Comparing randomized trial designs to estimate treatment effect in rare diseases with longitudinal models: a simulation study showcased by Autosomal Recessive Cerebellar Ataxias using the SARA score

Comparing randomized trial designs to estimate treatment effect in rare diseases with longitudinal models: a simulation study showcased by Autosomal Recessive Cerebellar Ataxias using the SARA score

Abstract Parallel designs with an end-of-treatment analysis are commonly used for randomised trials, but they remain challenging to conduct in rare diseases due to small sample size and heterogeneity. A more powerful alternative could be to use model-based approaches. We investigated the performance...

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Bibliographic Details
Main Authors: Niels Hendrickx, France Mentré, Alzahra Hamdan, Mats O. Karlsson, Andrew C. Hooker, Andreas Traschütz, Cynthia Gagnon, Rebecca Schüle, Matthis Synofzik, Emmanuelle Comets, ARCA Study Group, EVIDENCE-RND consortium
Format: Article
Language:English
Published: BMC 2025-07-01
Series:BMC Medical Research Methodology
Subjects:
Non-linear Mixed effect models
Simulation study
Rare disease
Clinical trial design
Model-based analysis
Online Access:https://doi.org/10.1186/s12874-025-02626-x
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https://doi.org/10.1186/s12874-025-02626-x

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