NHS England to fund drug for rare conditions

NHS England is to fund a life-changing treatment for people with rare conditions causing repeated bouts of fever, joint pains and swelling.

Periodic fever syndromes (PFS) are a group of rare genetic conditions where the person’s immune system overreacts, leading to frequent inflammation ‘flares’, involving chest or joint pains, headaches, mouth ulcers and skin rash.

The NHS has now confirmed a deal has been struck for canakinumab, which modifies the immune system, turning off the inflammation process, significantly reducing the number of ‘flares’ children and adults experience. The deal forms part of a major programme to ramp up access to innovative treatments, while freeing up funding for frontline patient care through smarter procurement.

There are around 168 patients in England with these diseases, both children and adults, and around 80 per cent of these would be prescribed canakinumab.

The agreement brings into the NHS a more effective, convenient drug with fewer side effects than existing treatments, and follows contracts agreed in recent months which introduced new drugs for restoring sight and cystic fibrosis.

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. This latest new treatment has the potential to significantly improve the lives of children who bear the burden of bouts of crippling pain which blights lives and puts strain on families. It’s the latest in a line of major deals NHS England has successfully negotiated, which show that when drug companies play ball with the NHS, taxpayers get a fair deal and patients get cutting-edge treatments.”