OBJECTIVES: Single-arm trials (SAT) have been increasingly used to support oncology reimbursement submissions worldwide. In a previous study, the authors assessed the use of SAT-based oncology technical appraisals by the National Institute for Health and Care Excellence (NICE) from 2017 to 2022, approach to generate comparative evidence, and recommendations from committee reviews. The objective of this study was to assess the use of SATs in Canadian Agency for Drugs and Technologies in Health (CADTH) reviews of oncology drugs.

METHODS: The same method employed by the NICE study was used; submissions only using SATs as clinical evidence were identified from CADTH reimbursement recommendations published in the past five years. Final recommendations and clinical/economic guidance reports were reviewed to extract information on interventions, indications, trial design, external comparator data source, and committee commentaries.

RESULTS: Of 132 oncology CADTH reviews identified between January 2018 and August 2022, 29 (22%) used SATs as primary clinical evidence. Comparative evidence was generated from various sources, including 13 comparator trials, 12 real-world evidence studies, and 3 using both. More than one source or approach was applied in some submissions: 13 (45%) naïve comparisons and 20 (69%) adjusted indirect treatment comparison (ITC), including 14 (48%) matching-adjusted indirect comparisons, 7 (24%) propensity score analyses, and 4 (14%) others. In spite of the uncertainty around treatment benefits generated from SATs, the committee granted recommendations to 62% (18 of 29) of SAT-based reviews compared with 77% by NICE. Similar to NICE, the decisive factor was the convincingness of net clinical benefit and plausible cost-effectiveness, which are determined by the relative treatment effect demonstrated through ITC. Patient-based values and adoption feasibility were also considered.

CONCLUSIONS: SAT-based submissions to CADTH have a slightly lower acceptance rate compared with NICE. Generally, clinical evidence based on rational ITC approaches can lead to positive reimbursement decisions.