Health Canada is shortly to send out for public consultation new proposals aimed at streamlining patients' access to orphan drugs and spurring research into the development of such treatments.

The agency says it is developing a modern framework for the designation, authorisation and monitoring of orphan drugs, with a key focus on international information-sharing and collaboration for the development and regulation of such products. "Enabling Canadian scientists and regulator to participate with trusted global counterparts will make better use of scarce resources and benefit Canadian patients," says Health Canada.

The new framework will maintain evidence requirements based on clinical trials and will be supported by greater information-sharing amongst international partners who are committed for pooling scarce resources for maximum benefit. Once authorised, treatments will continue to be closely monitoring for effectiveness and safety while in use, the agency says.

Announcing the proposals, Health Minister Leona Aglukkag also reported that Canada is set to become the first country in North and South America to launch Orphanet, a comprehensive database of information and services for rare diseases.

"Too often, Canadians dealing with rare diseases are faced with difficulties in accessing the information and medication they need," she said, and the proposed new approach "will better support the development and authorisation of drugs for rare diseases and launch of a new web portal to assist patients in finding the information and services they need." 

Currently, when an orphan drug is not available in Canada, doctors can apply individually for each patient through Health Canada's Special Access Programme (CAP). While facilitating access, CAP also represents a significant burden to the healthcare system, and a regulatory framework designed and used specifically to approve drugs to treat small, vulnerable populations will more effectively address this need, say government officials.

Based on existing international experience and knowledge, the new framework will provide Health Canada with new tools to gather and share information, including the registration of clinical trials. It will also facilitate patient participation in this highly-specialised area, they add.

The plans have been welcomed by the Canadian Organization for Rare Disorders (CORD), which points out that they would reverse a 16-year-old Health Canada policy "denying the need for an orphan drug policy in Canada." The moves also come nearly 30 years after the US passed the world's first orphan drug act and 12 years after the European Union (EU) did for the same for its 27 member states.

In the decade before 1980, there were only 10 new drugs for rare diseases but, since the 1983 US legislation, there have been more than 300 such treatments. However, until now, Canada's 2.8 million people with rare disorders have had access to only about half of these, and usually several years later, says CORD.

Moreover, most Canadian provinces and the Common Drug Review (CDR) - the pan-Canadian process for reviewing the clinical, cost-effectiveness and patient evidence for drugs conducted by the Canadian Agency for Drugs and Technologies in Health (CADTH) - routinely deny funding for drugs for rare diseases, even after they are approved by Health Canada, it says. Ontario, British Columbia and Alberta do have "special pathways" for rare-disease drugs but there is no consistency, so patients in one province may receive treatment while their relatives in another province will be denied, says the group, which calls instead for something similar to the "risk-pooling" scheme announced by private drug plans earlier this year that will significantly benefit patients with rare diseases and others requiring high-cost innovative therapies.