Children with cystic fibrosis live longer as treatment advances

Philip Watt addresses a protest over exclusion of children from access to the drug Kaftrio. Picture: Gareth Chaney/Collins Photos

Children born with cystic fibrosis are now expected to live longer than ever before but many challenges remain, including around accessing modern treatments, Cystic Fibrosis (CF) Ireland has said.

In its annual report, CF Ireland said children born with cystic fibrosis between 2016 and 2021 are now expected to reach 51.8 years old. Only 60 years ago, most such children died before 12 or 13 years of age.

“The number of adults with CF over 40 years of age in 2021 is now 13.4%, an increase of 4.4% since 2010,” the report says.

Over the last 10 years, progress has hinged on new drug therapies which treat the underlying cause of CF, not just the symptoms.

The median predicted survival age is rising by around one year annually because of improvements in CF care in Ireland, the report shows. 

The first of these new drugs was Kalydeco in 2013, followed by Orkambi in 2017, and the most recent and most powerful, Kaftrio in 2020.

Access to Kaftrio became the subject of public  protest when 35 children were deemed ineligible  through the public health system. Picture: Gareth Chaney/Collins Photos

However, this latter drug has been the subject of public debate and protest. Some 35 young children were initially deemed by drug company Vertex to fall outside the eligibility for access through the public health system.

This issue was only recently resolved following a year-long battle, during which doctors with the National Clinical Programme for Cystic Fibrosis claimed children were being used as pawns in a financial disagreement.

Writing in the annual report, CEO Philip Watt described the dispute as "the unexpected and cruel exclusion of 35 children aged 6-11" from the agreement. 

"In 2022 there were significant improvements in CF care in Ireland, but also existing and new challenges," he said.

He also welcomed progress towards building an adult cystic fibrosis outpatient unit in Galway, saying the Galway branch had raised €700,000 towards this.

CF Ireland holds its 60th conference on Saturday in Co Meath.