Received: 28 March 2019
Accepted: 3 July 2019
First Online: 22 July 2019
Ethics approval and consent to participate
: This study used anonymized German claims data. Therefore, no ethics approval was needed by an independent ethics committee or institutional review board.The utilized database addresses all data protection regulations in Germany. To ensure the protection of individual data and privacy, regions that are smaller than federal states or patient cohorts with less than 100 individuals were not analyzed in a granular way. Furthermore, patient counts below 5 were reported as “< 5”.
: Not applicable.
: IA, MJ, AJ, PL, and CS are full-time employees and stockholders of BioMarin Europe Ltd. KK, JA, CJ, and SB are full-time employees of Xcenda, acting as contractors of BioMarin Europe Ltd. for the execution of this study. FT received honoraria for presentations from BioMarin. AM has been a member of scientific advisory boards supported by BioMarin Europe Ltd. and has received honoraria as a speaker for BioMarin Europe Ltd. FR has received speaker and consulting honoraria from BioMarin Europe Ltd.