A methodological framework for drug development in rare diseases

Abstract : Developing orphan drugs is challenging because of their severity and the requisite for effective drugs. 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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Article dans une revue
Orphanet Journal of Rare Diseases, BioMed Central, 2014, 9 (164), pp.1-10. <10.1186/s13023-014-0164-y>
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https://hal.inria.fr/hal-01097224
Contributeur : Vitaly Volpert <>
Soumis le : vendredi 19 décembre 2014 - 10:48:12
Dernière modification le : jeudi 9 février 2017 - 16:58:48

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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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