Sarac Sinan B, Kiely Peter, Stankeviciute Simona, Camarero Jorge, McKee Amy
Parexel International, Durham, North Carolina, USA.
MSD, Madrid, Spain.
Br J Clin Pharmacol. 2025 Mar;91(3):672-677. doi: 10.1111/bcp.16383. Epub 2025 Jan 10.
Early approval mechanisms, such as conditional approval in the EU, have been used extensively to provide timely access to therapeutic innovations to cancer patients with unmet medical needs. While based on promising early evidence, such approvals are challenging from many perspectives due to the lack of comprehensive data. The limitation typically relates to data that demonstrates clinical benefit via early endpoints and is only acceptable when the early evidence is particularly convincing to assume that the benefits of early access are greater than the potential harms. This paper describes the requirements for conditional approval and presents common pitfalls in oncology, such as misunderstandings about the strength of evidence from exploratory trials and secondary analyses, lack of planning and opportunities to improve communication. Thereafter, we present a framework ('EDGE') on how to improve the submission and evaluation of drug applications for conditional approval in the EU.
早期批准机制，如欧盟的有条件批准，已被广泛用于为有未满足医疗需求的癌症患者及时提供治疗创新药物。虽然基于有前景的早期证据，但由于缺乏全面数据，此类批准从多个角度来看都具有挑战性。这种局限性通常与通过早期终点证明临床获益的数据有关，只有当早期证据特别有说服力，能够假定早期用药的益处大于潜在危害时，此类数据才是可接受的。本文描述了有条件批准的要求，并指出了肿瘤学领域常见的陷阱，比如对探索性试验和二次分析证据强度的误解、缺乏规划以及改善沟通的机会。此后，我们提出了一个框架（“EDGE”），阐述如何改进欧盟有条件批准药物申请的提交和评估。
