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Feature Drug Regulation

Cancer drugs: high price, uncertain value

BMJ 2017; 359 doi: (Published 04 October 2017) Cite this as: BMJ 2017;359:j4543
  1. Deborah Cohen, associate editor, The BMJ
  1. dcohen{at}

A study published in The BMJ this week shows how most new cancer drugs are failing to deliver any clinically meaningful benefit. It’s time for Europe to raise the evidence bar before market approval, finds Deborah Cohen

Most cancer drugs recently entering the European market do so without clear evidence of extending or improving quality of life, new research published in The BMJ has found.1

The findings raise serious questions about why the current regulatory environment supports the approval of cancer drugs that may leave patients at risk of experiencing toxicity and reduced quality of life without deriving meaningful benefit.

Out of the 68 cancer drug indications approved by the European Medicines Agency during 2009-13, 57% (39) entered the market without evidence of a survival or quality of life benefit. Even when drugs did show survival gains over available treatment options, most of these were not clinically meaningful, researchers found.

Many of the drugs were approved on the basis of surrogate endpoints despite evidence that these are not a reliable indicator of overall survival or quality of life for most cancer treatments.

“When expensive drugs that lack clinically meaningful benefits are approved and reimbursed within publicly funded healthcare systems, individual patients may be harmed, important resources wasted, and the delivery of equitable and affordable care is undermined,” the researchers say.

The study comes at a time when European governments are starting to seriously challenge the high cost of drugs. While it’s hard to know how much healthcare systems are paying for cancer drugs because prices are often negotiated behind closed doors, the total amount spent on cancer care is growing, partly because of the cost of drugs.

Inadequate evidence

The research found that the EMA is basing many approval decisions on uncontrolled study designs or surrogate endpoints, which don’t always translate …

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