Modern Adaptive Randomized Clinical Trials: Statistical and Practical Aspects

摘要

In the last few years, many trials have failed to achieve their objectives, with even the most promising compounds not reaching the shelves, causing an enormous waste of investment and potentially missed health opportunities for many patients. Many have started to blame the 'gold standard' randomized clinical trial and its rigid protocol. It did not come as a surprise that, admidst these criticisms, a more flexible design, the adaptive randomized trial, has gained momentum. Its undoubtedly promising features, like the dynamic allocation of patients to treatment with higher probabilities of response and the better statistical efficiencies, sound like a breath of fresh air for drug developers.