Mum begs pharmaceutical company to give her son
So today, Lorraine is making a plea directly to the drug company Vertex to grant her dying son Trikafta now, on compassionate grounds.
「Vertex, if you have any compassion at all, you won’t let my amazing son die,」 Lorraine says. 「Help me get Trikafta for Joe… I don’t know what else can help him now.」
I first spoke to Lorraine and Joe two years ago in March 2018 when they were fighting for the life-changing drug Orkambi.
Joe was 14 and extremely unwell. Orkambi wasn’t available then on the NHS because of its hefty price tag, and the Barnes family were among those fighting a three-year battle to get Vertex, NHS England and NICE to agree a deal so it could be prescribed in the UK.
The Orkambi campaign was eventually successful, with the drug finally made available in the UK last October, but campaign group Just Treatment believe up to 200 people may have died waiting for the row to be resolved.
Orkambi helped Joe for a while, but he is now extremely ill again, and all the evidence suggests Trikafta could help. So now his exhausted family are facing another David and Goliath fight for a tiny pill that could save their son’s life.
Currently, there is no cure for CF and half of people in the UK with it die before they are 32. The life-limiting condition clogs the lungs with sticky mucus as well as affecting other organs. But in the very few cases in the UK where Trikafta has been made available, they have had almost miraculous results.
「Joe is too poorly for a lung transplant,」 Lorraine says. 「He can’t go to school, he gets breathless just going upstairs. He’s suffering with chest pains, coughing up blood, he can’t gain weight, he’s depressed. He’s gone through more in his 16 years that most people will go through in a lifetime.」
Lorraine says Joe’s doctor is in the process of applying for compassionate Trikafta for her son and seven other patients, but that the paperwork is prohibitive.
「It took his doctor 11 hours spread over six weeks to fill in one form,」 she says. 「If there are eight to apply for that will take 48 weeks to get through them all and then it’s up to Vertex who gets the drug.」
Trikafta campaigners know the drugs are expensive, but say the cost is nothing compared with the cost to the NHS of looking after a child going through end-stage CF.
CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. Treatment can easily take four hours a day including physiotherapy, and patients can be on up to 70 tablets every day.
A spokeswoman for Vertex said: 「Triple combination therapy is not yet licensed in the EU, and we are restricted by law in how and to whom we can supply this potential medicine in a compassionate use programme.
「Due to the severity of cystic fibrosis and the high unmet medical need of CF patients, we voluntarily provide some of our unapproved medicines to patients, if requested by their doctors, via a 「compassionate use」 (CU) programme and under strict conditions in line with the law.
「Making decisions about requests for compassionate use of an unlicensed medicine is complex. We endeavour to make these decisions as ethically, fairly and quickly as possible, in line with the various medically driven clinical criteria, information about the potential medicine as well as the patient’s overall medical circumstances.
「To date, over 550 patients outside of the US have been approved to receive CU triple combination treatment including over 170 patients in the UK. We cannot lawfully comment on a specific patient’s circumstances.」
A spokesman for the Department of Health and Social Care said: 「Cystic fibrosis is a terrible condition and we will never underestimate the bravery of the people fighting it.
「The deal we struck with Vertex for Orkambi is already transforming hundreds of lives. We urge Vertex to help as many people as possible access Trikafta on compassionate grounds ahead of licensing.」标签：