Sixty-eight cancer drug indications won European approval between 2009 and 2013—but most of them did so without showing they could extend or improve life, a new study shows.
According to results published in the British Medical Journal on Tuesday, 57% of indications approved during that period hit the market based on endpoints other than overall survival or improved quality of life. Instead, they won their green lights via “surrogate” endpoints such as tumor shrinkage or progression-free survival.
And after a median of five years on the market, only eight drug indications had managed to post data showing a survival or quality of life benefit.
What’s more, in many situations where new meds did show benefits over existing therapies, those benefits were marginal, researchers from King’s College London and the London School of Economics said.
And the way they see it, that’s not acceptable considering the products’ high prices.
"When expensive drugs that lack clinically meaningful benefits are approved and paid for within publicly funded healthcare systems, individual patients can be harmed, important societal resources wasted, and the delivery of equitable and affordable care undermined,” they said in a statement.
So what can be done? According to Vinay Prasad, M.D., an assistant professor at Oregon Health & Science University and an outspoken Twitter user, the use of surrogate endpoints should be “the exception not the rule.”
He also wants to see “rigorous testing against the best standard of care in randomized trials powered to rule in or rule out a clinically meaningful difference in patient-centered outcomes in a representative population,” he wrote in an editorial linked to the study results, adding that “the expense and toxicity of cancer drugs means we have an obligation to expose patients to treatment only when they can reasonably expect an improvement in survival or quality of life.”
The results hit not long after two confirmatory trials failed to show that high-profile immuno-oncology meds could improve overall survival. Roche’s Tecentriq was the first to flop, failing to extend survival among bladder cancer patients, and Merck’s Keytruda came next with a miss in head and neck cancer.