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Phases of methodological research in biostatistics—Building the evidence base for new methods

Heinze, Georg; Boulesteix, Anne‐Laure; Kammer, Michael; Morris, Tim P; White, Ian R; (2023) Phases of methodological research in biostatistics—Building the evidence base for new methods. Biometrical Journal , Article 2200222. 10.1002/bimj.202200222. Green open access

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Abstract

Although new biostatistical methods are published at a very high rate, many of these developments are not trustworthy enough to be adopted by the scientific community. We propose a framework to think about how a piece of methodological work contributes to the evidence base for a method. Similar to the well-known phases of clinical research in drug development, we propose to define four phases of methodological research. These four phases cover (I) proposing a new methodological idea while providing, for example, logical reasoning or proofs, (II) providing empirical evidence, first in a narrow target setting, then (III) in an extended range of settings and for various outcomes, accompanied by appropriate application examples, and (IV) investigations that establish a method as sufficiently well-understood to know when it is preferred over others and when it is not; that is, its pitfalls. We suggest basic definitions of the four phases to provoke thought and discussion rather than devising an unambiguous classification of studies into phases. Too many methodological developments finish before phase III/IV, but we give two examples with references. Our concept rebalances the emphasis to studies in phases III and IV, that is, carefully planned method comparison studies and studies that explore the empirical properties of existing methods in a wider range of problems.

Type: Article
Title: Phases of methodological research in biostatistics—Building the evidence base for new methods
Open access status: An open access version is available from UCL Discovery
DOI: 10.1002/bimj.202200222
Publisher version: https://doi.org/10.1002/bimj.202200222
Language: English
Additional information: © 2023 The Authors. Biometrical Journal published by Wiley-VCH GmbH. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
Keywords: biostatistics, methodological research, reproducibility
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology > MRC Clinical Trials Unit at UCL
URI: https://discovery.ucl.ac.uk/id/eprint/10164401
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