Pediatric requirements in Europe stymie help for hemophilia
Flora Peyvandi, Frits R Rosendaal, Brian O’Mahony, Pier Mannuccio Mannucci
The European Medicines Agency requires that drug developers submit a ‘pediatric investigational plan’ to ensure that there is
adequate information about how children fare on experimental medication for many indications before they go to market. But this
requirement places an undue constraint on the makers of new hemophilia drugs and threatens to create an unreasonable delay inaccess to these therapies among adults with this disorder in the EU.