Eligible Age for Kaftrio for Kids: The UK Approval Process
Written by Marc Cotterill
Introduction
In this article, I aim to answer some of the key questions that CF parents have asked me since I published my last article about NICE and Vertex a few days ago.
Understandably many families are eagerly awaiting news about whether the eligible age for Kaftrio will be lowered in line with guidance in the US, from 6 years old to 2 years old. It has been talked about a lot over the last few months, and it is especially relevant since the news from NICE was announced last week, but what is the process and where are we with it?
If you are confused about the eligible age for Kaftrio, whether it will be lowered, and how a decision about this relates to the ongoing NICE process, this is the post for you.
Within the article, I cover the following:
The Current European Medicines Agency (EMA) position
The role of the Medicines and Healthcare products Regulatory Agency (MHRA)
The role of Vertex within these processes
How these processes intersect with the ongoing NICE process
And the possible outcomes for families
Before I begin, I want to mention that if you are finding these processes and the news last week too much to deal with right now, I completely understand. You do not have to read this or share and discuss it online.
For those who are ready, I wanted to offer you an easy way to understand this often overwhelming and complex world.
Finally, it is important to note that this is my interpretation of the information available online, with some consultation with CF group leaders who are involved in these processes.
A simple breakdown
Current EMA Position
A committee within the European Medicines Agency (EMA) has given a ‘positive opinion’ that Kaftrio should be approved for use in younger children, ages 2 to 5 years. But what does that mean?
A ‘positive opinion' from the EMA is a significant step forward, signalling that formal approval could be on the horizon. Following a positive opinion like this, it usually leads to formal approval within 6-8 weeks - so this is expected imminently.
MHRA’s Role
The UK has its own regulatory body, the Medicines and Healthcare products Regulatory Agency (MHRA), which decides whether to follow the EMA's recommendations and allow the same use of medicines in the UK.
For context, in the past for other modulators, it has.
For MHRA to approve the use of Kaftrio for the lower age group, Vertex has to submit an application to the MHRA for approval. Vertex has previously stated that it had submitted this application as covered here, and ‘CF Voices’ Facebook group leaders have emailed Vertex this morning (08/11/23) for confirmation, to which they have confirmed adding:
“We are expecting the MHRA decision late November / early December” - Vertex
(Link to CF Voices and other advocacy groups below).
Given that Vertex has submitted its application, the MHRA will be reviewing it. As mentioned above, historically, if the EMA has licensed new uses for a drug, the MHRA has quickly allowed doctors in the UK to prescribe it for those uses.
NICE Funding Review
Meanwhile, as we know, NICE is assessing whether the NHS should fund Kaftrio. This process looks at cost-effectiveness but is separate from the MHRA's decision on who can be prescribed the drug.
MHRA don’t provide timescales or updates, they simply make announcements when decisions are made, but it's likely to happen shortly after the EMA finalises its decision (based on past experiences) and based on the message above, that is expected by Vertex by late November or early December this year.
Possible Outcomes for CF Families
As things stand, we are in a waiting period.
We know that Vertex has applied to MHRA, MHRA now needs to review the application (which we assume is in progress) and decide whether to approve it (and as stated they usually do so in line with EMA), but then this entirely separate process intersects back into the ongoing NICE process in the following way:
AND THIS IS KEY!
If MHRA approves Kaftrio for the younger age group before NICE reaches a decision on the future funding of modulators, then doctors could start prescribing Kaftrio to eligible patients within the younger age group of 2 to 5 years old immediately.
This is because as things stand, patients would be covered under the current NHS deal. Nothing changes here until the NICE process concludes.
However, if MHRA approves Kaftrio for the younger age group after NICE reaches a decision on funding modulators, then access to Kaftrio for eligible patients within this younger age group of 2 to 5 years old, for those who are not yet on any modulator, will be entirely dependent on the NICE outcome.
HOWEVER - A REMINDER:
Suppose a child is already on a modulator therapy (like for example, Orkambi) before the NICE decision is finalised. In that case, they are protected by the current deal, and will therefore have access to the ‘full range of licenced meds going forward’ (quoted from the NICE documentation).
In other words:
If a child is already on a modulator (before the decision from NICE), they will be able to ‘upgrade’ to Kaftrio when they reach the eligible age (or immediately if that eligible age is agreed to be lowered by MHRA) regardless of the outcome of the NICE process.
It's important to note that the NICE process could take some months—which is a good thing for those who are waiting to ‘upgrade’ or for those hoping for the eligible age to be dropped. An appeal process is built in before the final guidance is published which of course takes time. Equally, it could conclude positively sooner than that if 'confidential discounts' were to be agreed with Vertex that makes the drugs more ‘cost-effective’ in NICE’s review.
So whilst the two processes (1: eligible age groups, and 2: NHS funding) are separate, there is a connection.
I hope this provides clarity.
Final thoughts
It is a vastly complex group of processes, but it's essential to recognise the core obstacle in our path: that is the high prices set by Vertex for modulator therapies. These costs not only challenge the allocation of NHS resources but also set a precedent that affects the global accessibility of life-changing treatments.
The UK, with its robust healthcare advocacy and stringent evaluation systems, is uniquely positioned to influence change. By uniting our voices and efforts, we can and should, amplify the call for more reasonable pricing.
Our collective action can extend beyond our borders, advocating for a change that could ripple out to improve access to modulator therapies worldwide, quite literally breathing life into the lungs of this global community.
Whilst it will always be our instinctive priority to secure these medications for our loved ones here in the UK, we must also do what we can to contribute to the global conversation that will benefit every person with cystic fibrosis, regardless of where they happen to have been born.
If you have not already, please sign the global access petition.
By doing so, we send a clear message: the health of patients should take precedence over profit margins. Together, we can apply the necessary pressure to address the root cause of this issue — the high prices of modulator therapies — for the benefit of all who are touched by cystic fibrosis.
Let's stand in solidarity and champion the right to accessible treatment that promises a better quality of life for those affected by this condition.
Reminder of Groups to Follow:
As we keep fighting for access both in the UK and globally, these are the groups to follow to collaborate in finding solutions and taking action:
Just Treatment:
Website: https://justtreatment.org/
Instagram: https://www.instagram.com/justtreatment/
Facebook: https://www.facebook.com/justtreatment
X (Twitter): https://twitter.com/JustTreatment
Vertex Save Us:
Instagram: https://www.instagram.com/vertexsaveus/
Facebook: https://www.facebook.com/VertexSaveUs/
X (Twitter): https://twitter.com/VertexSaveUs/
Strawfie: Prominent UK and Global Campaigner:
CF Voices:
Facebook: https://www.facebook.com/CFVoices
UK Kaftrio Group: