The debate raises so many interesting points, not least the question of what price you can put on a life? It also highlighted that middle ground that CF exists in; it is neither so rare that a high cost drug in a small population would be affordable, nor so common, like acute conditions, where a large population drives down the cost per patient.
What is apparent, is that NICE must change the way in which they perform cost benefit analysis on drugs for chronic conditions: Every night we spend on the ward costs the NHS more than £400, and in PICU, more than £2000; more than one of Isaac’s daily drugs cost £8,000+ per annum... and at every single clinic visit he requires the care not only of his CF nurse, his CF consultants, but also a dietician, physiotherapist, psychologist and the pharmacist. He has also at various times been under the care of ENT, the Gastro team and Immunology, all related to his CF symptoms. He has multiple tests each year, including X-rays, CT’s, ultrasounds and bone scans, not to mention the regular bloods and sputum samples the lab deals with..... what this adds up to I can’t imagine.
.... Orkambi has been proven to reduce admissions to hospital and reduce the decline in lung function by 42%. NICE agree the drug is effective, but not cost effective for the NHS (the original offer was over £100k per patient, per annum). I know none of this is simple, but the debate suggests to me that NICE is not fit to make that decision as they don’t fully understand the bigger picture in chronic conditions.
There are around 70,000 people with CF in the world, and the U.K. and Ireland have a much higher prevelance of the condition than normal (10,400 in the U.K. alone). Orkambi is available in Ireland, but not in England. Government intervention between NHS England and the manufacturer, Vertex, is urgently required. I have no doubt an agreement will now be found, but everyday it is delayed, children are losing their lives. Half of sufferers will lose their life to CF before the age of 31 and imagining how those at that end stage of the spectrum feel about this delay is simply crushing. Yes, the cost is high, and it’s easy to blame that on the pharmaceutical company, but the portfolio offer to NHS England (Vertex have newer, more effective options to Orkambi already in development, and this portfolio would guarantee future access to these drugs also) is better than deals made in other counties that are already prescribing Orkambi.
I’ve said before, Orkambi will not work for Isaac, nor will the next drug in the pipeline, Symdeko, nor even the one after that, as he has a rare and shitty mutuation of CF which will require a different kind of precision medicine to enable the correct movement of salt and water through his cells (the root problem in CF) but that doesn’t mean I’m not hugely passionate about access to the drug, and we are super excited about the paths it will open for further research that may one day help him, and move CF into a category like diabetes; a condition that you live with, rather than one that you die from.
In the meantime, have I mentioned lately that I’m throwing myself out of a plane soon? Everyone with CF deserves the chance of a longer, healthier life. Please support our skydiving fundraising by clicking here. Thank you x
