Estimands in clinical drug development: from design to regulatory assessment

Marian Mitroiu

doi:10.33540/723

Estimands in clinical drug development: from design to regulatory assessment

Marian Mitroiu

Julius Center, dept. Data Science & Biostatistics

Research output: Thesis › Doctoral thesis 1 (Research UU / Graduation UU)

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Abstract

In this thesis, I investigated the (implicit) use of estimands in the pre-estimand era in clinical trials for regulatory evaluation and approval to understand what estimands may correspond to efficacy investigations and common efficacy analyses. Additionally, I developed data-generating models (DGMs) that take into account the association between clinical outcomes and intercurrent events.

Original language	English
Awarding Institution	University Medical Center (UMC) Utrecht
Supervisors/Advisors	Roes, Kit C.B., Primary supervisor Teerenstra, S., Co-supervisor, External person Oude Rengerink, K, Co-supervisor
Award date	12 Dec 2022
Publisher	Utrecht University
Print ISBNs	978-90-393-7528-0
DOIs	https://doi.org/10.33540/723
Publication status	Published - 12 Dec 2022

Keywords

Estimand
Intention-to-treat
Per protoco
Intercurrent event
Study withdrawal
Treatment discontinuation
Missing data
Sensitivity analysis
Data-generating models
Simulation study

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10.33540/723

proefschrift - 63886b7f9ebfbFinal published version, 10 MB

https://dspace.library.uu.nl/handle/1874/424348

Cite this

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title = "Estimands in clinical drug development: from design to regulatory assessment",

abstract = "In this thesis, I investigated the (implicit) use of estimands in the pre-estimand era in clinical trials for regulatory evaluation and approval to understand what estimands may correspond to efficacy investigations and common efficacy analyses. Additionally, I developed data-generating models (DGMs) that take into account the association between clinical outcomes and intercurrent events.",

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AB - In this thesis, I investigated the (implicit) use of estimands in the pre-estimand era in clinical trials for regulatory evaluation and approval to understand what estimands may correspond to efficacy investigations and common efficacy analyses. Additionally, I developed data-generating models (DGMs) that take into account the association between clinical outcomes and intercurrent events.

KW - Estimand

KW - Intention-to-treat

KW - Per protoco

KW - Intercurrent event

KW - Study withdrawal

KW - Treatment discontinuation

KW - Missing data

KW - Sensitivity analysis

KW - Data-generating models

KW - Simulation study

U2 - 10.33540/723

DO - 10.33540/723

M3 - Doctoral thesis 1 (Research UU / Graduation UU)

SN - 978-90-393-7528-0

PB - Utrecht University

ER -

Estimands in clinical drug development: from design to regulatory assessment

Abstract

Keywords

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