Evicenter
P&T meetings

Emcitate

MCT8 deficiency

Also known as tiratricol
78 sources

Section 5 of 6

Economic information and modeling report

12 evidence topics · 6 sources

Modeling overview

Summary

No identified source reports a budget impact model for tiratricol. One published model estimates a cost-based-plus annual price in the EU context. Its maximum-cost scenario yields €27,600 per person per year, and scenarios with lower costs or more treated individuals yield €5,300 to €16,500. The authors report that the number of patients and the cost of capital are the parameters with the largest influence on price, and they cite a company projection of €63,500 to €95,000 per person per year.

In Germany, the G-BA resolution estimates approximately 40 to 110 patients in the statutory health insurance target population and annual treatment costs of €44,216.13 to €972,754.87 per patient after statutory rebates. The lower limit uses the 175 μg/day starting dose, and the upper limit uses the maximum weight-based dose at an average body weight of 77.7 kg. The G-BA identifies a hint of a non-quantifiable additional benefit, with a high risk of bias from the single-arm, open-label design of Triac Trial I. IQWiG's assessment is limited to patient numbers and costs in the manufacturer's dossier. The French Transparency Committee rated the clinical benefit insufficient to justify public funding because efficacy was demonstrated for biological endpoints only, in a non-comparative study.

German eligible population and annual treatment costs

CharacteristicValue
Patients eligible for treatment in the statutory health insurance target population“Approx. 40 – 110 patients”
Annual treatment costs per patient, tiratricol“€ 44,216.13 - € 972,754.87”
Additionally required statutory health insurance services“Different from patient to patient”

Budget impact model

Approach and framework

No evidence found.

Perspective and time frame

No evidence found.

Epidemiology and eligible population inputs

No evidence found.

Cost assumptions

No evidence found.

Model outcomes

No evidence found.

Results

Base case

No evidence found.

Scenario analyses

No evidence found.

Budget impact model discussion

No evidence found.