Presentation (CTI)

OBJECTIVES: Access to new therapies varies internationally - the United States often enables access following a treatment’s inclusion in the National Comprehensive Cancer Network guidelines, Germany after a treatment receives regulatory approval, and Canada following public reimbursement decisions. Pembrolizumab monotherapy, supported by the single-arm KEYNOTE-158 trial (median overall survival 65.4 months, median follow-up 54.5 months), received regulatory approval from Health Canada in 2019, and Canada’s Drug Agency granted a positive reimbursement recommendation in 2023 for patients with microsatellite instability high (MSI-H) ​or deficient mismatch repair (dMMR) endometrial cancer. In a model-based study, pembrolizumab was estimated to be cost-effective in Canada versus chemotherapy. Using this case study, the humanistic burden on patients under various access scenarios was assessed.
METHODS: The published partitioned-survival Markov model based on KEYNOTE-158 provided core inputs, and was modified to assess the population‑level impact of hypothetical access immediately after regulatory approval vs. access at reimbursement approval, for Canadian women with MSI-H or dMMR endometrial cancer. Gradual uptake after access was assumed to be similar across scenarios. Population-level outcomes such as long-term survival, mortality/progression events avoided, and cumulative quality adjusted life years (QALYs) and life years (LYs) were evaluated. QALYs were assessed and discounted annually to the regulatory approval year.
RESULTS: Reimbursement immediately after regulatory approval, rather than a delay of 3 Years, resulted in a population level increase of 2,480 QALYs and 3,477 LYs compared with the base case. Other access scenarios resulted in smaller, yet meaningful, benefits. Earliest access delivered the greatest survival advantage, markedly increasing long-term and progression-free survival. The proportion of patients with access and time of access were the key drivers of survival gains.
CONCLUSIONS: Our model-based analysis demonstrates that timely access to therapies could improve patient outcomes. Future reimbursement decisions need to strike a balance between waiting for greater data certainty and minimizing patient burden.