Cystic fibrosis patient blasts postcode lottery after she is denied ‘miracle’ drug

A patient refused a ground-breaking cystic fibrosis drug has condemned the postcode lottery stopping her gaining its benefits.

Megan Kellett-Jones, 28, hoped she would be given Trikafta on compassionate grounds, like many sufferers across the country whose lives have been transformed.

But the hospital which has been treating her for 20 years is waiting for the treatment to be approved by drugs regulator NICE. A decision is not expected until next year.

Mrs Kellett-Jones, a keen horse rider, has a lung capacity of just 25 per cent, making everyday tasks very difficult.

She depends on a variety of inhalers simply to speak and needs 20 minutes to recover after a shower.

Her husband Phillip Jones, 46, wants the Royal Victoria Infirmary in Newcastle to reconsider and warned that the couple may seek a judicial review.

They are expecting their first child in June through a surrogacy as Mrs Kellett-Jones is too ill to carry a baby herself.

Trikafta is already available in the US and has been submitted for safety and clinical analysis with the European Medicines Agency.

Its maker Vertex claims it is ‘almost a cure’ for cystic fibrosis and it is effective for 90 per cent of patients. 

But drugs available on the NHS – Orkambi and Symkevi – work for only 50 per cent of sufferers. Mrs Kellett-Jones, of North Cowton, North Yorkshire, said: ‘I’m not eligible for a transplant due to the complex bacteriology that grows in my lungs so I was really hopeful about this breakthrough drug.

‘I was so disappointed to hear the Royal Victoria Infirmary, the hospital I have been attending for 20 years, wasn’t going to apply to the drugs company for compassionate access despite many, many other hospitals doing so.

‘Vertex were making the drug available for patients with less than 30 per cent lung function on compassionate grounds.

‘It’s just really sad that other people with cystic fibrosis who have been given Trikafta are really thriving and the patients at the RVI have effectively been left out.

‘We don’t have time to wait and are deteriorating all the time.

‘It would just be nice to think that the doctors who are meant to be advocating for their patients were able to do so.’

Mr Jones, an equine surgeon, said the couple could be left with no option but to ask to transfer to a hospital in Leeds where the drug is available. ‘It is a cruelty to withhold this drug from people who need it so desperately,’ he added. ‘It defies any kind of logic.

‘This is a life-changing drug that has seen people go from an intensive care bed to leading a normal life.

‘It’s transforming the lives of sufferers around the world. It’s available all over the UK but not to us. This affects dozens of patients and some are extremely ill.

‘Megan has become a professional at hiding her suffering from almost everybody apart from those closest to her.’

He claimed: ‘Six months after other hospitals started applying for and receiving the drug for free, RVI management are still denying their patients access.’

A hospital spokesman declined to comment on an individual case but added: ‘Making decisions about requests for compassionate use of an unlicensed medicine is very complex.

‘We endeavour to make these as ethically and fairly as possible with clinical and cost effectiveness taken into consideration.

‘Clinicians can make individual requests for a specific drug if they believe the patient’s circumstances are exceptional and they would gain clinical benefit from having the treatment and such requests are considered on a case-by-case basis.’