Although no cure, Orkambi could transform my world, with the potential to half the number of seemingly endless days in hospital having intravenous antibiotic treatment, improve my long-term health and help me in my fight to live a normal life, a life unlimited by cystic fibrosis.
I told NICE how I feel CF takes control of my life: the gruelling and often unplanned two weeks at a time in hospital having antibiotics pumped through my body, never knowing what the next day will bring for my health and being unable to plan ahead.
I described the physical and mental burden of my daily treatment: up to 50 pills, nebulisers, inhalers and punishing physio, all before I can even think about starting my day and on top of the many challenges that life brings for all of us.
I underlined that being 33 with a median life expectancy of 40, the prospect of fewer of the infections that disrupt my life and damage my long-term health would mean so much to me.
I explained how although the 3% increase in lung function Orkambi has been shown to provide may not sound like much, I don’t know many CFers who would turn their nose up at it. And I would be really worried if my lung function dropped by that much.
With negotiations yet to take place, we don’t know how much Orkambi will cost the NHS. But we know it won’t be cheap. So the big question is, is it worth it? Well of course I think I’m worth it (L’Oréal told me so)!
Some say better treatments may arrive. Well I and thousands like me don’t have the luxury of time. What’s more, a failure to invest in treatments like Orkambi threatens further investment in new treatments for cystic fibrosis.