According to the Canadian Organization for Rare Disorders (CORD), it has been more difficult to gain access to orphan drugs than it is in other developed countries. This main reason for this challenge is the complex drug approval system in Canada, where the federal government – through Health Canada – approves drug trials and the drugs, but the provinces determine whether they fund/reimburse those therapies, and declining it even if proven safe and effective by Health Canada.
This is already affecting thousands of rare disease patients in Canada, who in some cases already have a life-sustaining therapy available in the market. This is the case of Morgan MacIntyre, a 12-year old girl suffering from atypical hemolytic uremic syndrome, or aHUS, an ultra-rare disease which is now treated by the famous and recently approved orphan drug, Soliris, from Alexion Pharmaceuticals. The drug costs an average of $440,000 a year, and was approved by Health Canada to treat aHUS last spring, but while it is approved for funding in Quebec, it is not in Ontario.
Another layer to this complex reimbursement system in Canada is the Common Drug Review, an independent agency that assesses drug cost-effectiveness for all of the provinces except Quebec. The CDR makes its own assessment, and can recommend that a drug not be listed for provincial funding. For aHUS, the agency’s review panel had a number of reservations about the drug, including the fact that once patients start taking it, they must keep on taking it.
The reason why orphan drugs are constantly being rejected for funding is because orphan drug trials can’t possibly meet the same standards as those for drugs for large populations.
Earlier in April 2013, David Lee from Health Canada, spoke at the World Orphan Drug Congress USA, about the recent initiatives to align the reimbursement policies for orphan drugs in Canada, in a similar move to what’s been done in the European Union by CAVOMP. This shows how important it is to address orphan drug reimbursement differently than through the same guidelines for large population drugs.