Fondazione ReS (Ricerca e Salute), Research and Health Foundation, Via dei Due Macelli, 48, 00187, Rome, Italy.
Drugs and Health, Rome, Italy.
Clin Drug Investig. 2024 Aug;44(8):577-586. doi: 10.1007/s40261-024-01381-z. Epub 2024 Jul 20.
Compassionate drug use (CDU) provides early access to not yet authorised medicines and is funded by pharmaceutical companies. The observational retrospective study Compass-O monitored the CDU of onco-haematological drugs, managed by seven Italian units for cytotoxic drug preparations (Unità Farmaci Antiblastici [UFA]), between 1 January, 2016 and 31 December, 2021.
We aimed to evaluate the CDU of onco-haematological drugs managed by seven Italian UFA, between 2016 and 2021.
The seven UFA provided anonymised data concerning CDU approved in the study period. The early access and potential cost savings for the National Health System (Servizio Sanitario Nazionale [SSN]) were analysed for CDU concerning drug-therapeutic indication combinations with complete data and reimbursed by SSN up to December 2023 (date of study execution), according to the executive decision of the Italian Medicines Agency (Agenzia Italiana del Farmaco [AIFA]). Both analyses distinguished solid/liquid tumours and categorised the combinations as innovative (fully/conditionally) or non-innovative based on AIFA assessments.
Compass-O collected 783 CDU authorisations, with 572 (73.1%) analysable in terms of early access and cost savings. On average, early access amounted to 514 days and the total cost savings was €376,115,801. Compassionate drug use approvals involved mainly solid tumours (93.7% vs 6.3% for liquid tumours), and the combination of trastuzumab emtansine-breast cancer was the most dispensed (n = 73; early access = 426 days; potential cost savings: €610,388). Out of 572 CDU approvals, 200 (35%) were innovative drug-therapeutic indication combinations, with 598 days of early access and a total potential saving of €113,124,069.
The study Compass-O showed a significant economic burden of CDU and a relevant need for early access, particularly for innovative drugs. However, there is currently no structured monitoring of CDU in Italy, suggesting the need for a national observatory, of which Compass-O can be the pilot phase.
同情用药（CDU）提供了对尚未获得授权的药物的早期获取途径，并由制药公司提供资金。观察性回顾性研究 Compass-O 监测了 2016 年 1 月 1 日至 2021 年 12 月 31 日期间由意大利七个细胞毒性药物制备单位（Unità Farmaci Antiblastici [UFA]）管理的肿瘤血液学药物的 CDU。
我们旨在评估 2016 年至 2021 年期间意大利七个 UFA 管理的肿瘤血液学药物的 CDU。
七个 UFA 提供了研究期间批准的 CDU 的匿名数据。根据意大利药品管理局（Agenzia Italiana del Farmaco [AIFA]）的执行决定，对 CDU 进行了分析，这些 CDU 涉及药物-治疗指征组合，这些组合的数据完整且截至 2023 年 12 月（研究执行日期）由国民保健服务（Servizio Sanitario Nazionale [SSN]）报销，以评估 CDU 对国民保健服务（SSN）的早期准入和潜在成本节约。这两种分析均区分了实体/液体肿瘤，并根据 AIFA 评估将组合归类为创新（完全/有条件）或非创新。
Compass-O 共收集了 783 项 CDU 授权，其中 572 项（73.1%）可进行早期准入和成本节约分析。平均而言，早期准入时间为 514 天，总节省成本为 376,115,801 欧元。 CDU 批准主要涉及实体肿瘤（93.7%比液体肿瘤 6.3%），曲妥珠单抗恩美曲妥珠单抗-乳腺癌的联合治疗方案是最常用的（n = 73；早期准入=426 天；潜在节省成本：610,388 欧元）。在 572 项 CDU 批准中，200 项（35%）为创新药物-治疗指征组合，早期准入时间为 598 天，总潜在节省额为 113,124,069 欧元。
Compass-O 研究表明 CDU 存在显著的经济负担，并且对早期准入有很大的需求，特别是对创新药物。然而，意大利目前没有 CDU 的结构化监测，这表明需要建立一个国家观察站， Compass-O 可以作为试点阶段。
