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
2 LBBE - Laboratoire de Biométrie et Biologie Evolutive - UMR 5558
3 UBET - Unité de Biostatistique et d'Evaluation des Thérapeutiques
4 CIC CHU Lyon (inserm)
5 Centre Léon Bérard [Lyon]
6 DRACULA - Multi-scale modelling of cell dynamics : application to hematopoiesis
7 MMCS - Modélisation mathématique, calcul scientifique

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.

Domaines: Equations aux dérivées partielles [math.AP]

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⟩