2. Materials and Methods
The database on human medicines from European Medicines Agency was used and data from Rare disease (orphan) designations (https://www.ema.europa.eu/en/medicines/download-medicine-data#rare-disease-(orphan)-designations-section) and European public assessment reports (EPARs) (https://www.ema.europa.eu/en/medicines/download-medicine-data#european-public-assessment-reports-(epar)-section) were retrieved (20 May 2020) for the analysis.
A control with the data obtained from EMA database was performed for consistency with the Community Register of orphan medicinal products from EC (https://ec.europa.eu/health/documents/community-register/html/reg_od_act.htm?sort=a) in which all ODs are registered. (20 May 2020)
The ClinicalTrials.gov database and The European Union Clinical Trials Register were used to query all active studies on Haemophilia (20 May 2020). ClinicalTrials.gov provides information on medical studies in human volunteers. Most of the records on ClinicalTrials.gov describe clinical trials. The EU Clinical Trials Register contains information on interventional clinical trials on medicines conducted in the European Union (EU), or the European Economic Area (EEA).