rare-diseases-germany-2019

Potency of Orphan Drugs

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Orphan drugs are considered to be medicinal products intended for the diagnosis, prevention or the treatment of rare diseases. Approval of all drugs in rare diseases conditions must be based on effectivenessof substantial evidence of effectiveness in treating or preventing the condition with the evidence of safety for the treatment use. Over the last decade of 30 years, more than 400 medicines representing 447 separate medicinal indications have been approved to treat conditions and diseases symptoms rare diseases, compared to lesser than 10 in the 1970s