Approval of orphan-designated cancer drugs: FDA vs EMA


  • Dawn O'Shea
  • Univadis Medical News
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A new study reveals dramatic variation in new cancer drugs receiving orphan drug designation in the United States (US) and Europe.

The research, published in BMJ Open, reviewed cancer drugs newly approved with orphan drug designations by the US Food and Drug Administration (FDA) from 2008 through 2017. European public assessment reports were searched to determine whether these FDA-approved drugs were also approved by the European Medicines Agency (EMA).

Of 135 cancer drug indications receiving FDA orphan designation, 101 (75%) were also approved by the EMA, with (41%) or without orphan designation, by 1 August 2018.

Of these, 46 were approved for first-line therapy, while 55 were indicated for second-line, third-line or fourth-line therapy. Forty-five were for supplementary indications of already approved drugs.

A third were approved for biomarker-based indications in the US; however, only nine approved cancer drug indications by the EMA were biomarker-derived drugs.

The authors say one reason for the different application of orphan status in the US and the EU could be the different legal prerequisites; the EMA requires demonstration of ‘significant benefit’ compared with drugs already on the market targeting the same disease, while the FDA requirement of a positive benefit-risk assessment represents a lower threshold.