Lorraine asks "Why are we still waiting for Orkambi?"

Cystic fibrosis is a life-limiting condition that makes it difficult to breathe, causes vulnerability to infection and restricts your ambitions. Medication, physiotherapy and general health all have to be taken into account when planning the simplest activity, and being unwell frequently interferes with work and education.

Conventional CF treatments target the symptoms of CF. Precision medicines like Orkambi tackle the cause of the condition. Though Orkambi is not a cure, it has been found to slow decline in lung function, the most common cause of death for people with CF, by 42%.

Lorraine's story

Why are we are still waiting to get our hands on Orkambi? Every step towards getting it approved seems excruciatingly slow for families like mine desperate to grab a hold of something which might make a massive difference to the lives of our children. Time is ticking away and Cystic Fibrosis being degenerative by nature means it's not waiting for anyone. The health of both my sons has deteriorated considerably and it's very scary to think of a future for either of them.

Joseph

Joseph had surgery for a fundoplication procedure in October 2016 and since then his life has just become one round of treatments and even more horrible complications. He can no longer be sick, but he can retch when he feels sick, and he does it a lot. He suffers from terrible wind problems now and eats very little. The overnight feed was too much for him to begin with but that is slowly getting better. He has feelings of fullness when he's eaten barely a mouthful of food and meal times are a real battle to encourage him to eat just the tiniest amounts of food. Most days he eats nothing at all and his overnight feed is what is keeping him going. His weight gain since the surgery has been none existent.

We take one tiny step forward and then 10 steps backwards as soon as Joseph picks up a bug like a cold which he did a couple of months ago. His lung function now averages 38%. Imagine living a life aged just 14 years old with half lung capacity. He gets breathless just walking up stairs on really bad days. He has a poor quality of life for a child his age who should be out enjoying himself on his bike, walking, running and eating properly.

Joseph has one round of treatments after another, before and after school, which can last up to 2 hours. He has a ventilator now which blows air in to his lungs to open up the airways to make mucus clearance easier. He nebulises 3 drugs, twice a day - DNase to thin the mucus, hypertonic saline to help shift the mucus, and nebulised antibiotics. He has Insulin due to having CF related diabetes and Ursodeoxycholic acid for liver disease. He takes vitamins for bone density: D3, D, and vitamin E. Creon with all his food and overnight feed, probiotics as well as Domperidone for sickness. He has to endure various forms of physio such as percussion, acapella or PEP. He has nasal polyps so he needs spray up each nostril once a day. He has to have IV antibiotics every 3 months for 2-3 weeks at a time. His growth is poor and he is noticeably small for his age.

And yet he never complains but I know he hates CF and all it entails. He just wants to live a normal, happy, pain free life.

Daniel

Daniel, aged 22, has been undergoing treatment for a mycobacterium infection since September 2016. This began with 3 weeks of intensive IV antibiotics followed by 18 months of 3 different oral antibiotics a day along with various vitamins. He too has to endure weeks of IV antibiotics if he picks up a cold or chest infection. His lung function is around 66% but each set back could mean a worrying slide in his health.

Both of my sons are handsome, intelligent and popular young men. As their mother I want them to live a long, happy and healthy life. I fear for their futures so much now, the older they get the more care they need, the more medication they need, the bigger the worry. Cystic Fibrosis causes so much stress, not only to the sufferer, but to their family and friends who look after them and every infection my sons pick up these days leads to less and less lung function.

Orkambi, to us, would mean less worry, less stress, a better, more secure future. Better health would mean so much. I know Orkambi doesn't work for everyone but quite frankly, when your children are as ill as mine now are, I'm happy to grab hold of even the tiniest glimmer of improvement. I'd like to personally challenge any of the decision makers for approval for funding of Orkambi to come live with me and my family for 1 week. Experience what it's like to juggle all the medication my sons need daily. Their prescription list is vast. Try drawing up IV antibiotics for 3 weeks and administering them 3 times a day knowing how sick they are going to feel, but that their lungs are priority. Try listening out for the overnight feed pump alarming all night long even on nights it's not alarming because your brain is tuned in to listening out. Try changing soaked sheets when the overnight feed line disconnects. Try watching your 14 year old retching because he can't be sick, but he can feel sick, watching mucus and spit coming out of his nose, this sometimes ruptures a vessel in his nose which makes his nose bleed. Try cleaning up blood after a serious coughing fit following a lung bleed. Think how you'd feel watching your sons suffer every single day knowing you can't do anything to stop the decline no matter how many drugs you pump in to them, how many hours of treatments you do with them - because Cystic Fibrosis by nature is degenerative. Cystic Fibrosis makes you feel useless.

I joined the Cystic Fibrosis Trust in London to protest for Orkambi for my sons, for all those living with Cystic Fibrosis and for all the friends I've lost to this dreadful condition. Cystic Fibrosis is NOT just about Physio. It’s time more was known about it and all it entails because it is still robbing us of our young friends and I've lost countless over the past few years. Now is the time to do battle and to fight for the right to have access to these drugs. My sons lives are priceless to me and I'd gladly give my life for them both if it mean't them having a long, happy life, to see 50 and beyond.

Click here to sign the petition

In July 2016 the National Institute of Clinical Excellence (NICE) recognised Orkambi as an ‘important treatment’. They were, however, unable to recommend the drug for use within the NHS on grounds of cost effectiveness and a lack of long-term data.

The CF Community are calling on the British Government to call for a resolution to ongoing negotiations between Vertex Pharmaceuticals, NHS England and NICE as a matter of the utmost urgency. It is essential that a fair and sustainable agreement is found.