Monday, 10 December 2012


Many of you will have heard me mentioning about Kalydeco in previous blogs but a lot of people still have no idea what it is or how life changing it can be. So I thought I'd do a little blog on what it is about and the problems we are facing with getting Kalydeco.

Kalydeco treats the cause of's caused by two faulty genes and there are many different mutations on genes. Kalydeco works with gene G551D which is one of the genes I have! In America this medicine became approved and has been used since January. The problem we are finding in the UK is that it is VERY costly; approx £180,000 per person per year. The drug company (Vertex) aren't wanting to reduce their selling price but the Government wont provide any extra funding for this new drug & are therefore saying that people can't have it. NOTHING like this has ever come about for CF before - it is by no means a 'cure' but it will improve quality of life dramatically. What is the point in spending billions creating a medicine that in the end wont be used? The NHS & drug committees need to agree the funding. All the treatments we take for our CF treat the consequences of our illness whereas Kalydeco modifies the genes..this tablet fixes the fault that causes the problems.

At the moment everyone involved is meeting mid December to discuss the matter further.. They said in September they would make a definite decision but put it off for another few months..they cannot keep doing this. Some people desperately need this drug & do not have a 'few more months' to spare!

The improvements this drug can make is incredible. I have a few friends who have been giving this drug on compassionate grounds, either they had lung function below 40% for over 6 months or where on the transplant list. These people have come off permanent oxygen, stopped relying on wheelchairs to get about because they're too out of breath, been fitter and healthier. If Kalydeco is going to improve someones life THIS much then it is insane not to give it to the moment it feels as though they're dangling a thread going 'oo look at this lovely thing you could have that will basically stop you from having more permanent lung damage and dying, oh wait no you cant have it!' 
My doctor was saying it is SO frustrating as it is just right there yet we have to continue waiting.

This blog is essentially to help gain a little more awareness of what Kalydeco is, the problems we're having and how amazing it can be! Having to focus ALL the time on how you're breathing and noticing each breath as each one is a slight struggle even when healthy can be so mentally and physically draining but us CFers carry on fighting in hope that this drug will become available and help us.

PLEASE share this & tell people. If the decision comes through from the next meeting that we can have Kalydeco then it'll be life changing but if the decision comes through that they are NOT giving it then we will be pushing towards the press and will be doing everything we can. So until that day comes there is no more we can do but raise awareness and sit here and wait.

Below are a few links to either blogs of those who have had Kalydeco & you can see their amazing changes or links to newspaper articles

1 comment:

  1. Good to be aware of these things! I hope the right decision is made and if not I'm sure you will have plenty of support until it is. K xx