Background Few studies have been undertaken on off-label drug use in Spain since the introduction of new legislation in 2009 in which the responsibility for off-label use was transferred from the Spanish Agency for Medicines and Health Products to the physician.
Purpose To analyse which clinical units requested off-label drugs more often, for what treatments and indications they were used, and to establish the economic impact this had on a Spanish university hospital.
Materials and methods Descriptive observational study from October 2009 to December 2012. We included all individual requests for off-label drugs received in the pharmacy service. The request was submitted by physicians who indicated prior treatment received and the reasons deemed appropriate for the requested treatment. Individualised assessment reports were written with an analysis of efficacy, safety, convenience and cost, which were referred to the hospital’s medical administration to make the decision to authorise or deny their use.
Results A total of 512 requests were analysed, of which 72.7% were for antineoplastics (372), followed by the musculoskeletal system with 8.2% (42). The most-requested drugs were bevacizumab with 13.3% (68) and rituximab with 8.2% (42) of requests.
It was observed that the most frequent off-label indications were for glioblastoma with 7.8% (40) and breast cancer 5.3% (27) of requests.
Requests for adult units represented 80.5% (412), being mostly oncology at 43.9% (181) and haematology with 15.5% of requests (64). Paediatric clinical units performed 9.5% (100) of requests, of which the most frequent were onco-haematology with 49% (49) and rheumatology with 11% (11).
The cost of off-label drugs authorised was 4,938,808 € with a median cost per patient of 7,340 € [1,307 €, 16,728 €] and represented 3.03% of the total expenditure of the pharmacy service. Treatments that were rejected would have meant an expenditure of 1,656,644 € and this would have been 1.02% of drug spending.
Conclusions Off-label drugs were requested mostly in the field of oncology and haematology. The authorised off-label drugs are very expensive. Unapproved treatments would have increased costs with theoretically minimal health benefits.
No conflict of interest.
Statistics from Altmetric.com
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.