Lee-on-the-Solent family call for cystic fibrosis drug to be available on NHS

The family of 10-year-old Ethan Marvin, from Lee-on-the-Solent, are among hundreds of people who want to see the drug Orkambi free for those with the condition.

Currently, the drug is only available in England privately after the National Institute for Health and Care Excellence (Nice) deemed it too expensive last June.

The organisation was unable to recommend Orkambi for use by the NHS on cost grounds, even though it recognised the treatment as effective for managing cystic fibrosis. Treatment costs £104,000 per patient, per year.

Ethan’s parents Karina and Craig know the benefits of the drug after Ethan was given it while they lived in Dallas, in America. It was available to him through their health insurance.

They were given six months of the drug to use while they settled back into life in England, but as the drug runs out in October Ethan will be without it.

Karina said: ‘Ethan is an incredibly fit sporty boy. In America he played football at academy level, sometimes six matches in a week. He plays tournaments which are tough but nobody would know by looking at him that he has cystic fibrosis.

‘Despite his athletic prowess, Ethan has always been very skinny.

‘Thriving and gaining or maintaining weight is a problem for many cystic fibrosis patients.

‘Since he started on Orkambi he has been in excellent health. There have been significant changes in his height and weight.

‘Before taking this drug, Ethan would typically have at least one course of two weeks antibiotics per month.

‘Since February he has only had one course of antibiotics in nearly five months.’

Cystic fibrosis is a life-limiting genetic disease which causes the internal organs, especially the lungs and digestive system, to become clogged with thick, sticky mucus.

Recent results from a phase three clinical trial suggest that Orkambi can stop the damage to the lungs of people with the condition.

The results also show the drug can be beneficial for young children.

Karina added: ‘We are very concerned that Ethan may not be able to continue taking it. It seems so cruel to take it away. He is a bright young man and understands how important the drug is.

‘I am desperate for him to stay on it and for other children in the UK to be given the chance to start on it.’

A protest is being held in London on Monday and people with be marching at Downing Street to call for the NHS to provide Orkambi. As well as the protest, an online petition has been started.

James Barrow, head of external relations at the Cystic Fibrosis Trust, said: ‘Cystic fibrosis is a cruel and life-shortening condition which places a huge daily burden on people living with the disease.

‘Orkambi is a major step forward in the treatment of the illness which could help people live longer and has the potential to save the lives of thousands in the UK.’

This week is Cystic Fibrosis Awareness Week and tomorrow people around Portsmouth are being called to wear yellow to show their support.