Polak Tobias B, Cucchi David G J, van Rosmalen Joost
Erasmus MC, afd. Biostatistiek, Rotterdam.
Contact: Tobias B. Polak (
Ned Tijdschr Geneeskd. 2021 Feb 25;165:D5168.
Expanded access is a pathway to access unregistered medicines if there are no registered treatments available and patients cannot enroll in clinical trials. Expanded access may serve as a last resort for patients who are in dire need of treatment options and cannot await the completion of drug development and for patients who may benefit from treatments that are not (or not anymore) registered in their jurisdiction. Unregistered medicine can be acquired via named-patient pathways ('Leveren op Artsenverklaring') or via group programs ('Compassionate Use Programma's). We describe the origins of expanded access and its daily practice in the Netherlands. We observe an increasing trend in expanded access requests. The potential risks these treatments provide, the possibility of ceasing further treatment and the preferences of individual patients should all inform the decision whether or not to pursue expanded access.
扩大用药途径是在没有已注册治疗方法且患者无法参加临床试验的情况下获取未注册药品的一种途径。对于急需治疗选择且无法等待药物研发完成的患者，以及可能从其所在辖区未注册（或不再注册）的治疗方法中受益的患者，扩大用药途径可能是最后的手段。未注册药品可以通过指定患者途径（“凭医生声明供应”）或通过集体项目（“同情用药项目”）获得。我们描述了扩大用药途径在荷兰的起源及其日常实践。我们观察到扩大用药途径申请呈上升趋势。这些治疗方法带来的潜在风险、停止进一步治疗的可能性以及个体患者的偏好，都应作为决定是否寻求扩大用药途径的依据。
