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DI-028 Off-label use: a descriptive retrospective analysis
  1. S Buendía-Bravo,
  2. A Giménez Manzorro,
  3. B Monje García,
  4. M Sanjurjo Sáez
  1. Hospital General Universitario Gregorio Marañón, Hospital Pharmacy, Madrid, Spain


Background Off-label use is covered by the Spanish law, which establishes its evaluation and management in the hospital setting. Therefore hospital pharmacy budgets must include this financial burden.

Purpose To describe the frequency of off-label use, what drugs and indications are prescribed off-label and its financial burden in a hospital setting.

Material and methods A retrospective observational study was conducted. Off-label uses during 2013 were recorded. We collected the following variables: drug name, therapeutic use, number of patients and cost. The information was assessed by reviewing our Hospital Pharmacy off-label databases. Costs were calculated according to the purchase prices of our hospital.

Results We found 62 drugs in 131 off-label indications. These drugs were used by 809 patients. 18.3% were children. 29% of the drugs were used by 86% of the patients. The most frequent and the highest financial impact drug uses were: erythropoietins for myelodysplastic anaemia in adult patients (87 patients, €257,484) and sildenafil for children with pulmonary hypertension (87 patients, €143,460).

Other expensive off-label uses were: immunoglobulins for neuropathy (€283,863) and octreotide for angiodysplasia with bleeding (€142,489).

The most frequent diseases to be treated off label were: lupus (mycophenolate mofetil, 55 and rituximab, 75 patients), toxicity associated with hepatitis C treatment (filgrastim or erythropoietins, 88 patients) and pulmonary hypertension (sildenafil, 87 patients).

In all cases, there were no other therapeutic options.

Conclusion A large number of drugs are prescribed off-label and some of them are expensive. Therefore, off-label use is associated with a high financial burden.

References and/or acknowledgements No conflict of interest.

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