d65c534b2133b075ad2828dc8ad099fc  pmc_30442155.pdf
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Title:          Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties
Subject:        Orphanet Journal of Rare Diseases, 2018, doi:10.1186/s13023-018-0926-z
Keywords:       Orphan drug production,Rare diseases,Research design/methods,Research design/standards,Clinical trials as topic,Drug approval
Author:         Caridad Pontes
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