"Considering that many new targeted agents have fewer safety issues than the older cytotoxic (cell killing) treatments, we feel that the risk of accepting a few therapies that offer no benefit, but with a very low chance of a truly poorer outcome, for a greater long-term benefit at the end seems reasonable. The possibility of a small number of backward steps in the series of trials should not lead us to discard the whole process. The classical, large sample-size clinical trials are designed to avoid wrong decisions, but take a very long time to reach a definitive result when the disease is rare."
In rare diseases, the ability to pursue many of the promising agents available for clinical testing is hindered by the need to invest much effort in a single direction by running a large trial over many years, the researchers say. However, they believe that statisticians and regulators may be more difficult to convince of the need for change. "The culture within these groups is very risk-averse," said Dr. Le Deley. "Their conservative approach is reasonable; the higher the strength of evidence, the lower the risk of wrong decision. But in rare diseases, which could include many small subsets of cancers, this approach is counterproductive as soon as many new agents become available for clinical testing. We need a more flexible process to cater for this situation."
Dr Le Deley makes it clear that her recommendation to decrease the sample size and relax the decision criteria makes sense only if there is no way to increase the trial accrual rate, and thus that the proposal should not be taken as an encouragement to abandon collaborative projects with potential for broad participation. The traditional, large-scale clinical trial will still be needed, but smaller, targ
|Contact: Mary Rice|
ECCO-the European CanCer Organisation