The majority of cancer treatments approved in Europe between 2009 and 2013 launched without clear evidence that they improved survival or quality of life for patients, a study in the BMJ said.
Of 68 cancer indications approved by the European Medicines Agency during this time, 57% launched without definitive evidence of survival or quality of life gains, researchers from King's College London and the London School of Economics said.
After a median of five years on the market, eight additional cancer indications showed gains in survival or quality of life. However, when the drugs showed an improvement, it was often marginal, researchers said.
For the 33 remaining indications, there is still uncertainty over whether the drugs extend survival or improve quality of life.
Cancer drugs that reported quality of life gains at the time of their approval included AstraZeneca PLC's Iressa and Boehringer Ingelheim's Gilotrif, each for non-small cell lung cancer, as well as Sanofi's Caprelsa for thyroid cancer.
According to the researchers' data, Roche Holding AG's Rituxan for leukemia and Herceptin for a gene-marked breast cancer did not show gains when they were approved, though Herceptin has since shown overall survival and quality of life improvements since launch.
After launch, Pfizer Inc. and EMD Serono's Zalkori was able to show quality of life gains for non-small cell lung cancer patients, as was Roche and Astellas Pharma Inc.'s Tarceva. Novartis AG's Tasigna and Arzerra also showed improvements for leukemia patients, while its Votrient did for kidney cancer.
The researchers said their findings raise the possibility that regulatory evidence standards are "failing to incentivize drug development that best meets the needs of patients, clinicians, and healthcare systems."
"The use of uncontrolled study designs or surrogate endpoints should be the exception not the rule," said Vinay Prasad, assistant professor at Oregon Health & Science University, in a linked editorial. Prasad is the author of another recent report that argued the cost of developing cancer drugs is far below what pharmaceutical companies have claimed.
European governments are starting to seriously challenge the high cost of drugs, Deborah Cohen, associate editor at The BMJ, wrote in an accompanying feature.