The First International Rare Diseases Day was celebrated in Canada, where it was also the occasion for a member of parliament (MP) from Vancouver to unveil a motion calling for a national rare disease policy.

Don Bell, a MP from North Vancouver, lost a grandson to a rare disorder last July, according to CORD, the Canadian Organisation for Rare Diseases. Mr. Bell has created private member Motion M-426, presented at a House of Commons Debate in mid April.

The motion, following an initiative promoted by CORD, seeks to: establish a definition for serious rare disorders as those with a prevalence of less than 1 in 2000 Canadians; examine the feasibility of a national “Chance for Life Fund” equivalent to 2% of the total annual public drug expenditure to be designated for therapies for rare disorders; consider the establishment of a multi-stakeholder advisory body, including treaters and patients, to recommend treatment access for life-threatening or serious rare disorders, based on scientific standards and social values; consider the establishment of centres of reference for specific rare disorders, comprised of national and international experts, who will develop criteria for treating patients based on scientific evidence and patient impact and provide on-going surveillance into the real-world safety and effectiveness of these treatments on individual and group basis; consider options to provide incentives through orphan drug regulation and policy, to assure Canadian organisations and researchers are motivated to conduct research and development into treatments for rare and neglected disorders; support internationally accepted standards for conduct of clinical trials in rare disorders appropriate for the challenges inherent to very small patient populations; consider ensuring that Health Canada’s progressive licensing framework provide appropriate support to the design of clinical trials for very small patient populations and appropriate review of evidence submitted from these trials; and report the progress accomplished to the House within six months.

The motion has been worded deliberately to ensure it could be immediately “votable” and not require “government expenditure or other procedural restrictions to its passage”. Canada is amongst a dwindling handful of developed countries that do not yet have rare disease and orphan medicine policies in place to encourage the development of rare disease research and treatments. Whether or not a medicinal product is funded varies from region to region under the current health scheme. The Canadian press recently reported widely the case of a young Hunter syndrome patient who could not access an expensive but effective treatment that was not funded in his home province of Ontario, though it is funded in other regions of the country, such as British Columbia. The House of Commons approved Motion M-426 on 7 May. It is now up to the government to examine and respond specifically to the challenges delineated in the motion within a year.

Read the Motion hearing