More than half of cancer drugs may not work, study suggests

A number of cancer drugs entered the European market “without evidence of benefit on survival or quality of life”, researchers have said.

All new cancer drugs are given Europe-wide market approval through the European Medicines Agency (EMA).

But a new study, published in The British Medical Journal (BMJ), found that of cancer drugs approved by the EMA between 2009 and 2013, 57% (39/68) had no supporting evidence of better survival or quality of life when they entered the market.

After an average of five years of follow up, only half had shown a survival or quality of life gain over existing treatments or placebo.

For the remaining 33 (49%), “uncertainty remains over whether the drugs extend survival or improve quality of life”, according to the authors from King's College London and the London School of Economics and Political Science (LSE).

And of the 23 drugs with a survival benefit that could be scored with a validated tool, only 11 (48%) were judged to offer a clinically meaningful benefit, they added.

As a result of the findings, the researchers have called on the EMA to increase the evidence bar for the market authorisation of new cancer drugs.

Study author Huseyin Naci, assistant professor in LSE's Department of Health Policy, said: “It is remarkable that so few cancer drugs enter the European market without any clear data on outcomes that matter to patients and their doctors: longer survival and better quality of life.

“There is a clear need to raise the bar for approving new cancer drugs.”

Lead author, Dr Courtney Davis, a medical and political sociologist in the Department of Global Health and Social Medicine at King's, added: “We evaluated the evidence base for all new drugs entering the market over a five year period and found that the majority came onto the market without clear evidence that they improved patients' survival or quality of life.”

An EMA spokesman said: “EMA has had no time yet to analyse the findings of the BMJ study properly. Therefore we cannot comment on this.

“What we can say is that EMA has discussed the evidence underpinning cancer medicines widely and welcomes further debate on this.”

Cancer Research UK said the study may not reflect the situation of cancer drugs in the UK.

The National Institute for Health and Care Excellence (Nice) aims to review all new cancer drugs within 90 days of them being licensed for use in England.

After looking at the clinical and cost effectiveness, Nice then decides whether the drug should be routinely used across the NHS.

Some drugs are approved for widespread use and others are rejected.

If a drug shows promise but there is not enough data Nice can, under certain circumstances, recommend that the drug is available through the Cancer Drugs Fund, which allows more time for evidence to be collected before a final decision is made.

Commenting on the research, Emma Greenwood, Cancer Research UK's director of policy, said: “This study doesn't necessarily show us what's happening here in the UK.

“While the European Medicines Agency (EMA) decides which new drugs are safe to be sold in Europe, it's national bodies like Nice that decide which drugs should be made available to patients.

“Nice makes these decisions based on the clinical effectiveness and the cost of a drug to determine whether it will bring value to patients and the NHS.

“But the study does highlight the importance of using real-world evidence from patients, on top of data from clinical trials, to build our understanding of how drugs work in a real life setting.

“We're already starting to see this happen through the Cancer Drugs Fund in England, where patients can access promising new drugs while more data is collected on their effectiveness.

“This type of evidence is becoming increasingly important as more innovative and targeted treatments are developed.”

PA