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-001)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  The study has been designed to assess whether SCENESSE® can be used safely within designated treatment centres and to generate data to support the clinical effectiveness of SCENESSE®.

Non-interventional study

• Non-interventional study design:
  Other

Study drug and medical condition

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

Population studied

• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

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

Study design details

• Main study objective:
  • Gather long-term safety data of SCENESSE®• Evaluate compliance with the risk minimization measures
• Outcomes:
  • Gather long-term safety data of SCENESSE®• Evaluate compliance with the risk minimization measures, • Evaluate adherence with the controlled distribution program• Generate data to contribute to knowledge about clinical benefits and to add data on potential clinical effectiveness of SCENESSE
• Data analysis plan:
  All patients enrolled in the registry will be included in the safety assessment. In general longitudinal comparisons will be within Treated Groups (including separate groups that take into account patients who commence or discontinue treatment) and between groups, Treated versus Untreated.The number of participants with treatment-emergent adverse events will be summarized by MedDRA PT and body system. Adverse events will be further summarized by intensity, seriousness and outcome. Adverse events will also be summarized by 6 monthly time intervals from entry into the registry to assess the longitudinal effect of the treatment and EPP.Assessment of Clinical EffectivenessContinuity on treatment - together with logs detailing reasons for discontinuing (and those recommencing)Quality of Life (EPP-QoL) – will be measured using the EPP-QoL questionnaire, provided to the patient for completion at baseline prior to treatment in Year 1 and at each subsequent visit.