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A methodological framework for drug development in rare diseases

Patrice Nony 1 Polina Kurbatova 2 Agathe Bajard 3 Salma Malik 1, 4 Charlotte Castellan 2 Sylvie Chabaud 5 Vitaly Volpert 6, 7 Nathalie Eymard 7 B. Kassai 1, 4 Catherine Cornu 4, 1 
1 Evaluation et modélisation des effets thérapeutiques
Département biostatistiques et modélisation pour la santé et l'environnement [LBBE]
6 DRACULA - Multi-scale modelling of cell dynamics : application to hematopoiesis
CGPhiMC - Centre de génétique et de physiologie moléculaire et cellulaire, Inria Grenoble - Rhône-Alpes, ICJ - Institut Camille Jordan [Villeurbanne]
7 MMCS - Modélisation mathématique, calcul scientifique
ICJ - Institut Camille Jordan [Villeurbanne]
Abstract : 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.
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Submitted on : Friday, May 20, 2022 - 10:16:42 AM
Last modification on : Tuesday, October 25, 2022 - 4:21:55 PM


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Patrice Nony, Polina Kurbatova, Agathe Bajard, Salma Malik, Charlotte Castellan, et al.. A methodological framework for drug development in rare diseases. Orphanet Journal of Rare Diseases, 2014, 9 (164), pp.1-10. ⟨10.1186/s13023-014-0164-y⟩. ⟨hal-01097224⟩



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