Orkambi and Vertex: the horror of hope

Your child is diagnosed with the life-shortening, life-limiting, chronic illness that is Cystic Fibrosis. You’re told that your child’s life expectancy is early 30s, the age you are when you receive this punch-in-the-gut news. The median age of survival in Ireland is only 27, YOUNGER than you are at the time. You want to vomit. But you’re also told that now is the best time for a child with CF to be born as we’re entering an unprecedented age of progress in treatments. And that is what gets you through – you cling to this glimmer of hope with gorilla glue fingers; it becomes everything to you.

I remember sitting on the couch on the evening of Eva’s Diagnosis Day, my heart on the floor; feeling empty, hopeless and so very lost. The Six One News was on in the background and a piece on a breakthrough CF drug from Vertex Pharmaceuticals came on. The drug was called ‘Kalydeco’. But the NCPE had advised against funding it. So had the HSE. And the CF Community was fighting for access. It was the first drug to treat the underlying cause of the condition for people with the G551D gene, the reporter said.

I decided the timing was a sign. A sign that my child would defy all the odds and stats because she was born in the year of Breakthrough A.D. She didn’t have the genetics that Kalydeco treated, but surely this drug was only the beginning. This ‘sign’ became my lifeline. I was convinced that she was diagnosed on the day this was all over the news for good reason. This was my reassurance and this was to be (and still is) my drive every single day of her life. A miracle was within reach – I could feel it, I could smell it, I could imagine it. I wouldn’t have to bury my child a quarter way through a lifetime – she would bury me as children are supposed to bury their parents. My husband would walk her down the aisle. She would have children of her own. CF was not a death sentence. Not any more. This news report was the only reason I could pretend to deal with her diagnosis. It was the only reason I could face the new reality we found ourselves in.

Kalydeco was finally approved when Minister James Reilly bravely overruled the funding recommendation and today, the people who are on it confirm it is utterly life-changing. His decision has improved and extended the lives of those patients and I can’t wait to see how many more years it adds to their lives. It’s the most inspiring medical breakthrough I have ever seen. (Of course I am slightly biased.)

Fast-forward almost four years and the CF Community is again fighting for access to a breakthrough drug, from Vertex once more. This one is called ‘Orkambi’. And a lot of history is repeating itself: once again my daughter doesn’t have the genetics it treats (this one is for ‘double deltas’, the most common genetic combination). Once again the NCPE and HSE have recommended against funding it (the final HSE ruling is due on December 12th I believe). And once again, the CF Community finds itself fighting for the lives of its family and friends.

So many things have changed for me personally though. I’ve lost my naivety and innocence. I’ve now seen the lows of CF first hand through my daughter’s exacerbations. I now know the people in the community who are fighting. I’ve met so many of them. I speak to a lot of them daily. I feel their pain on an extremely personal level. And this time, I’m fighting alongside them.

I feel sick when I see parents having to beg for medical breakthroughs for their children. I’m horrified at the idea that people who are on Orkambi from the trials aren’t guaranteed that they can stay on it for life. I’m disgusted at the way the HSE and Vertex have engaged in public mud-slinging of each other’s negotiation tactics, leaving patients drowning and terrified in the middle. Leaks to newspapers. Downplaying of the drug in interviews. Scaremongering that makes patients feel like they have to air every detail of their difficulties in public to make the case for why their lives matter. And of course I’m petrified that if this drug doesn’t get through, that I’ll never see a drug to treat my own precious little lady.

Is the HSE the big bad wolf for not paying for it? Is Vertex the big bad wolf for demanding a huge price tag? Are they both as bad as each other? Surely it’s not impossible to reach a fair, middle ground? We face a world where our knight in shining armour, Vertex, the company that could save our children’s lives, is also painted as greedy and immoral. This conflict is incomprehensible. We love them. We hate them. But boy do we need them. Regardless of how any of us feel or who we ultimately blame for this debacle, it doesn’t change the fact that patients are getting sicker while a drug that could change their lives sits on a shelf somewhere other than in their pharmacy.

My daughter is nearly 4 and is only starting to ask questions about her CF. But how is this circus affecting teens with CF? How is it affecting young adults? Facing the worst of their illness blasted all over the media. Finding their lives discussed in monetary terms of what they are ‘worth’. I can’t begin to estimate the psychological damage that this storm will leave behind. Every single one of us is being damaged by the cruel game we’re caught in the middle of.

I truly feel for the people waiting for this drug. And I truly feel for those who don’t even have a drug to fight for yet, ourselves included. Our groups may have different genetics and be facing different waits for new therapies, but one thing unites us all: we’re all clinging to hope. And it would seem that hope is perhaps the cruellest feeling of them all.


Image courtesy of the #YesOrkambi campaign, photographer: Simon Burch. The beautiful little girl featured is not my daughter – but she is one of the little ladies who deserves to try Orkambi.