Public Reimbursement of Prescription Drugs Used For Off-Label Indications in Ontario.

BACKGROUND: A Canadian Agency for Drugs and Technologies in Health (CADTH) therapeutic review concluded that bevacizumab and ranibizumab have similar efficacy and safety in treating retinal conditions and recommended bevacizumab be used as preferred initial therapy based on a cost-saving perspective. Such use would be off-label because bevacizumab is not approved for these conditions and has a serious safety warning (SSW) in its Product Monograph (PM) about intravitreal use.

OBJECTIVE: To evaluate whether the Ontario Public Drug Programs (OPDP) reimbursement is provided only for off-label use for serious, life-threatening or severely debilitating conditions and not when the drug's PM contains a SSW against such use.

METHODS: OPDP reimbursement criteria for non-palliative drugs from its frequently-requested Exceptional Access Program (EAP) and Limited Use (LU) lists were compared with approved indications and SSWs in the drugs' PMs.

RESULTS: Of 125 unique frequently-requested non-palliative EAP drugs, 12 included off-label use for serious conditions for which no alternative treatment exists. Eight of the 12 had SSWs, but only one warning for orally-administered sirolimus related directly to the OPDP-reimbursed off-label use. Of 29 injectable non-palliative LU drugs, one had off-label LU criteria allowing reimbursement for an unapproved indication and a SSW unrelated to the reimbursable indication.

CONCLUSION: Presently, OPDP only reimburse drugs for off-label use for the treatment of serious, life-threatening or severely debilitating conditions for which no alternative treatment exists. OPDP should not diverge from this approach by allowing cost-savings to trump appropriate drug use, which would set a unique and unprecedented example.