Main Article Content

Nigel S B Rawson
Arpit Chhabra


Off-label drug use, drug reimbursement, drug coverage, Ontario


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.

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.
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.

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.
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.

Abstract 1169 | pdf Downloads 466


1. Rawson NSB. Including off-label drug indications in HTA jeopardizes patient health and discourages innovation. [Internet] Can Health Policy; 2016. Available at:
2. Anti-vascular endothelial growth factor (VEGF) drugs for the treatment of retinal conditions: recommendations report. [Internet] CADTH; 2016. Available at:
3. Hoffman La Roche Ltd. Avastin Product Monograph [Internet] 2018.
4. Curtis LH, Hammill BG, Schulman KA, Cousins SW. Risks of mortality, myocardial infarction, bleeding, and stroke associated with therapies for age-related macular degeneration. Arch Ophthalmol 2010;128:1273–9.
5. Gower EW, Cassard C, Chu L, Varma R, Klein R. Adverse events following intravitreal injection of Avastin or Lucentis for treating age-related macular degeneration (abstract 6644). Association for Research in Vision and Ophthalmology Annual Meeting; May 2011.
6. CATT Research Group. Ranibizumab and bevacizumab for neovascular age-related macular degeneration. N Engl J Med 2011;364:1897–908.
7. Novartis Pharmaceuticals. Lucentis Product Monograph. 2017. [Internet]. Available at:
8. Bayer Inc. Eylea Product Monograph. 2018. [Internet]
9. Wittich CM, Burkle CM, Lanier WL. Ten common questions (and their answers) about off-label drug use. Mayo Clin Proc 2012;87:982–90.
10. Carton L, Cottencin O, Lapeyre-Mestre M, et al. Off-label prescribing of antipsychotics in adults, children and elderly individuals: a systematic review of recent prescription trends. Curr Pharm Des 2015;21:3280–97.
11. Saiyed MM, Ong PS, Chew L. Off-label drug use in oncology: a systematic review of literature. J Clin Pharm Ther 2017;42:251–8.
12. Culshaw J, Kendall D, Wilcock A. Off-label prescribing in palliative acre: a survey of independent prescribers. Palliat Care 2013;27:314–9.
13. Choonara I, Conroy S. Unlicensed and off-label drug use in children: implications for safety. Drug Saf 2002;25:1–5.
14. Eguale T, Buckeridge DL, Verma A, et al. Association of off-label drug use and adverse drug events in an adult population. JAMA Intern Med 2016;176:55–63.
15. Government of Ontario. Get coverage for prescription drugs [Internet]. 2018. Available at:
16. Government of Ontario. Formulary search [Internet] 2016. Available at:
17. Government of Ontario. Exceptional Access Program (EAP) reimbursement criteria [Internet] 2018. Available at:
18. Rawson NSB. What does the new Ontario pharmacare plan offer children and young adults with rare disorders? J Popul Ther Clin Pharmacol 2017;24:90–8.
19. Government of Ontario. EAP reimbursement criteria for frequently requested drugs [Internet] 2017. Available at:
20. Government of Ontario. Telephone request service reimbursement criteria Exceptional Access Program [Internet] 2015. Available at:
21. Health Canada. Drug product database online query [Internet] 2018. Available at: