In Japan, since April 2019 selected pharmaceutical products and medical devices have required health technology assessment (HTA) of their cost-effectiveness (CE) by the Central Social Insurance Medical Council (Chuikyo), from which list-price adjustments are made. This formal HTA process was implemented in response to unsustainable healthcare costs, with Japan having some of the highest drug costs outside the US. This research aims to compare the CE outcomes of the first two pharmaceutical products that have been assessed in Japan (Fluticasone/umeclidinium/vilanterol [Trelegy] and Tisagenlecleucel [Kymriah]) with other CE markets.

Publicly-accessible assessments of Trelegy and Kymriah by six CE-HTA bodies (Chuikyo, NICE, SMC, CADTH, PBAC/MSAC) up to June 2021 were extracted and compared.

Chuikyo’s ICER threshold per QALY is €38k-€76k compared to €23k-€35k for NICE, with SMC, CADTH and PBAC/MSAC not having any explicit ICER thresholds. Trelegy (H1-category) and Kymriah (H3-category) were both recommended by Chuikyo for list price reductions based on their CE (Trelegy -0.5%, Kymriah -4.3%). Trelegy for COPD and pulmonary empheysema was stratified into 12 sub-populations, with only three sub-populations deemed cost-effective with added benefit and ICERs between €2.5k-€14k. NICE has not yet appraised Trelegy, whilst SMC, CADTH and PBAC have all restricted their recommendations to specific sub-populations. Kymriah was stratified into three sub-populations for B-ALL (ICER €17k-€21k), and two sub-populations for DLBCL (ICER €61k-€95k) by the Chuikyo. NICE has recommended both indications for the CDF, whilst SMC has recommended both with PASes. CADTH has given both conditional recommendations requiring price reduction. Finally, MSAC has recommended both using risk-sharing agreements.

The first two products to be subject to CE evaluation in Japan have not been deemed acceptably cost-effective and price reductions have been demanded. These products have also had challenges in being deemed cost-effective in other mandatory cost/QALY markets based on the evidence at submission.