Funding for this research was provided by:
Received: 20 December 2020
Accepted: 9 March 2021
First Online: 20 March 2021
: This study was approved by the Research Ethics Sub Committee of the Faculty of Health and Social care within the University of Chester and conducted in correspondence with regional and relevant guidelines, in partnership with the National Hemophilia Foundation (NHF) in the United States. Consultative discussions regarding research design were held with the NHF to ensure relevance and adequacy of the research questions, objectives and methods. Patient consent for use of clinical data was not required (as per European Pharmaceutical Market Research Association (EPhMRA) guidelines). Patient consent was obtained via tick box selection for the patient-reported element of the study.
: Not applicable.
: TB, SA and JO are employees of HCD Economics, which received funding for this study from uniQure Inc. EKS and NL are employees of uniQure Inc, which sponsored the study.