EU Orphan Drug Designation – overcoming regulatory challenges

Since the introduction of the EU orphan legislation in 2000, treatments for rare diseases now account for approximately 25% of all marketing authorization applications in the EU (EMA annual report, 2020). Clearly, the incentives offered by the ODD process have encouraged sponsors to pursue innovations in this domain. Yet while orphan status has been granted to more than 2,300 medicines, only 192 have received marketing authorization. There is no question that success or failure depends on multiple factors. But sponsors can amplify their prospects for gaining authorization by better understanding the regulatory considerations involved in bringing their innovations to patients awaiting them.

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