Drug could give Esme an extra 23 years of life – if NHS and drug firm end price battle

The health service has been locked in a tense pricing battle with manufacturer Vertex since 2016 over the medication Orkambi, which has been hailed as a breakthrough for those with cystic fibrosis (CF).

There are more than 10,000 people with CF in the UK and one of them is 17-month-old Esme Ives. While she's currently too young to take Orkambi, without it little Esme's life expectancy could be capped at around 47.

With it, parents Claire and Mark have been told it could give her an extra 23 years as it slows the deterioration of the lungs.

CF causes sticky mucus to build up in the lungs of sufferers, increasing their chances of serious lung infections and over time causing scarring that makes it harder to get oxygen.

Claire and Mark Ives, with Willow, three, and 17-month-old Esme who has cystic fibrosis. The family hope a new drug, Orkambi, can be approved by the NHS which could extend Esme's life by 23 years. Picture: DENISE BRADLEY (Image: Archant)

But the health service has refused to fund the drug, which costs £104,000 a year, except in exceptional circumstances.

Campaigners argue the cost is offset with reducing expensive hospital admissions, where CF patients must have their own rooms and specialist care.

Mrs Ives, a teacher at Thomas Bullock Primary Academy in Shipdham, said: 'As a family I think we are able to look forward to a longer future with Esme if she can take Orkambi. If she is able to take the drug from a young age before too much lung damage it will really help her chances.

'She currently has her first case of pseudomonas (a kind of chest infection) which was scary at first but also a relief as it allowed us to have a reason why she had been losing weight and coughing so much.

Esme Ives, 17-months-old, who has cystic fibrosis, and her family hope a new drug, Orkambi, can be approved by the NHS which could extend Esme's life by 23 years. Picture: DENISE BRADLEY (Image: Copyright: Archant 2019)

'One of the hardest things is that she doesn't look ill and it can be so hard for people to understand CF.'

Mrs Ives added: 'Vertex have offered a pipeline of drugs to NHS England at a lower cost than anyone else, however it's still been rejected as too expensive despite us having the fifth largest economy in the world and being available in most other European countries, even at a higher price.

'NICE are responsible for the rejection as its processes are 20-plus years old and don't apply in the modern era of new precision medicines.'

Orkambi has been licensed in the UK for more than three years, but is not available on the NHS.

Esme Ives, 17-months-old, who has cystic fibrosis, and her family hope a new drug, Orkambi, can be approved by the NHS which could extend Esme's life by 23 years. Picture: DENISE BRADLEY (Image: Copyright: Archant 2019)

Campaigners say 220 people eligible for the drug have died in this time and around half of UK sufferers could benefit from it.

A Commons Health Select Committee is currently investigating the situation and is expected to report its findings at the end of March, meaning that patients and their families face many more months of anxious waiting.

Mrs Ives, 34, said: 'If Esme was able to take Orkambi it would change her life.'

She said her daughter's recent bout of illness had hammered home to her just how devastating the illness can be.

Mrs Ives and husband Mark, who live in Bracken Drive and have another daughter Willow, got married last year to ensure Esme would be there, a sign of how unpredictable CF can be.

A spokesman for NHS England said: 'We understand how difficult it must be for families affected by cystic fibrosis – this is why it is so important that as with all companies seeking NHS approval for treatments of this kind, Vertex must fully engage with the NICE process.'

While a NICE spokesman added: 'We have been working over the past month clarifying our process with Vertex. We are still very keen for the company to engage with us but as it stands they are not doing that.'

A spokesman for Vertex said: 'Out of respect for the Parliamentary process, we will not be providing further updates at this time. Vertex welcomes the committee's interest in access to cystic fibrosis medicines and we look forward to continuing to support its inquiry into this important issue.'

A petition previously reached more than 100,000 signatures, prompting a debate in parliament on the issue. Another petition has now been started due to the lack of progress, and can be visited at petition.parliament.uk/petitions/231602

What is cystic fibrosis?

Cystic fibrosis (CF) is caused by a defective gene, and one in 25 people carry the faulty DNA.

When two carriers have a child, there is a one in four chance they will have CF.

The condition affects more than 10,400 people in the UK.

The faulty CF gene controls the movement of salt and water in and out of cells, which means those with CF have a build-up of mucus in the lungs, digestive system, and other organs.

Two people with the condition should never meet face-to-face because their lungs carry specific bugs which could cause a serious infection in someone else also affected. This means at CF clinics strict segregation is in place.

People with CF often need to take more than 50 tablets a day to help digest food and keep respiratory symptoms in check, as well as undergoing daily physiotherapy, antibiotics, and taking enzyme tablets with food.