Canada is one of the few developed countries without a specific regulatory framework for drugs for rare diseases, often termed “orphan drugs”. Over the past few years, the federal government has been working on revising the Canadian Food and Drug Regulations to implement a specific pathway for these drugs. As reported in our earlierPharma-in-Brief in October 2012, the government has been taking steps to implement a regulatory framework, including the government’s review and powers of oversight over this class of drugs. A key focus of the new approach will be on international information-sharing and collaboration for the development and regulation of orphan drugs.
In August 2014, the Minister of Health Rona Ambrose announced a pilot project targeting patient input from Canadians to help inform future reviews of orphan drugs. The project will simulate how input from patients will be gathered and incorporated into the drug submission review process once the Orphan Drug Framework is in effect. While the regulatory framework has yet to be announced and there is no set definition as yet as to what qualifies as a “rare disease”, communications from the government suggest the definition to be in line with that used by other jurisdictions, namely, that a rare disease is “a life-threatening, seriously debilitating, or serious chronic condition that only affects a very small number of patients (typically less than 5 in 10,000 persons).”
In a journal article published in February 2014 (J Popul Ther Clin Pharmacol Vol 21 (1): e42-e46: February 23, 2014) it was noted that of the 99 drugs with orphan indications approved by the U.S. Food and Drug Administration (FDA) from 2008 to 2013, as of May 2013, only 51 of these had been approved (through the regular submission process) in Canada. Another 21 were approved for sale in Canada under the special access programme. The article also explains the intended approach of Health Canada, noting that regulations will set the criteria for designation, aligned to the greatest extent possible with those used by the European Medicines Agency and the FDA, will outline the requirements for market authorization and will mandate post-market oversight. Flexibility in the design of trials and evaluation of results will be allowed to accommodate smaller patient populations.
There is currently no timeline established for introduction of the new Regulations. However, given that amendments to the Food and Drugs Act are still before Parliament (in Bill C-17, an Act to Amend the Food and Drugs Act, introduced on December 6, 2013), which is directed towards patient safety and includes many broad regulation-making authorities, publication of orphan drug regulations for comment could well be awaiting passage of that Bill. In terms of steps to finalization, the Regulations will need to be first published for comment in Canada Gazette Part I, with comments received then taken into account for final publication of the regulations in Canada Gazette Part II.
Link to the government news releases: