Data from: Health Canada’s use of accelerated review pathways and therapeutic innovation, 1995-2016: a cohort study

Objectives: This study examines the use of accelerated approval pathways by Health Canada over the period 1995 to 2016 inclusive and the relationship between the use of these pathways and the therapeutic gain offered by new products. Design: Cohort study. Data sources: Therapeutic Products Directorate, Biologics and Genetic Therapies Directorate, Notice of Compliance database, Notice of Compliance with conditions web site, Patented Medicine Prices Review Board, La revue Prescrire, World Health Organization (WHO) Anatomical Therapeutic Chemical (ATC) classification system. Interventions: None. Primary and secondary outcomes: Percent of new drugs evaluated by Health Canada that went through an accelerated pathway between 1995 and 2016 inclusive. Kappa values comparing the review status to assessments of therapeutic value for individual drugs. Results: 438 (70.3%) drugs went through the standard pathway, 185 (29.7%) an accelerated pathway. Therapeutic evaluations were available for 509 drugs. Health Canada used an accelerated approval pathway for 159 of the 509 drugs whereas only 55 were judged to be therapeutically innovative. The Kappa value for the entire period for all 509 drugs was 0.276 (95% CI 0.194, 0.359) or fair. Conclusion: Health Canada’s use of accelerated approvals was stable over the entire time period. Its ability to predict which drugs will offer a major therapeutic gain is relatively poor. The findings in this study should provoke a discussion about whether Health Canada should continue to use these pathways and if so how their use can be improved.研究目的：本研究旨在考察1995年至2016年（含首尾年份）加拿大卫生部（Health Canada）加速审批路径的使用情况，以及该路径使用与新药所提供的治疗获益之间的关联。 研究设计：队列研究（Cohort study）。 数据来源：治疗产品局（Therapeutic Products Directorate）、生物制品与基因治疗局（Biologics and Genetic Therapies Directorate）、合规通知书数据库（Notice of Compliance database）、附条件合规通知书官方网站（Notice of Compliance with conditions web site）、专利药品价格审查委员会（Patented Medicine Prices Review Board）、《Prescrire》评论（La revue Prescrire）、世界卫生组织（World Health Organization, WHO）解剖学治疗学及化学（Anatomical Therapeutic Chemical, ATC）分类系统。 干预措施：无。 主要与次要结局指标：1995至2016年期间，经加拿大卫生部评估的新药中采用加速审批路径的占比；用于对比审评状态与单个药物治疗价值评估一致性的Kappa值。 结果：共438种（70.3%）药物采用标准审批路径，185种（29.7%）采用加速审批路径。共获取509种药物的治疗评估数据，加拿大卫生部对其中159种药物启用了加速审批路径，而仅55种药物被判定为具有治疗创新性。针对全部509种药物的整个研究周期的Kappa值为0.276（95%置信区间：0.194, 0.359），一致性水平为一般（fair）。 结论：加拿大卫生部的加速审批路径使用情况在整个研究周期内保持稳定。其预测何种药物可带来重大治疗获益的能力相对薄弱。本研究结果应引发关于加拿大卫生部是否应继续采用此类审批路径，以及若继续采用该如何优化其使用的讨论。