Drugmakers are already facing growing backlash to the perceived high prices of drugs, but a new study about the real benefits of cancer drugs is sure to add fuel to the fire. It says that follow-up studies of cancer drugs that won speedy approvals based on early results found most did not prolong or improve life in patients.
The study was of 18 cancer drugs approved by the FDA between 2008 and 2012. It found that of 13 where further studies were available, none of the drugs were determined to prolong life, and only one had enough evidence to say it improved quality of life.
Despite that, the study said the FDA rescinded only one approval for Genentech's Avastin in treating breast cancer, although it is approved for use in other cancers.
“We were shocked to find that these drugs don’t save lives and don’t improve quality of lives," lead author Diana Zuckerman told Reuters. Zuckerman is also president of the National Center for Health Research and the Cancer Prevention and Treatment Fund in Washington, D.C.
The study said that the FDA’s aim is to approve drugs that can improve survival or quality of life and that about a third of cancer drugs show a “demonstrated improvement in overall survival in randomized clinical trials.” But it said that the FDA also considers so-called “surrogate endpoints” for life threatening conditions with few alternative treatment options, things like how soon a cancer begins to progress after a drug has been started. The other two-thirds of approvals were based on these surrogate endpoints, the story said.
The authors acknowledged that there have been “transformative cancer drugs with large and meaningful clinical benefits.” But most of the cancer drugs they looked at showed only marginal improvements for patients. They said that of 71 consecutive approvals for drugs treating solid cancers they looked at, the median improvement in overall survival in pivotal trials was just 2.1 months. They argued for making overall survival a surrogate endpoint in trials as well.
The drugs they looked at ranged from $20,000 to $70,000 as average prices for what Medicare would pay. They could be higher or lower in different regions of the country, and when paid by other providers.
For years now, what are sometimes seen as the extraordinary prices of new cancer drugs have been getting criticism from doctors who treat cancer patients. Sanofi in 2012 actually cut in half the price of its then-new colorectal cancer drug Zaltrap after an op-ed piece by doctors from Memorial Sloan-Kettering Cancer Center said they wouldn’t use it because its limited benefits didn’t justify its price.
More recently, Astellas' prostate cancer drug Xtandi has been held up as an example by lawmakers as a drug whose price should be looked over by federal officials. They pointed out that its U.S. list price is about $129,000 per treatment course compared with $39,000 in Sweden and in Astellas' native Japan.
The FDA in a statement to Reuters defended its decision to approve cancer drugs based on surrogate endpoints. “It has been widely accepted that benefit can be demonstrated by a number of endpoints, not just overall survival," the FDA said.
But Dr. Vinay Prasad, another cancer doctor and researcher, said that too often, “We don't really know if people live longer or improve based on those outcomes. I think their results are very sobering," he told Reuters, referring to the new study.
Editor's Note: The story was updated to change the average price range that Medicare paid for the drugs.