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External control arms for rare diseases: building a body of supporting evidence
Comparator arms in randomized clinical trials may be impractical and/or unethical to assemble in rare diseases. In the absence of comparator arms, evidence generated from external control studies has been used to support successful regulatory submissions and health technology assessments (HTA). Howe...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer US
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10673956/ https://www.ncbi.nlm.nih.gov/pubmed/37095406 http://dx.doi.org/10.1007/s10928-023-09858-8 |