The ERN-EuroBloodNet is mapping the accessibility of Paroxysmal nocturnal hemoglobinuria (PNH) drugs (Eculizumab, Ravulizumab and anti-complement drugs or other biosimilar complement inhibitors) in European Member States.
The scientific committee of this study is formed by: ERN-EuroBloodNet Coordination Team, Régis Peffault de Latour (Assistance Publique-Hôpitaux de Paris, Hôpital Saint-Louis), Carlo Dufour (IRCCS Institute Giannina Gaslini - Genova) and Antonio M Risitano (AOU Federico II - Naples), Jens Peter Panse (University Hospital RWTH Aachen), Maria Piggin (PNH Support and PNH Global Alliance) and Pascale Burmester (Lichterzellen and PNH Global Alliance).
The purpose of this study is to map the countries within the European Union with access to PNH drugs, to assess the modalities of accessibilities and their reimbursement status.
The survey is circulating among ERN-EuroBloodNet members, Patients Associations, the ERN Board of Member States and National Contact Points.
The results of this study will be used for a policy report delivered to policymakers and regulatory bodies.
The survey, which takes 5 minutes for answering, is available here until the 16th of June 2023!