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

November 30, 2021

[C2H2001] Summary of cost-effectiveness evaluationof posaconazole (Noxafil)
1. Indications
Prevention of invasive fungal infection (IFI)
2. Price of the drug
Posaconazole is being reimbursed since April 2020 at JPY 3,109.1 (as of November2021). The price is calculated using a cost calculation method. This product isdesignated as an H1 cost-effectiveness evaluation item.
3. Scope of cost-effectiveness evaluation
This product has been observed to help in the prevention of IFI among (a) thepatients who have undergone allogeneic hematopoietic stem-cell transplantation and(b) those who have or are anticipated to have neutropenia. Hereafter, the scope ofevaluation agreed upon at the first session of the Expert Committee of CostEffectiveness Evaluation (ECCEE) is described. The target population comprises
population (a) and (b) described above. The cheapest fluconazole (capsule) in
population (a) and itraconazole (oral solution) in population (b) are considered as thecomparators.

(a) Patients who have undergone allogeneic hematopoietic stemPopulation

cell transplantation(b) Patients who have or are anticipated to have neutropenia

Comparators(a) fluconazole (capsule)(b) itraconazole (oral solution)

4. Evaluation of additional benefitsAs a result of the systematic review, two randomized control trials (RCTs)
corresponding to the research questions were detected: C/I98-316 trial for
population (a) and P01899 trial for population (b). These are non-inferiority trials
through which the superiority of posaconazole is tested if non-inferiority is proven.
In the randomized C/I98-316 trial, patients were allocated to the posaconazole(N=301) and fluconazole (N=299) groups. Similarly, in the randomized P01899 trial,
patients were allocated to the posaconazole (N=304) and fluconazole/itraconazole(N=298) groups. The primary endpoint is to ensure the prevention of IFI. In
population (A), the incidence of IFI was 5.3% in the posaconazole group and 9.0%
in the fluconazole group. The odds ratio was 0.56 [95%CI 0.30-1.07, P=0.07]. Inpopulation (B), the incidence of IFI was 2.3% in the posaconazole group and 8.4%
in the fluconazole group. The difference between both the incidences was -6.09%[95%CI -9.68—2.50%, P=0.0009].
Manufacturers insisted

that in population (A), in the secondary endpoint inwhich the observation period is changed, posaconazole significantly reduced theincidence of IFI, although the superiority of posaconazole was not shown, considering
the results of the primary endpoint.
The academic group concluded as follows.
･ (a) Population A: According to the results of the C/I98-316 trial, the superiority ofposaconazole for all fungal infections was not supported. However, the trial shows asignificant improvement in the prevention of aspergillosis infection. Considering theseresults, the group concluded that posaconazole has an additional benefit that helps
in the prevention of the aspergillosis infection.
･ (b) Population B: Posaconazole has an additional benefit because the P01899 trial
shows that it is superior to the comparators….
5. Results of the cost-effectiveness analysis
The manufacturer performed a cost-effectiveness analysis. The academic group used
the model adopted by the manufacturer, but some parameters such as the probabilityof IFI and demographic information, were changed. The ECCEE accepted the following.
Population
Patients who have undergone allogeneic
hematopoietic stem-cell transplantation
Patients who have or are anticipated tohave neutropenia

ICER (JPY/QALY)
9,813,704
1,271,646