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.

European cost-based-plus pricing model: objective

“This study aims to evaluate the potential of a cost-based-plus pricing model for repurposed tiratricol and, in doing so, contribute to the development of socially responsible pricing strategies for rare disease therapies that balance affordability with sufficient incentives for sustainable drug repurposing.” (opens the source at this quote in a new tab)

European cost-based-plus pricing model: company price projection

“Despite public contributions to its repurposing, the current marketing authorization holder, Egetis Therapeutics, has projected a price of €63.500–€95.000 per-patient-per year, significantly exceeding historical costs and straining already limited healthcare budgets.” (opens the source at this quote in a new tab)

European cost-based-plus pricing model: cost inputs

“Manufacturing costs are benchmarked at €0,65 per tablet, based on tiratricol’s reported cost under Cenexi.” (opens the source at this quote in a new tab)
“Since a real discount rate is used, inflation is not separately included in the calculations.” (opens the source at this quote in a new tab)
“A profit margin of 8% is applied, aligning with the AIM model (2021) and roughly with the inflation-adjusted average return of the S&P 500 index, adjusted for inflation” (opens the source at this quote in a new tab)

European cost-based-plus pricing model: results

“Under the maximum cost scenario, assuming fewer patients than projected by the company itself, a price of €27.600 per-patient-per-year is derived.” (opens the source at this quote in a new tab)
“Minimum cost scenarios and those projecting a higher number of patients yield prices ranging from €5.300 to €16.500.” (opens the source at this quote in a new tab)

European cost-based-plus pricing model: sensitivity analysis

“The tornado charts show that across all scenarios, the number of patients and the WACC are the most sensitive parameters driving price deviations.” (opens the source at this quote in a new tab)
“Cost-of-capital exerts by far the largest influence across all scenarios, illustrating the substantial financial impact of capital charges on development investments.” (opens the source at this quote in a new tab)

German eligible population and annual treatment costs

Characteristic	Value
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”

German cost calculation basis and uncertainty in patient numbers

“The starting dose of 175μg/day for children weighing less than 10 kg is used as the lower limit.” (opens the source at this quote in a new tab)
“The product information specifies a maximum dosage of 50 μg/day/kg body weight (BW) for subjects weighing more than 60 kg.” (opens the source at this quote in a new tab)
“Overall, the specified number of patients in the SHI target population is subject to uncertainty.” (opens the source at this quote in a new tab)
“Uncertainties exist in the calculation of the prevalence of MCT8 deficiency (AHDS) due to the unclear methodological procedure for deriving the lower limit.” (opens the source at this quote in a new tab)

German assessment of patient numbers and treatment costs

“In accordance with §35a (1) Sentence 11 Social Code Book (SGB) V, the added medical benefit of orphan drugs is deemed as proven by the fact that they have been approved.” (opens the source at this quote in a new tab)
“On behalf of the Federal Joint Committee (G-BA), IQWiG therefore solely assesses the information on patient numbers and costs in the pharmaceutical company's dossier.” (opens the source at this quote in a new tab)

German health technology assessment

“There is a hint for a non-quantifiable additional benefit of tiratricol for the treatment of peripheral thyrotoxicosis in subjects with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley syndrome), from birth, since the scientific data does not allow quantification.” (opens the source at this quote in a new tab)
“Quantification of the extent of the additional benefit on the basis of these data is not possible due to the lack of comparison.” (opens the source at this quote in a new tab)
“For the TRIAC I study presented, there is a high risk of bias at study level due to the single-arm, open-label study design, thus not allowing a comparative assessment.” (opens the source at this quote in a new tab)

French reimbursement assessment

“The clinical benefit of EMCITATE (tiratricol) dispersible tablets is insufficient in the MA indication to justify public funding.” (opens the source at this quote in a new tab)

French Transparency Committee rationale

“The efficacy/adverse effects ratio has not been adequately established given the efficacy demonstrated for biological endpoints only, in the absence of evidence of an improvement for clinically relevant endpoints, during a non-comparative study.” (opens the source at this quote in a new tab)
“EMCITATE (tiratricol) has no role in the care pathway.” (opens the source at this quote in a new tab)
“EMCITATE (tiratricol) is unlikely to have an additional impact on public health.” (opens the source at this quote in a new tab)

Emcitate (tiratricol)

Modeling overview