DTB: Vol 23, No 1

Orphan drugs: regulation and controversies

Encouragement of research and development of drugs for rare diseases is merited, but should be directed to the most relevant ones. Given the difficulty involved, research on orphan drugs should be carried out under maximum quality standards. Research funding should be reviewed in order to make it sustainable and to ensure that equitable access to drugs is guaranteed. Keywords: orphan drugs, rare diseases


  • Javier Garjón. Drug Prescribing Service. Navarre Health Service. Spain


 - Link to the video "Orphan Drugs"


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