Posted by Michael Wonder on 06 Mar 2018
Access to new drugs for rare disorders in Canada
2 March 2018 - McMillan and Campbell believe that Canada needs a regulatory framework for the introduction of orphan drugs for rare disorders.
We agree. However, the need is unlikely to be filled in the foreseeable future because, in October 2017, the present federal government deleted, without notice or consultation, all references to the previous government’s 2012 Orphan Drug Regulatory Framework.
Access to a new drugs for rare disorders not only depends on its regulatory approval but also on the drug successfully passing the Canadian Agency for Drugs and Technologies in Health’s (CADTH’s) Common Drug Review (CDR) process, negotiations regarding price between the pan-Canadian Pharmaceutical Alliance (pCPA) and the manufacturer, the Patented Medicine Prices Review Board price assessment process, and individual drug plan evaluations and negotiations with the manufacturer.
Although CADTH has considered establishing a focused review process for DRDs, it chose to integrate their review into the usual CDR process. Nevertheless, Richter et al. from CADTH recently analysed CDR recommendations for DRDs made between 2004 and 2016 and suggested that it may be inappropriate to apply the same appraisal standards to drugs for ultra-rare disorders (those with a prevalence of ≤1 per 100,000).
