Vertex: Make Orkambi Affordable For The NHS. Save the Lives of Cystic Fibrosis Sufferers.

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My friend Maisie Baker suffers with a life threatening illness called (CF) Cystic Fibrosis, she is just 19 years old.
Half of people with Cystic Fibrosis are less than when 29 years old when they die. 

She has been given the chance, of a gift of hope. She has been granted access to the "First of its Kind Drug, Ever," to be made in the history of the illness Cystic fibrosis, since the gene was discovered in 1989.

This Wonder drug is called, Orkambi and it treats the root cause of the (CF) and not just the symptoms.

It is only available for her genetic mutation of Cystic Fibrosis, which is DDF508, she is taking Orkambi on the compassionate use programme, as there are currently no other options available for her, as a lung transplant is not advisable.

Maisie's  Story.  Life Before Orkambi.

In recent years Maisie's health has been in serious decline. For the last two years she has been on continuous IVs (intravenous drugs), "My lung function was at its lowest point at 29%.
Every day is dominated by her CF treatment regime – pills, needles, inhalers, nebulisers and physiotherapy take several hours a day to complete.  

Maisie also has to take enzyme tablets before every meal as without them her body cannot digest fat and process her food to maintain a healthy weight.

Her bedtime routine can take up to an hour as she repeats her treatment regime as well as preparing her overnight feed.

“I feed through a PEG (percutaneous endoscopic gastrostomy) tube overnight to help me gain weight”, said Maisie “This means that at night I have to get the feed ready, set up machines and flush out my PEG with sterilised water.”

Not surprisingly, Maisie has struggled both mentally and physically under this huge treatment burden, but her most crushing moment came when at the age of just 19 she was told that a lung transplant – at that point seemingly her only hope of a better quality of life – was too dangerous for her, So Orkambi was plan B.

“I’m hoping that Orkambi will work and improve my lung function, give me more energy and allow me to do simple things without getting breathless.”

Maisie's Miracle Story With life-saving drug Orkambi.

“Within a week I started to notice massive improvements. I feel so amazing. My resting heart rate used to be around 110-120 bpm now for the first time in ages my resting heart rate is normal at 70-80 bpm.

“For the last four years I have slept sitting up with the help of a wedge as my lungs couldn’t handle me lying down flat – it caused me to struggle with my breathing.

“But for the first time last night I slept without my wedge and it was amazing to sleep lying flat. I could breathe.

And the biggest change for me is that I haven’t had one of my ‘coughing fits’ since starting it.

Usually my morning consists of me sitting up in bed, struggling to catch my breath whilst I cough, going bright red in the face and feeling dizzy. But that’s all stopped. I no longer cough until I can’t breathe!

“On the 18th of August just 10 days after coming home I did my first set of blows (lung function). When I left hospital the last FEV1 I did was 36%. The blows I did on the 18th came out at 50%!! 

I’ve never had an FEV1 that good since I was 8/9 years old.  I never thought in a million years I would be able to get a lung function past 40%. Never mind 50%!”

Maisie is naturally delighted to have been given access to Orkambi herself. 

But her thoughts are with her fellow CF sufferers when she says “As happy as I am to have started on Orkambi, I still wish that more people had access to the drug and the hope of a better quality of life that it offers.”
That a drug like Orkambi has been developed is akin to a miracle for those people who have faced a lifelong daily battle against the many health issues cystic fibrosis can cause.

To have that miracle dangled just out of the reach of the people who could potentially have their life transformed by it seems cruel beyond belief.

But the clock is ticking, people have been waiting for more than 2 years to access this life-changing treatment. And as time passes, people are experiencing irreversible lung damage.

NICE, the government body responsible for appraising medicines, recognises Orkambi as “important and effective for managing the condition”. However, with a UK list price of £104,000 per person, per year, it was unable to recommend Orkambi because the treatment is too expensive.

Thanks to the remarkable efforts of the cystic fibrosis community - with 26,000 people taking action - the NHS has opened its door to negotiations with Vertex. It’s now up to Vertex to seize this opportunity and present a bold offer that will work for the NHS. Vertex pharmaceuticals who make the drug Orkambi, must lower the price while there is still time to negotiate with the NHS.

If these negotiations fail, Orkambi will continue to be out of reach for those who so desperately need it. And children & young adults, they die sooner.

We have to act urgently to convince Vertex to make the right decision and Make Orkambi affordable to the NHS.

It’s impossible to put a price on life. But it is not impossible to put a fair price on life-extending drugs.

Producing drugs that are unaffordable is unethical.
Act now and demand that Vertex lower the cost of Orkambi before it’s too late.



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