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For this reason, in earlier phases of drug development and rare diseases with limited sample sizes, there is a tendency to omit\u00a0control groups and to replace them with historical information for perceived feasibility arguments. This has significant implications for interpretation of trial results. We present headline results from a trial in stem cell transplantation, where fludarabine and clofarabine were investigated for induction chemotherapy in high\u2010risk acute myeloid leukaemia or advanced myelodysplastic syndrome. The trial was initially designed as single\u2010arm trial (SAT) and is discussed here from the planning and assessment perspective. Changes in the treatment setting or patient selection may lead to \u2018promising results\u2019 in SATs, which are difficult to identify and may raise unwarranted hopes which cannot be confirmed in a subsequent randomized trial. RCTs should be used to achieve a straightforward interpretation of the results, even if the sample size is small. 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