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

March 31st, 2023

[C2H2110] Summary of cost-effectiveness evaluationof Teduglutide (Revestive)
1. Indications
Short Bowel Syndrome (SBS)
2. Price of the drug
Teduglutide has been reimbursed since November 2021 at JPY 79,302 (as ofMarch 2023). The price was calculated using a method that included ausefulness premium (II) of 5%. This product was designated as the H2 item forcost-effectiveness evaluation.
3. Scope of the cost-effectiveness evaluation
This product is indicated for the treatment of SBS. The scope of evaluationagreed upon during the first session of the Expert Committee of CostEffectiveness Evaluation (ECCEE) is described below.
Population
Comparator(a) Adult SBS patients(b) Pediatric SBS patients
Standard care
(Selected technology: Teduglutide + Standard care)

4. Evaluation of additional benefits
In the systematic review, the manufacturer detected a clinical trial for SBS in
populations (a) and (b) and found that teduglutide had an additional benefit overcomparator technologies, significantly improving the response rate. In response,
the academic group added other relevant publications. Additional benefits inpopulation (a) were primarily evaluated in the CL0600-020 trial (STEPS trial), a
randomized controlled trial (RCT) involving adult patients with SBS.

The results of the STEPS trial demonstrated that the teduglutide arm wassignificantly superior to the standard care arm in terms of the proportion ofpatients who experienced a reduction of at least one day of PS per week,
improving the response rate and PS change.
Moreover, additional benefits in population (b) were evaluated primarily in
the TED-C14-006 trial (006 trial), an RCT involving pediatric patients with SBS.
The 006 trial was an open-label trial, which may have led to an imbalance inpatient backgrounds. However, teduglutide’s benefit was evaluated in a post-hoc
analysis that reported a statistically significant increase in the number of
responders in the teduglutide arm than in the standard care arm. Moreover,there was a trend toward the superiority of the teduglutide arm in terms of thenumber of days of PS performed per week and decreasing the amount of PS
needed.
Based on these results, the post-hoc analysis determined additional
benefits for teduglutide in both populations (a) and (b).
5. Results of the cost-effectiveness analysis
The manufacturer conducted a cost-effectiveness analysis using a Markov modelcomprising nine health states for population (a) and five health states for
population (b), based on the number of days per week of PS administered.
In response, the academic group added a long-term setting based on thelong-term observational results of the CL0600-004 trial (004 trial), in whichsome PS withdrawals continued teduglutide treatment. This was because of thethe assumption that patients who withdrew from PS would maintain theirdiscontinued teduglutide treatment is clinically inappropriate.
Additionally, the manufacturer assumed that the long-term effects on thereduction in PS implementation days would continue for over two years forchildren and five years for adults. However, the rationale and validity of thissetting were unclear. Therefore, the academic group conducted an analysis
assuming that no state transitions occurred after the observation period in theSTEPS-2 or 006 trials for populations (a) and (b), respectively.
C2H deemed further reanalysis necessary because we should consider thelarge uncertainty can not be avoided in rare pediatric diseases. Therefore, C2H
continued to review the analytical model, and inquire about the manufacturer.
As a result, populations (a) and (b) were analyzed in a setting where acertain percentage of all SBS patients in the model would continue teduglutide
treatment in the long term, based on the results of the long-term observational

study of the 004 trial and unpublished information. However, regarding thelong-term effects on the reduction, manufactures’ suggestion was accepted.
C2H submitted the resulting ICERs mentioned below for final analysis.

Population

ICER (JPY/QALY)

Adult SBS patients

88,522,201

Pediatric SBS patients

9,533,412