DI-011 Analysis of off-label use in biological therapies

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Biologics are usually prescribed off-label in severe immune mediated inflammatory diseases. Unfortunately, off-label prescription is sometimes hampered in these diseases due to a lack of evidence of effectiveness.


To assess the risk and outcome of biological therapies on off-label practices in the pharmacy department of a tertiary hospital.

Material and methods

This study included all patients treated between January 2014 and June 2016 with an off-label biological prescription. The data were collected from the clinical history of the patients and from the pharmacy programmes: athos, prisma and cafydim. We analysed the following variables: treatment time, doses, adverse drug reactions (ADRs) and previous/subsequent treatments. Treatment repercussion were evaluated. The data were analysed through a statistical descriptive study.


A total of 5 types of off-label biologics were requested and administered to 30 patients for 9 different diseases.


In 80% (24) of cases the patient had been treated previously with corticosteroids and/or methotrexate. In 20% (6), the previous treatment was a biologic (not off-label prescription). Most of the prescriptions were adalimumab: 20 (67%), of which 8 (40%) were treatment for Behcet’s syndrome, 5 (25%) for uveitis in children, 5 (25%) for sarcoidosis (1 of whom was a child of 11 years old), 1 (5%) for mesenteric panniculitis and 1 (5%) for systemic lupus erythematosus (SLE). All other prescriptions were: 2 golimumab for synovitis and sarcoidosis, 2 tocilizumab for SLE, 1 anakinra for familial Mediterranean fever, 3 ustekinumab for enteritis, Crohn’s disease and Wegener’s granulomatosis, and 2 certolizumab for uveitis and enteritis. Only for 1 patient (3.3%) was treatment not effective: adalimumab for sarcoidosis, moreover it produced ADRs (increased morning stiffness and pain in joints) after 30 months so the patient returned to methotrexate monotherapy. 9 (30%) patients developed ADRs: 6 from adalimumab (of which 3 were in children), the remaining 3 ADRs were from golimumab, tocilizumab and anakinra treatment. All were mild/moderate and were not grounds for treatment discontinuation.


In our assessment, off-label biological therapies were effective in most patients (96.6%) and were safe (70%). Evaluation of the cost of off-label biological therapies, in terms of medication risk and cost to healthcare, will be essential to their widespread clinical utility.


No conflict of interest

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