Economic information and modeling report Budget impact model

Budget impact model discussion

2 evidence topics · 5 sources

Summary: implications of the comparator model for a zilurgisertib budget impact estimate

No budget impact model of zilurgisertib exists, so the Canadian palovarotene analysis is the only modeled precedent for FOP. In that analysis, palovarotene acquisition was the only cost modeled, because no standard-of-care costs were included, and the 3-year budget impact ranged from $143,363 with a 99% price reduction to $25,600,609 with a prevalence of 1 in 1 million, against a CADTH base case of $14,336,341 (Canadian dollars) for an eligible population of about 19 patients. CADTH identified the number of eligible patients, uptake, flare-up dosing, and the omitted standard-of-care costs as the principal uncertainties. Two of these differ for zilurgisertib: its labeled dosage is a fixed 100 mg once daily, supplied as 25 mg tablets in bottles of 120, with no weight-based or flare-up regimen, and the U.S. population stated by the manufacturer is approximately 300 people, of whom only those aged 12 years and older fall within the labeled indication. A plan-level estimate for zilurgisertib requires the launch price, which Mirum plans to announce at the launch expected in October 2026, and the plan's own count of members with FOP.

Palovarotene budget impact analysis: sources of uncertainty identified by CADTH

“The number of patients eligible for palovarotene is uncertain, owing to uncertainty in the prevalence of FOP in Canada, the proportion of patients accurately diagnosed with FOP, and the proportion of patients who would have public coverage for palovarotene.” (opens the source at this quote in a new tab)
“The uptake of palovarotene may be higher than expected by the sponsor.” (opens the source at this quote in a new tab)
“Palovarotene drug acquisition costs are uncertain and may be underestimated.” (opens the source at this quote in a new tab)
“The cost of SoC was not captured in the estimated budget impact and may vary between those receiving palovarotene plus SoC versus SoC alone.” (opens the source at this quote in a new tab)
“The estimated budget impact is highly sensitive to the number of patients eligible for palovarotene and assumptions about its uptake.” (opens the source at this quote in a new tab)