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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-...
Autores principales: | Contesse, Marielle G., Valentine, James E., Wall, Tracy E., Leffler, Mindy G. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Healthcare
2019
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Materias: | |
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 |
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