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To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases

BACKGROUND: Over the past 30 years, the healthcare industry has increasingly turned its attention to rare diseases. Regulators have emphasized the need for clinical research in this area to be patient-centered. However, there is a lack of evidence concerning whether this need is actually met. In thi...

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Detalles Bibliográficos
Autores principales: Lanar, Sally, Acquadro, Catherine, Seaton, James, Savre, Isabelle, Arnould, Benoit
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7268539/
https://www.ncbi.nlm.nih.gov/pubmed/32493385
http://dx.doi.org/10.1186/s13023-020-01400-0