Guiding the willingness to pay for repurposed tiratricol in the treatment of Allan-Herndon-Dudley syndrome using a cost-based-plus pricing framework

Background: Tiratricol, initially marketed as Téatrois®, has been repurposed as an orphan drug for Allan-Herndon-Dudley syndrome, a rare genetic disorder affecting thyroid hormone transport. Despite public contributions in 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. This raises concerns regarding affordability, pricing justification, and the willingness to pay for repurposed medicines where public funding has supported development. This study evaluates a cost-based-plus pricing approach for repurposed tiratricol, applying a novel pricing framework across distinct scenarios. By transparently outlining key cost components—including research and development, manufacturing costs, and cost-of-capital—alongside patient population estimates, it proposes more socially acceptable pricing that reflects genuine investment requirements and a reasonable profit margin. Results Under the maximum cost scenario, assuming fewer patients than projected by the company itself, a price per patient per year of approximately €9,600 is derived. Minimum cost scenarios and those projecting a higher number of patients yield prices ranging from €2,600 to €6,100. Conclusion: Given the substantial public role in tiratricol’s repurposing, this study argues in favour of the lower end of the price range, while acknowledging the need for sustainable access through formal registration, justifying a price higher than that of the original product. This research highlights the broader ethical and economic implications of orphan drug pricing, advocating for evidence-based policies that promote transparency in cost structures. Such approaches can ensure a predictable and sustainable business model for repurposed medicines, thereby justifying willingness to pay and informing responsible reimbursement decisions.