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Randomized clinical trials remain the gold standard to establish efficacy and safety of new treatments. In acute myeloid leukemia, large trials have been associated with gradual improvement in outcome over 2 decades in younger patients without major differences emerging between treatments. By contrast, in older patients, improvement has been minimal, which justifies a new approach to identifying effective treatments. Given the urgent unmet need, and with the emergence of several novel agents or combinations that are likely to be expensive, large benefits are probably required to change clinical practice. To address this issue, we have evolved a "Pick a Winner" randomized progressive design with a rolling incorporation of novel treatments (drug X), which has been tested in older patients with acute myeloid leukemia. The rationale, operational characteristics, and initial experience of such an approach in the context of the United Kingdom National Cancer Research Institute AML16 trial are presented.

Original publication




Journal article



Publication Date





2389 - 2394


Adenine Nucleotides, Aged, Aminoglycosides, Antibodies, Monoclonal, Antibodies, Monoclonal, Humanized, Antineoplastic Combined Chemotherapy Protocols, Arabinonucleosides, Arsenic Trioxide, Arsenicals, Clinical Trials, Phase III as Topic, Clofarabine, Cost-Benefit Analysis, Cytarabine, Cytosine, Drug Discovery, Early Termination of Clinical Trials, Follow-Up Studies, Humans, Leukemia, Myeloid, Acute, Middle Aged, Multicenter Studies as Topic, Oxides, Patient Selection, Quality-Adjusted Life Years, Quinolones, Randomized Controlled Trials as Topic, Research Design, Sample Size, Software Design, Treatment Outcome, United Kingdom