When Canada’s draft Orphan Drug Regulatory Framework suddenly disappeared from the government website, my immediate reaction was “it’s the kiss of death” for rare disease drugs in Canada.  Not because we disbelieve Health Canada’s claim that they are able to approve orphan drugs without new legislation ­— but without an Orphan Drug Policy, there has been little incentive to do orphan drug research, to conduct clinical trials, and to bring in drugs in a timely fashion.

Only half of USA-approved orphan drugs come to Canada,  and often with long delay.  Which is why, in 2007, the Canadian Organization for Rare Disorders drafted an Orphan Drug Policy, and why we were thrilled when, in 2012, the Health Minister produced an Orphan Drug Regulatory Framework.  This framework would allow clinical trials and orphan drug approvals to take place in Canada at the same time as in the USA and the European Union.  But the Conservative government failed to implement it and the Liberal government has now removed the framework from its website.

Even worse, more than half of Canadian – approved orphan drugs are not funded by the public drug plans.

In many countries, orphan drugs are reimbursed through their own review process.  In contrast, the Canadian Agency for Drugs and Technologies in Health, which reviews drugs for funding, disproportionately recommends against rare disease drugs, primarily because they apply, inappropriately, standards for evidence and cost-effectiveness designed for “common” drugs.  The result leaves more than half of Canadian approved orphan drugs not funded by public drug plans.

Even OHIP+, Ontario’s total pharmacare program for those under 25, will not cover most rare disease drugs.

What is more frightening is the likelihood that access will get worse under the government’s proposed changes to the Patented Medicines Prices Review Board, which will create additional barriers for new drug entry, especially rare disease drugs.  Now, more than ever, an Orphan Drug Policy is needed to ensure that Canada does not fall even further behind other developed countries in serving some of the most vulnerable patients, those affected by rare diseases.