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Improved Decision-Making Confidence Using Item-Based Pharmacometric Model: Illustration with a Phase II Placebo-Controlled Trial.

ABSTRACT: This study aimed to illustrate how a new methodology to assess clinical trial outcome measures using a longitudinal item response theory-based model (IRM) could serve as an alternative to mixed model repeated measures (MMRM). Data from the EXACT (Exacerbation of chronic pulmonary disease tool) which is used to capture frequency, severity, and duration of exacerbations in COPD were analyzed using an IRM. The IRM included a graded response model characterizing item parameters and functions describing symptom-time course. Total scores were simulated (month 12) using uncertainty in parameter estimates. The 50th (2.5th, 97.5th) percentiles of the resulting simulated differences in average total score (drug minus placebo) represented the estimated drug effect (95%CI), which was compared with published MMRM results. Furthermore, differences in sample size, sensitivity, specificity, and type I and II errors between approaches were explored. Patients received either oral danirixin 75 mg twice daily (n = 45) or placebo (n = 48) on top of standard of care over 52 weeks. A step function best described the COPD symptoms-time course in both trial arms. The IRM improved precision of the estimated drug effect compared to MMRM, resulting in a sample size of 2.5 times larger for the MMRM analysis to achieve the IRM precision. The IRM showed a higher probability of a positive predictive value (34%) than MMRM (22%). An item model-based analysis data gave more precise estimates of drug effect than MMRM analysis for the same endpoint in this one case study.

SUBMITTER: Llanos-Paez C

PROVIDER: S-EPMC8172506 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:BackgroundEthical decision-making confidence develops from clinical expertise and is a core competency for nurse leaders. No tool exists to measure confidence levels in nurse leaders based upon an ethical decision-making framework.AimsThe objective of this research was to compare ethical decision-making among nurse leaders in the U.S. and three German-speaking countries in Europe by developing and testing a newly constructed Ethical Decision-Making Confidence (EDMC) scale.MethodsThe cross-sectional survey included 18 theory-derived questions on ethical decision-making confidence which were used to develop the scale.ParticipantsA convenience sample of nurse leaders from the U.S. and three German-speaking countries in Europe who self-identified as holding a leadership position.Ethical considerationsEthical approval was given by the IRB Board of a U.S. university. Participation in the survey implied voluntary consent.ResultsThe scale's item structure dimensionality and subscale's reliability were analyzed and compared between nurse leaders from all four countries. A principal component analysis (PCA) produced a 15-item bi-dimensional EDMC scale yielding a skill-related (9-item) and a behavior-related (6-item) confidence dimension. EDMC subscales showed good-to-excellent internal consistency. In both subscales, U.S. nurse leaders rated their mean EDMC score higher than their German-speaking counterparts in Europe.DiscussionThis exploratory study is the first of its kind to focus on nurse leaders' confidence regarding ethical decision-making in an international context. An overarching factor structure was identified, which is shared by the two samples of nurse leaders and to examine (sub)scales' psychometric properties.ConclusionThis newly developed scale is an effective tool for measuring ethical decision-making confidence in nurse leaders. The promising results of this study should be replicated to ensure validity and reliability of the EDMC scale measuring skill-related and behavior-related concepts and include nurse leaders from various cultural, social, and demographic groups.

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Improved Decision-Making Confidence Using Item-Based Pharmacometric Model: Illustration with a Phase II Placebo-Controlled Trial.

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