Cost-effectiveness of vorasidenib in treatment of isocitrate dehydrogenase-mutated low-grade glioma

Cancer. 2026 Jun 1;132(11):e70463. doi: 10.1002/cncr.70463.

Abstract

Background: Vorasidenib is Food and Drug Administration-approved for treatment of grade 2 isocitrate dehydrogenase (IDH)-mutant gliomas and has demonstrated a benefit in progression-free survival compared to a placebo. This study assessed the cost-effectiveness of this treatment strategy compared to standard treatment: observation or chemoradiation (CRT).

Methods: The authors used an integrated Markov model to compare quality-adjusted life-years (QALY) gained and cost-effectiveness of patients with IDH-mutated low-grade glioma (LGG) treated with vorasidenib, observation, or CRT. Outcomes were expressed as incremental cost-effectiveness ratios (ICERs) per QALY gained. Treatment is considered cost-effective if it meets the commonly accepted willingness-to-pay threshold of $100,000 per QALY gained.

Results: Based on model parameters, vorasidenib demonstrated an ICER of $356,756 per QALY when compared to CRT. When compared to observation, vorasidenib demonstrated an improved ICER of $215,191 per QALY. On one-way sensitivity analysis compared to observation, median cost of vorasidenib and median overall survival were most likely to affect ICER with only marginal changes on analysis of other model factors. For ICER to become ≤$100,000 compared to observation, the cost of vorasidenib would need to drop to below $14,072 monthly or have a median survival gain of at least 11.9 years. The probabilistic sensitivity analysis demonstrated that vorasidenib was favored in none of 5000 Monte Carlo iterations.

Conclusion: Using the best available data, the current price of vorasidenib does not meet commonly accepted cost-effectiveness thresholds in treatment of IDH-mutated LGGs.

Keywords: IDH mutation; cost‐effectiveness; low‐grade glioma; quality‐adjusted life years; vorasidenib.

MeSH terms

  • Antineoplastic Agents* / economics
  • Antineoplastic Agents* / therapeutic use
  • Brain Neoplasms* / drug therapy
  • Brain Neoplasms* / economics
  • Brain Neoplasms* / genetics
  • Brain Neoplasms* / mortality
  • Cost-Benefit Analysis
  • Cost-Effectiveness Analysis
  • Glioma* / drug therapy
  • Glioma* / economics
  • Glioma* / genetics
  • Glioma* / mortality
  • Glioma* / pathology
  • Humans
  • Isocitrate Dehydrogenase* / genetics
  • Markov Chains
  • Mutation
  • Quality-Adjusted Life Years
  • Triazines* / economics
  • Triazines* / therapeutic use

Substances

  • Isocitrate Dehydrogenase
  • Triazines
  • Antineoplastic Agents