Authorization of off-label drugs in a tertiary hospital: 5-year perspective.

IF 1.3 Q4 PHARMACOLOGY & PHARMACY

FARMACIA HOSPITALARIA Pub Date : 2025-07-07 DOI:10.1016/j.farma.2025.05.018

Lupe Rodríguez-de Francisco, Ángela María Villalba-Moreno, Eva Rocío Alfaro-Lara

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引用次数: 0

Abstract

Introduction: The off-label use of drugs requires scientific support to balance risk/benefit, being limited to exceptional cases in which there are no therapeutic alternatives.

Methods: Retrospective descriptive study of the reports of the Pharmacy and Therapeutics Committee between 2018 and 2022 in a third level hospital; analyzing the requests, drugs, final opinion, reasons, level of evidence and economic impact.

Results: A total of 124 reports were analyzed, highlighting oncohematological (41.9%) and autoimmune (27.4%) diseases as main indications. Oncology (37.1%) and Pediatrics (18.5%) were the main applicants, with 87.9% for antineoplastic and immunomodulatory drugs. A total of 74.2% of the applications were approved due to lack of alternatives and solid evidence (phase II-III trials), while 25.8% were denied due to the availability of therapeutic options or insufficient evidence. In terms of cost, 53% of oncohematological drugs cost between €10,000-50,000/treatment and 62.1% of non-oncohematological drugs cost between €1,000-10,000/year. Approval of the rejected treatments would have generated an additional expenditure of €2,272,603.

Conclusion: An increase of up to four times in the evaluation of off-label use drugs was evidenced, with a high approval rate.

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三级医院超说明书用药的授权：5年展望

药物的超说明书使用需要科学支持来平衡风险/收益，仅限于没有治疗替代方案的特殊情况。方法：对某三级医院2018 - 2022年药学与治疗学委员会报告进行回顾性描述性研究；分析请求、药品、最终意见、原因、证据水平和经济影响。结果：共分析124例报告，以血液病（41.9%）和自身免疫性疾病（27.4%）为主要适应症。肿瘤（37.1%）和儿科（18.5%）是主要的申请人，抗肿瘤和免疫调节药物占87.9%。共有74.2%的申请由于缺乏替代方案和可靠的证据（II-III期试验）而被批准，而25.8%的申请由于治疗方案的可用性或证据不足而被拒绝。在费用方面，53%的血液肿瘤药物费用在1万-5万欧元/次治疗之间，62.1%的非血液肿瘤药物费用在1000 -1万欧元/年之间。批准被拒绝的治疗将产生2,272,603欧元的额外支出。结论：超说明书用药的审评增加了4倍以上，审评合格率较高。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

FARMACIA HOSPITALARIA PHARMACOLOGY & PHARMACY-

CiteScore

1.90

自引率

21.40%

发文量

审稿时长

37 days

期刊介绍： Una gran revista para acceder a los mejores artículos originales y revisiones de la farmacoterapia actual. Además, es Órgano de expresión científica de la Sociedad Española de Farmacia Hospitalaria, y está indexada en Index Medicus/Medline, EMBASE/Excerpta Médica, Alert, Internacional Pharmaceutical Abstracts y SCOPUS.