Evaluating health policy and legal responses : how to reduce barriers and improve access to orphan drugs for rare diseases in Canada

Abstract : Rare diseases are debilitating conditions often leading to severe clinical manifestations for affected patients. Orphan drugs have been developed to treat these rare diseases affecting a small number of individuals. Incentives in the legal framework aimed to recoup the research and development cost of orphan drugs for pharmaceutical companies have been implemented in the United States and the European Union. At the present time, Canada is still lacking a legal and policy framework for orphan drugs. Several problems at the federal and provincial levels remain: lack of research funds for rare diseases, discrepancies on orphan drug policies between provinces, difficulties to access and reimburse these high price drugs. Recommendations and measures are proposed, such as a pan-Canadian (national) scientific committee to establish evidence-based guidelines for patients to access orphan drugs uniformly in all provinces with a disease specific registry, a formal agreement for a centralized Canadian public funding reimbursement procedure, and increasing the role of “guardian” for prices by the Patented Medicines Review Board in Canada. These recommendations and measures will be beneficial for the implementation of a policy framework for orphan drugs in Canada.

How are Canadian universities training and supporting undergraduate medical, physiotherapy and occupational students for global health experiences in international low-resource settings?

Abstract : OBJECTIVES : Canadian medical (MD), physiotherapy (PT) and occupational therapy (OT) students increasingly show an interest in global health experiences (GHEs). As certain moral hazards can occur as a result of student GHEs, a growing consensus exists that universities must have an established selection process, in-depth pre-departure training (PDT), adequate onsite supervision and formal debriefing for their students. This study aimed at identifying current practices in Canadian MD, PT and OT programs and discussing areas for improvement by comparing them with recommendations found in the literature. METHODS : Canadian MD, PT and OT programs (n = 45) were invited to answer an online survey about their current practices for GHE support and training. The survey included 24 close-ended questions and 18 open-ended questions. Descriptive statistics and a thematic analysis were performed on the data and results were discussed with recommendations found in the literature. RESULTS : Twenty-three programs responded to the survey. Student selection processes varied across universities; examples included using academic performance, interviews and motivation letters. All but 1 MD program had mandatory PDT; content and teaching formats varied, as did training duration (2-38 hours). All but 1 MD program had onsite supervision; local clinicians were frequently involved. Debriefing, although not systematic, covered similar content; debriefing was variable in duration (1-8 hours). CONCLUSIONS : Many current practices are encouraging but areas for improvement exist. Integrating global health content into the regular curriculum with advanced study option in global health for students participating in GHEs could help universities standardize support and training.