Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations

Patients with rare diseases often have limited or no options for approved treatments or participation in clinical trials. In such cases, expanded access (or “compassionate use”) provides a potential means of accessing unapproved investigational medicines. It is also possible to capture and analyze c...

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Detalles Bibliográficos
Autores principales: Polak, Tobias B., Cucchi, David G. J., van Rosmalen, Joost, Uyl-de Groot, Carin A., Darrow, Jonathan J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Pharmacology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9168458/
https://www.ncbi.nlm.nih.gov/pubmed/35677436
http://dx.doi.org/10.3389/fphar.2022.913567

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9168458/
https://www.ncbi.nlm.nih.gov/pubmed/35677436
http://dx.doi.org/10.3389/fphar.2022.913567

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