Background Off-label prescription is legal and common, but often not supported by strong evidence.
Purpose To analyse off-label treatments with biological therapies, its indications and to determine its economic impact within the total cost of these drugs.
Material and methods This observational descriptive study was conducted in a reference hospital from October 2015 to March 2016. Treatments codified in the dispensation system (Savac) according to the diagnosis ‘Treatments not included in data sheet’ and those who had a defined diagnosis but were not reflected in the data sheet were included.
Drug, diagnosis and clinical service prescriber were collected from the electronic clinical record (Selene) and request reports sent to the pharmacy. The number of dispensations and spending (both off-label and total spending on biological therapy in the study period) was recorded from Savac.
Results During the study period 34 off-label treatments were prescribed to 32 patients. Applicant services were: internal medicine (35.3%), allergology (26.5%), digestive (12.7%), dermatology (11.8%), ophthalmology (8.8%) and rheumatology (2.9%). 11 drugs were used in 18 different indications. The most employed was omalizumab with 26.5% (33.33% food allergy, 33.33% anaphylaxis and the rest for atopic dermatitis, allergic rhinitis and urticaria acute recurrent) followed by adalimumab with 14.7% (uveitis non-infectious), ustekinumab with 11.8% (75% Crohn’s disease and 25% atopic dermatitis), tocilizumab with 11.8% (spondyloarthritis, papilophlebitis, thyroid associated orbitopathy and uveitis), and the rest had low values. In terms of economic impact, off-label use represented 4.73% (€122 482.7) of total expenditure on biological therapies during the study period; 1.19% at the cost of omalizumab. Regarding the total cost of each drug, off-label use was 57.34% of total expenditure for tocilizumab; 11.55% for rituximab, 10.85% for omalizumab, 8.91% for ustekinumab and less than 5% for the rest.
Conclusion Our study results showed that off-label use of biological agents has important economic and healthcare impact; some biologicals represented >50% of expenditure (eg, tocilizumab). A monitoring mechanism should be set up to evaluate the efficacy and safety of these treatments due to insufficient clinical experience with them.
No conflict of interest
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