标准方面，存在改善沟通和更好地达成一致的机会。
