Holding our Breath

Lorcán is now over 10 months old. As his birthday approaches and I start to think about the sorts of gifts that would be best for a child with cystic fibrosis (woodwind instruments, bouncy toys, climbing frames) I’ve started to reflect on what a strange year it’s been. I’ve looked back on pictures from this time last year when I was pregnant, when the blood test showed no likelihood of the conditions it looked for, when the 20 week scan showed a perfect baby, who we discovered with excitement was going to be a boy. By that point we had started to relax, it was all going to be ok. To be honest I don’t think I’ll ever get over the shock of the diagnosis of cystic fibrosis so soon after he was born. I still think back on it with a cold horror.

I will never take anything for granted again, and nor should you, because this could happen to anyone. With one in 25 people a carrier in Great Britain and 1 in 19 people a carrier in Ireland, babies with CF will continue to be born. When they are wouldn’t it be so incredibly wonderful for their parents to hear, ‘your child has cystic fibrosis but there is a precision medication available for them to take when they’re old enough that will slow the decline in lung function by up to 42%’ rather than, ‘the drugs available on the NHS will only help alleviate the symptoms of CF, they won’t address the cause’? Currently Lorcán is not eligible for Orkambi, only those who are 12 and over can take it, however trials will soon begin for two year olds. Imagine if it was available when he turned two! If we could just try to keep him healthy(ish) until then, it’s only another year and a bit! However nothing is moving quickly and unless negotiations open up it won’t be available when he’s two, and maybe not even six, I can’t bear to think that far ahead or beyond, it hurts too much.

I am lucky that Lorcán is so young and currently so healthy, but what about the people who so desperately need Orkambi now? The people who are heading towards lung transplants that they may not be able to have, the people with children of their own that they may not live to see grow up. Orkambi is not a cure but it could give these people years. It’s also a bridge between what we’ve got now (which can be painfully insufficient for those who suffer the most) and the drugs that are currently being developed that will help 90% of people with CF, Orkambi can only help 40% of people with CF, those with two copies of the most common gene like Lorcán. So it can only slow lung decline by up to 42%, so what? Tell that to the people who only have a handful of years left to live. What’s the alternative? There isn’t one.

The Cystic Fibrosis Trust is striving for a ‘portfolio’ approach to reimbursement with Vertex Pharmaceuticals that will guarantee access to Orkambi now as well as incorporating future drugs as part of a long-term deal. Just across the border in the Republic of Ireland a deal like this is in place. This kind of approach could ensure that future treatments can be accessed more quickly within the current system. This would reduce the cost of Orkambi, allow more assurance for fiscal planning in CF in the future, and create a brighter future for Lorcán and thousands of others like him. Supported and driven by the Cystic Fibrosis Trust, people all over the UK have been campaigning for this. If we get a deal in one part of the UK, it will put pressure on the other parts to follow suit, so the fight for one is a fight for all.

In Wales there is a petition initiated by Rhian Barrance, a dear friend of mine. This petition to the Welsh Assembly calls for a resolution to negotiations between NHS Wales and Vertex. If the petition gets over 5000 signatures it will automatically be considered for a debate at the Welsh Assembly. Anyone in the world can sign it so please do- https://www.assembly.wales/en/gethome/e-petitions/Pages/petitiondetail.aspx?PetitionID=1266

In Scotland a debate is anticipated at the Scottish Parliament to discuss Orkambi and the wider issue of access to medicines in Scotland, in relation to the Montgomery review.

In England there was parliamentary event held on Wednesday 29 November in the Palace of Westminster.

As for Northern Ireland, well, the CF Trust drafted a cross-party letter to Jeremy Hunt and Richard Pengelly calling for a timely resolution to the ongoing negotiations between Vertex and Health and Social Care Northern Ireland. I am pleased as punch to say that, following a deluge of requests from campaigners, 66 MLAs from a number of different parties have signed it. We are currently waiting for a response.

There have been many ups and downs throughout these ten months but one of the things that has helped immensely has been seeing the person Lorcán is growing into every day, a funny, sweet, clever little person who currently has absolutely no idea that he has a life-limiting condition. He doesn’t know that in 2016 the median age of death across the UK for those with CF was just 31 years of age. Other people, sadly, do. The families of those that have gone before and the older people living with CF, who with every year of approaching that number find it more and more difficult to breathe, these people are more than aware.

The picture attached to this blog is of my little merman holding his breath under water. We’re all holding our breath for Orkambi. Please help us break the surface, end the deadlock, and make Orkambi available now. What a wonderful birthday gift for Lorcán that would be, I can’t think of a better one.


C7EFAE92-11AB-4BFC-9B72-DA5CD132C8DELorcán at Little Flippers, photography by Stephanie Williamson.