Cancer is one of those diseases that cause dread in its victims and their families. Though many are treatable or even curable, many others are not and there is great pressure on medics to offer any source of hope the those suffering from hard to treat varieties of the disease.

And modern cancer drugs are often very expensive:

...the average cancer drug costs in excess of $100 000 (£75 000; €85 000) per year of treatment...

This makes decisions about whether health systems can afford them very controversial.

But a recent editorial in the BMJ claims than many modern cancer drugs get approved despite the fact that the evidence that they work is very weak. The editorial argues (quoting a recent European study that extended work done on approvals by the USA's FDA and reached similar conclusions):

In their study of cancer drugs approved by the European Medicines Agency between 2009 and 2013, 57% (39/68) had no supporting evidence of better survival or quality of life when they entered the market. After a median of 5.9 years on the market, just six of these 39 (15%) agents had been shown to improve survival or quality of life.

They expand this further (quoting both US and European studies):

Although we are approving cancer drugs at a rapid pace, few come to market with good evidence that they improve patient centred outcomes. If they do, they often offer marginal benefits that may be lost in the heterogeneous patients of the real world. Most approvals of cancer drugs are based on flimsy or untested surrogate endpoints, and postmarketing studies rarely validate the efficacy and safety of these drugs on patient centred endpoints.

It looks like our fear of cancer is giving drug companies an easy path to the approval of marginal or useless drugs.

Is it true that most modern cancer drugs offer minimal benefits in the real world?

  • 2
    Regarding the comment at the end, it's not just fear getting drugs to market; a lot of researchers in this area push for this as they want to help dying people. If these drugs are then being misapplied, the solution wouldn't be making it harder to get them to market, but rather to increase information and encourage sound decision-making.
    – Nat
    Oct 20, 2017 at 3:50
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    @Nat Perhaps many researchers are motivated by their desire to help sick people. Others may just want to make money. Either way the regulations should ensure that we measure whether they actually help people. Noble motivation and false hope are not sufficient reasons to sell expensive but ineffective drugs.
    – matt_black
    Oct 20, 2017 at 13:03
  • @matt_black do people no longer come equipped with brains and responsibility for their own actions?
    – hobbs
    Oct 21, 2017 at 4:23
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    @hobbs Yes people should think for themselves. But the information they need is often complex, technical and controlled by the drug firms and regulators. Few people are equipped to judge the efficacy of the drugs. Moreover most of the treatments are paid for by insurers or health systems not the individuals. This creates a huge incentive and information asymmetry that doesn't make good decisions by individuals easy or likely.
    – matt_black
    Oct 22, 2017 at 16:06
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    This is a really important question but it's also one of those where I'm not sure how much we can add to what the likes of the British Medical Journal say. It's hugely difficult to assess and controversial even among researchers who agree on all the facts - especially with diseases like prostate cancer where even basic survival statistics are hotly disputed because so many elderly people die with it not of it... Oct 23, 2017 at 17:07


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