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

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.

KEYWORDS:

• Orphan drugs
• health technology assessment
• drug prices
• drug insurance
• Canada

Declaration of interest

Over the past three years, NSB Rawson received research and consultation fees from AbbVie Canada, Canadian Cancer Survivor Network, Canadian Health Policy Institute, Fraser Institute, Macdonald-Laurier Institute and 3Sixty Public Affairs, and an article processing expense from RAREi (a network of Canadian biopharmaceutical companies committed to improving the lives of rare disease patients by researching, developing and commercializing rare disease treatments). During the same period, J Adams received research and consulting fees from the Alliance for Safe Online Pharmacies, Canadian PKU and Allied Disorders Inc. and Macdonald-Laurier Institute, and a fee from BioMarin Pharmaceuticals for speaking to European PKU patient leaders. No conflict of interest exists between these activities and the present work. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or material discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contribution statement

Both authors made a significant contribution to the work reported in the conception, study design, acquisition of data, analysis and interpretation. Both take responsibility and accountability for the contents of the article and share responsibility to resolve any questions raised about the accuracy or integrity of the work.

Data availability statement

The data used in this analysis are available from publicly accessible resources [8–23, 25–31,53–56].

Additional information

Funding

This paper was not funded.