Cystic Fibrosis scandal: UK waits for wonder drug to save children's lives

Starting at 6.30am, Charlotte Taylor, three, has a relentless routine of pills, therapies, physiotherapy and diet-specific meals - and she is deemed one of the UK's healthier CF youngsters. Currently, the NHS cannot afford Orkambi's list price of £105,000 per patient a year charged by US manufacturer Vertex and a £500million offer for the firm's portfolio of CF drugs was rebuffed. Families of some of Britain's 10,000 CF sufferers are urging Vertex and UK health chiefs to end the stalemate and the Daily Express is crusading for Orkambi to be made available.

Our online petition has attracted more than 62,000 signatures.

In the three years Orkambi has been unavailable on the NHS, it is feared up to 220 adults and young children have died needlessly.

But thanks to our hard-hitting campaign, both sides have agreed to try to reach a funding agreement.

Charlotte, of Cobham, Surrey, takes an average of 14 pancreatic enzyme tablets a day: Two at breakfast; one with her snack; five at lunch; one with an afternoon snack; four with her dinner and one with her evening milk.

But her food must be eaten within 15-20 minutes of taking the pills and fussy eater Charlotte's mother, Mary, faces a daily battle to get her to eat meals in time.

But Mrs Taylor, 38, said: "Believe it or not, we are the lucky ones. "Charlotte is actually quite healthy at the moment, so a poorly CF child her age would have a lot more treatments packed in. "It is exhausting for child and parents.".

Even Charlotte's five year-old sister Allie gets involved, trying to encourage her to eat her meals and sitting with her during physiotherapy.

On top of all her pills and supplements, she has half an hour of gruelling "bubble-pep" physio to clear mucus from her lungs, along with 15 minutes of back-slapping "percussion" treatment given by her mother. 

She goes to bed exhausted at 6.30pm, leaving Mrs Taylor and husband Simon, 40, to begin preparing her next day's meals and medication

Mrs Taylor said: "There are routines and rules and you have to stick to them, or else your child will get ill very quickly - it is that scary.

"All CF families live our lives on a daily knife-edge and it is terrifying." Mrs Taylor said of the Orkambi wait: "We need action to be taken now so this life-limiting condition becomes life unlimiting."

In August, family friend Amanda Sharples is taking part in an English Channel relay swim for the Cystic Fibrosis Trust.

Anyone who would like to help can go to www.justgiving.com/fundraising/channelswimforcharlotte 

A daypacked with pills and physio

6.30am: woken to take prophylactic antibiotic... unable to eat for two hours before and one hour after to be effective.

7am: bubble PEP physio - 100 breaths with huffing to shift the mucus on her lungs.

7.30am: breakfast, plus two tablets of omeprazole (reflux medicine); one syringe with 2ml a day of DEKAS, a cystic fibrosis-specific multivitamin; and one sachet of Movicol to prevent constipation; plus pancreatic enzyme supplement Creon, measured by the amount of fat in the meal. Also the non-prescription supplements Sambucol and Allicin Max (garlic) are taken to boost immune system.

10am: high-calorie snacks with Creon, as people with CF burn more calories.

11.30am/12pm: lunch with Creon and extra salt added to replace salt lost due to CF.

1pm: extra snack with Creon.

2.45pm: percussion physio in the afternoon for 15 minutes.

3pm: prophylactic antibiotic taken, so no food now for two hours.

5pm: dinner with Creon and extra salt.

6.30pm: milk with Creon and additional omeprazole reflux medication and immune-boosting Sambucol.

6.45pm: Bed

Throughout the day, Charlotte must drink copious amounts of water, a minimum of two pints a day to prevent digestive issues along with Dioralyte rehydration salts.

'NO MORE LIVING IN FEAR AND PAIN, MY BOYS ARE JOYFUL'

A SINGLE mother of two has told of the suffering before her two sons were given the CF wonder drug Orkambi

Dutch teacher Chantal Welling was housebound for two years because of the stress and burnout from dealing with the genetic disease

And her boys Keano, 20, and Miklo van Boogaart, 17, wanted to end their lives because of the pain and suffering it caused

Chantal, 46, from Brunssum, near Maastricht, said: "It was a life with pain and anxiety versus almost a normal life.

"For people who do not feel this, it is difficult to understand that things like breathing, eating and sleeping is a struggle every minute of every day.

"Living in fear and pain has changed and now they live a joyful life.

"I wanted to shout, scream, dance - my heart exploded from joy when my boys got Orkambi."

COURAGE OF GYMNAST, 5

FIVE year-old Pattie Dobbyne is determined not to let cystic fibrosis rule her life - or ruin her dream to be a Team GB gymnastics star.

The youngster was diagnosed as a baby before being treated in hospital for infections like pneumonia.

But she joined a gymnastics club near her home in Girton, Cambridgeshire, at two-and-a-half, initially just to boost her fitness and lung function.

Since then she has not been back to hospital - - and her family are insistent the gymnastics is keeping her fit and well.

On top of daily training on high bars, vault, floor exercises and trampoline, Pattie also takes 22 tablets, has two hours of physio and four nebulisers.

Her mother Georgie Dobbyne, 35, saids: "Pattie is amazingly competitive, which is great as it means she wants to train herself.

"After her difficult early years we read that exercise was good for CF sufferers as it keeps their lungs clear so we tried mini-gym.

"Now it is all she wants to do all day and night and she is obsessed. She even practises cartwheels in our living room. 

"We are positive that all the gym work is helping to keep her healthy and out of hospital."

Pattie is now in the development squad for five-year-olds at the Cambridge Gymnastics Academy. Her dream is to be a British gymnast like Alice Kinsella, 17, who last year won a gold medal at the 2018 Commonwealth Games in Australia.

Georgie said NHS England's stalemate with Vertex made no financial sense because getting Orkambi for children like Pattie now would save the health service millions in the long-run.

The mother-of-two added: "Think of all the millions of pounds that could be saved on lung transplants and months of hospital stays for CF patients in their later years if they can get Orkambi when they are children.

"Health deterioration for children with cystic fibrosis isn't just a possibility, it's an absolute eventuality.

"Orkambi would 'stop the clock' of deterioration So the healthier they are and the earlier they get it means they can maintain that hopefully for the rest of their lives Surely that is common sense.

"Yet NICE (the National Institute for Health and Care Excellence) and the NHS don't seem to be taking that potential future cost-saving into account.

They are simply focused on how much a deal will cost them now."