Jan R. Boehnke

Alt: a woman in a red dress (Sandra Oh) and a man in a tuxedo (Andy Samberg) standing next to each other, shouting something, with the caption reading "Flu Shots!!!"

YouTube video by European Association of Science Editors (EASE)

YouTube video by European Association of Science Editors (EASE)

Nur 70 Prozent der größten Kliniken bieten kostenloses WLAN an. Ein knappes Drittel nimmt Geld fürs Surfen am Krankenbett – bis zu 5 Euro am Tag oder 30 Euro pro Woche. Wo es für Patienten am teuers...

Nur 70 Prozent der größten Kliniken bieten kostenloses WLAN an. Ein knappes Drittel nimmt Geld fürs Surfen am Krankenbett – bis zu 5 Euro am Tag oder 30 Euro pro Woche. Wo es für Patienten am teuers...

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Background Published in 2019, a new addendum to the ICH E9 guideline presents the estimand framework as a systematic approach to ensure alignment among clinical trial objectives, trial execution/conduct, statistical analyses, and interpretation of results. The use of the estimand framework for describing clinical trial objectives has yet to be extensively considered in the context of patient-reported outcomes (PROs). We discuss the application of the estimand framework to PRO objectives when designing clinical trials in the future, with a focus on PRO outcomes in oncology trial settings as our example. Main We describe the components of an estimand and take a naïve PRO trial objective to illustrate how to apply attributes described in the estimand framework to inform construction of a detailed clinical trial objective and its related estimand. We discuss identifying potential post-randomization events that alter the interpretation of the endpoint or render its observation impossible (also defined as intercurrent events) in the context of PRO endpoints, and the implications of how to handle intercurrent events in the construction of the PRO objective. Using a simple objective statement, “What is the effect of treatment X on patient’s quality of life?”, we build up an example estimand statement and also use a previously published phase III oncology clinical trial to illustrate how an estimand for a PRO objective could have been written to align to the estimate framework. Conclusion The use of the estimand framework, as described in the new ICH E9 (R1) addendum guideline will become a key common framework for developing clinical trial objectives for evaluating effects of treatment. In the context of considering PROs, the framework provides an opportunity to more precisely specify and build the rationale for patient-focused objectives. This will help to ensure that clinical trials used for registration are designed and analysed appropriately, enabling all stakeholders to accurately interpret conclusions about the treatment effects for patient-focused outcomes.

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. Previous reviews of publications and published protocols have found that few trials explicitly defined the primary estimand. To obtain a more complete picture of how the use of estimands has changed over time, whether trials are using estimands correctly (i.e. correctly defining the five attributes of an estimand), and which strategies are being used to handle intercurrent events, we obtained access to an extensive database of original research protocols (n = 29,212) submitted to the United Kingdom’s Health Research Authority, which oversees ethical review of clinical trials. Methods Protocols were eligible for review if they included the term ‘estimand’ and attempted to define at least one attribute of the primary estimand. For eligible protocols, we extracted information on trial characteristics such as whether the trial was randomized and the therapeutic area, as well as whether the estimand attributes used for the primary outcome were correctly defined, and which strategies were used to handle intercurrent events. Results We found that the number of protocols defining a primary estimand increased starkly with publication of the ICH E9(R1) Addendum (approximately 3 protocols/year pre-ICH E9(R1) Addendum vs. 18 protocols / year during the consultation period vs. 23 protocols in the year following the adoption of the ICH E9(R1) Addendum). However, the description of the primary estimand was suboptimal; many protocols failed to mention specific attributes (such as population or treatment conditions) in the estimand description, and many protocols incorrectly defined estimand attributes (e.g. by describing the estimand population based on their analysis population). Conclusions Although release of the ICH E9(R1) Addendum has dramatically increased the use of estimands in clinical trials, their reporting is suboptimal. There is still work to be done to ensure estimands reach their full potential in bringing clarity and focus to research questions.

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Abstract. In recent years, there has been considerable policy and academic interest in the existence of ethnic inequalities in the Criminal Justice System.

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This paper analyses the changing geographies of ethnic diversity and segregation in England and Wales using Census data covering the last 30 years (1991–2021). Residential segregation of all ethnic g....

PDF | This was an invited presentation to delineate five challenges and a workflow to develop common scoring algorithms between mental health assessment... | Find, read and cite all the research you n...

Objective: Although evidence for benefits of psychotherapy is substantial, less is known about potential harm. Therefore, we systematically summarized randomized controlled trials (RCTs) to compile...