Data collection for the sake of data collection 171 8♦ analysis of that data, should be seen. Even more provoking is the fact that some European Union member states simply do not allow data collection within compassionate use programs.50 Fourth, the authors state that one might avoid using (placebo) controls, i.e.,‘putting a real patient at risk’, by using statistical techniques. Advanced statistical techniques may attenuate biases in analyses of observational (expanded access) data. However, their results are not as robust as those of randomized controlled trials – and robust results are needed in times of crisis. Statistical methods applied to observational data cannot fully replace randomized controlled trials.92 Furthermore, a majority of drugs still fail to demonstrate effect in randomized controlled trials, hence, patients might have been better off being randomized to a control group. We support the authors in their call for regulatory guidance and alignment as it comes to the opportunity expanded access programs bring to collect real-world data. We should not fail to learn from expanded access data, but by failing to randomize patients in the first place, we might learn nothing at all.
RkJQdWJsaXNoZXIy MTk4NDMw