Irene Dell'Anno, Leonardo Dondi, Immacolata Esposito, Annamaria Mascolo, Annalisa Capuano, Giulia de Marchi, Adriano Cristinziano, Domenico Tarantino, Marcello Pani, Carla Masini, Caterina Donati, Elisabetta Rossin, Antonio Serafini, Gabriele Bagaglini, Gabriella Bonanni, Tommaso Gregori, Arturo Cavaliere, Roberta Matocci, Alessandro D'Arpino, Nello Martini, Carlo Piccinni
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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.</p><p><strong>Objective: </strong>We aimed to evaluate the CDU of onco-haematological drugs managed by seven Italian UFA, between 2016 and 2021.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>The study Compass-O showed a significant economic burden of CDU and a relevant need for early access, particularly for innovative drugs. 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引用次数: 0
摘要
背景：同情用药（CDU）为尚未获得授权的药物提供早期使用机会，由制药公司资助。Compass-O观察性回顾研究对2016年1月1日至2021年12月31日期间由意大利七家细胞毒性药物制剂单位（Unità Farmaci Antiblastici [UFA]）管理的抗肿瘤药物的CDU进行了监测：我们的目的是评估 2016 年至 2021 年间意大利七个细胞毒性药物制剂单位管理的抗肿瘤药物 CDU：这七家 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]）的行政决定，对截至 2023 年 12 月（研究执行日期）具有完整数据并由国家卫生系统（Servizio Sanitario Nazionale [SSN]）报销的药物治疗适应症组合 CDU 的早期使用和潜在成本节约情况进行了分析。这两项分析对实体瘤/液体瘤进行了区分，并根据 AIFA 的评估结果将组合药物分为创新型（完全/有条件）和非创新型：结果：Compass-O共收集了783项CDU授权，其中572项（73.1%）可从早期使用和成本节约角度进行分析。平均而言，提前使用时间为 514 天，节约成本总额为 376,115,801 欧元。批准的同情用药主要涉及实体瘤（93.7% 对比起液体瘤的 6.3%），其中曲妥珠单抗-乳腺癌联合用药最多（n = 73；早期使用 = 426 天；潜在节约成本：610,388 欧元）。在获得批准的 572 个 CDU 中，有 200 个（35%）是创新药物治疗适应症组合，提前使用时间为 598 天，潜在节省费用总额为 113,124,069 欧元：Compass-O 研究表明，CDU 带来了巨大的经济负担，而且需要尽早使用，尤其是创新药物。然而，意大利目前还没有对 CDU 进行结构性监测，这表明有必要建立一个国家观察站，Compass-O 可以作为该观察站的试点阶段。
The Early Access and the Potential Cost Savings by the Compassionate Use of Onco-haematological Drugs: Results from the Italian Study Compass-O.
Background: 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.
Objective: We aimed to evaluate the CDU of onco-haematological drugs managed by seven Italian UFA, between 2016 and 2021.
Methods: 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.
Results: 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.
Conclusions: 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.
期刊介绍：
Clinical Drug Investigation provides rapid publication of original research covering all phases of clinical drug development and therapeutic use of drugs. The Journal includes:
-Clinical trials, outcomes research, clinical pharmacoeconomic studies and pharmacoepidemiology studies with a strong link to optimum prescribing practice for a drug or group of drugs.
-Clinical pharmacodynamic and clinical pharmacokinetic studies with a strong link to clinical practice.
-Pharmacodynamic and pharmacokinetic studies in healthy volunteers in which significant implications for clinical prescribing are discussed.
-Studies focusing on the application of drug delivery technology in healthcare.
-Short communications and case study reports that meet the above criteria will also be considered.
Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Clinical Drug Investigation may be accompanied by plain language summaries to assist readers who have some knowledge, but non in-depth expertise in, the area to understand important medical advances.
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