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Original Investigation
February 22, 2021

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

Author Affiliations
  • 1Department of Medicine, University of Calgary, Calgary, Alberta, Canada
  • 2School of Population and Public Health, University of British Columbia, Vancouver, British Columbia, Canada
  • 3Leeds, Greenville and Lanark District Health Unit, Brockville, Ontario, Canada
  • 4Division of Cancer Care and Epidemiology, Queen’s University Cancer Research Institute, Kingston, Ontario, Canada
  • 5Department of Oncology, Queen’s University, Kingston, Ontario, Canada
  • 6Department of Public Health Sciences, Queen’s University, Kingston, Ontario, Canada
JAMA Intern Med. 2021;181(4):499-508. doi:10.1001/jamainternmed.2020.8588
Key Points

Question  What are the differences in the clinical evidence and benefit between cancer drugs that received a positive vs those that received a negative reimbursement recommendation from the pan-Canadian Oncology Drug Review (pCODR)?

Findings  In this cohort study of 104 reimbursement recommendation decisions, 78 received a positive recommendation from the pCODR. Of these, 72 were based on phase 3 randomized clinical trials and 39 had proven overall survival benefit with median gains of 3.7 months.

Meaning  Results of this study found that only half of the pCODR-recommended cancer drugs showed improved survival, and the survival gains were usually modest; these findings suggest that, despite the pCODR framework, cancer drugs without meaningful patient benefit continue to be reimbursed in the Canadian market.

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 < .001) and have substantial benefit according to the ESMO-MCBS scores (61.5% [48 of 78] vs 19.2% [5 of 26]; P < .001). The most common primary end points associated with the successful submissions were PFS (53.9%) and OS (32.1%). Overall, 39 of 78 submissions (50.0%) that received a positive recommendation had shown OS benefit, with median (interquartile range) OS gains of 3.7 (2.7-6.5) months.

Conclusions and Relevance  This cohort study found that, although the pCODR takes into account the magnitude of clinical benefit, only half of the cancer drugs that received a positive recommendation had evidence of improved OS and the survival gains were usually modest. These results suggest that, although the pCODR helps filter out some cancer drugs with low quality of evidence and low magnitude of benefit, cancer drugs without meaningful patient benefit continue to enter the Canadian market; these findings are important for making reimbursement policy decisions globally.

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