Budget Impact of Metyrapone for the Treatment of Cushing’s Syndrome in France
Author(s)
Mariam Sbeity, Market Access1, Henry Dummett, Politics and Economics2, Théo Favard, PharmD3.
1Global Pricing and Market Access, ESTEVE RD France, Paris, France, 2Esteve, London, United Kingdom, 3ESTEVE RD France, Paris, France.
1Global Pricing and Market Access, ESTEVE RD France, Paris, France, 2Esteve, London, United Kingdom, 3ESTEVE RD France, Paris, France.
OBJECTIVES: Endogenous Cushing’s syndrome (CS) is a rare endocrine disorder characterized by chronic excess of cortisol and loss of cortisol circadian rhythm. Patients who fail surgery, or are ineligible, require pharmacological agents such as steroidogenesis inhibitors (metyrapone, ketoconazole, osilodrostat) to control cortisol levels and reduce associated comorbidities. This study assesses the budget impact of metyrapone use for CS from a French healthcare system perspective.
METHODS: The target population includes adults with CS treated with metyrapone or osilodrostat. A 5-year budget impact model was developed comparing two scenarios:
1. All patients treated with metyrapone vs. all treated with osilodrostat
2. A gradual shift in treatment from metyrapone to osilodrostat, with osilodrostat market share increasing from 0% to 75% over 5 years and metyrapone share decreasing accordingly. The model captures drug acquisition, routine medical care, and adverse event (AE) management costs. CS prevalence was estimated using published epidemiological data. Daily treatment doses were: 1,500 mg/day for metyrapone and (a maximum of) 10 mg/day for osilodrostat. Assumptions for net drug costs reflect typical rebate levels in France, including those applied to products with a minor clinical added value rating.
RESULTS: In Scenario 1 (100% metyrapone vs. 100% osilodrostat), the 5-year per-patient drug cost was €46,035 for metyrapone vs. €110,875 for osilodrostat, resulting in a 58% cost saving with metyrapone. AE management costs were 81% lower with metyrapone (€1,338 vs. €6,869). The net incremental 5-year budget impact for the whole population was €73,035,706, representing a 54% cost saving with metyrapone. In Scenario 2, shifting a portion of patients to osilodrostat, reaching 75% market share, results in a cumulative-5-year incremental budget impact of €42,522,900 which is 41% higher compared with maintaining 100% metyrapone use.
CONCLUSIONS: Treatment of CS patients with metyrapone results in a lower budget impact compared with osilodrostat, driven by lower drug acquisition and AE management costs.
METHODS: The target population includes adults with CS treated with metyrapone or osilodrostat. A 5-year budget impact model was developed comparing two scenarios:
1. All patients treated with metyrapone vs. all treated with osilodrostat
2. A gradual shift in treatment from metyrapone to osilodrostat, with osilodrostat market share increasing from 0% to 75% over 5 years and metyrapone share decreasing accordingly. The model captures drug acquisition, routine medical care, and adverse event (AE) management costs. CS prevalence was estimated using published epidemiological data. Daily treatment doses were: 1,500 mg/day for metyrapone and (a maximum of) 10 mg/day for osilodrostat. Assumptions for net drug costs reflect typical rebate levels in France, including those applied to products with a minor clinical added value rating.
RESULTS: In Scenario 1 (100% metyrapone vs. 100% osilodrostat), the 5-year per-patient drug cost was €46,035 for metyrapone vs. €110,875 for osilodrostat, resulting in a 58% cost saving with metyrapone. AE management costs were 81% lower with metyrapone (€1,338 vs. €6,869). The net incremental 5-year budget impact for the whole population was €73,035,706, representing a 54% cost saving with metyrapone. In Scenario 2, shifting a portion of patients to osilodrostat, reaching 75% market share, results in a cumulative-5-year incremental budget impact of €42,522,900 which is 41% higher compared with maintaining 100% metyrapone use.
CONCLUSIONS: Treatment of CS patients with metyrapone results in a lower budget impact compared with osilodrostat, driven by lower drug acquisition and AE management costs.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE110
Topic
Economic Evaluation, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Budget Impact Analysis
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Rare & Orphan Diseases