The Case for the Use of Patient and Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic Overview

INTRODUCTION: The traditional model of evaluating treatments based primarily on primary outcome measures has stumbled in its application to rare disease. Rare disease clinical trials face the methodological challenges of small, heterogeneous patient populations and relatively few validated, disease-...

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Detalles Bibliográficos
Autores principales: Contesse, Marielle G., Valentine, James E., Wall, Tracy E., Leffler, Mindy G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2019
Materias:
Commentary
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6824378/
https://www.ncbi.nlm.nih.gov/pubmed/30879250
http://dx.doi.org/10.1007/s12325-019-00920-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6824378/
https://www.ncbi.nlm.nih.gov/pubmed/30879250
http://dx.doi.org/10.1007/s12325-019-00920-x

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