The potential of the estimands framework for clinical pharmacology trials: Some discussion points

Ring, Arne; Wolfsegger, Martin J.

doi:10.1111/bcp.14233

Cited by 4 publications

(1 citation statement)

References 11 publications

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“…The framework defines estimands as ‘a precise description of the treatment effect reflecting the clinical question posed by the trial objective.’7 Recent publications discussing how this framework can be applied in oncology,8 9 palliative care,10 clinical pharmacology11 and substance use disorder12 indicate that this development is a consideration for many different health contexts in which RCTs are used.…”

Section: Introductionmentioning

confidence: 99%

Missing data in palliative care research: estimands and estimators

Roydhouse

Floden

Braat

et al. 2022

BMJ Support Palliat Care

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There are several methodological challenges when conducting randomised controlled trials in palliative care. These include worsening function and high mortality, leading to treatment discontinuation, some of which will be unrelated to the intervention being evaluated.Recently, a new framework for handling postrandomisation events, such as attrition, has been released. This framework aims to align trial objectives, design, conduct and analysis by clarifying what and how to estimate treatment effects in the presence of data affected by postrandomisation events.The purpose of this paper is to introduce palliative care researchers to this framework and how it can guide trial design, and efficacy and safety analysis in a palliative care context where individual withdrawal from the trial is common.In this paper, we describe the estimand framework and the background for it. We also consider postrandomisation events that are frequently encountered in palliative care trials and how these might affect objectives of interest. We then construct efficacy and safety estimands for a trial in palliative care. Better trial design and alignment of objectives with analysis can improve our understanding of what treatments do and do not work in palliative care.

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Section: Introductionmentioning

confidence: 99%

Missing data in palliative care research: estimands and estimators

Roydhouse

Floden

Braat

et al. 2022

BMJ Support Palliat Care

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Current developments of the estimand concept

Fierenz,

Zapf

2024

Pharmaceutical Statistics

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Since the introduction of the estimand in therapeutical studies, several adaptions have been developed. This short article highlights the important aspects of the estimand concept. A literature research was conducted to identify different extensions to this framework. Different modified strategies for intercurrent events are presented, as well as examples of methods to implement the estimand in clinical studies. The article reflects that the estimand is an ongoing research field with further exploration.

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A first step towards best practice recommendations for the design and statistical analyses of pragmatic clinical trials: A modified Delphi approach

Palin

Staa

Steels

et al. 2022

Brit J Clinical Pharma

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Aim Pragmatic clinical trials (PCTs) are randomized trials implemented through routine clinical practice, where design parameters of traditional randomized controlled trials are modified to increase generalizability. However, this may introduce statistical challenges. We aimed to identify these challenges and discuss possible solutions leading to best practice recommendations for the design and analysis of PCTs. Methods A modified Delphi method was used to reach consensus among a panel of 11 experts in clinical trials and statistics. Statistical issues were identified in a focused literature review and aggregated with insights and possible solutions from experts collected through a series of survey iterations. Issues were ranked according to their importance. Results Twenty‐seven articles were included and combined with experts' insight to generate a list of issues categorized into participants, recruiting sites, randomization, blinding and intervention, outcome (selection and measurement) and data analysis. Consensus was reached about the most important issues: risk of participants' attrition, heterogeneity of “usual care” across sites, absence of blinding, use of a subjective endpoint and data analysis aligned with the trial estimand. Potential issues should be anticipated and preferably be addressed in the trial protocol. The experts provided solutions regarding data collection and data analysis, which were considered of equal importance. Discussion A set of important statistical issues in PCTs was identified and approaches were suggested to anticipate and/or minimize these through data analysis. Any impact of choosing a pragmatic design feature should be gauged in the light of the trial estimand.

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The potential of the estimands framework for clinical pharmacology trials: Some discussion points

Cited by 4 publications

References 11 publications

Missing data in palliative care research: estimands and estimators

Missing data in palliative care research: estimands and estimators

Current developments of the estimand concept

A first step towards best practice recommendations for the design and statistical analyses of pragmatic clinical trials: A modified Delphi approach

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