Regorafenib carries high incremental cost and delivers small incremental benefit, providing low value for patients with metastatic colorectal cancer, researchers reported online Aug. 24 in the Journal of Clinical Oncology.
Compared with best supportive care, use of regorafenib resulted in a gain of 6 weeks of life, which dropped to 2 quality-adjusted weeks of life (0.04 quality-adjusted life years [QALYs]) in light of its significant toxicity profile. With the cost of treatment estimated between $32,000 and $43,000, depending on the dose, the incremental cost-effectiveness ratio (ICER) was $730,000-$980,000 per QALY, Dr. Daniel Goldstein and his associates reported (J Clin Oncol. 2015 Aug 24. doi: 10.1200/JCO.2014.61.9569).
“With increasing deductibles and copays, our patients are now bearing a significant burden of the cost of drugs,” wrote Dr. Goldstein of the department of hematology and medical oncology at Emory University, Atlanta, and his colleagues. There is a need, they wrote, for new strategies “in patient selection for clinical trials, guideline development, and payer coverage determination that address clinical value in addition to statistically significant clinical benefit.”
The CORRECT (Colorectal Cancer Treated With Regorafenib or Placebo After Failure of Standard Therapy) trial indicated an overall survival benefit of 1.4 months for regorafenib, compared with placebo, in patients with colorectal cancer who progressed after standard regimens. Adverse events of grade 3 or 4 occurred in 54% of the regorafenib arm, compared with 14% in the placebo arm.
The analysis based on the CORRECT data indicated a low likelihood that regorafenib would be considered cost effective at a willingness to pay threshold less than $600,000 per QALY. Regorafenib exceeds typical accepted values for cost-effectiveness.
Model parameters that most influenced ICER were regorafenib cost, probability of stopping treatment due to an adverse event, and baseline utility value. The ICER remained greater than $550,000 per QALY across a broad range of each of these variables.
Given the increasing amount of out-of-pocket costs for patients, the investigators recommend “a careful discussion between physicians and patients regarding the additional benefit and potential total drug cost before starting regorafenib.”
The study was supported by internal funds and by a grant from the National Institutes of Health. Dr. Goldstein reported having no disclosures. Three of his coauthors reported ties to industry.