国立保健医療科学院 保健医療経済評価研究センター
Center for Outcomes Research and Economic
Evaluation for Health (C2H), National Institute of
Public Health (NIPH) | URL:http://c2h.niph.go.jp

2023 年 4 月 4 日[C2H2105] Summary of cost-effectiveness evaluationof amikacin sulfate (ARIKAYCE®)
1. Indications
Nontuberculous mycobacterial (NTM) lung infections caused by Mycobacterium
avium complex (MAC)
2. Price of the drug
Amikacin sulfate (ARIKAYCE®) has been reimbursed from May 2021 at JPY
42408.40 (as of January 2022). The price is calculated using a cost calculation
method with a usefulness premium (II) of 10%. This product s designated as aH1 cost-effectiveness evaluation item.
3. Scope of cost–effectiveness evaluation
This product is indicated for the treatment of non-tuberculous mycobacterial(NTM) lung infections caused by Mycobacterium avium complex (MAC) as part ofa combination antibacterial drug regimen in patients who do not achieve negative
sputum cultures after a minimum of 6 consecutive months of multidrug
background regimen therapy*. The scope of evaluation agreed upon at the firstsession of the Expert Committee of Cost-Effectiveness Evaluation (ECCEE) isdescribed below.
Patients with MAC lung disease after a minimum of 6Populationconsecutive months of a multidrug background regimen
therapyComparator

Multidrug regimen (Selected technology ：ARIKAYCE® ＋Multidrug regimen)*3-drug regimen including rifamycin, ethambutol, and clarithromycin

4. Evaluation of additional benefits
In this systematic review, the randomized INS-212 trial was detected. The INS212 trial is a Phase 3 trial involving patients with refractory MAC lungdisease(n=336). Patients were randomized to receive either ARIKAYCE plus abackground regimen (n=223) or a background regimen alone (n=112). The
primary endpoint of sputum culture conversion was significantly greater for
ARIKAYCE plus a background regimen than for a background regimen alone(29.0% vs. 8.9%; adjusted odds ratio, 4.22; 95% CI, 2.08–8.57; P < 0.001).
In addition, the number of participants who achieved sustained culture
conversion at the end of treatment was greater in the ARIKAYCE plus backgroundregimen arm.
Consequently, the academic group concluded that ARIKAYCE has additional
benefits for the comparator.
5. Results of the cost-effectiveness analysis
The manufacturer estimated the cost effectiveness using the simulation model.
For QOL values, the manufacturer evaluated the presented health scenario (MACnegative/MAC-positive) for the general population using the TTO (time-trade off)
method. The Guideline for Preparing Cost-Effectiveness Evaluation to the Central
Social Insurance Medical Council states that “it is difficult to directly collect QOL
scores from subjects, it is acceptable for general people to evaluate the presented
health scenario by direct methods." However, because QOL values weremeasured using the EQ-5D-3L in the INS-212 trial, the academic group concluded
that it was not difficult to measure QOL values for patients with MAC lung disease.
In addition, the Guideline for preparing Cost-Effectiveness Evaluation state that
"When the QOL score is assessed by PBM, the subjects’ own QOL responses
should be used.” Thus, the academic group applied the results of the INS-212
trial in which QOL values were measured using the EQ-5D-3L.
The academic group modified the quality of life (QOL) score and recurrence
rate in patients with MAC lung disease. The ECCEE accepted the following criteria:
Population

Comparator

ICER (JPY/QALY)

Multidrug regimen

11,135,395

Patients with MAC lung disease
after a minimum of 6 consecutive
months of multidrug backgroundregimen therapy