This chapter illustrates the use of the estimand framework in clinical trials for four diseases in immunology and inflammation: generalized pustular psoriasis (GPP), rheumatoid arthritis (RA), atopic dermatitis (AD), and non-radiographic axial spondylarthritis (nraSpA). These diverse diseases necessitate varied trial designs to achieve specific treatment objectives. The estimand framework is crucial for trial design, addressing confounding factors, and examining key questions like symptom reduction, disease remission rates, maintenance, and flare control. Each case study includes a detailed trial design with clear objectives, well-defined estimands, and strategies for addressing intercurrent events. The description of the analysis approaches includes the statistical models and methods being used, often completed by sensitivity and supplementary analyses. These case studies highlight the complexities of applying the estimand framework in immunology and inflammation, emphasizing the importance of articulating clear trial objectives and estimands for interpretable treatment effects.

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Applying the Estimand Framework: Case Studies in Immunology and Inflammation

  • Leslie Meng,
  • Peter Mesenbrink,
  • Fan Wang,
  • Chang Chen,
  • Hongjie Deng

摘要

This chapter illustrates the use of the estimand framework in clinical trials for four diseases in immunology and inflammation: generalized pustular psoriasis (GPP), rheumatoid arthritis (RA), atopic dermatitis (AD), and non-radiographic axial spondylarthritis (nraSpA). These diverse diseases necessitate varied trial designs to achieve specific treatment objectives. The estimand framework is crucial for trial design, addressing confounding factors, and examining key questions like symptom reduction, disease remission rates, maintenance, and flare control. Each case study includes a detailed trial design with clear objectives, well-defined estimands, and strategies for addressing intercurrent events. The description of the analysis approaches includes the statistical models and methods being used, often completed by sensitivity and supplementary analyses. These case studies highlight the complexities of applying the estimand framework in immunology and inflammation, emphasizing the importance of articulating clear trial objectives and estimands for interpretable treatment effects.