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...

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Detalles Bibliográficos
Autores principales: Maeda, Kojiro, Kaneko, Masayuki, Narukawa, Mamoru, Arato, Teruyo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
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

Internet

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

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