The cost-effectiveness analysis of immune checkpoint inhibitors for microsatellite instability-high/mismatch repair

Pei Yang1,2,3, Zhengda Pei4, Yuan Zhou5,6

  • 1Department of General Surgery, Xiangya Hospital, Central South University, Changsha, 410008, Hunan, China.

Scientific Reports
|December 3, 2025
PubMed

Insights

First-line immunotherapy, including nivolumab plus ipilimumab, is cost-effective for advanced colorectal cancer (CRC) with MSI-H/dMMR mutations in the USA. Dual immunotherapy demonstrates preferable outcomes compared to single immunotherapy or chemotherapy.

Area of Science:

  • Oncology
  • Health Economics
  • Clinical Research

Background:

  • Immune checkpoint inhibitors (ICIs) offer novel treatment avenues for colorectal cancer (CRC) patients, potentially avoiding chemotherapy.
  • Evaluating the economic viability of these therapeutic innovations is crucial for real-world application.
  • This study focuses on microsatellite-instability-high/mismatch repair deficient (MSI-H/dMMR) advanced CRC, a specific subtype.

Purpose of the Study:

  • To compare the cost-effectiveness of first-line dual-immunotherapies, single immunotherapy, and chemotherapies for MSI-H/dMMR advanced CRC.
  • To analyze treatment outcomes from the perspective of American healthcare systems.
  • To inform clinical and economic decision-making for CRC treatment strategies.

Main Methods:

  • Utilized individual patient data (IPD) from KEYNOTE-177 and CheckMate-8HW trials.
  • Developed a 30-year Markov model with three health states.
  • Evaluated cost-effectiveness using metrics like QALYs, ICER, and INHB at a $100,000/QALY willingness-to-pay threshold.

Main Results:

  • Nivolumab plus ipilimumab and pembrolizumab demonstrated increased cost and effectiveness compared to chemotherapy.
  • The incremental cost-effectiveness ratio (ICER) for nivolumab plus ipilimumab versus chemotherapy was $13,670/QALY, with favorable incremental net-health benefits (INHB).
  • Nivolumab plus ipilimumab was also found to be preferable to pembrolizumab in terms of ICER and INHB.

Conclusions:

  • First-line immunotherapeutic treatments, particularly dual-immunotherapy regimens like nivolumab plus ipilimumab, appear cost-effective for MSI-H/dMMR advanced CRC in the USA.
  • The findings support the integration of immunotherapy into standard care for this patient population.
  • The study provides a robust economic evaluation framework for novel cancer therapies.

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