Outcomes of off-label drug uses in hospitals: a multicentric prospective study

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 €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.Electronic supplementary materialThe online version of this article (doi:10.1007/s00228-014-1746-2) contains supplementary material, which is available to authorized users.

Off-label use of targeted therapies in oncology

“…Among targeted therapies, rituximab is the agent that probably carries the greatest potential for off-label indications mostly beyond oncology. Indeed, a Spanish prospective investigation reported that rituximab was the most frequently used agent off-label (21.1%) among 232 drugs (considering all therapeutic classes) in 5 tertiary hospitals during one year (2011-2012) [14] . In addition, a prospective Australian national study found that offlabel use of rituximab covers 63 different diagnosis with 89% of off-label use outside oncology in the year 2012 [15] .…”

Off-Label Uses of Omalizumab

“…Recently, a multicentric prospective cohort study of 226 patients treated with off-label medicines was carried out in five tertiary hospitals from May 2011 to May 2012 [9]. Omalizumab was used in 6 % of patients.…”

Evaluation of Knowledge, Attitudes and Experience of off-label Drug Prescribing Practice among Physicians in Baghdad City Hospitals

“…However, it has been noted that healthcare professionals commonly prescribe medicines off-label with levels of evidence considered to be low. This is principally problematic because off-label use with inadequate strong scientific evidence may be associated with higher rates of adverse events that may harm the patient (2)(3)(4) . Off-label prescribing is not certainly bad.…”