Background Off-label treatments are characterised as those that do not correspond to the labelled indications. They are intended to respond to the unmet medical needs of poorly studied populations or not studied at all in trials. Off-label treatments are steadily increasing in the onco-haematology area.

Purpose The aim of this study was to describe off-label treatments prescribed in the oncology-haematology area of a third level hospital.

Material and methods This was an observational retrospective study including all off-label drugs prescribed from March 2015 to September 2016. Analysed data were: demographic (age, sex) and treatment related (drug involved, off-label indication, treatment duration and economic impact). Data were collected from the pharmacy electronic prescription programme and multidisciplinary oncology–haematology commission database.

Results 63 off-label drug uses were approved out of 67 requested during the study period (6% rejected due to lack of evidence); oncology was the area that solicited the highest quantity of off-label medications, 53/67 (79.1%); haematology requested 14/67 (20.9%). Patient median age was 55 years (range: 25–85); 27 men (demographic data lacking in 7/63). The top four off-label drugs prescribed were: (i) bevacizumab (n=25), (ii) crizotinib (n=5), (iii) paclitaxel–albumina (n=5) and (iv) nivolumab (n=6). The most frequent off-label indications solicited were: (i) treatment of glioblastoma for bevacizumab (18/25); (ii) NROS mutated non-small cell lung cancer for crizotinib (3/5); (iii) neoadjuvant treatment of breast cancer for paclitaxel–albumina (3/5); (iv) Hodgkin refractory lymphoma for nivolumab (2/6). Median treatment duration was: 4.2 months (range 0.3–16.1), 3.6 months (range 1.6–11.0), 2.8 months (range 1.5–7.3) and 2.8 months (range 0.3–3.1), respectively. Median cost per patient and drug was: €18 819.80 (range €5790.70–€72 384) for bevacizumab; €18 705.60 (range €9352.80–€56 116.80) for crizotinib; €3469.76 (range €1301.20–€4770.90) for paclitaxel–albumina; €8192 (range €2048–€12 288.80) for nivolumab.

Conclusion Off-label drug use is a common practice in the onco-haematology area. It is very important to analyse the clinical outcomes of off-label drug uses in order to improve decision making, protocol generation and to estimate the economic impact on our healthcare system.

References and/or acknowledgements Levêque D. Off-label use of targeted therapies in oncology. World J Clin Oncol2016;7:253–7.

No conflict of interest