Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease

Abstract In this study, we explored the feasibility of using real-world data (RWD) from a large clinical research network to simulate real-world clinical trials of Alzheimer’s disease (AD). The target trial (i.e., NCT00478205) is a Phase III double-blind, parallel-group trial that compared the 23 mg...

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Autores principales:	Zhaoyi Chen, Hansi Zhang, Yi Guo, Thomas J. George, Mattia Prosperi, William R. Hogan, Zhe He, Elizabeth A. Shenkman, Fei Wang, Jiang Bian
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Computer applications to medicine. Medical informatics R858-859.7
Acceso en línea:	https://doaj.org/article/612dc9dac3b54a81922e281028b3e3fd
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spelling	oai:doaj.org-article:612dc9dac3b54a81922e281028b3e3fd2021-12-02T16:50:22ZExploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease10.1038/s41746-021-00452-12398-6352https://doaj.org/article/612dc9dac3b54a81922e281028b3e3fd2021-05-01T00:00:00Zhttps://doi.org/10.1038/s41746-021-00452-1https://doaj.org/toc/2398-6352Abstract In this study, we explored the feasibility of using real-world data (RWD) from a large clinical research network to simulate real-world clinical trials of Alzheimer’s disease (AD). The target trial (i.e., NCT00478205) is a Phase III double-blind, parallel-group trial that compared the 23 mg donepezil sustained release with the 10 mg donepezil immediate release formulation in patients with moderate to severe AD. We followed the target trial’s study protocol to identify the study population, treatment regimen assignments and outcome assessments, and to set up a number of different simulation scenarios and parameters. We considered two main scenarios: (1) a one-arm simulation: simulating a standard-of-care (SOC) arm that can serve as an external control arm; and (2) a two-arm simulation: simulating both intervention and control arms with proper patient matching algorithms for comparative effectiveness analysis. In the two-arm simulation scenario, we used propensity score matching controlling for baseline characteristics to simulate the randomization process. In the two-arm simulation, higher serious adverse event (SAE) rates were observed in the simulated trials than the rates reported in original trial, and a higher SAE rate was observed in the 23 mg arm than in the 10 mg SOC arm. In the one-arm simulation scenario, similar estimates of SAE rates were observed when proportional sampling was used to control demographic variables. In conclusion, trial simulation using RWD is feasible in this example of AD trial in terms of safety evaluation. Trial simulation using RWD could be a valuable tool for post-market comparative effectiveness studies and for informing future trials’ design. Nevertheless, such an approach may be limited, for example, by the availability of RWD that matches the target trials of interest, and further investigations are warranted.Zhaoyi ChenHansi ZhangYi GuoThomas J. GeorgeMattia ProsperiWilliam R. HoganZhe HeElizabeth A. ShenkmanFei WangJiang BianNature PortfolioarticleComputer applications to medicine. Medical informaticsR858-859.7ENnpj Digital Medicine, Vol 4, Iss 1, Pp 1-9 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Computer applications to medicine. Medical informatics R858-859.7
spellingShingle	Computer applications to medicine. Medical informatics R858-859.7 Zhaoyi Chen Hansi Zhang Yi Guo Thomas J. George Mattia Prosperi William R. Hogan Zhe He Elizabeth A. Shenkman Fei Wang Jiang Bian Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease
description	Abstract In this study, we explored the feasibility of using real-world data (RWD) from a large clinical research network to simulate real-world clinical trials of Alzheimer’s disease (AD). The target trial (i.e., NCT00478205) is a Phase III double-blind, parallel-group trial that compared the 23 mg donepezil sustained release with the 10 mg donepezil immediate release formulation in patients with moderate to severe AD. We followed the target trial’s study protocol to identify the study population, treatment regimen assignments and outcome assessments, and to set up a number of different simulation scenarios and parameters. We considered two main scenarios: (1) a one-arm simulation: simulating a standard-of-care (SOC) arm that can serve as an external control arm; and (2) a two-arm simulation: simulating both intervention and control arms with proper patient matching algorithms for comparative effectiveness analysis. In the two-arm simulation scenario, we used propensity score matching controlling for baseline characteristics to simulate the randomization process. In the two-arm simulation, higher serious adverse event (SAE) rates were observed in the simulated trials than the rates reported in original trial, and a higher SAE rate was observed in the 23 mg arm than in the 10 mg SOC arm. In the one-arm simulation scenario, similar estimates of SAE rates were observed when proportional sampling was used to control demographic variables. In conclusion, trial simulation using RWD is feasible in this example of AD trial in terms of safety evaluation. Trial simulation using RWD could be a valuable tool for post-market comparative effectiveness studies and for informing future trials’ design. Nevertheless, such an approach may be limited, for example, by the availability of RWD that matches the target trials of interest, and further investigations are warranted.
format	article
author	Zhaoyi Chen Hansi Zhang Yi Guo Thomas J. George Mattia Prosperi William R. Hogan Zhe He Elizabeth A. Shenkman Fei Wang Jiang Bian
author_facet	Zhaoyi Chen Hansi Zhang Yi Guo Thomas J. George Mattia Prosperi William R. Hogan Zhe He Elizabeth A. Shenkman Fei Wang Jiang Bian
author_sort	Zhaoyi Chen
title	Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease
title_short	Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease
title_full	Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease
title_fullStr	Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease
title_full_unstemmed	Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease
title_sort	exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of alzheimer’s disease
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/612dc9dac3b54a81922e281028b3e3fd
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Exploring the feasibility of using real-world data from a large clinical data research network to simulate clinical trials of Alzheimer’s disease

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