CYSTIC FIBROSIS PATIENTS LEFT TO DIE IN WALES

Welsh Health Specialised Services Committee deny compassionate use of new drug Orkambi for critically ill 

Fifteen patients in Wales are awaiting a potentially life-saving decision from the Welsh Health Specialised Services Committee to allow them treatment that could significantly delay the progression of cystic fibrosis. Clinicians are unable to prescribe the new drug Orkambi to patients urgently in need, despite it being made available for compassionate use in Wales by the drug’s manufacturer.

Patients in England, Scotland and Northern Ireland already have access to this treatment for compassionate use. The drug has been shown to improve lung function and reduce outbreaks of infection common in those with cystic fibrosis.  Evidence shows it can cut hospitalisations by over 60%. This reduction in outbreaks is important as frequent infections are associated with accelerated decline in lung function and worse survival.

One of the fifteen who could benefit from Orkambi in Wales is Mike Boyle, from Cardiff. The 43 year old father of two said; “This is life and death for me. My cystic fibrosis has left me with severely damaged lungs, kidney disease and CF related diabetes. I’m in desperate need for a kidney and lung transplant but have been rejected from two transplant lists based on the severity of my condition.

“My lungs wouldn’t cope with a kidney transplant, and my kidney would fail if I had a lung transplant. My only hope is to improve my lung function enough to survive a kidney transplant and Orkambi could potentially help – a small increase lung function could make all the difference. There are no other options left for me – I’m desperate. You can’t appreciate the strain on my mental health trying to deal with these circumstances, but I still have hope – I want to see my twin girls grow up. When I was born my parents were told I would not live to see my tenth birthday, I’m just hoping that developments that have increased my life thus far, continue to help me.”

James Barrow, Head of External Affairs at Cystic Fibrosis Trust said “It is unacceptable that people in desperate need of a treatment that could improve their health are being denied access because of where they live. If the pharmaceutical company has made the treatment available on compassionate grounds doctors should be able to prescribe it. It is a critical situation and there is no time to waste – lives could be snatched earlier as a direct result of bureaucratic delay.”

The Cystic Fibrosis Trust has written to the Minister for Health & Social Services requesting this issue is investigated urgently.