Last night, Health Secretary Matt Hancock tweeted the news that the Cystic Fibrosis drug, Orkambi, would finally be available on the NHS, giving sufferers hope for a better future.
For almost four years Paul and Catherine Upstone , whose 14-year-old daughter Cerys has Cystic Fibrosis (CF), have been working with MP Andrea Leadsom to make the drug available on the NHS.
The condition causes a build-up of thick sticky mucus in the lungs, digestive system and other organs, which requires a lifetime of medications and physiotherapy to manage.
Cerys has to take antibiotics for flu like symptoms and has two sessions of physiotherapy and four nebulisers every single day. The condition has also meant Cerys has had to take considerable amounts of time away from school.
Three years ago there was a breakthrough in the treatment of CF when the drug Orkambi was approved. The drug was licensed in the UK and available in many countries around the world.
Known as a precision drug, Orkambi has been shown to improve a patient’s health by targeting the underlying cause of CF and not the symptoms.
The joy, however at the possibility of an improved treatment regime for Cerys and the thousands of sufferers across the country quickly grew into frustration as the manufacturer, the NHS and the National Institute for Health and Care Excellence (NICE) became embroiled in a dispute preventing its access.
That dispute has now been resolved giving families a new hope for a brighter future.
Catherine said: "Cerys and I are completely overwhelmed by the news. It gives up such hope for the future. We are very proud of all the work the cf community have done to campaign for these drugs.
"The news was a total shock but we were so hopeful after Scotland got access in September. I have spoken to Andrea's office to thank them for their support over the past four years. Her team seemed to be unaware of the deal.
"We are looking forward to celebrating the news tonight as a family and hope Wales and Northern Ireland do not have to wait too long for their deal.
"The deal says that consultants should be able to start prescribing within 30 days.
She added: "The best early Christmas present ever."
MP Andrea Leadsom said via her website: "I am absolutely delighted that the Health Secretary has announced today that NHS England has secured agreement with Vertex Pharmaceuticals to make available all three of their UK-licensed cystic fibrosis medicines.
"I have a number of constituents whose children are CF sufferers, and who have campaigned tirelessly and bravely for this outcome; I am so pleased for them.
"This agreement means NHS patients will now have full access to Orkambi, Symkevi and Kalydeco, and around 5000 people may now take up these treatments.
"There is no cap on patient numbers, and each and every patient in England who might benefit can now get these treatments, free on the NHS. Clinicians will be able to begin prescribing these drugs within 30 days.
"This is a life-saving achievement and, on behalf of all my constituents living with cystic fibrosis, I would like to thank everyone who has campaigned for this to happen, as well as to Vertex and the NHS for reaching an agreement.
"It also wouldn’t have happened without the personal efforts of Matt Hancock, and his determination to see this through."