Benefit of Liver Cancer Drug Disputed in Real World Setting

Sorafenib for advanced hepatocellular carcinoma may not offer the significantly lengthened life expectancy reported in clinical trials.

Findings from a recent study suggest that the FDA-approved drug sorafenib for advanced liver cancer may not offer patients the longer life expectancy that found in clinical trials.

During the trials, the median survival was approximately 11 months, whereas patients in the current study had a median survival of 3 months.

Researchers found that the drug was not as effective in Medicare patients with more extensive cancer and serious liver disease, compared with patients in the trials, according to the study published in The Oncologist.

"No drug that results in a 3-month survival can be thought to be offering a meaningful life expectancy," said study author Hanna K. Sanoff, MD, MPH. "This doesn't mean that we shouldn't prescribe it, but we should be mindful that the broader population of liver cancer patients is sicker than the patients in the landmark trial. Our patients deserve to know that the promise of nearly a year of life may not be their reality."

The drug sorafenib was approved in 2007 for advanced hepatocellular carcinoma after a phase 3 clinical trial suggested the drug increased the survival of patients by 2.8 months. Patients in the study were in good physical condition and had well-controlled cirrhosis.

"This drug was tested in a clinical trial with patients with mild cirrhosis who were pretty fit," Dr Sanoff said. "Because of concurrent cirrhosis, it may be that the gap between the trial population and the average liver cancer patient may be greater than in some other cancers."

Researchers in the current study analyzed data from Medicare patients diagnosed between 2001 and 2008. It was discovered that the median survival of patients receiving the drug was 3 months after the first prescription, which was not much longer than the survival of patients who did not receive the drug.

The researchers believe the lower survival was likely because of a sicker population. They also found, however, that patients in the study with an earlier stage of the disease could benefit more from the drug.

Before prescribing the medication, physicians should take into account side effects and cost, which is approximately $10,811 per month for patients and insurers. The high cost can lead to an increase in out-of-pocket costs for patients, even among patients with Medicare Part D, who would pay $540 per month for the drug, according to the study.

"This is obviously going to present financial challenges for many patients,” concluded study co-author Stacie Dusetzina, PhD. “This underscores the fact that establishing effectiveness of therapies outside of trial settings is complicated but important, if we want to really understand the value of cancer therapies. Translating the benefits of treatments into a 'real world' setting isn't always easy."