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Collaborate on real-world evidence

What we commit to

We will continue to engage with health systems and patient communities to jointly collect patient experience data to embed the goals and outcomes that matter to patients as we develop therapeutic solutions and support.

What we have done so far

Sobi actively collaborates with the Patient Community to gather real-world evidence and deploy patient preference studies. This involves engaging with people living with Rare Diseases and healthcare professionals to understand their experiences and perspectives. The data collected from these interactions provides valuable insights into the effectiveness and impact of treatments in real-world settings, beyond the controlled environment of clinical trials. This evidence is crucial for improving patient care and advancing medical research. Examples of cooperative projects are: INTO-HLH Registry (US), REAL-AVA, ADHERENCE (EHA 2024), DevelopAKUre, Trapeze, PNH Burden of Illness Study and many others.



Up to 2025, we co-designed and collected patient input into

19

Real World Evidence studies, involving 25 Patient Organisations in the process.

Measurement

We will measure impact through:

  • Number of Real World Evidence studies designed involving patients or executed involving Patient Organisations (where applicable).

Involvement

The Sobi departments involved are:

  • Value and Access
  • Patient Engagement
  • Medical
  • Compliance
  • Legal