On October 3, 2012, Health Canada issued a press release announcing that it will develop a framework for the marketing approval of drugs to treat rare life-threatening, seriously debilitating or serious chronic rare diseases that are commonly referred to as "orphan drugs."
Currently, orphan drugs are approved in Canada through the same regulatory pathways as other drugs. Because of the small patient population for orphan drugs, other jurisdictions, including the United States and Europe, have created legislative incentives to encourage research into rare diseases. It appears from the press release that Health Canada also recognizes this need and has committed to implementing a new approach to help bring orphan drugs to the Canadian market. These changes will not address the pricing for or reimbursement of orphan drugs.
Changes to the Food and Drug Regulations will be required to implement the new regulatory framework. Once proposed regulations are published, the public will have an opportunity to comment before final regulations are in force. Health Canada has previously consulted with stakeholders, including the Canadian Organization for Rare Disorders, in making its decision to support orphan drug development. At this stage, it is unclear how an orphan drug will be defined in Canada.
In the United States, the Orphan Drug Act defines a rare drug as (i) one that affects less than 200,000 persons in the United States, or (ii) one that affects more than 200,000 in the United States but there is no reasonable expectation that the cost of developing the drug for the rare disease will recovered from sales in the United States. If a drug is deemed to be a rare drug, the U.S. Food and Drug Administration cannot approve another drug for the same indication for seven years from the approval date of the orphan drug. The Act also provides for certain tax incentives for clinical trials.
The details of Canada’s regulations will be important for manufacturers to consider, particularly given that the population in Canada is smaller than that in the United States.