Background In Canada, reimbursement recommendations on drugs for common and rare diseases are overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH) and made through the pan-Canadian Oncology Drug Review (pCODR) and the Common Drug Review (CDR). While the agency specifies information requirements for the review of drug submissions, how that information is used by each process to formulate final reimbursement recommendations, particularly on drugs for rare diseases (DRDs) in which per patient treatment costs are often high, is unclear. The purpose of this study was to determine which factors contribute to recommendation type for DRDs. Methods Information was extracted from CDR and pCODR recommendations on drugs for diseases with a prevalence < 1 in 2000 from January 2012 to April 2018. Data were tabulated and multiple logistic regression was applied to explore the association between recommendation type and the following factors: condition/review process (cancer vs non-cancer), year, prevalence, clinical effectiveness (improvements in surrogate, clinical and patient reported outcomes), safety, quality of evidence (availability of comparative data, consistency between population in trial and indication, and bias), clinical need, treatment cost, and incremental cost-effective ratio (ICER). Two-way interactions were also explored. Results A total of 103 recommendations were included. Eleven were resubmissions, all of which received a positive recommendation. Among new submissions ( n = 92), DRDs that were safe or offered improvements in clinical or patient reported outcomes were more likely to receive positive reimbursement recommendations. No associations between recommendation type and daily treatment cost, cost-effectiveness, or condition (cancer or non-cancer) were found. Conclusions Clinical effectiveness, as opposed to economic considerations or whether the drug is indicated for cancer or non-cancer, determine the type of reimbursement recommendation.

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Objective: Robot-assisted surgery is a minimally invasive approach for repairing the mitral valve. This study aimed to assess its safety and clinical efficacy when compared with conventional sternotomy, partial sternotomy, and right minithoracotomy. Methods: A systematic review of peer-reviewed studies comparing robot-assisted mitral valve repair with conventional sternotomy, partial sternotomy, and right minithoracotomy was conducted following Cochrane Collaboration guidelines. Meta-analyses were performed where possible. Results: The search strategy yielded 15 primary studies, of which 12 compared robot-assisted with conventional sternotomy, 2 compared robot-assisted with partial sternotomy, and 6 compared robot-assisted with right minithoracotomy. The overall quality of evidence was low, and there was a lack of data on long-term outcomes. Individual studies and pooled data demonstrated that robotic procedures were comparable to conventional sternotomy and other minimally invasive approaches with respect to the rates of stroke, renal failure, reoperation for bleeding, and mortality. Robot-assisted mitral valve repair was superior to conventional sternotomy with reduced atrial fibrillation, intensive care unit and hospital stay, pain, time to return to normal activities, and physical functioning at 1 year. However, robot-assisted mitral valve repair had longer cardiopulmonary, aortic cross-clamp, and procedure times compared with all other surgical approaches. Conclusions: Based on current evidence, robot-assisted mitral valve repair is comparable to other approaches for safety and early postoperative outcomes, despite being associated with longer operative times. Ideally, future studies will be randomized controlled trials that compare between robot-assisted surgery, conventional surgery, and other minimally surgery approaches focusing on hard clinical outcomes and patient-reported outcomes.