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).