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Journal Articles Orphanet Journal of Rare Diseases Year : 2014

A methodological framework for drug development in rare diseases

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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hal-01097224 , version 1 (20-05-2022)

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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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