Over the last few weeks we have had a few people mention to us about the news they have been seeing on the tv, the drug orkambi.
Well it does, and with Stanley’s gene type being double F508DEL he is infact the perfect match for the drug to work!, Cystic Fibrosis is such a complex condition which can almost make it difficult to explain exactly what it is to people. but putting it in basic terms the faulty gene affects the way salts and water move around the body causing a build up of thick sticky mucus this then clings to the lungs causing infections blocks the pancreas meaning enzymes are needed to help digest food, and in 97% of men with the condition leaves them unable to have children naturally.
Orkambi works by allowing the protein within the cells to rise to the surface to move salt in and out of the cell, keeping a healthy balance of salt and water in the lungs and other organs, and so far research has shown that it reduces outbreaks of infection, and can cut the number of hospital admissions by more than 60 per cent. Trials took place involving 1,100 CF patients in Europe, the United States and Australia found lung function improved after 24 weeks in ALL patients taking Orkambi.
sounds almost to good to be true? well there’s a catch.. it costs £104,000 per patient per year in the UK and after NICE assessing there criteria it was deemed to be not cost effective. I’m sure people can imagine the frustration for patients living with the condition and parents of children with CF when your basically told that there’s something that could potentionally help your child but there not allowed to have it. Putting it bluntly they are effectively putting a price on people’s life’s. They are putting s price on OUR child’s life. I can only imagine the anger that causes the people living with this awful condition themselves day in day out. Imagine hearing on the news everyday people discussing the life you yourself has to live, the treatments that have to be endured each and every day, endless medicines,physio,hospital stays.. discussing a drug that could help even if only a little bit and then having all hope taken away because of a price tag and a battle between companies.
Companies are now in talks in the hope that they can come to an agreement in which the price is reasonable and makes it more accessible for people with the condition to have it. Tomorrow there will be a debate in parliament to decide ultimately what will happen. The truth is some people that are living with CF won’t have the time to wait for something better to come along orkambi is the only thing they have left to fight for in order to keep them well for now. Time unfortunately is not on some people’s sides.
Obviously to us as parents there is no “reasonable price” how can you put a price on someone’s life and the quality of it? In all honesty we’re not even sure if it’s something Stanley would be able to have in the long run at the moment, it’s not something that has come up and we’ve not spoken to his team about it, however the fact that there are drugs shown to work being rejected that could offer help to people living with this condition.. well it can be very frustrating, it almost seems like it’s pointless in doing trials If they just can’t get past the last hurdle and get the drugs into the NHS. These drugs need to be made more accessible.
But it’s not just a drug there taking away – it’s hope, I believe our little boy has been born at the best time he ever could should you ever have to be born with cystic fibrosis, the trials and research taking place is unlike anything that’s happened before, new developments are taking place all the time, new names of drugs coming up, new treatments and advances. I believe if not a cure,then a drug that helps to lead a normal life has got to be made available in the near future. It’s all so close yet so far away. In some ways it just seems like another cruel twist – people are literally being made to fight for the right to live and lead a “normal” life and it’s wrong.
we as parents desperately hope that drugs like orkambi and any future breakthroughs in trials are made more accessible for people with CF. Orkambi has got to be just the start, bigger and better things have got to happen but whilst orkambi is the best thing around it needs to be made available.
I recently read a quote.. “we shouldn’t have to fight for our children’s life’s, but believe us if you make us that fight will be relentless” these words could not be more true.
Our promise to you Stanley will always be that whilst you have breath in your body, me and daddy will fight til our last breath for yours. The purpose of our lives will always be to protect yours. We along with every other CF parent out there won’t give up without a fight #yesorkambi and yes to finding a cure. 💜
More than ever the fundraising we and so many others will be doing for the CF trust will help push towards finding that all important breakthrough for Stanley and for others living with CF. So please take the time to donate anything you can – ❤️