Yesterday I attended NICE’s final appraisal to discuss Orkambi. This time, as an observer rather than an expert patient. Admittedly, it was hard: sat there quietly, knowing that the initial recommendation was to not recommend the treatment in England for those like myself who have cystic fibrosis and are homozygous F508Del.
At the meeting I was particularly frustrated to hear the committee did not fully appreciate the treatment’s potential to slow decline and increase lung function. Many of us with the condition have – literally – waited a lifetime for a treatment like this – one that targets the underlying cause of cystic fibrosis.
Furthermore, I was disappointed to hear that current quality life measures are still being regarded as an effective way of capturing what it is to have cystic fibrosis.
Have you ever sat in clinic with a clipboard and some questions about how you feel that day? These measures don’t tell of the hours spent taking treatments, the emotional and physical strain of juggling CF with everyday life or the impact this can have on those closest to us, be it families, carers or partners.
Apparently, we’re too upbeat for our own good! How can they measure improvement, if we keep saying we’re doing just fine – however hard it gets.
It seems in this case that in our quest to strive for a life unlimited, our ‘keep calm, carry on’ attitude has seemingly worked against us. There needs to be a better way of measuring this.
Aside from the cost, another key sticking point seems to be that although NICE accepts the treatment is clinically effective, the data cannot fully describe the potential longer term benefits.
Therefore, I was reassured to hear as the meeting drew to a close that there was enthusiasm around the Trust’s proposal to use the UK CF Registry, which could better capture these outcomes. It seemed to me that it was supported from all sides of the table.
In the meantime, (back in the real world) CF continues to be life threatening with a huge treatment burden to boot. A drug like Orkambi could mean that I can go on that dream holiday, or I could start planning a family or have the energy to take the dog for a walk. Things that most other people take for granted.
And whilst carrots are being dangled and the cost of life calculated, the stark reality is that I and many others with cystic fibrosis continue to watch our condition decline.
Reassuringly whilst we endure a long, torturous wait for the final decision from NICE in the summer, the Cystic Fibrosis Trust continues to campaign for access to the drug. It’s not about stamping our feet and saying “that’s not fair”, it’s about finding robust solutions to enable this to happen.
Until then, I remain hopeful and will continue to do what we do best; keep calm and carry on.
Read more on NICE's appraisal meeting.