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Kalydeco hope for children in England

NHS England announces provisional funding for transformational precision medicine for children age two to five with rare CF mutations, mounting pressure on the other UK nations to follow suit.

NHS England’s announcement could have life-changing implications for young children with one of nine gating mutations, and provide a major boost for ‘Stopping the Clock’, the Cystic Fibrosis Trust’s campaign for access to personalised medicines.

The treatment, which is currently only funded across all nations in the UK for those over six years old, has been shown to significantly increase lung function, slow the progression of lung disease and halve hospital admissions.

Research published earlier this year in ‘The Lancet Respiratory Medicine’ journal suggested that pre-school children with one of the nine mutations (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) could also benefit from improved pancreatic function, weight gain and movement of salt between cells.

The decision is one of many that will not be confirmed until a dispute over funding for HIV prevention medicines is resolved. Similar decisions in Wales and Northern Ireland are yet to take place.

Scotland has a separate approval process through the Scottish Medicines Consortium, who in May opted not to recommend Kalydeco for two- to five-year-olds to NHS Scotland. In response, the Cystic Fibrosis Trust proposed a solution for funding for the drug to be considered by the Scottish New Medicines Fund.

Emma Foord  is one of many mums in England who will be hoping the provisional decision goes ahead as soon as possible. She said: “My son is 18 months old, with a copy of the G551D. He's had quite the journey in his little life, suffering three major abdominal operations as a result of cystic fibrosis. Having access to a drug such as Kalydeco could dramatically improve his quality of life."

Keith Brownlee, Director of Impact at the Trust, said: “The NHS in England has shown the way with this very positive decision and we hope the legal issues are quickly resolved. We ask other nations in the UK to extend funding without delay: it is inconceivable that a drug that has been shown to slow the progression of cystic fibrosis would be denied for younger people, because of where they live, who stand to benefit so much at such a crucial time in their development, fighting a progressive disease.”