• Timothée Olivier, Alyson Haslam, Patricia Burke, Isabelle Boutron, Florian Naudet, IoannidisJohn P AJPA0000-0003-3118-6859Meta-Research Innovation Center at Stanford (METRICS), Stanford University, Stanford, California, USA.Department of Medicine, Stanford University School of Medicine, Stanford, California, USA.Department of Epidemiolo, and Vinay Prasad.
• Department of Oncology, Geneva University Hospital, Geneva, Switzerland.
• Eur. J. Clin. Invest. 2024 Oct 1; 54 (10): e14267e14267.

BackgroundMethodological limitations affect a significant number of oncology and haematology trials, raising concerns about the applicability of their results. For example, a suboptimal control arm or limited access to best care upon progression may skew the trial results toward a benefit in the experimental arm. Beyond the fact that such limitations do not prevent drugs reaching the market, other assessment tools, such as those developed by professional societies-ESMO-MCBS and ASCO Value Framework-do not integrate these important shortcomings.MethodsWe propose creating a novel framework with the scope of assessing registration cancer clinical trials in haematology and oncology (randomized or single arm)-that is trials leading to a marketing authorization. The main steps of the methods are (1) assembling a scientific board; (2) defining the scope, goal and methods through pre-specified, pre-registered and protocolized methodology; (3) preregistration of the protocol; (4) conducting a scoping review of limitations and biases affecting oncology trials and assessing existing scores or methods; (5) developing a list of features to be included and assessed within the framework; (6) assessing each feature through a questionnaire sent to highly cited haematologists and oncologists involved in clinical trials; and (7) finalizing the first version of framework.ResultsNot applicable.ConclusionsOur proposal emerged in response to the lack of consideration for key limitations in current trial assessments. The goal is to create a framework specifically designed to assess single trials leading to marketing authorization in the field of oncology and haematogy.© 2024 The Author(s). European Journal of Clinical Investigation published by John Wiley & Sons Ltd on behalf of Stichting European Society for Clinical Investigation Journal Foundation.

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