Dersimelagon (MT-7117)

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.