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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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https://hal.inria.fr/hal-01097224
Contributor : Vitaly Volpert <>
Submitted on : Friday, December 19, 2014 - 10:48:12 AM
Last modification on : Wednesday, April 14, 2021 - 3:31:36 AM

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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, BioMed Central, 2014, 9 (164), pp.1-10. ⟨10.1186/s13023-014-0164-y⟩. ⟨hal-01097224⟩

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