Dersimelagon

No budget impact model, cost-effectiveness analysis, or health technology assessment of dersimelagon has been published. No United States price has been announced, and the United Kingdom horizon-scanning briefing records the cost as confidential. The only forward cost estimate is the Dutch horizon scan value of €71,000 per patient per year, which the site states is an imputation from the median cost of medicines in Anatomical Therapeutic Chemical class D02BB in the Netherlands rather than a manufacturer price. The relevant payer precedent is the appraisal of afamelanotide, the only approved therapy and the closest comparator: the National Institute for Health and Care Excellence did not recommend it, reporting a company base case of £305,244 per disability-adjusted life year averted and evidence review group estimates from £133,748 to £1,958,162 per quality-adjusted life year gained at a stated implant cost of £13,209 excluding value added tax. Germany accepted an annual cost of €51,875.85 to €69,167.80 after concluding the data did not permit quantification of additional benefit, Norway declined funding, and the Netherlands and Scotland recommended access. A budget impact model for dersimelagon would need to be built against that record, and against a claims-based observation that 7.6% of diagnosed patients in the United States currently receive any treatment for the condition.

No product-specific budget impact model exists for dersimelagon, so the inputs a payer would need are not established. Three are missing entirely: a United States acquisition cost, an eligible population count, and an uptake assumption. The eligible population is uncertain because the number of people with either disorder in the United States is not known from any epidemiological study; the largest identified cohorts are 322 participants enrolled in the Porphyrias Consortium longitudinal study and 696 patients identified in a national claims database over 2016 to 2023. Current treated prevalence is low, with 7.6% of claims-identified patients receiving any treatment and 3.9% receiving afamelanotide at a median treated duration of 5.68 months, so an oral product taken daily and available to adolescents would change both the treated population and the duration of treatment per patient. Offsetting costs are documented but not modeled: mean all-cause costs were $71,714 per patient per year compared with $18,646 in matched comparators, with 46.3% of patients using narcotics.