Cost of Regorafenib versus Benefit Analyzed in Patients with Metastatic Colorectal Cancer

According to a recent cost-effectiveness analysis, third-line therapy with regorafenib (Stivarga) in patients with previously treated metastatic colorectal cancer (CRC) far ­exceeded accepted willingness-to-pay thresholds based on incremental cost-effectiveness ratio (ICER) and quality-adjusted life-years (QALYs). Presented at the 2015 Gastrointestinal Cancers Symposium, the results showed that regorafenib provided an additional 0.04 QALYs, at a cost of $39,391.

“The incremental cost-effectiveness ratio was almost $900,000 per QALY, which is way above the benchmark value,” said Daniel A. Goldstein, MD, Hematology Oncology Fellow at the Winship Cancer Institute, Emory University, Atlanta. “Basically, you’re not getting a lot of bang for your buck.”

Regorafenib was approved by the FDA in 2012 for patients with CRC and is now the standard of care as third-line therapy in patients with metastatic CRC, improving the median overall survival by only 1.4 months. Dr Goldstein was quick to note its considerable side-effect profile and high cost.

“It has a lot of side effects—hand-foot syndrome, fatigue, diarrhea—and patients who have already received 2 lines of treatment are often not in a high enough performance status to receive it,” he said.

In addition, when it is administered, there are extraordinary costs to payers. Citing the lack of any published US-based cost-effectiveness studies of regorafenib for the treatment of metastatic colorectal cancer, Dr Goldstein’s outcomes were obtained by merging publicly available clinical trial data with publicly available cost data for Medicare.

According to estimates from the Centers for Medicare & Medicaid Services, the base-case value for the drug was $11,364 per cycle. (The drug is administered on the first 21 days of a 28-day cycle.) The management of adverse events included in Dr Goldstein’s assessment was based on accepted standards.

“We used the CORRECT trial data that were published for patients who were treated with regorafenib versus placebo in a third-line setting,” said Dr Goldstein, “and they found that regorafenib had a 6-week median overall survival benefit. And so, when you incorporate quality-adjusted life-years into that, because the quality of life for patients who are in third-line treatment is not great, the drug provided patients only 0.04 quality-adjusted life-years gained.”

And those 0.04 QALYs proved costly. Including adverse events, the price of regorafenib equaled $39,391 for the total treatment duration.

“In all 1-way sensitivity analyses,” noted Dr Goldstein, “the ICER of regorafenib was greater than $700,000 per QALY. And the ICER of regorafenib was greater than $200,000 per QALY in more than 99% of probabilistic sensitivity analyses.”

At a willingness-to-pay threshold of $100,000 per QALY, the use of regorafenib in patients with previously treated metastatic CRC is not cost-­effective.

“If there is not a precise number for willingness-to-pay value, it’s been approximated in the past at around $50,000 per QALY, perhaps $100,000 per QALY, and maybe even $150,000 per QALY,” emphasized Dr Goldstein, “but this number of $900,000 per QALY is way above that benchmark value….It’s the highest number I’ve ever seen in any of my personal studies.”

When asked whether this drug is still used at Emory, Dr Goldstein acknowledged, “When we do have patients who have run out of all FDA-approved options, we may offer this drug, although, admittedly, it doesn’t have that much benefit in unselected patients.”

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