Roche Holding AG and a group of outside investigators may share raw data from conflicting trials of Avastin to help determine whether the $7 billion-a-year treatment really helps patients with deadly brain cancer.
The data is from two trials that both found the drug didn’t help patients with glioblastoma live longer, but differed on a more subjective measure: quality of life. In results published yesterday in the New England Journal of Medicine, researchers who led a Roche-sponsored trial said Avastin improved or maintained quality of life and brain function. An independent study dubbed RTOG 0825 said Avastin patients were worse off on both counts.
The outcome may help determine how broadly Avastin is used for glioblastoma, a cancer whose patients have few other options for treatment. Roche, based in Basel, Switzerland, got accelerated approval for Avastin in glioblastoma in 2009 based on earlier, smaller trials. The drug, with sales of 6.25 billion Swiss francs ($7.04 billion) last year, is also used to treat tumors including metastatic colorectal and kidney cancers.
The difference between the studies is “neither trivial nor academic,” Howard Fine, deputy director of the New York University Cancer Institute, wrote in an editorial published with the results. If Avastin improves patients’ quality of life and brain function, “then a strong argument can be made for its use as part of the initial treatment of glioblastoma, regardless of its effect on survival.”
If Avastin actually damages brain function, there’s no argument for its wide use, especially since it wasn’t shown to help patients live longer in the trials, he wrote.
“The first question is, is the outcome different just because they used different statistical methodologies?” Fine said in a telephone interview. Fine wasn’t involved in either study published today, though he has led previous research on Avastin in brain cancer. “You can apply different statistical tests and get very different statistical answers.”
It’s common for cognitive function to decline as patients’ brain tumors get worse, Fine said. Considering quality of life and brain function improvements when reviewing a cancer drug would be new ground for U.S. regulators, he said.
Roche is open to sharing its trial data and is already in talks with researchers from the RTOG study as well as other parties, spokesman Daniel Grotzky said in an e-mail.
Roche put a new policy on sharing trial data into effect last year, Grotzky said. The drugmaker had faced criticism for previously not releasing full details on studies of its flu treatment Tamiflu, and the Cochrane Collaboration, a health-care analysis group, is now examining the efficacy and safety of the treatment.
The accelerated approval for Avastin in glioblastoma required the Swiss company to file trial data showing a clinical benefit for patients by the end of 2015. Roche is discussing the results of the latest studies with the Food and Drug Administration, Holli Dickson, a spokeswoman for Roche unit Genentech, said in an e-mailed statement.
Initial results from the two trials and a separate study of Avastin in cervical cancer, also published today in the medical journal, were presented last June at a meeting of the American Society of Clinical Oncology. Roche is planning to file for approval for Avastin for cervical cancer this year.