Evaluation of the Clinical Benefit of Cancer Drugs Submitted for Reimbursement Recommendation Decisions in Canada

Abstract

Importance: Cancer drugs approved by the US Food and Drug Administration have come under scrutiny for marginal clinical benefits; however, the clinical benefits of cancer drugs recommended for reimbursement in Canada have not been adequately studied. Objective: To assess the differences in the clinical evidence and benefit of cancer drugs that received a positive vs a negative recommendation for provincial reimbursement in Canada. Design, Setting, and Participants: This cohort study obtained publicly available regulatory documents from the pan-Canadian Oncology Drug Review (pCODR) and corresponding clinical trial documentation. All cancer drugs with a solid tumor indication that were submitted from the inception of the pCODR (July 2011) to February 2020 were evaluated. To be included, submissions had to have a final reimbursement recommendation; submissions that were incomplete, were withdrawn, or had a pending decision were excluded. Exposures: A completed reimbursement recommendation decision from the pCODR. Main Outcomes and Measures: Final reimbursement recommendation (positive vs negative); trial characteristics; and relevant clinical outcomes (ie, overall survival [OS] and progression-free survival [PFS]), including the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS) scores available at the time of pCODR assessment. Results: Between 2011 and 2020, the pCODR issued 104 reimbursement recommendation decisions for cancer drugs with a solid tumor indication. Among these drug submissions, 78 (75.0%) received a positive recommendation, of which 72 (92.3%) were conditional. Drugs that received a positive recommendation compared with those with a negative recommendation were more likely to have phase 3 randomized clinical trial design (92.3% [72 of 78] vs 53.8% [14 of 26]; P