NICE appraisal of modulator therapies

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Part of the NHS deals to grant access to modulator therapies included the agreement that data would be collected on their effectiveness. With the period of data collection ending in 2023, NICE is soon to embark on an appraisal of modulator therapies. Cystic Fibrosis Trust has provided feedback to the NICE consultation on how to approach this appraisal.

In June 2020 NHS England agreed a deal with Vertex Pharmaceuticals that granted access to the CFTR modulator treatment Kaftrio. It was rapidly followed by deals in Wales, Scotland and Northern Ireland. This followed a lengthy campaign by the CF community and Cystic Fibrosis Trust, which had previously seen Orkambi, Symkevi and Kalydeco made available on the NHS in all nations of the UK through the same process, called an “interim access agreement”.

The 2021 UK Cystic Fibrosis Registry’s annual report found that 5,321 people were taking Kaftrio, 515 Symkevi, 942 Orkambi and 606 Kalydeco.  We expect those numbers to show a further increase when the 2022 data is reported, as more people have become eligible.

NHS leaders and politicians across the UK have highlighted the importance and transformational impact of these medicines for people with CF.

The NHS deals confirmed that if the license for these modulator treatments was extended, access would automatically be granted to eligible patients in the UK, which NHS England described as “amongst the most comprehensive access arrangements of any country in Europe.”  In January 2022, access was extended to eligible children aged 6-11 and later in 2023 we hope to see a licensing decision on extending Kaftrio to children aged 2-5 from licensing organizations in the US, UK and Europe. 

As part of the NHS deals it was also agreed that data would be collected on the effectiveness of modulator treatments through a partnership between the Trust (via the UK Cystic Fibrosis Registry), NHS England, the National Institute for Health and Care Excellence (NICE), and Vertex Pharmaceuticals. The treatments would then be looked at by NICE, the government body that assesses the cost effectiveness of treatments (usually before access is granted but in this case as part of the interim access arrangements).

The data collection period ends in 2023 and in advance of that NICE has been consulting on how it will approach the assessment of modulator therapies. The Trust responded to this consultation and provided feedback to NICE covering:

  • background information on CF and the different types of medicines people with CF need to take
  • which existing medicines to compare the modulator therapies against to understand how effective they are
  • whether the appropriate outcome measures have been included that will capture all the benefits of modulator therapies
  • the incredible impact of these medicines for most people with CF who are eligible to take them.

What next?

We expect NICE to publish the timetable for the next stage of their process during 2023. The Trust will be able to submit evidence on the impact of modulator therapies, including the stories of people with CF and their families. In the meantime, we are working with the NHS in all nations of the UK to ensure that the impact of modulator treatments is understood and that specialist care for people with CF is protected.

We won’t stop until everybody can live without the limits of CF.

Find out more from NICE here

Updated 8 February 2022

Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects more than 10,800 people in the UK. One in 25 of us carries the faulty gene that causes it, usually without knowing.

Since 1964, we've supported people with cystic fibrosis to live longer, healthier lives - and we won’t stop until everyone can live without limits imposed by CF.




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