Reflections on the MHRA announcement and NICE process
David Ramsden, Chief Executive of Cystic Fibrosis Trust said: “Following yesterday’s approval of Kaftrio for 2–5-year-olds, I wanted to give some reflections. The first thing to highlight is what a fantastic piece of news it is. We were delighted to see the NHS followed the announcement by confirming that prescribing can start shortly, and that access will be long term.
"We also met with the NHS earlier in the week to raise the community’s concerns about the uncertainty that the NICE process is causing. I’m pleased to say that they offered us assurance that everybody on [modulator] drugs continues to have access and also that they were ready to play their full part in ensuring that a comprehensive deal is put in place for all future access.
"The NICE process is ongoing, and there are a few key milestones ahead, not least the close of the consultation next Friday, the 24 November, and then a committee meeting on the 14 December. We don’t know exactly what will happen after that committee meeting, but we do have an expectation that we actually might not hear very much– in fact there may be a period where we hear very little while discussions are ongoing behind closed doors.
"What I can assure you of is that we will keep you fully updated as things develop and also of our commitment to ensure that there are comprehensive and permanent arrangements in place so that everyone who can benefit from highly effective modulators does but also that we continue our focus on ensuring that all people with CF have the treatments they need to live a long and healthy life. We won’t stop.”
As part of the NICE appraisal, NICE have asked for people to submit their feedback to the NICE consultation by 24 November, sharing the views and experiences of people with CF and their families.
What happens next?
Regulatory approval is an important step towards children aged 2–5 with eligible CF mutations receiving Kaftrio. The drug manufacturer will now need to make it available to UK CF teams, who can then start to prescribe Kaftrio for eligible children. It will be up to individual CF teams to organise this process, and this is something they have experience of already from previous roll-outs of modulator therapies to specific age groups.
Does this mean that NICE have changed their recommendation about whether Kaftrio is cost-effective?
The NICE appraisal process and regulatory approval of Kaftrio for 2–5-year-olds are two separate and different processes. MHRA approval means the drug is safe and effective for its intended use. The NICE process looks at how well a treatment works, and whether it is cost-effective, to decide whether it should be funded by the NHS.
The NICE appraisal recommendation shared by NICE on 3 November is a first stage, draft recommendation. We are hopeful that there will be a resolution to the uncertainty that announcement has created and we are urging all parties to work together quickly to find a solution.
Eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment, as clinically appropriate.
When I heard the news that Kaftrio had been licensed for eligible children here in the UK from age 2 I was so happy and felt a sense of relief. Since Ava’s diagnosis we’ve looked forward to this day “Kaftrio day” as a sense of security for her future. It’s amazing that she can access modulators from such a young age. CF is a life-long condition but my hope is that Kaftrio helps her future be more manageable, longer and healthier. Ava’s future looks different now and as a family we’re just so grateful for that.Carley, mum to 2-year-old Ava who has CF