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Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis

Decisions about health interventions are often made using limited evidence. Mathematical models used to inform such decisions often include uncertainty analysis to account for the effect of uncertainty in the current evidence base on decision‐relevant quantities. However, current uncertainty quantif...

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Autor principal: Iskandar, Rowan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9290849/
https://www.ncbi.nlm.nih.gov/pubmed/34528265
http://dx.doi.org/10.1002/sim.9195
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author Iskandar, Rowan
author_facet Iskandar, Rowan
author_sort Iskandar, Rowan
collection PubMed
description Decisions about health interventions are often made using limited evidence. Mathematical models used to inform such decisions often include uncertainty analysis to account for the effect of uncertainty in the current evidence base on decision‐relevant quantities. However, current uncertainty quantification methodologies, including probabilistic sensitivity analysis (PSA), require modelers to specify a precise probability distribution to represent the uncertainty of a model parameter. This study introduces a novel approach for representing and propagating parameter uncertainty, probability bounds analysis (PBA), where the uncertainty about the unknown probability distribution of a model parameter is expressed in terms of an interval bounded by lower and upper bounds on the unknown cumulative distribution function (p‐box) and without assuming a particular form of the distribution function. We give the formulas of the p‐boxes for common situations (given combinations of data on minimum, maximum, median, mean, or standard deviation), describe an approach to propagate p‐boxes into a black‐box mathematical model, and introduce an approach for decision‐making based on the results of PBA. We demonstrate the characteristics and utility of PBA vs PSA using two case studies. In sum, this study provides modelers with practical tools to conduct parameter uncertainty quantification given the constraints of available data and with the fewest assumptions.
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spelling pubmed-92908492022-07-20 Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis Iskandar, Rowan Stat Med Research Articles Decisions about health interventions are often made using limited evidence. Mathematical models used to inform such decisions often include uncertainty analysis to account for the effect of uncertainty in the current evidence base on decision‐relevant quantities. However, current uncertainty quantification methodologies, including probabilistic sensitivity analysis (PSA), require modelers to specify a precise probability distribution to represent the uncertainty of a model parameter. This study introduces a novel approach for representing and propagating parameter uncertainty, probability bounds analysis (PBA), where the uncertainty about the unknown probability distribution of a model parameter is expressed in terms of an interval bounded by lower and upper bounds on the unknown cumulative distribution function (p‐box) and without assuming a particular form of the distribution function. We give the formulas of the p‐boxes for common situations (given combinations of data on minimum, maximum, median, mean, or standard deviation), describe an approach to propagate p‐boxes into a black‐box mathematical model, and introduce an approach for decision‐making based on the results of PBA. We demonstrate the characteristics and utility of PBA vs PSA using two case studies. In sum, this study provides modelers with practical tools to conduct parameter uncertainty quantification given the constraints of available data and with the fewest assumptions. John Wiley and Sons Inc. 2021-09-15 2021-12-20 /pmc/articles/PMC9290849/ /pubmed/34528265 http://dx.doi.org/10.1002/sim.9195 Text en © 2021 The Authors. Statistics in Medicine published by John Wiley & Sons Ltd. https://creativecommons.org/licenses/by/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Research Articles
Iskandar, Rowan
Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis
title Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis
title_full Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis
title_fullStr Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis
title_full_unstemmed Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis
title_short Probability bound analysis: A novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis
title_sort probability bound analysis: a novel approach for quantifying parameter uncertainty in decision‐analytic modeling and cost‐effectiveness analysis
topic Research Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9290849/
https://www.ncbi.nlm.nih.gov/pubmed/34528265
http://dx.doi.org/10.1002/sim.9195
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