A methodological framework for drug development in rare diseases
Résumé
Developing orphan drugs is challenging because of their severity and the requisite for effectivedrugs. The small number of patients does not allow conducting adequately powered randomized controlled trials(RCTs). There is a need to develop high quality, ethically investigated, and appropriately authorized medicines,without subjecting patients to unnecessary trials.
| Origine | Fichiers produits par l'(les) auteur(s) |
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