Category Ranking

98%

Total Visits

921

Avg Visit Duration

2 minutes

Citations

20

Article Abstract

The ICH E9(R1) Addendum on Estimands and Sensitivity Analysis provides a framework for defining the treatment effect a trial intends to estimate-the estimand. The addendum is widely adopted in pharmaceutical research. However, it remains underutilized in trials investigating internet-based interventions (IBIs). This manuscript introduces the addendum to IBI researchers. It concludes that estimands are essential to improve the interpretability, relevance, and validity of effect estimates derived in IBI trials.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC12368201PMC
http://dx.doi.org/10.1038/s41746-025-01936-0DOI Listing

Publication Analysis

Top Keywords

ich estimand
4
estimand framework
4
framework improve
4
improve interpretation
4
interpretation treatment
4
treatment effects
4
effects internet
4
internet interventions
4
interventions ich
4
ich e9r1
4

Similar Publications

Since the first decentralized clinical trial (DCT) was conducted in 2011, there has been an increased usage of DCT due to its benefits of patient-centricity and generalizability of findings. This trend was further expedited by the global COVID-19 pandemic. We identified 23 case studies across various therapeutic areas and grouped them into different categories according to their purposes-by necessity, for operational benefits, to address unique research questions, to validate innovative digital endpoints, or to validate decentralization as a clinical research platform.

View Article and Find Full Text PDF

The estimand framework as outlined in ICH E9(R1) has been extensively discussed and implemented in clinical trials of therapeutic products. However, there is limited literature on the application of the framework in preventive vaccine trials, which has many unique characteristics, including emphasis on estimating the per-protocol or "biological" effect. We provide a comprehensive review of the application of the framework to preventive vaccine trials evaluating clinical outcome and immunogenicity, focusing on commonly encountered intercurrent events including but not limited to: noncompliance with vaccination schedule and blood sampling window, infection not meeting protocol definition, death, and use of prohibited products.

View Article and Find Full Text PDF

Background: The use of estimands in clinical trials was formalised with the adoption of the final International Conference on Harmonisation E9 Addendum on Estimands and Sensitivity Analysis in Clinical Trials (ICH E9(R1) Addendum) in November 2019. The declared objective of the ICH E9(R1) Addendum is to bring clarity and transparency to the research question of interest. For this to be achieved, the estimand must be described in accordance with the requirements of the ICH E9(R1) Addendum so that the target treatment effect is clear to all stakeholders.

View Article and Find Full Text PDF

The creation of the ICH E9 (R1) estimands framework has led to more precise specification of the treatment effects of interest in the design and statistical analysis of clinical trials. However, it is unclear how the new framework relates to causal inference, as both approaches appear to define what is being estimated and have a quantity labeled an estimand. Using illustrative examples, we show that both approaches can be used to define a population-based summary of an effect on an outcome for a specified population and highlight the similarities and differences between these approaches.

View Article and Find Full Text PDF

The ICH E9(R1) Addendum on Estimands and Sensitivity Analysis provides a framework for defining the treatment effect a trial intends to estimate-the estimand. The addendum is widely adopted in pharmaceutical research. However, it remains underutilized in trials investigating internet-based interventions (IBIs).

View Article and Find Full Text PDF