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Category: Project

Description: Background: Methodological limitations afflict a significant number of oncology and hematology 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 drug to reach the market, other assessment tools, such as those developed by professional societies – ESMO-MCBS and ASCO Value Framework – do not address these important shortcomings either. Methods: We propose creating a novel framework which scope is to assess registration cancer clinical trials in hematology and oncology (randomized or single arm) – i.e. 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) pre-registration 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 hematologists and oncologists involved in clinical trials; and 7) finalizing the first version of framework. Results: not applicable. Conclusions: Our proposal emerged in response to the lack of consideration for key limitations in current trial assessments. Its goal is to create a framework specifically designed to assess single trials leading to marketing authorization in the field of oncology and hematogy.