Protocol version for countries where patients treated off-label cannot be included in the disease registry: A Post-Authorisation Disease Registry Safety Study to Generate Data on the Long-Term Safety and Clinical Effectiveness of SCENESSE® (Afamelanotide 16mg) in Patients with Erythropoietic Protoporphyria (EPP). (SCENESSE® PASS-002)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Non-Interventional Study

Study drug and medical condition

• Name of medicine:
  SCENESSE
• Medical condition to be studied:
  Porphyria non-acute

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)
• Estimated number of subjects:
  200

Study design details

• Main study objective:
  The study has been designed to assess whether SCENESSE® can be used safely within porphyria treatment centres and to generate data on long term safety and outcome endpoints derived from the use of SCENESSE®. The recommended risk minimization measures and the effectiveness of the controlled distribution of SCENESSE® will also be assessed.
• Outcomes:
  Gather long-term safety data on SCENESSE®, Evaluate compliance with the risk minimization measures
• Data analysis plan:
  Safety assessment as outlined above will occur via the European EPP Disease Registry. Treatment-emergent adverse events and the use of concomitant medications will be assessed at every visit to the treatment centre and recorded using electronic CRFs. Compliance with the risk minimization measures will be assessed against the designated criteria throughout the study while the effectiveness of the controlled distribution of SCENESSE® will be evaluated every 12 months.Analysis of Clinical EffectivenessAll patients enrolled in the registry will be included in the assessment of clinical effectiveness so long as data are available. In general, baseline will be the data recorded on entry into the Registry. In general, clinical effectiveness will also be summarised by the subgroup variables, SCENESSE exposure at entry and Season of enrolment.