Orphan drugs approved in Canada: health technology assessment, price negotiation, and government formulary listing

Abstract

ABSTRACT Background The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have incentives to stimulate the development and marketing of orphan drugs. Health Canada has none. Methods We identified 82 FDA and/or EMA-designated orphan drugs approved by one or both agencies between 2015 and 2020 that were also authorized in Canada. We tracked the drugs through health technology assessments (HTAs), price negotiations, and listing in government drug plans to assess the time required for these processes. Results Median times for HTAs and price negotiations suggest a delay of around a year, but the median wait time between marketing authorization and price negotiation completion was over 18 months. Conclusions Listing of orphan drugs in Canadian government drug plans is closely aligned with reimbursement recommendations and outcomes of price negotiations. Medicines with unsuccessful price negotiations are not listed. However, not all drugs with successful negotiations are listed by all provinces and listing does not guarantee patient access. Compared with Americans and some western Europeans, Canadians with rare disorders continue to suffer from a lack of timely and equitable access to innovative treatments. A comprehensive orphan drug policy would improve Canadians’ access to the innovative treatments on the research horizon.