NHS England has decided to fund a life-changing treatment for certain individuals with periodic fever syndromes (PFS), a group of rare conditions characterised by fever, joint pains and swelling. Patients with PFS often experience inflammatory ‘flares’ involving chest or joint pains, headaches, mouth ulcers and skin rash.
On the occasion of World Rare Diseases Day on 29 February 2020, the NHS secured a deal with Novartis for using the immunomodulatory drug canakinumab to reduce the frequency of ‘flares’ in children and adults with PFS.
An estimated 168 individuals in England have PFS. Around 80 per cent of them would be eligible to receive canakinumab, including patients with familial Mediterranean fever, mevalonate kinase deficiency (hyperimmunoglobulinaemia D syndrome) and tumour necrosis factor receptor-associated periodic syndrome.
Current treatments for PFS include anti-inflammatory and immune-suppressing drugs which are not always effective and some are associated with unfavourable side effects such as weight gain, thinning of skin and bone and increased risk for diabetes and infections.
Sir Simon Stevens, chief executive of NHS England, said: "Some illnesses affect a small number in a big way, which is why the NHS is increasingly bringing innovative, specialised and targeted treatments to people who need them."