Are current cancer drug trials fit for purpose?

New analysis published in the British Medical Journal has found that almost half of pivotal trials forming the basis of European Medicines Agency approvals of new cancer drugs were at high risk of bias based on their design, conduct or analysis. What exactly are the problems with cancer trial design and administration, and what needs to change?

GlobalData’s Verdict pharmaceutical technology journalist Chris Lo writes: “Surrogate measures such as progression-free survival and treatment response rates have become increasingly prominent over the gold standard endpoint of overall survival (OS) over the last two decades, and it appears that this trend is driving a lack of clarity around the actual clinical benefits of new cancer drugs coming on to the market.

Study co-author Dr Huseyin Naci, assistant professor of health policy at the London School of Economics tells GlobalData: “In this paper, we were interested in using an established, robust tool (the Cochrane risk-of-bias tool) to look into the validity of these trials. While we know randomised controlled trials are the gold standard, they still can have potential problems. We wanted to evaluate those issues.