Kelly Sarah A, Ronan Karine E, Zameer Mohammed, Brown Jennifer, Johnston Grainne, Adams Ruth, Murphy Dearbhla, Kelly Deirdre, Darwish Waseem, McCaffery John, Coyne Geraldine O'Sullivan, Harrold Emily, Iqbal Shahid, Cowzer Darren, Duffy Austin G
School of Medicine, University College Dublin, Dublin, Ireland.
Department of Medical Oncology, Mater Misericordiae University Hospital, Dublin, Ireland.
Ir J Med Sci. 2025 Jun;194(3):807-815. doi: 10.1007/s11845-025-03930-7. Epub 2025 Mar 21.
Despite the rise in the number of approved novel oncology drugs, just over half of all new cancer medicines approved by the EMA between 2017 and 2021 were granted reimbursement in Ireland by the HSE. Compassionate access programmes (CAPs) are a means of providing managed access to drugs which are of proven benefit but have not yet received full approval for reimbursement by the state, or where the requested indication has not been yet been authorised/licensed.
A retrospective review was performed of patients attending The Mater hospital for treatment of advanced malignancy who availed of a CAP between August 2012 and July 2022. Clinical data collected included disease type, treatment received, duration of treatment received, and best response to treatment. To categorize outcome "Clinical Benefit" was defined as a radiological complete, partial, or stable disease response to treatment.
One hundred and thirteen patients were included in the study. Ninety-three received at least one dose of CAP treatment. Treatment duration ranged from 0 to 112 months, with 12 patients on treatment for ≥ 2 years. N = 47 (42%) experienced a Clinical Benefit. Of these, N = 7 experienced a complete response [CR]. Thirty patients (27%) did not receive a planned treatment or died within 3 months of treatment.
In this review of a decade of CAPs at our institution we observed that a significant proportion of patients derived a clinical benefit from CAP treatment. Unfortunately, however, a significant proportion of patients did not receive a planned treatment due to disease progression or died within 3 months of treatment suggesting availability came too late. While CAPs can provide meaningful benefit, they are not a substitute for timely approval of novel agents.
尽管获批的新型肿瘤药物数量有所增加，但2017年至2021年间欧洲药品管理局（EMA）批准的所有新型癌症药物中，只有略超过一半在爱尔兰获得了卫生服务局（HSE）的报销。同情用药计划（CAPs）是一种提供对已证明有益但尚未获得国家全额报销批准的药物进行管理性获取的方式，或者是针对尚未获得授权/许可的所请求适应症的药物。
对2012年8月至2022年7月期间在圣母医院接受晚期恶性肿瘤治疗并使用了同情用药计划的患者进行回顾性研究。收集的临床数据包括疾病类型、接受的治疗、接受治疗的持续时间以及对治疗的最佳反应。为了对结果进行分类，“临床获益”被定义为对治疗的放射学完全缓解、部分缓解或疾病稳定反应。
113名患者纳入研究。93名患者接受了至少一剂同情用药计划治疗。治疗持续时间从0到112个月不等，12名患者治疗时间≥2年。N = 47（42%）患者有临床获益。其中，N = 7名患者有完全缓解[CR]。30名患者（27%）未接受计划治疗或在治疗3个月内死亡。
在对我们机构十年的同情用药计划的回顾中，我们观察到相当一部分患者从同情用药计划治疗中获得了临床获益。然而，不幸的是，相当一部分患者由于疾病进展未接受计划治疗或在治疗3个月内死亡，这表明药物可及性来得太晚。虽然同情用药计划可以提供有意义的益处，但它们不能替代新型药物的及时批准。
