New Cancer Drugs May Not Improve Survival

Many cancer drug approvals based on indirect measures, study finds.

The efficacy of newly-approved cancer drugs has been called into question due to limited or no gains in survival and quality of life for patients, according to a new study published by The BMJ.

The authors found that many novel cancer drugs received approval based on indirect measures, which may not be able to predict whether patients will live longer or have a better quality of life. These findings may result in questions about new drug approval standards, according to the study.

"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,” the authors wrote.

Included in the study were cancer drugs approved by the European Medicines Agency (EMA) between 2009 and 2013.

Of the 68 cancer indications approved, 57% were authorized based on a surrogate endpoint. These approvals were found to lack evidence that they improved survival or improved quality of life, according to the study.

After 5 years, only 8 additional drugs showed a survival benefit or gains in quality of life.

Out of the 68 indications granted during the 4-year period, only 35 were found to improve survival or quality of life, while the benefits of 33 drugs related to survival and quality of life were uncertain, according to the study.

While the authors noted there can be some limitations to their study design, they believe regulatory standards may be failing to meet the needs of patients, providers, and clinicians, according to the study.

In a linked editorial, Vinay Prasad, MD, MPH, said that "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.”

Dr Prasad added that using surrogate endpoints should not be the norm and only acceptable on occasion.

"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,” Dr Prasad wrote.

Both the FDA and EMA are looking to change their regulatory processes to combat high drug costs.

In an accompanying feature, Debra Cohen, MBChB, associate editor of The BMJ, said that there are methodological issues related to study design, analysis, and reporting.

"The fact that so many of the new drugs on the market lack good evidence that they improve patient outcomes puts governments in a difficult position when it comes to deciding which treatments to fund," Dr Cohen wrote. "But regulatory sanctioning of a comparator that lacks robust evidence of efficacy, means the cycle of weak evidence and uncertainty continues."