title: Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?
abstract: BACKGROUND
  Over the past 20 years, the number of therapies developed for rare diseases has rapidly increased. Often, these therapies represent the only active treatment for debilitating and/or life-threatening conditions. However, they create significant challenges for public and private payers. Because they target small patient populations, clinical evidence of efficacy/effectiveness is typically limited, while the cost per patient is high. In Canada, each province/territory establishes its own mechanisms for determining which drugs for rare diseases (DRDs) to provide.
  
  
  OBJECTIVE
  To compare current mechanisms across provinces and territories, and explore their impact on access.
  
  
  METHODS
  A systematic review of relevant published and unpublished documents was performed. Electronic bibliographic databases, the internet, and government websites were scanned using structured search strategies. Information was extracted independently by two researchers, and included aspects such as program type, condition/patient/therapy eligibility criteria, role of health technology assessment (HTA), decision options, ethical assumptions, and stakeholder input. It was validated through member-checking with provincial/territorial policy experts and tabulated to facilitate qualitative analyses. Impact on access was assessed through a cross-province/territory comparison of the coverage status of all non-cancer therapies reviewed by the Common Drug Review for indications affecting <1/2,000 Canadians using the Kappa statistic. Reasons for variations were explored using qualitative techniques.
  
  
  RESULTS
  Each province/territory has formal and informal mechanisms through which such therapies may be accessed. In most cases, formal mechanisms constitute the centralized HTA processes that also apply to common therapies. While several provinces have established dedicated processes/programs, whether they have affected access is not clear. Despite broadly comparable approaches, there is less than perfect agreement on publicly funded DRDs across jurisdictions.
  
  
  CONCLUSIONS
  Individual jurisdictions have developed different approaches to providing access to these therapies. However, as the number increases, a more systematic approach to decision-making may be needed.
authors.affiliation: Professor, Health Technology and Policy Unit, School of Public Health, University of Alberta, Edmonton, AB.
authors.forename: Devidas
authors.initials: D
authors.lastname: Menon
authors.affiliation: Research Assistant, Health Technology and Policy Unit, School of Public Health, University of Alberta, Edmonton, AB.
authors.forename: Derek
authors.initials: D
authors.lastname: Clark
authors.affiliation: Director, Health Technology and Policy Unit, School of Public Health, University of Alberta, Edmonton, AB.
authors.forename: Tania
authors.initials: T
authors.lastname: Stafinski
mesh_terms: D002170:Canada; D005380:Financing, Government / Q000379:methods / Q000592:standards; D006297:Health Services Accessibility / Q000191:economics* / Q000592:standards; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics* / Q000592:standards / Q000706:statistics & numerical data; D035583:Rare Diseases / Q000188:drug therapy / Q000191:economics*; D012051:Reimbursement Mechanisms / Q000592:standards* / Q000639:trends; D013219:State Government; D013222:State Medicine / Q000191:economics*; D011795:Surveys and Questionnaires; D013673:Technology Assessment, Biomedical / Q000191:economics / Q000458:organization & administration / Q000592:standards*