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.

Working with physical therapists to develop and evaluate an evidence-based online module for Developmental Coordination Disorder (DCD): bridging the knowledge-to-practice gap

Aims: Developmental Coordination Disorder (DCD) is a chronic condition with potential negative health consequences. Clinicians working with children with DCD need access to tailored, synthesized, evidence-based DCD information; however a knowledge-to-practice gap exists. The aim of this study was to develop and evaluate an evidence-based online DCD module tailored to physical therapists’ (PTs) identified needs. Methods: Guided by the Knowledge to Action framework, we interviewed PTs working with children with DCD (n=9) to identify their information needs. Their recommendations, along with synthesized DCD research evidence, informed module development. PTs (n=50) responded to scaled items and open-ended questions to evaluate module usefulness. Results: The module incorporated important PT DCD content areas including: 1) Identification; 2) Planning Interventions and Goals; 3) Evidence-Based Practice; 4) Management; and, 5) Resources. Case scenarios, clinical applications, interactive media, links to resources, and interactive learning opportunities were also embedded. PTs perceived the module to be comprehensive and useful and provided feedback to improve module navigation. Conclusions: Involving end-users throughout the development and evaluation of an online PT DCD module contributed to its relevance, applicability, and utility. The ongoing clinical use of this module may have the potential to improve the quality of PT DCD services.