Orphan drug clinical development - 16/04/20
, Marie-Noelle Lefebvre a, b, Olivier Rascol c, Joëlle Micallef aBienvenido a EM-consulte, la referencia de los profesionales de la salud.
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Summary |
Clinical development for orphan drugs is extremely demanding but fascinating. There is no single aspect that is really specific to it but instead it gathers most of the hurdles: design, outcomes, recruitment, ethics, cost, probability and predictability for success. To overcome these difficulties, there has to be a great collaboration between academic centers, small and large pharma companies, patients’ representatives as well as health authorities to provide support and innovative approaches. The ultimate goal is to give access to patients with unmet medical needs to drugs with a favorable benefit-risk ratio. We review and discuss here the pillars for a successful clinical development for orphan drugs.
El texto completo de este artículo está disponible en PDF.Keywords : Rare diseases, Orphan drugs, Market authorization, Clinical trial, Patient
Esquema
Vol 75 - N° 2
P. 141-147 - avril 2020 Regresar al número
Artículo precedente
- Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases
- Joëlle Micallef, Olivier Blin
- A methodological framework for drug development in rare diseases. The CRESim program: Epilogue and perspectives
- Patrice Nony, Behrouz Kassai, Catherine Cornu, for the CRESim, Epi-CRESim research groups
Bienvenido a EM-consulte, la referencia de los profesionales de la salud.
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Bienvenido a EM-consulte, la referencia de los profesionales de la salud.
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