Battle to get ‘wonder drug’ that improves cystic fibrosis in days
YOUNG people blighted by the incurable lung disease cystic fibrosis (CF) have had their health transformed in as little at two weeks by a new ‘wonder drug’.
Experts believe that using the tablets daily could stop the illness, which currently kills many sufferers by their mid-30s, from becoming severe later in life.
However, campaigners hit out last week at health chiefs who have denied NHS patients access to the £100,000-a-year medication because it is ‘not cost-effective’.
British charity the CF Trust, which represents sufferers, claimed the full economic case for the drug has not been taken into account. A spokesman said: ‘This is a truly outstanding drug that will save the NHS money in the long run and holds out the hope of patients living potentially a normal lifespan in the future.’
Orkambi is a combination of two drugs – lumacaftor and ivacaftor – which tackle the gene mutation that causes the inherited disease, rather than just relieving its symptoms. It has been hailed by researchers at Imperial College London as a gamechanger which, while no cure, could make the disease a chronic condition rather than a killer.
Cystic fibrosis is caused by a faulty gene that a child inherits from both carrier parents.
The gene, known as the cystic fibrosis transmembrane conductance regulator (CFTR), is responsible for controlling the movement of water in and out of cells.
The fault leads to the mucus produced throughout the body becoming thick and building up in the lungs and digestive system.
Classic complications caused by the condition – which tend to present in infancy – include chronic infections, breathlessness, digestive problems and even infertility.
Orkambi works by correcting the gene fault, keeping a healthy balance of salt and water in mucus so it can function normally. Research has shown that it reduces the incidence of infection and can cut the number of hospital admissions by more than 60 per cent by keeping patients’ lungs clear and free of infection and the scarring that eventually kills.
SUFFERERS and their doctors have battled for two years to get the drug funded by the NHS. They believe the results of new trials in children – results of which were published last month in the journal Lancet Respiratory Medicine – should help ensure that Orkambi is made available to all CF patients.
But NHS prescribing watchdog the National Institute for Health and Care Excellence (NICE) has not yet recommended routine use due to the extraordinarily high cost. It has also been rejected for use by the Scottish Medicines Consortium.
Orkambi manufacturers Vertex have made financial deals in other EU countries that have led to the drug being given the green light, and campaigners hope the same will now happen here.
The paediatric trial, involving more than 200 children aged six to 11, some from the UK, showed that the drug improved lung function within two weeks of starting treatment. Professor Felix Ratjen, from The Hospital for Sick Children in Toronto, who led the study said the results were ‘exciting’. He added: ‘The findings suggest that this drug combination may help to preserve lung health in children with cystic fibrosis, potentially slowing the progression of the disease and allowing these children betterquality, longer lives.
‘Further research into long-term effects will be needed. If successful, giving the drug treatment soon after diagnosis will be crucial to ensure that the disease is slowed as soon as possible.’
The failure to license Orkambi in adults in the UK is leading to distress among sufferers, campaigners argue. Some have already been prescribed it on compassionate grounds while others who appear equally in need have not been given access.
ONE person who has benefited already, though, is Daniel Bodio, 34, an engineer from Swindon, who was diagnosed when he was two months old. He said: ‘Until my early 20s I had no problems, regularly running cross-country and swimming. I was very disciplined with my medication and sport keeps the body strong.
‘My first major infection was in 2011, after I had my wisdom teeth out. The infection was resistant to antibiotics so I never fully recovered, meaning I was extremely breathless from doing the smallest activity such as brushing my teeth or having a shower.’
Before Daniel took Orkambi in July this year, his lung function was just 25 per cent and he was facing a lung transplant. Within three weeks, it had improved to 39 per cent. Although he is still battling the persistent infection, he feels that his health is improving.
‘I’m coughing less, and sleeping better. I’d lost weight due to illness, but that has come back. I can now do all the little things that other people take for granted, like going up stairs, without getting breathless.’
He adds: ‘The drug should be available to everyone – it brings normality to what is otherwise a difficult existence.’