A non-interventional Post Authorization Safety Study (PASS) to evaluate long-term safety of Orfadin treatment in hypertyrosinemia type 1 (HT-1) patients in standard clinical care (OPAL)

ENCePP Code of conduct

No

Data sources

• Data sources (types):
  Other
• Data sources (types), other:
  Prospective patient-based data collection, In addition to prospective data collection retrospective data was collected from an ongoing Post-marketing surveillance program (PMS). The PMS is replaced by the non-interventional PASS.

Use of a Common Data Model (CDM)

• CDM mapping:
  No

Data quality specifications

• Check conformance:
  Unknown
• Check completeness:
  Unknown
• Check stability:
  Unknown
• Check logical consistency:
  Unknown

Data characterisation

• Data characterisation conducted:
  No