Public reimbursement of prescription drugs used for off-label indications in Ontario

Abstract

Background A Canadian Agency for Drugs and Technologies in Health 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’s Product Monograph (PM) has a serious safety warning that the drug is not formulated or authorized for intravitreal use. Objective To evaluate whether the Ontario Public Drug Programs (OPDP) reimbursement is provided only for off-label use for serious conditions or when the drug’s PM contains a serious safety warning. Methods Comparisons were made between the OPDP reimbursement criteria for non-palliative drugs from its frequently requested Exceptional Access Program (EAP) list and for non-palliative injectable drugs from its Limited Use (LU) list and approved indications and serious safety warnings 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 serious safety warnings, but only one had a serious safety warning directly related to the OPDP-reimbursed off-label use. Of 29 non-palliative injectable LU drugs, one had off-label LU criteria allowing reimbursement for an unapproved indication and a serious safety warning unrelated to the reimbursable indication. Conclusion Presently, OPDP only reimburse drugs for off-label use for the treatment of serious conditions for which no alternative treatment exists. OPDP should not diverge from this approach by allowing cost-savings to trump appropriate drug use.