Challenges in Assessing the Cost-effectiveness of Cancer Immunotherapy

Mené dans un contexte américain, cette étude analyse le rapport coût-efficacité du pembrolizumab en traitement de deuxième ligne chez des patients atteints d'un carcinome hépatocellulaire

JAMA Network Open, Volume 4, Numéro 1, Page e2034020-e2034020, 2021, commentaire en libre accès

Résumé en anglais

Chiang et al examine the cost-effectiveness of pembrolizumab for the second-line treatment of patients with advanced hepatocellular carcinoma previously treated with sorafenib, on the basis of the results of the pivotal phase 3 trial KEYNOTE-240. The study finds that pembrolizumab for this indication is not cost-effective at the reference willingness-to-pay threshold in the US of $150 000 per quality-adjusted life-year. This result was consistent even in the optimistic setting, which assumed that patients living beyond 30 months were cured with pembrolizumab. Using a 2-way sensitivity analysis, Chiang et al conclude that to be cost-effective, the median survival benefit vs placebo would need to be longer than 12 months, instead of the benefit of 3.3 months demonstrated in KEYNOTE-240. Alternatively, the list price of pembrolizumab could be reduced by more than half (58%) from $6915 to $2925 per cycle. This work by Chiang et al adds to a growing body of literature demonstrating high cancer drug costs relative to the benefits provided to patients treated on a large scale. There is a concern that such costs, given the widespread use of novel agents, such as immunotherapy, in oncology and an aging US demographic threaten the financial sustainability of our health care system.