Summary: available economic evidence and its limits

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.

United Kingdom horizon scanning of dersimelagon

“Dersimelagon for previously untreated erythropoietic protoporphyria and X-linked protoporphyria Company/Developer Mitsubishi Tanabe Pharma Europe Ltd” (opens the source at this quote in a new tab)
“Estimated Cost The cost of dersimelagon was confidential at the time of producing this briefing” (opens the source at this quote in a new tab)
“There are currently no pharmaceutical treatment options recommended by the National Institute for Health and Care Excellence (NICE) for the treatment of EPP or XLP.” (opens the source at this quote in a new tab)

National Institute for Health and Care Excellence appraisal of the approved comparator

“Afamelanotide is not recommended, within its marketing authorisation, for preventing phototoxicity in adults with erythropoietic protoporphyria (EPP).” (opens the source at this quote in a new tab)
“The cost-effectiveness analyses for afamelanotide are very challenging. Based on the available evidence provided by the company, the cost-effectiveness estimates are all substantially higher than the range normally considered acceptable for highly specialised technologies.” (opens the source at this quote in a new tab)

Acquisition cost and dosing frequency assumed in that appraisal

“Afamelanotide has not been launched in England, but the company has stated that the cost of an implant will be £13,209 (excluding VAT).” (opens the source at this quote in a new tab)
“The marketing authorisation recommends 3 implants per year, depending on the length of protection needed. The maximum number of implants recommended in the marketing authorisation is 4 per year.” (opens the source at this quote in a new tab)

Incremental cost-effectiveness estimates for the approved comparator

“the company's base case: £305,244 per DALY averted (£305,151 per QALY gained when converted to a QALY-based ICER using the ERG's simple QALY adaptation)” (opens the source at this quote in a new tab)
“the ERG's exploratory base case (3 implants per year, gradual onset and 2-month attenuation of the relative treatment effect): £1,759,833 per QALY gained” (opens the source at this quote in a new tab)
“the ERG's exploratory base case assuming a maximum of 4 implants per year: £1,958,162 per QALY gained.” (opens the source at this quote in a new tab)
“The ICERs associated with these analyses ranged from £133,748 to £253,676 per QALY gained. The committee considered that £133,748 per QALY gained was a potentially plausible ICER although this was the most optimistic scenario it had been presented with.” (opens the source at this quote in a new tab)
“Over the lifetime of someone with EPP, the undiscounted QALYs gained with afamelanotide in the ERG's original exploratory base case were 0.56, and did not exceed 0.8 in the ERG's sensitivity analyses.” (opens the source at this quote in a new tab)

German benefit assessment and annual cost of the approved comparator

“Hint for a non-quantifiable additional benefit, since the scientific data does not allow a quantification.” (opens the source at this quote in a new tab)
“Adult patients with erythropoietic protoporphyria for the prevention of phototoxicity approx. 540 to 1,090 patients” (opens the source at this quote in a new tab)

Norwegian assessment of the approved comparator

“The cost of Scenesse, based on list price (max PSP ex VAT), three implants and administration costs per year is 415 000 NOK per patient per year.” (opens the source at this quote in a new tab)
“It has not been possible to establish an incremental cost-effectiveness ratio (ICER), since the health-related quality of life gain of afamelanotide is given on a scale based on a non validated disease specific questionnaire which was developed for the purpose of the afamelanotide studies.” (opens the source at this quote in a new tab)
“However, by using an approximation of utility based on mapping from DLQI to EQ-5D by NICE, the NoMA has estimated the absolute shortfall of EPP to be approximately 11 QALYs.” (opens the source at this quote in a new tab)

National reimbursement decisions for the approved comparator

“In 2016, Dutch authorities issued a positive recommendation for reimbursement for afamelanotide. In contrast, until now, England has maintained their negative assessment” (opens the source at this quote in a new tab)
“In 2018, Norway based their negative recommendation for funding afamelanotide on the preliminary negative recommendation in England” (opens the source at this quote in a new tab)
“In contrast, in Scotland the existing evidence was assessed as sufficient to recommend access within the ultra-orphan pathway in 2021, allowing for the collection of additional real-world data for the reassessment of the decision after three years” (opens the source at this quote in a new tab)

Manufacturer-reported annual cost and pricing policy for the approved comparator

“The annual costs of therapy with SCENESSE® range between €56,404 and €84,606 per EPP patient per annum.” (opens the source at this quote in a new tab)
“CLINUVEL has adopted a uniform global pricing policy and provides its clear rationale, acknowledging the facts that patients are migrating across borders to seek treatment, expert physicians are associated through porphyria networks, and hospitals collaborate internationally to purchase pharmaceutical products for orphan diseases.” (opens the source at this quote in a new tab)

United States coverage arrangements for the approved comparator

“In the U.S., SCENESSE® is reimbursed by over 100 private insurers through prior authorization mechanisms, with patients receiving authorization for treatment on an annual basis. Clinuvel has reached agreements on pricing with U.S. government payors for SCENESSE® for Medicare and Medicaid, as well as patients receiving treatment through the Veterans' Affairs.” (opens the source at this quote in a new tab)
“Beyond statutory requirements, Clinuvel does not provide sales rebates or discount incentives on SCENESSE® sales and aims to maintain uniform pricing within each jurisdiction.” (opens the source at this quote in a new tab)

Published cost-effectiveness analysis of the approved comparator

“The base case lifetime DALYs averted via the use of afamelanotide was 1.87 and the Incremental Cost-Effectiveness Ratio (ICER) was £373,000 per DALY averted. When hereditary angioedema was used as a proximate condition the ICER was £401,000 per QALY gained.” (opens the source at this quote in a new tab)
“Differing proxy sources for DALY weights generated estimates of DALYs averted ranging from 0.72 to 2.50. Alternative sources for utility weights generated results ranging from £208,000 to £1.1M per QALY gained. The model showed sensitivity to the number and the cost of each dose.” (opens the source at this quote in a new tab)

Dersimelagon (MT-7117)

Modeling overview