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Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs
BACKGROUND: The unmet medical needs of individuals with very rare diseases are high. The clinical trial designs and evaluation methods used for ‘regular’ drugs are not applicable in the clinical development of ultra-orphan drugs (<1000 patients) in many cases. In order to improve the clinical dev...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2017
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5569502/ https://www.ncbi.nlm.nih.gov/pubmed/28835285 http://dx.doi.org/10.1186/s13023-017-0690-5 |