Last Sunday I was devastated to hear that Ireland had declined a potentially life saving drug to help the majority of cystic fibrosis patients. Why? Cost. This is the first drug EVER to address the root cause of CF so it is potentially life changing. Up until now all CF associated symptoms have been managed and treated, not stopped. This drug, Orkambi, corrects the basic defect, therefore, treating the underlying cause. For me and my one year old daughter who is eligible for this medication, this is everything. If her decline in lung function could be stopped and she could live a full, long, happy and healthy life then how can a price be put on that?
Jillian McNulty, a leading campaigner for the drug, penned this open letter to highlight the importance of approving these drugs for all suitable CF patients…
‘I’ve just sent this letter to Tony O’Brien & NCPE… I feel like saying so much more, I’m angry, I’m heart broken, I’m frustrated, I’m crying as I type. My life can’t go back to what it was, it just can’t…We need to get Orkambi VIRAL. SHAME them.
To the NCP/HSE…..How much is my life worth? Why put a price tag on my life?
Orkambi. To most people this word means nothing. To anyone connected to the Cystic Fibrosis world, it’s the pot of gold at the end of the rainbow. Two years ago I was one of 8 patients over 14 in Ireland to be chosen to take part in Phase III of Orkambi trials. The day I got the phone call, I cried, I then smiled so hard my jaws hurt. I had won the lotto, I may not have had millions in my bank account, but I was being given a chance of LIFE, I had won the health lotto.
In the few years previous my Cystic Fibrosis had been in a sharp decline, my lung functions had a massive 20% drop in the year before Orkambi. And while I was as active as I could be; running, cycling it took its toll on my body, a 2km run took me treble the time it took a healthy person, I needed naps to get through the day, life was a struggle, I could never plan anything more than a week or 2 in advance because CF would always get in the way. My hospital admissions were every 4-5 weeks, most of the time I ended up in hospital for a minimum of 6 weeks but my average stays were 7-8 weeks, but at times they lasted up to 15 weeks, I had spent most of my year in hospital, with bad exacerbations came horrendous symptoms. Pre Orkambi I managed, I pushed through, my stubborn nature made sure of it. If you’re healthy person reading this, I bet you never give a second though to washing your hair or having a shower? Something so simple but a daily necessity became such a painful battle within my body when I had an exacerbation. Taking a shower at times became so difficult it hurt, I needed a chair to sit down on, my whole body ached as I gasped trying to take in as much breath as I can to actually function. For months at a time I didn’t have the energy to wash my own hair, spent a fortune in the hairdressers as I wasn’t physically capable of washing my own hair.
Then came Orkambi, within a few months, my body started changing, during my runs I could breathe deeper, it’s not something I can explain correctly because I don’t think I’ve ever known what it’s like to breathe properly. But I was no longer coughing & spluttering so much after running, gradually my need for naps became less and less and one day it just hit me that I hadn’t napped that week at all and I was like OH MY GOD Orkambi is working, Orkambi is changing me, Orkambi is curing my body. Suddenly I had boundless energy and little things no longer took it out of me! My hospital admissions were happening every 3 months, while I still didn’t plan I was hopeful things were changing. Fast forward to December 2015, I have been out of hospital for 11 months, I’ve had no decline in over 2 years, I am physically stronger, I can now plan things months in advance. I no longer need daily naps to function. I have a life, a life that doesn’t revolve around CF & constant hospital stays. I feel “normal”. No one can argue with these FACTS. Orkambi has transformed my life.
Why am I writing this? While I have known for a long time now how much Orkambi has transformed my life, this week I got a very unequivocol reminder how quickly things can change. Last week I officially finished phase III of the trial, which means I would gain access to Orkambi on an open access programme until it becomes available in Ireland. However I am still waiting for the marketed dose to arrive, I have been without Orkambi for just over a week and I am struggling, really struggling, my life is back to 2 years ago, I am extremely breathless, even walking a few steps exhausts me and brings on coughing attacks while I try to catch my breath. My lungs just feel heavy again, chest physiotherapy is getting harder by the day, mucous isn’t moving the way it has been on Orkambi, I began having these symptoms within 24 hours of being off Orkambi, I am fearful that I will end up in hospital. This is heart breaking as I am almost a year out of hospital, the longest period of time I’ve ever had hospital free since my hospital admissions began. And my fear is the longer I am without Orkambi the worse I will get. I know I will have it within a few more days so I will be ok. But for those who haven’t even had the opportunity to give this drug a try, the wait for them is agonising while the battle for recommendation/reimbursement continues.
So, can someone please tell me why my life and the lives of other CF patients in Ireland have a price tag on them & why we have to endure a harrowing wait while a decision that will affect our lives is being made? The news today that the HSE has ruled out funding for Orkambi is utterly devastating. There are people that desperatly need to start this drug now, they are desperatly clinging to the hope that one pill may help them live a near normal life without CF getting in the way, a chance to perhaps stop progression in it’s tracks and get to spend longer with their families & loved ones. We are young generation just wanting to have a quality of life we deserve, CF is a cruel unforgiving illness and when there is an opportunity to acquire a drug like Orkambi it should be fast tracked and made available to patients within a few weeks of European approval, CF will not wait for a decision to be made over months and months, CF will progress while people are made wait, CF will steal more lives while the decision to put a price tag on our lives is being discussed. I’ve seen first hand the difference one pill can make to my life, I now have a quality of life and quality of life is priceless.
Cystic Fibrosis Campaigner & Fundraiser.’
Ireland’s refusal now makes it very easy for the NHS in England to follow suit. As Jillian says we need to fight and get this drug to patients now as time will sadly cost lives. The drug has been approved in America and Germany. Patients are already benefitting. We need to follow suit.
When the time comes – and it is coming – I hope to be able to count on YOUR support in shouting very loudly about this to all and sundry!
To read more about Orkambi click here.
To read more about Cystic Fibrosis click here.