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Activity Number:
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77
- Contributed Poster Presentations: Biopharmaceutical Section
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Type:
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Contributed
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Date/Time:
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Monday, August 3, 2020 : 10:00 AM to 2:00 PM
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Sponsor:
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Biopharmaceutical Section
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Abstract #312289
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Title:
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Quantitative Determination of Threshold in Clinical Outcome Assessments (COA) for a Clinically Meaningful Change
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Author(s):
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Wenting Cheng* and Zijuan Chen and Kun Chen and Li Zhu and Chunlei Ke
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Companies:
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Biogen and Texas A&M University and Biogen and Biogen and Biogen
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Keywords:
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drug development;
endpoint development;
clinical outcome assessment;
neurological disease;
threshold determination
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Abstract:
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One of the most important questions in drug development is that if the effects seen in clinical trials can be interpreted and communicated as a clear clinical benefit that is meaningful to patients. Statistical significance can sometimes be achieved for small between-group mean differences; however, statistical significance alone does not indicate whether an individual patient has experienced a meaningful clinical benefit. Traditional approaches to identifying the threshold for a clinically meaningful change are a combination of qualitative and quantitative methods called psychometric analysis but there is no systematic statistical approach available for determining the threshold. We will develop a novel statistical path to set the threshold for a clinically meaningful change, based on the combination of statistical methodology and the basic idea of psychometric analysis. We propose three threshold determination approaches by linking patient reported outcomes and COA: latent variable approach, Rasch model and mixture model. We will illustrate our methodology through simulations and a motivating case study in multiple sclerosis clinical trials.
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Authors who are presenting talks have a * after their name.
