Stephen Hepburn MP: Orkambi drug to help little Harriet should be available on the NHS
I've been greatly affected by a warm and loving Jarrow family with a young girl diagnosed with Cystic Fibrosis.
I popped in to see Emma and Chris Corr and their two beautiful children, Nancy and Harriet.
Little Harriet, aged just three, has been through more than most of us will in our entire life since she was born.
At just five days old she needed an operation to unblock her bowel, and it was then her parents were told she had Cystic Fibrosis.
Emma and Chris spend hours every day ensuring Harriet receives the exercise and regular physiotherapy needed to fight a debilitating and life-threatening genetic disease which affects about 10,400 every year in the UK.
Cystic Fibrosis affects the lungs, digestive system and other organs. Sufferers live a shortened life. The build-up of mucus clogs lungs, impeding breathing and leaving them prone to chronic infections.
No parent wants their baby born with such an illness but the likes of Emma and Chris fight like tigers for a child who deserves all our support.
There is, alas, no cure for Cystic Fibrosis, but medical science is advancing all the time and a new drug called Orkambi now offers real hope.
Available in the Republic of Ireland, Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, Greece and the United States, I want you to join me and Emma and Chris to see it provided by our National Health Service for Harriet and others like her.
The drug is said to attack the root cause of Cystic Fibrosis instead of treating only the symptoms.
Trials resulted in astonishing results. It helps roughly half of all sufferers with a specific genetic mutation. And for them the decline of lung function was slowed by up to 42%.
The Cystic Fibrosis Trust calculated after a study some 2,834 people in the UK could benefit.
Other new medications are also being trialled.
The fly in the ointment is Orkambi is expensive and not currently available on the NHS. It should be.
Our National Institute for Clinical Excellence admits it’s “clinically effective and important for managing cystic fibrosis” whilst refusing to fund it. That’s not acceptable.
I’m asking you for the sake of Harriet and others to join the campaign to make Orkambi available to give them a longer, more comfortable life and who knows what will turn up in the future.
I addressed a demonstration outside Downing Street last year called by the Cystic Fibrosis Trust and recently in Parliament I reaffirmed my support at a roundtable meeting with MPs to distribute this valuable medication in our country.
We must do everything humanly possible to give gorgeous children such as Harriet the best chance of a full and normal life.
To help them go to school full-time, help them find jobs, help them do everything everybody else does and help them live to a ripe old age.
The petition to make Orkambi available on the NHS has attracted more than 114,000 signatures and I’ll be joining a debate in Parliament on Monday, March 19.
Can public pressure actually make a difference? I’d like to think so. Yes we can for Harriet.