Generalized pairwise comparisons of prioritized outcomes are a powerful and patient-centric analysis of multi-domain scores

BACKGROUND: Generalized pairwise comparisons (GPC) can be used to assess the net benefit of new treatments for rare diseases. We show the potential of GPC through simulations based on data from a natural history study in mucopolysaccharidosis type IIIA (MPS IIIA). METHODS: Using data from a historic...

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Detalles Bibliográficos
Autores principales: Deltuvaite-Thomas, Vaiva, De Backer, Mickaël, Parker, Samantha, Deneux, Marie, Polgreen, Lynda E., O’Neill, Cara, Salvaggio, Samuel, Buyse, Marc
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10571482/
https://www.ncbi.nlm.nih.gov/pubmed/37828533
http://dx.doi.org/10.1186/s13023-023-02943-8

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