May 16, 2016
For advanced liver cancer, there’s a single approved drug shown to offer patients a chance at longer life. But a new study co-authored by two researchers in the UNC Gillings School of Global Public Health found that this drug was notably less effective in a group of Medicare patients, who likely had more extensive cancer and serious liver disease, than it was for patients included in clinical trials.
The researchers from the Gillings School are Jennifer Lund, PhD, assistant professor of epidemiology, and Stacie Dusetzina, PhD, assistant professor of health policy and management at UNC Gillings and assistant professor in the UNC Eshelman School of Pharmacy. Both are members of the UNC Lineberger Comprehensive Cancer Center.
In the journal The Oncologist, the researchers report today that the median survival for a group of Medicare patients on the drug sorafenib was three months, which is significantly lower than the median survival of nearly 11 months for patients treated with the drug during a phase III clinical trial. As the drug comes with significant side effects and a cost to patients and insurers of more than $10,000 a month, researchers are questioning the value of the drug for all patients.
The U.S. Food and Drug Administration approved sorafenib – known commercially as Nexavar – for the treatment of advanced hepatocellular carcinoma in 2007. In a phase III clinical trial, patients with advanced liver cancer had a median survival of 10.7 months on the drug, which was 2.8 months more than patients who didn’t receive the treatment. But patients in that study also were in good physical condition and their level of cirrhosis, which nearly universally accompanies liver cancer, was well controlled.
In the new study, the researchers analyzed survival data for a group of patients insured by Medicare – a government health insurance program for people aged 65 years and older, or with disabilities – and who were diagnosed between 2008 and 2011. Of the 27 percent of 1,532 patients given sorafenib, median survival from the first prescription was three months, which was not statistically longer than survival of untreated patients.
The research team concluded that lower survival in the Medicare study group was likely due to a generally sicker population. Further analysis suggested that patients in the study with earlier stage disease might be more likely to benefit from taking the drug. The researchers pointed to issues of cost, both financial and in terms of side effects, as factors that patients and doctors should consider when deciding on a course of treatment.
In previous studies, researchers have found that the median monthly price for the drug across all available Medicare part D plans in 2014 was $10,811 per month, said study co-author Dusetzina.
That price tag can mean thousands of dollars in out-of-pocket costs for patients, she explained, as most plans require cost sharing of at least 25 percent when filling the drug’s prescription. Even for patients who have reached the catastrophic spending level in Medicare Part D – when the amount they are expected to pay out-of-pocket decreases – they would still pay $540 per prescription fill each month.
“This is obviously going to present financial challenges for many patients,” Dusetzina said. “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.”
Patient data was drawn from the National Cancer Institute’s Surveillance, Epidemiology and End Results Program Medicare linkage.