Three people in Wales with rare mutations of the cystic fibrosis gene are missing out on the transformational precision drug ivacaftor, five months after the Welsh Government granted access.
Ivacaftor has been proven to significantly improve lung function and slow the progression of cystic fibrosis, and in December 2015 the Government in Wales approved its use for people over six years old who have one of the eight non-G551D gating mutations. However, while the drug received European regulatory approval in July 2014 and patients in England with these mutations have been receiving it since July 2015, the three people eligible for it in Wales are yet to receive it.
The treatment has also been shown to reduce time spent in bed on intravenous antibiotics from five weeks to less than five days a year.
Ed Owen, Chief Executive at the Cystic Fibrosis Trust said: “It is completely unacceptable that patients should be made to wait for this life-changing treatment, risking serious deterioration in their condition in the process. We call upon the Welsh Government to resolve this issue without delay.”
The Cystic Fibrosis Trust has written to Vaughan Gething AM, Cabinet Secretary for Health & Social Services, requesting a meeting and for this issue to be investigated urgently.