Given financial constraints, drug manufacturers are required to provide pharmacoeconomic evaluations to demonstrate the value for money of their drug compared to current treatment when requesting reimbursement by publicly funded health care systems. This thesis is a retrospective examination of pharmacoeconomic evaluations submitted for review to the Common Drug Review process. Its purpose was to determine the pattern of adherence to guidelines, trends in methodological quality, and transparency, changes in the adoption and practice of sensitivity analysis and probabilistic methods, use of indirect treatment comparison, and identify methodological factors−determinants of recommendations. Using an instrument that was developed and tested, information from 201 pharmacoeconomic evaluations was collected and analysed. Pharmacoeconomic evaluations may have improved over time in terms of adherence, methodological quality, transparency, use of probabilistic sensitivity analysis and indirect treatment comparison. However, such improvements have been minimal and further efforts are needed to better improve pharmacoeconomic evaluations in the future.

Introduction Identifying key differences between the manufacturer's submitted analysis and the Canada's Common Drug Review (CDR) economic reanalysis is a crucial step toward creating more appropriate models by manufacturers. We compared manufacturers’ submitted analysis to CDR reanalysis in order to identify any variations in incremental costs utility ratio (ICUR) and 3-year Budget impact analysis (BIA). We assessed the willingness to pay (WTP) threshold and CDR critiques on manufacturers’ clinical and economic reports. Method A pair of reviewers extracted data regarding therapeutic category, percent price reduction requested by CDR, WTP, and the critiques on the manufacturers' clinical and economic reports in publicly available CDR reports from 2018 to 2022. We used Wilcoxon rank test to assess the difference between mean incremental QALY, ICUR, and BIA in manufacturers and CDR reanalysis reports and chi-square tests and logistic regression to assess the relationship between the variables and the final CDR recommendation. Results Of 178 reports assessed, 31 received "do not reimburse" recommendation and 147 received "reimburse with criteria or conditions". The median ICUR in manufacturer's analysis was $138,658/QALY and significantly lower than ICUR reanalyses by CDR of $380,251/QALY. The ICUR in manufacturers' submitted reports was 2.5-fold lower than in the CDR reanalysis ($138,658/QALY versus $380,251/QALY). The CDR reanalysis median for 3-year BIA was $4,575,102 which was 27% higher than the manufacturers submitted 3-year BIA (p value<0.001). The most frequent CDR critiques were clinical effectiveness and the uncertainty of evidence in cost-effectiveness analysis and miscalculations in the population of patients and the percentage of market share in BIA. From 2018 to 2020, $100,000 was the most frequent WTP threshold followed by a $50,000 threshold, but during 2021 and 2022, the CDR only used $50,000 as a WTP threshold. Conclusion Manufacturers may tend to underestimate the costs or overestimate the effect of their medications. / Thesis / Master of Science (MS)