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expert reaction to MHRA extending the license of cystic fibrosis medication to children between two and five years old

Scientists react to MHRA licensing cystic fibrosis medication for younger children. 


Kevin Southern, Professor of Child Health, Chair, UK CF Medical Association, Director of the European CF Society Standards of Care Committee, said:

“The initial draft of the NICE evaluation of CFTR modulator therapy concludes that elexacaftor-tezacaftor-ivacaftor (KaftrioTM) is clearly effective for people with cystic fibrosis (CF) who are eligible but, given current thresholds, not cost effective.  This accurately reflects both the impact on patients (which has been profound) and the high cost of this drug.  Next year NHS England (on behalf of the UK) will negotiate with Vertex (the pharmaceutical company that developed and manufactures E-T-I) a new contract for this drug.  The NICE evaluation will inform this process, as will the significant voice of the CF community.  The transparent nature of the NICE evaluation and the robust conclusions have been deeply unsettling for people with CF and their families, many of whom have experienced the life changing benefits of this therapy.  Whilst the UK needs a mechanism to rigorously evaluate new therapies, the Secretary of State should consider the impact for people with the condition of these processes, especially when a new therapy has already been successfully implemented.”


David Sheppard, Professor of Physiology at the University of Bristol, said:

“It is excellent news to learn that the license for the cystic fibrosis medicines Kalydeco (ivacaftor) and Kaftrio (elexacaftor-tezacaftor-ivacaftor) has been extended to children with cystic fibrosis aged two to five years old carrying gene defects that respond to these medicines.

“Cystic fibrosis is caused by gene defects that disable or destroy a protein called CFTR.  The disease affects many organs in the body with damage starting early in life.  For some organs, such as the pancreas, damage is already severe at birth.

 “Kalydeco and Kaftrio are drugs taken by mouth that target all organs affected by cystic fibrosis and the root cause of the disease, faulty CFTR proteins. Starting treatment with Kalydeco and Kaftrio early in life limits organ damage, leading to longer healthier lives.

 “Of the 11,000 people with cystic fibrosis in the UK, around 10,000 have gene defects eligible for treatment with Kalydeco and Kaftrio.  For the rest, some very rare gene defects might be identified as responsive to Kaftrio with laboratory testing.  But others require different treatments to target the root cause of disease that either rescue or replace faulty CFTR proteins.”


Dr Jane Chudleigh, Senior Lecturer (Research & Teaching) in Child Health at King’s College London, said:

“This is very welcome news and important in terms of ensuring children can receive early access to modulator therapies that can improve long term health outcomes. This decision will hopefully also inform the final NICE recommendations.”


David Ramsden, Chief Executive at Cystic Fibrosis Trust, said:

“This is great news and another important step in ensuring access to life changing modulator drugs for all of those with cystic fibrosis who could benefit. I’m also reassured that NHS England has confirmed all children eligible today can be confident about their long-term access to these treatments. Today’s news reinforces the need to ensure that the current NICE process rapidly results in a comprehensive deal to end the uncertainty for all who could benefit in the future.”



Declared interests

Kevin Southern: ‘No Conflicts of Interest’

David Shepperd: Current research grants from the Cystic Fibrosis Trust and a previous Vertex Innovation Award (2017-2019) from Vertex Pharmaceuticals

Dr Chudleigh: Principal Investigator in the Exploring Perceptions of the Proposed Cystic Fibrosis Screening Protocol Incorporating Next Generation Sequencing project.

David Ramsden: No DOIs 

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