Early Stage Health Economic Evaluation of IMMUTOL Gene Therapy for Relapsing/Remitting Multiple Sclerosis in the Netherlands
Author(s)
Attila Imre, PharmD1, Rok Hren, PhD2, Balázs Nagy, PhD2.
1Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary, 2Syreon Research Institute, Budapest, Hungary.
1Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary, 2Syreon Research Institute, Budapest, Hungary.
OBJECTIVES: Relapsing-remitting multiple sclerosis (RRMS) causes substantial lifelong health and societal burdens. IMMUTOL is a novel gene therapy in pre-clinical development aimed at restoring immune tolerance and achieving durable remission. Early health economic assessment is essential to define value-based pricing and support development strategies prior to market entry. The objective is to explore how one-time price and clinical efficacy of IMMUTOL influence cost-effectiveness relative to a clinically relevant comparator, an early highly effective treatment sequence (OCR-NAT-CLA-ALE) under Dutch guidelines.
METHODS: A lifetime patient-level microsimulation (ErasmusMC/iMTA MS model) of 10,000 RRMS patients was conducted from societal and healthcare perspectives. A €50,000/QALY willingness-to-pay threshold was applied; and varied IMMUTOL’s relapse rate reduction (IRR 0.0-0.3), disability progression reduction (RR 0.0-0.3), and one-time prices (€50,000-€2,000,000). Incremental cost-effectiveness ratios (ICERs) and heatmaps were generated to identify price-efficacy combinations that meet the cost-effectiveness criteria in the Netherlands.
RESULTS: IMMUTOL was dominant (higher QALYs at lower costs) across all efficacy scenarios at prices ≤ €200,000. At €500,000, IMMUTOL remained cost-effective (ICER < €50,000/QALY) if IRR ≤ 0.2 and RR ≤ 0.1. At prices ≥ €1,000,000, only the idealized “magic pill” scenario (IRR = RR = 0.0) approached cost-effectiveness. Heatmap visualizations delineated clear thresholds for value-based pricing under Dutch guidelines.
CONCLUSIONS: A one-time price up to €200,000 yields dominant economic outcomes for IMMUTOL gene therapy. At €500,000, cost-effectiveness requires substantial relapse and progression reductions (IRR ≤ 0.2; RR ≤ 0.1). These insights guide pricing strategy and development planning for emerging gene therapies in RRMS.
METHODS: A lifetime patient-level microsimulation (ErasmusMC/iMTA MS model) of 10,000 RRMS patients was conducted from societal and healthcare perspectives. A €50,000/QALY willingness-to-pay threshold was applied; and varied IMMUTOL’s relapse rate reduction (IRR 0.0-0.3), disability progression reduction (RR 0.0-0.3), and one-time prices (€50,000-€2,000,000). Incremental cost-effectiveness ratios (ICERs) and heatmaps were generated to identify price-efficacy combinations that meet the cost-effectiveness criteria in the Netherlands.
RESULTS: IMMUTOL was dominant (higher QALYs at lower costs) across all efficacy scenarios at prices ≤ €200,000. At €500,000, IMMUTOL remained cost-effective (ICER < €50,000/QALY) if IRR ≤ 0.2 and RR ≤ 0.1. At prices ≥ €1,000,000, only the idealized “magic pill” scenario (IRR = RR = 0.0) approached cost-effectiveness. Heatmap visualizations delineated clear thresholds for value-based pricing under Dutch guidelines.
CONCLUSIONS: A one-time price up to €200,000 yields dominant economic outcomes for IMMUTOL gene therapy. At €500,000, cost-effectiveness requires substantial relapse and progression reductions (IRR ≤ 0.2; RR ≤ 0.1). These insights guide pricing strategy and development planning for emerging gene therapies in RRMS.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE348
Topic
Economic Evaluation
Topic Subcategory
Work & Home Productivity - Indirect Costs
Disease
Neurological Disorders