MO19-5 The association between prices and clinical benefit of approved drugs for solid tumors in Japan

Abstract

In other countries, prices of approved cancer drugs have not reflected their clinical benefit (Vokinger KN. Lancet Oncol, 2020). We aimed to examine the association between the prices and clinical benefit of approved cancer drugs in Japan. We retrospectively evaluated drugs approved for solid tumors between Jan 2010 and Dec 2019 in Japan. Hormonal agents and an implanted drug were excluded. We calculated the monthly treatment cost for each cancer drug as per the prescription and dose information on the Pharmaceuticals and Medical Devices Agency label, with the first approved prices and the commonly used standard patient value (bodyweight 60 kg and body surface area 1.62 m2). Information about progression-free survival (PFS), overall survival (OS), and response rate (RR) was recorded from each pivotal study. Clinical benefit was defined as absolute or percentage difference (δ) and ratio of PFS, OS, or RR and hazard ratio (HR) of PFS or OS between the treatment and control groups in each pivotal study. Moreover, we evaluated pivotal studies using ESMO-MCBS. We identified 39 approved drugs for solid tumors (cytotoxic agent/molecularly targeted agent/immune checkpoint inhibitor, N = 5/28/6). We observed no correlation between the monthly treatment cost and the clinical benefit; δPFS, N = 31, R2 < 0.0001; δOS, N = 20, R2 = 0.0959; δRR, N = 29, R2 = 0.0461; PFS ratio, N = 30, R2 = 0.0290; OS ratio, N = 20, R2 = 0.0666; RR ratio, N = 27, R2 = 0.0545; PFS HR, N = 33, R2 = 0.0009; OS HR, N = 26, R2 = 0.1792. The ESMO-MCBS scores showed no strong association with the monthly cost; score = 1/2/3/4/5, N = 4/9/8/15/3; average monthly costs, 484,029/926,342/508,609/807,921/816,068 yen; p = 0.4066. Our study suggests that in Japan, the first approved prices of drugs for solid tumors did not reflect clinical benefit. Standard measures to evaluate the clinical benefit should be considered to determine the prices of cancer drugs.