A novel framework to assess haematology and oncology registration trials: The THEOREMM project.

Autor: Olivier T; Department of Oncology, Geneva University Hospital, Geneva, Switzerland., Haslam A; Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USA., Burke P; Patient, Liverpool, UK., Boutron I; Université Paris Cité and Université Sorbonne Paris Nord, Inserm, INRAe, Centre for Research in Epidemiology and Statistics (CRESS), Paris, France.; Centre d'Épidémiologie Clinique, Hôpital Hôtel Dieu, Paris, France.; Cochrane France, Paris, France., Naudet F; Inserm, CIC 1414 (Centre d'Investigation Clinique de Rennes), Rennes 1 University, Rennes, France.; Inserm, Irset (Institut de recherche en santé, environnement et travail), Rennes 1 University, Rennes, France.; Institut Universitaire de France, Paris, France., Ioannidis JPA; Meta-Research Innovation Center at Stanford (METRICS), Stanford University, Stanford, California, USA.; Department of Medicine, Stanford University School of Medicine, Stanford, California, USA.; Department of Epidemiology and Population Health, Stanford University School of Medicine, Stanford, California, USA., Prasad V; Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USA.
Jazyk: angličtina
Zdroj: European journal of clinical investigation [Eur J Clin Invest] 2024 Oct; Vol. 54 (10), pp. e14267. Date of Electronic Publication: 2024 Jun 27.
DOI: 10.1111/eci.14267
Abstrakt: Background: Methodological 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.
Methods: We 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.
Results: Not applicable.
Conclusions: Our 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.)
Databáze: MEDLINE
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