The Aplastic Anaemia Trust and PNH Support are very pleased to share the news that ravulizumab has now been recommended for use in the NHS as treatment for adults with Paroxysmal Nocturnal Haemoglobinuria (PNH).

Clinical trial evidence has shown that Ravulizumab (brand name, ULTOMIRIS®) is as safe and as effective in treating PNH as existing treatment, eculizumab (SOLIRIS®), however it is administered with infusions every eight weeks compared to every two weeks. NICE’s Final Appraisal document recommends ravulizumab as a PNH treatment for adult patients with haemolysis with clinical symptoms suggesting high disease activity, or whose disease is clinically stable after having eculizumab.

NICE’s decision to recommend ravulizumab is a game changer for PNH patients, their families and carers. Our community will immediately feel and see the benefits of having the option of an eight-weekly infusion. This level of independence from our lifelong chronic disease will enable us to live a life less impacted by the physiological and psychological burdens we all have to endure.

Alex Naylor, Patient Expert and Trustee of PNH Support

This is a significant result for PNH Support and The Aplastic Anaemia Trust, who have been participating in the NICE appraisal process since 2018.

Working together

PNH Support and the AAT took a joint approach to the appraisal process to best amplify the voice of our patient communities. Together, we nominated clinical experts and patient experts to provide evidence to the NICE appraisal committee, including Alex Naylor and Maria Piggin from PNH Support. PNH Support collected survey data from 54 patients and 20 carers on unmet need, quality of life and the advantages and disadvantages of ravlizumab, and both of our organisations used this data for submissions to NICE.

“It is wonderful to be able to celebrate this victory with our friends at PNH Support and our patient community. Having aplastic anaemia is the only known risk factor for developing PNH and more than 10% of people with aplastic anaemia will develop this connected condition. This result shows the value of our organisations working together to champion patient’s needs and improve their quality of life.”

Stevie Tyler, CEO of The Aplastic Anaemia Trust