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Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications
BACKGROUND: The evaluation of clinical evidence takes account of health benefit (efficacy and safety) and the degree of certainty in the estimate of benefit. In orphan indications practical and ethical challenges in conducting clinical trials, particularly in paediatric patients, often limit the ava...
Autores principales: | Wex, Jaro, Szkultecka-Debek, Monika, Drozd, Mariola, King, Sarah, Zibelnik, Natasa |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10360248/ https://www.ncbi.nlm.nih.gov/pubmed/37474954 http://dx.doi.org/10.1186/s13023-023-02701-w |
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