Woman’s fight to see ‘miracle’ drug available on the NHS

Charlotte and Michael Joslin will celebrate their first wedding anniverary next month.

Charlotte and Michael Joslin will celebrate their first wedding anniverary next month. - Credit: Archant

A woman who suffers with an incurable genetic condition is taking part in a drug trial which she says is having “miraculous” results.

Some of the equipment Charlotte needs every day.

Some of the equipment Charlotte needs every day. - Credit: Archant

Charlotte Joslin, from St Ives, has cystic fibrosis, which means she needs daily physiotherapy and a nebuliser, to clear the constant build-up of mucus on her lungs, as well as taking up to 50 tablets, including large amounts of intravenous antibiotics.

This exhausting routine takes between three and four hours and Charlotte relies heavily on her husband, Michael, aged 34, for practical and emotional support.

The couple will celebrate their first wedding anniversary next month and when Charlotte’s health deteriorated at the end of last year, Michael gave up work to become her full-time carer.

“When I am poorly it can take five or six hours to do everything and Michael helps me, as sometimes I am too tired. My step-son, who is 12, is amazing and has also adapted so well too. He even helps when I am unwell or when I am in hospital. I am truly lucky to be blessed to have such an amazing supportive family beside me which also includes my mother and father-in-law.”

Charlotte also has osteoporosis and a fracture in her lower back and has developed hearing problems due to the side-effects of such large does of medication and now wears hearing aids. In October 2016, Charlotte’s lung function dropped to 30 per cent and she spent two months, firstly in Hinchingbrooke Hospital and later at Papworth.

“That was the lowest my lung function had ever been and, if it had gone any lower, they would have started to talk about a transplant,” she explained. “But luckily there is a new drug out called Orkambi, although the NHS is not funding it. It’s like they are putting a price on people’s lives.”

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The NHS will only provide the drug in “rare cases” and “on compassionate grounds” and Charlotte has only been able to receive it as part of a trial.

Orkambi is what is known as a precision medicine, which means it treats the underlying genetic mutation that causes the condition rather than just the symptoms, which can have a huge impact on the sufferer.

“Since being on this new drug my lung function has gone from 30 per cent to 74 per cent, which is the highest it has ever been,” adds Charlotte. “I would normally have intravenous antibiotics every two to three months for two to four weeks but I haven’t had any since last December. It’s a miracle drug and I am so glad I got to start it. I just wish I hadn’t been so poorly to be given it in the first place.”

Charlotte is urging the NHS to make the drug widely available for cystic fibrosis patients and has joined the campaign to put pressure on the government and the drug company.

On June 26, the Cystic Fibrosis Trust held a day of protests across the UK calling on Vertex Pharmaceuticals, the NHS and the governments of England, Scotland, Wales and Northern Ireland to speed up their negotiations and reach an agreement on Orkambi.

On its website, it says: “Despite having been licensed for use in the UK for people with CF over the age of 12 who have two copies of the F508del mutation, NICE rejected its use on the NHS. It is currently only being prescribed to people on compassionate grounds from just 25 CF centres.

Compassionate use means that Vertex Pharmaceuticals (the manufacturer of Orkambi) has an arrangement in place allowing people who are seriously ill to take the drug free of charge. There are certain requirements that have to be met for compassionate use to be considered.”

According to the 2014 UK Cystic Fibrosis Registry Report, there are 2,834 people in England, 243 people in Scotland, 118 people in Wales and 101 people in Northern Ireland who could stand to benefit from Orkambi, a total of 3,296 people.

More information about the campaign to persuade NICE to fund the drug is available at: cysticfibrosis.org.uk.