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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Frontiers Media S.A.
2022
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| Materias: | |
| 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 |