Aligning Estimation with the Clinical Question of Interest

Overview

James Bell and Sue McKendrick will present on behalf of the Estimands Implementation Working Group, sponsored by EFPIA and EFSPI, and hosted by PSI.

This event will focus on the estimation aspects of the estimand framework. Having clearly defined the clinical question of interest through the estimand, it is equally important to ensure that the estimation approach is appropriately aligned with that question. In particular, how do different strategies for handling intercurrent events influence the choice of methods used to estimate the treatment effect?

The webinar will explore the challenges that arise when data are missing, particularly when missingness occurs disproportionately following treatment discontinuation or other intercurrent events. We will consider how different intercurrent event handling strategies affect the analysis, the assumptions required, and ultimately the interpretation of the estimated treatment effect.

The session will draw on insights from the work of EIWG estimation subteam, including simulation-based investigations of estimation methods under different intercurrent event strategies.

Reference:

Bell, J., Drury, T., Mutze, T., Pipper, C.B., Guizzaro, L., Mitroiu, M., Rantell, K.R., Wolbers, M., & Wright, D. (2024). Estimation methods for estimands using the treatment policy strategy: a simulation study based on the PIONEER 1 Trial. Pharmaceutical Statistics, 24(2). https://doi.org/10.1002/pst.2472

Speaker details

Speaker

Biography

James Bell, Contract Methodology Statistician, Elderbrook Solutions

James is a contract methodology statistician working for Elderbrook Solutions and chairs the estimation subteam of the Estimands Implementation Working Group (EIWG). His main areas of research are missing data handling, estimands and holistic approaches to clinical trial design.

Sue McKendrick, Statistical Science Director, Biostatistics - PPD, Thermo Fisher Scientific

Sue has over 30 years’ experience of statistical consultancy across drug development including collaborating with clinical colleagues on developing estimands in protocols. Sue chairs the Training Subteam of EIWG.

David Wright_cropped

David Wright, Head of Statistical Innovation, Astrazeneca

David Wright is Head of Statistical Innovation at AstraZeneca, previously worked for the MHRA and led the revision of the CHMP guideline on missing data in confirmatory clinical trials. An active member of EIWG.

Judith Anzures-Cabrera, Statistical Science Lead, Astellas

Judith is Statistical Science Lead at Astellas and has been actively involved in shaping the Estimands Academy for Trial Teams webinar series.

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