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Expensive cancer drugs have little impact on survival

Analysis questions whether vast sum spent on these drugs is appropriate

Ingrid Torjesen

Thursday, 10 November 2016

Expensive cancer drugs approved within the last 10 years may only increase life by a few months so spending the amount spent on them “may be inappropriate”, says an analysis* published in The BMJ today.

The author, Peter Wise, a former consultant at Charing Cross Hospital in London, calls for stricter drug approval criteria and improved consent processes “to achieve ethical treatment and reduce cancer costs.”

Cancer survival has improved in recent decades, he explains. In the US, for example, five-year relative survival in adults with solid cancer increased from 49% to 68% over 40 years.

But how much of that improvement in cancer survival can be attributed to new drugs is questionable, he says, because many drugs approved in the last decade prolong life by just one to two months.

“The approval of drugs with such small survival benefits raises ethical questions, including whether recipients are aware of the drugs’ limited benefits, whether the high cost:benefit ratios are justified, and whether trials are providing the right information,” says Wise.

He draws attention to limitations of cancer drug trials, such as the use of surrogate endpoints that allow earlier approval of new drugs, but are not always true indicators of survival benefit. And he warns that the marginal responses in clinical trials may not even apply to the majority of patients treated outside trials.

“The threshold for approval of new and existing cancer drugs needs to be raised - using more meaningful disease specific criteria of risk-benefit and cost-benefit,” he argues. He is hopeful that the recent integration of the Cancer Drugs Fund into the National Institute of Health and Care Excellence (NICE) in England might make it possible to monitor the “real world benefit” of these drugs.

He also raises concern over the US Food and Drug Administration (FDA)'s accelerated and “breakthrough” category which, he says, compounds the risk of premature approval on limited evidence.

“The low bar of approval for these expensive drugs ignores the ethical principle of fairness and equity,” he says. “By promoting marginally better treatment of poorly responsive cancers, it diverts valuable resources that might be better employed for other health needs, within and outside cancer care.”

A lack of fully informed consent for cancer treatment is also a concern, often leading to misinformed patients with unrealistic expectations, he adds. “Good cancer care demands empowerment of patients with accurate, impartial information followed by genuinely informed consent in both the clinical trial and therapeutic settings,” he says. “Ethical impediments to sound practice need to be addressed and corrected.”

* Wise PH. Cancer drugs, survival, and ethics. BMJ 2016;355:i5792. doi: 10.1136/bmj.i5792

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