ArticleEuropean journal of clinical pharmacology2014
Outcomes of off-label drug uses in hospitals: a multicentric prospective study.
Article in European journal of clinical pharmacology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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Who cites it
18 citing papers in PubMed.
- A model for the clinical management of the introduction of new medicines in a large University Hospital in Spain.Journal of pharmaceutical policy and practice · 2026Article
- Trends and off-label use of botulinum toxin type A in Chinese neurological practice from 2016 to 2023: a real-world study.Frontiers in pharmacology · 2026Article
- Botulinum toxin treatment in a resource-limited setting: Experiences from a public institution in a developing countryBiomedica : revista del Instituto Nacional de Salud · 2025Article
- Paediatric off-label use of drugs in Gansu, China: a multicentre cross-sectional study.BMJ open · 2024Article
- Real-world treatment outcomes of immune checkpoint inhibitors used off-label in oncology: A comprehensive cancer institution experience.Pharmacology research & perspectives · 2024Article
- Real-world treatment outcomes of medicines used in special situations (off-label and compassionate use) in oncology and hematology: A retrospective study from a comprehensive cancer institution.Cancer medicine · 2023Article
- Off-label use of rituximab in patients with systemic lupus erythematosus with extrarenal disease activity: a retrospective study and literature review.Frontiers in medicine · 2023Article
- Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain).Journal of clinical medicine · 2022Article
- Off-Label Use of Rituximab in Patients with Different Types of Nephropathies in a Tertiary Hospital: A Retrospective Study.Journal of clinical medicine · 2021Article
- Indications for Rituximab Use in an Integrated Health Care Delivery System.Journal of managed care & specialty pharmacy · 2020Observational
- Use of non-formulary high-cost medicines in an Australian public hospital.International journal of clinical pharmacy · 2019Article
- [Safety and efficacy of off-label use of biologic therapies in patients with inflammatory rheumatic diseases refractory to standard of care therapy : Data from a nationwide German registry (GRAID2)].Zeitschrift fur Rheumatologie · 2018Article
- Postmarketing studies for novel drugs approved by both the FDA and EMA between 2005 and 2010: a cross-sectional study.BMJ open · 2017Article
- Therapeutic enquiries about biological agents as a tool to identify safety aspects and patterns of use.European journal of hospital pharmacy : science and practice · 2016Article
- Off-label use of targeted therapies in oncology.World journal of clinical oncology · 2016Review
- Off-Label Uses of Omalizumab.Clinical reviews in allergy & immunology · 2016Review
- A Case of Interstitial Lung Disease Probably Related to Rituximab Treatment.Drug safety - case reports · 2015Article
- It is time to review how unlicensed medicines are used.European journal of clinical pharmacology · 2015Review
Corrections and comments
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Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeThe study aims to assess the clinical evidence, outcome and cost of off-label use of medicines in the hospital setting.
methodsA multicentric prospective cohort study of patients treated with off-label medicines was carried out in five tertiary hospitals from May 2011 to May 2012. Information on clinical characteristics of patients, drugs, outcomes and costs was collected. Patients were followed up to 6 months, and information was assessed by reviewing clinical records and interviewing physicians.
resultsA total of 226 patients were included. The median (interquartile range (IQR)) age of patients was 46 (33-62) years; 59 % were women. Patients had received a median of three previous treatments, and a lack of response (or suboptimal) was the main reason for off-label use (72.1 %). A total of 232 off-label medicines were administered for 102 different indications. The most frequent medicines were rituximab (49; 21.1 %), botulinum toxin (25; 10.7 %) and omalizumab (14; 6.0 %). In 117 (51.8 %) cases, the level of clinical evidence for their use was low. A partial clinical response was observed in 82 patients (36.3 %), complete response in 71 (31.4 %) and stabilization in 11 (4.9 %). A total of 58 (26.5 %) patients had adverse effects, which in 11 (4.9 %) were severe. The median (IQR) cost per patient was <euro>2,943.07 (541.9-5,872.54).
conclusionsThere was a high variability of off-label medicines and indications. Although the clinical evidence of off-label medicines was often low, clinical response was observed in many patients with previous multiple treatment failure, but at the expense of some adverse effects and a high cost. Registers of patients would be helpful for clinical decisions, although clinical trials are needed.
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