Life changing fever, headache and painkilling drug for children on the NHS

NHS England is to fund a life-changing treatment for people with rare conditions causing repeated bouts of fever, joint pains and swelling, in an effort to help a small group of people who ‘suffer in silence’.

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

On World Rare Diseases Day (Saturday 29 February) the NHS has confirmed a deal has been struck for the drug – canakinumab – which modifies the immune system, turning off the inflammation process, significantly reducing the number of ‘flares’ children and adults experience.

The deal with a global pharmaceutical firm has been agreed after negotiations led by the NHS, as part of a major programme to ramp up access to innovative treatments, while freeing up funding for frontline patient care through smarter procurement.

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.”

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

The discounted price of canakinumab, developed by Novartis, is commercial in confidence.

Patients with three different types of periodic fever conditions will be eligible for treatment: familial Mediterranean fever, mevalonate kinase deficiency (also known as the hyperimmunoglobulinemia D syndrome), and tumour necrosis factor receptor–associated periodic syndrome (TRAPS).

Symptoms include high temperature, severe fatigue, severe abdominal, chest or joint pains, headaches, rashes and mouth ulcers, and flares can last several days or even weeks, with long term risk of organ failure or even death if not treated.

Current treatments which include anti-inflammatory and immune-suppressing drugs such as high dose steroids and ibuprofen are not always effective, and some may cause side effects including weight gain, skin and bone thinning and susceptibility to diabetes and infections.

Another treatment option is Anakinra which does not work for everyone and is taken daily via an injection which can be unpleasant for patients. Canakinumab is only injected every 8-12 weeks and is transformative – allowing patients to live more normal lives.

The NHS has struck a string of ‘smart deals’ struck to drive value for the taxpayer and benefits for patients. These include:

  • a deal to make ocrelizumab, an innovative multiple sclerosis drug available;
  • a ground-breaking deal which could eliminate Hepatitis C;
  • a £300 million saving after negotiating deals with five manufacturers on low cost versions of the health service’s most costly drug, adalimumab, for arthritis;
  • striking the first full access deal in Europe for CAR-T therapy via the Cancer Drugs Fund, which can potentially cure some children and adults with blood cancers where other treatments have failed;
  • reaching a deal to make the life-extending lung cancer drug pembrolizumab available for routine use on the NHS;
  • a deal to make nusinersen available – the first treatment that targets the underlying cause of spinal muscular atrophy.