PNH Patient and Carer Survey for the NICE Appraisal of Ravulizumab (Ultomiris)

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1. Introduction to this survey

Ravulizumab (or Ultomiris) is being appraised by the National Institute of Health and Care Excellence (NICE) to decide whether it will be funded as a treatment for adult PNH patients (18 years plus) in England, Wales and Northern Ireland. The criteria for being able to be treated with Ravulizumab are:

Adults with paroxysmal nocturnal haemoglobinuria
• who have haemolysis with clinical symptom(s) indicative of high disease activity or
• whose PNH is clinically stable after having eculizumab for at least 6 months

PNH Support (registered Charitable Incorporated Organisation no.1161518) has been invited to take part in the appraisal process and is collecting evidence for our submission to NICE about patients' and carers/family members' views of Ravulizumab and the impact on them of treatment with Ravulizmab (or being able to access this drug). For those who are not aware, Ravulizumab inhibits the complement part of the immune system (alike Eculizumab or Soliris) and is delivered by 8 weekly intravenous infusion. If you would like to read more about Ravulizumab please see here. Patients being treated with Ravulizumab in the UK are recommended to take prophylactic antibiotics (as is the case for Eculizumab). It is very important that the PNH patient/carer community has a say in this appraisal so we really value your input. The survey should only take about 15 mins to complete and  will close on 21 August 2020.