That, in the opinion of the House, the government should respond specifically to the challenges faced by Canadians with rare diseases and disorders, in collaboration with provinces and territories and stakeholders by: ( a) examining options for defining serious rare diseases; (b) examining options, including the possible creation of a specific fund, to improve access to rare disease treatments, building on recent work undertaken by federal and provincial/territorial governments under the National Pharmaceuticals Strategy; (c) considering the establishment of a multi-stakeholder advisory body, including the Common Drug Review, treaters and patients, to recommend treatment access for life-threatening or serious rare disorders, based on scientific standards and social values; (d) exploring options to consider national and international expert advice in developing criteria for treating patients based on scientific evidence and patient impact, and to link these activities with ongoing post-market monitoring of real world drug safety and effectiveness; (e) considering options to encourage research and development into treatments for rare diseases and other unmet health needs; (f) considering internationally accepted standards for conduct of clinical trials in rare disorders appropriate for the challenges inherent to very small patient populations; (g) considering how Health Canada’s work on a progressive licensing framework could provide appropriate support to the design of clinical trials for very small patient populations and appropriate review of evidence submitted from these trials; and (h) reporting the progress accomplished to the House within 12 months.
