Cost-effectiveness Analysis of Regorafenib and TAS-102 in Refractory Metastatic Colorectal Cancer in the United States

Clin Colorectal Cancer. 2018 Dec;17(4):e751-e761. doi: 10.1016/j.clcc.2018.08.003. Epub 2018 Aug 24.

Abstract

Background: Regorafenib and TAS-102 are standard treatment options in refractory metastatic colorectal cancer based on improvement in overall survival by 6 and 8 weeks, respectively, when compared with best supportive care alone (BSC). Given the small incremental clinical benefit, we evaluated their cost-effectiveness from a United States payer's perspective.

Materials and methods: A Markov model was constructed to compare costs and effectiveness of regorafenib, TAS-102, and BSC. Model inputs for clinical efficacy and adverse events were from the CORRECT trial (Regorafenib monotherapy for previously treated metastatic colorectal cancer: an international, multicentre, randomised, placebo-controlled, phase 3 trial) for regorafenib and the RECOURSE trial (Randomized, Double Blind, Phase 3 Study of TAS-102 plus Best Supportive Care [BSC] versus Placebo plus BSC in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapies) for TAS-102. The incremental cost-effectiveness ratios (ICERs) were reported to compare treatments. Model robustness was checked with univariate and probabilistic sensitivity analyses as well as a scenario analysis using the CONCUR trial data for regorafenib.

Results: In our base case, regorafenib and TAS-102 had the ICERs of $395,223 per quality-adjusted life year (QALY) and $399,740 per QALY versus BSC, respectively. Compared with regorafenib, TAS-102 provided an additional 0.041 QALY at the cost of $16,608 or $406,104 per QALY, but the differences were not robust in sensitivity analyses. The most influential parameters on the ICERs were efficacy and health state utility parameters as well as the cost of treating neutropenia. In probabilistic sensitivity analysis using cost-effectiveness acceptability curves, BSC was more cost-effective than both regorafenib and TAS-102 in 50% of repetitions at the willingness-to-pay threshold of $330,000 per QALY.

Conclusion: Neither TAS-102 nor regorafenib are cost-effective at standard willingness-to-pay thresholds (ie, $150,000 per QALY) relative to BSC. There is no clear evidence that either treatment has better relative value.

Keywords: Colorectal cancer; Cost-effectiveness analysis; Regorafenib; Sensitivity analysis; TAS-102.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / economics*
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Colorectal Neoplasms / drug therapy
  • Colorectal Neoplasms / economics*
  • Colorectal Neoplasms / pathology
  • Cost-Benefit Analysis*
  • Double-Blind Method
  • Drug Combinations
  • Drug Resistance, Neoplasm*
  • Follow-Up Studies
  • Humans
  • Liver Neoplasms / drug therapy
  • Liver Neoplasms / economics*
  • Liver Neoplasms / secondary
  • Markov Chains
  • Palliative Care / economics*
  • Phenylurea Compounds / administration & dosage
  • Prognosis
  • Pyridines / administration & dosage
  • Pyrrolidines
  • Quality-Adjusted Life Years
  • Salvage Therapy*
  • Survival Rate
  • Thymine
  • Trifluridine / administration & dosage
  • United States
  • Uracil / analogs & derivatives

Substances

  • Drug Combinations
  • Phenylurea Compounds
  • Pyridines
  • Pyrrolidines
  • trifluridine tipiracil drug combination
  • regorafenib
  • Uracil
  • Thymine
  • Trifluridine