Regulatory approval and public drug plan listing of new drugs for rare disorders in Canada and New Zealand

J Popul Ther Clin Pharmacol. 2020 Jun 1;27(2):e58-e67. doi: 10.15586/jptcp.v27i2.673.


A previous assessment of the alignment of health technology assessments and price negotiations for new drugs for rare disorders in Canada completed between 2014 and 2018 demonstrated that it is working for governments but has yet to lead to improved access in a timely manner for all appropriate patients in all provinces. In this analysis, drugs for rare and ultra-rare disorders with a completed price negotiation or no negotiation between 2014 and 2018 in Canada, and their reimbursement recommendations and listings in Canadian public drug programs are compared with their regulatory approval in New Zealand and listing in the New Zealand National Formulary. The results show that pharmaceutical manufacturers generally seek regulatory approval for rare disorder drugs in Canada before New Zealand, and fewer rare disorder medicines receive regulatory approval in New Zealand. One reason for this difference might be New Zealand's smaller population. However, another reason is likely the restrictive drug formulary in New Zealand. Drugs not given coverage in New Zealand are frequently made unavailable by the manufacturer. Planned changes to Canada's pricing regulations and guidelines will significantly diminish the country's attractiveness as a place in which pharmaceutical companies want to do business, which has the potential to negatively impact the health of all Canadians irrespective of whether they have private or public drug coverage.

Keywords: Canada; New Zealand; drugs; formulary listing; rare disorders; regulatory approval.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Canada
  • Drug Approval / legislation & jurisprudence*
  • Drug Costs
  • Drug Industry / economics
  • Drug Industry / legislation & jurisprudence
  • Formularies as Topic
  • Guidelines as Topic
  • Health Services Accessibility
  • Humans
  • New Zealand
  • Orphan Drug Production / economics
  • Orphan Drug Production / legislation & jurisprudence*
  • Rare Diseases / drug therapy*
  • Rare Diseases / economics
  • Reimbursement Mechanisms
  • Technology Assessment, Biomedical