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Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation

Real‐world data and patient‐level data from completed randomized controlled trials are becoming available for secondary analysis on an unprecedented scale. A range of novel methodologies and study designs have been proposed for their analysis or combination. However, to make novel analytical methods...

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Autores principales: Eichler, Hans‐Georg, Koenig, Franz, Arlett, Peter, Enzmann, Harald, Humphreys, Anthony, Pétavy, Frank, Schwarzer‐Daum, Brigitte, Sepodes, Bruno, Vamvakas, Spiros, Rasi, Guido
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7158212/
https://www.ncbi.nlm.nih.gov/pubmed/31574163
http://dx.doi.org/10.1002/cpt.1638
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author Eichler, Hans‐Georg
Koenig, Franz
Arlett, Peter
Enzmann, Harald
Humphreys, Anthony
Pétavy, Frank
Schwarzer‐Daum, Brigitte
Sepodes, Bruno
Vamvakas, Spiros
Rasi, Guido
author_facet Eichler, Hans‐Georg
Koenig, Franz
Arlett, Peter
Enzmann, Harald
Humphreys, Anthony
Pétavy, Frank
Schwarzer‐Daum, Brigitte
Sepodes, Bruno
Vamvakas, Spiros
Rasi, Guido
author_sort Eichler, Hans‐Georg
collection PubMed
description Real‐world data and patient‐level data from completed randomized controlled trials are becoming available for secondary analysis on an unprecedented scale. A range of novel methodologies and study designs have been proposed for their analysis or combination. However, to make novel analytical methods acceptable for regulators and other decision makers will require their testing and validation in broadly the same way one would evaluate a new drug: prospectively, well‐controlled, and according to a pre‐agreed plan. From a European regulators' perspective, the established methods qualification advice procedure with active participation of patient groups and other decision makers is an efficient and transparent platform for the development and validation of novel study designs.
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spelling pubmed-71582122020-04-20 Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation Eichler, Hans‐Georg Koenig, Franz Arlett, Peter Enzmann, Harald Humphreys, Anthony Pétavy, Frank Schwarzer‐Daum, Brigitte Sepodes, Bruno Vamvakas, Spiros Rasi, Guido Clin Pharmacol Ther Reviews Real‐world data and patient‐level data from completed randomized controlled trials are becoming available for secondary analysis on an unprecedented scale. A range of novel methodologies and study designs have been proposed for their analysis or combination. However, to make novel analytical methods acceptable for regulators and other decision makers will require their testing and validation in broadly the same way one would evaluate a new drug: prospectively, well‐controlled, and according to a pre‐agreed plan. From a European regulators' perspective, the established methods qualification advice procedure with active participation of patient groups and other decision makers is an efficient and transparent platform for the development and validation of novel study designs. John Wiley and Sons Inc. 2019-10-01 2020-04 /pmc/articles/PMC7158212/ /pubmed/31574163 http://dx.doi.org/10.1002/cpt.1638 Text en © 2019 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals, Inc. on behalf of American Society for Clinical Pharmacology and Therapeutics. This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
spellingShingle Reviews
Eichler, Hans‐Georg
Koenig, Franz
Arlett, Peter
Enzmann, Harald
Humphreys, Anthony
Pétavy, Frank
Schwarzer‐Daum, Brigitte
Sepodes, Bruno
Vamvakas, Spiros
Rasi, Guido
Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation
title Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation
title_full Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation
title_fullStr Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation
title_full_unstemmed Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation
title_short Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation
title_sort are novel, nonrandomized analytic methods fit for decision making? the need for prospective, controlled, and transparent validation
topic Reviews
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7158212/
https://www.ncbi.nlm.nih.gov/pubmed/31574163
http://dx.doi.org/10.1002/cpt.1638
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