Objective: To assess the extent of off-label prescribing in specialty headache practice.
Methods: A prospective record was kept of all prescriptions written during a 30-day period in a tertiary care headache program affiliated with two teaching hospitals. Each drug was categorized as "on-label," defined as approved by the FDA for a headache or general pain indication, and used in accordance with label instructions, or "off-label," defined as any use of a drug not covered in the FDA-approved package insert.
Results: A total of 379 prescriptions were written during a 30-day period. One hundred and seventy-eight prescriptions (47%) met the criteria for off-label use. In all, 23 categories of off-label treatment were prescribed during the study, but just 4 accounted for over half of all off-label prescriptions: newer antiepileptic drugs such as topiramate and lamotrigine (each accounted for n = 26, 15% of off-label prescriptions), newer antidepressants, especially venlafaxine (n = 27; 15% of off-label prescriptions), and botulinum toxin type A (n = 13; 7% of off-label prescriptions). Two hundred and one prescriptions met criteria for on-label use. The largest percentages of prescriptions written for approved, on-label indications were for triptans (n = 74; 37% of on-label prescriptions), and nonsteroidal anti-inflammatory drugs (n = 64; 32% of on-label prescriptions).
Conclusions: Off-label prescribing is common in the specialty management of headache conditions. We conclude that it is within the current standard of care, and an integral part of practice, to use off-label medications in the treatment of complex headache conditions.