BUDGET IMPACT OF INTRODUCING SELUMETINIB FOR SYMPTOMATIC INOPERABLE PLEXIFORM NEUROFIBROMAS IN CHILDREN WITH NEUROFIBROMATOSIS TYPE 1: A PUBLIC PAYER PERSPECTIVE
Author(s)
Yanis OUABDESSELAM, Jr., PharmD1, Lina Ould amrouche, PharmD1, Abdelaziz Rouina, Sr., PharmD2, Mohamed Yacine Achouri, Sr., PharmD, PhD3.
1Pharmacy, University of Health sciences, Algiers, Algeria, 2Algiers, Algiers, Algeria, 3University of Health Sciences, Algiers, Algeria.
1Pharmacy, University of Health sciences, Algiers, Algeria, 2Algiers, Algiers, Algeria, 3University of Health Sciences, Algiers, Algeria.
OBJECTIVES: To estimate the budget impact of introducing selumetinib for symptomatic inoperable plexiform neurofibromas in children with neurofibromatosis type 1 (NF1), from a public payer perspective over a five-year horizon (2026-2030).
METHODS: An epidemiological budget impact analysis was conducted following International Society for Pharmacoeconomics and Outcomes Research (ISPOR) good-practice principles, structured as a case study. Scenario A (current care, including off-label imatinib and symptomatic management) was compared with scenario B, in which selumetinib was adopted progressively (from 20% to 100%). The eligible population was derived from a local selection cascade. Body surface area was projected using Mosteller's formula and a linear regression, and treatment persistence was extrapolated from the SPRINT trial using Weibull and simple-probability approaches. Selumetinib was dosed at 25 mg/m² twice daily in 28-day cycles. Only drug-acquisition costs were valued, and no discounting was applied. A univariate sensitivity analysis was performed on the net cumulative impact.
RESULTS: The eligible population increased from 15 to 31 children, and treated patients rose from 6 to 27 as uptake increased from 20% to 100%. Selumetinib unit prices were €3,707.70 (60×10 mg) and €9,269.25 (60×25 mg). The cumulative cost was €47,342 for scenario A versus €10,425,016 for scenario B, yielding a net cumulative budget impact of approximately €10.38 million over five years; the annual net impact rose from approximately €0.68 to €3.60 million. The net impact was most influenced by the number of incident patients (≈€5.2M-€15.6M) and the eligible population size (≈€8.5M-€12.3M), and less by price and exchange rate; drug acquisition remained the main cost driver.
CONCLUSIONS: Introducing selumetinib would entail a substantial and growing net expenditure for the payer, mainly governed by eligible population size and acquisition price. The analysis informs affordability rather than efficiency; managed access, price-volume agreements and a national registry could be considered to support decision-making.
METHODS: An epidemiological budget impact analysis was conducted following International Society for Pharmacoeconomics and Outcomes Research (ISPOR) good-practice principles, structured as a case study. Scenario A (current care, including off-label imatinib and symptomatic management) was compared with scenario B, in which selumetinib was adopted progressively (from 20% to 100%). The eligible population was derived from a local selection cascade. Body surface area was projected using Mosteller's formula and a linear regression, and treatment persistence was extrapolated from the SPRINT trial using Weibull and simple-probability approaches. Selumetinib was dosed at 25 mg/m² twice daily in 28-day cycles. Only drug-acquisition costs were valued, and no discounting was applied. A univariate sensitivity analysis was performed on the net cumulative impact.
RESULTS: The eligible population increased from 15 to 31 children, and treated patients rose from 6 to 27 as uptake increased from 20% to 100%. Selumetinib unit prices were €3,707.70 (60×10 mg) and €9,269.25 (60×25 mg). The cumulative cost was €47,342 for scenario A versus €10,425,016 for scenario B, yielding a net cumulative budget impact of approximately €10.38 million over five years; the annual net impact rose from approximately €0.68 to €3.60 million. The net impact was most influenced by the number of incident patients (≈€5.2M-€15.6M) and the eligible population size (≈€8.5M-€12.3M), and less by price and exchange rate; drug acquisition remained the main cost driver.
CONCLUSIONS: Introducing selumetinib would entail a substantial and growing net expenditure for the payer, mainly governed by eligible population size and acquisition price. The analysis informs affordability rather than efficiency; managed access, price-volume agreements and a national registry could be considered to support decision-making.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE412
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Budget Impact Analysis
Disease
Neurological Disorders, Pediatrics, Rare & Orphan Diseases