Access to medicines update

Blog - Anna Evans, Policy and Evidence Manager -

In this access to medicines update, Anna Evans, Policy and Evidence Manager, writes about the latest developments in access to CFTR modulators and the Trust’s work with the NHS and Vertex to ensure people across the UK can access the drugs they need.

Access across the UK

We’re now approaching six months since people across the UK with one copy of F508del and a minimal function mutation started to be prescribed Kaftrio. The rollout of the treatment has been encouraging and the vast majority of eligible people with CF in the UK have received their initial prescriptions. It’s been wonderful to see people experiencing the benefits this drug offers and that together with the community, all of our efforts ensured that the UK gained access as early as possible.

The innovative nature of the deals agreed for the portfolio of Vertex drugs has also meant that when new indications for any of them have been licensed, clinicians have been able to prescribe these drugs immediately. In November, two new indications were approved. Firstly, Kalydeco for children aged 4–6 months with one of nine gating mutations, shortly followed by approval of Symkevi for children aged 6–11 who are either homozygous for F508del or have one copy of F508del and a residual function mutation.

These agreements and the urgency given to making Kaftrio available on the NHS shows the lasting legacy of our campaigning for access to life saving drugs. This was an incredible collaborative effort and again, we would like to say a huge thank you to everyone who protested, signed petitions, submitted evidence to the Health and Social Care Committee and took countless other actions with us along the way.

But, we aren’t stopping here. We will continue to work to change and improve access, and we will pursue our mission so that drugs are available for everyone that could benefit from them.

Recently we have focused on supporting access to Kaftrio, Symkevi, and Kalydeco for people with rare mutations and those with one copy of F508del and any other mutation, and access to Kaftrio for children aged 6–11. Read on for an update.

NB: We have updated this blog based on further UK-wide discussions and will continue to update as things develop.

Access to Kaftrio, Symkevi, and Kalydeco for people with rare mutations (beyond EMA licence)

The U.S. Food and Drug Administration (commonly referred to as the FDA) has approved the use of Trikafta (Kaftrio)Symdeko (Symkevi), and Kalydeco for mutations that are too rare to allow for a traditional clinical trial but have been found to be responsive to CFTR modulators through ‘theratyping’ in which rare mutations are tested through laboratory studies to predict whether people with CF will respond to approved CFTR modulators.

It is ground-breaking for the FDA to license a drug for a population where no clinical trials have taken place and other regulators have not licensed CFTR modulators in this way.

In the UK, the deals struck with Vertex in 2020 which granted access to Kaftrio were also ground-breaking. Firstly, they grant access to all new indications as soon as they are licensed, and secondly because they included a limited provision to support ‘off-label’ or unlicensed access to CFTR modulators. This meant that UK clinicians could prescribe CFTR modulators for some people with rare mutations where there were laboratory studies to support this, but not regulator’s approval in Europe.

We have been working with Vertex and the NHS to support the expansion of the existing ‘off-label’ policies to include these additional mutations licensed in December. In some of the devolved nations clinicians can apply for people who have one of these rare mutations approved by the FDA in December to access CFTR modulators. Unfortunately, in other parts of the UK there is no access at this time. If you think you could benefit from access to a CFTR modulator, it is important to speak to your clinical team about your specific circumstances.

Access to Kaftrio for children aged 6–11

In September 2020, Vertex announced positive trial results for the use of Kaftrio in children between the ages of 6 and 11 with cystic fibrosis. The 24-week Phase 3 study, which was evaluating the safety and efficacy of Kaftrio in this age group, showed that the drug was generally well tolerated, safe, and participants showed improvements in their lung function (FEV), sweat chloride, and body mass index (BMI).

On 26 January, Vertex announced that they have submitted Kaftrio for children aged 6–11 to the FDA. Vertex have said they hope to submit to both the European Medicines Agency (EMA) and UK Medicines Healthcare Regulatory Authority (MHRA) in the first half of this year, though we do not know exact dates or timings.

Access to Kaftrio for people with one copy of F508del

A licence for Kaftrio for people aged over 12 with one copy of F508del and any other mutation is expected within the next six months. In some of the devolved nations clinicians can apply for people who have one copy of F508del and any other mutation to access Kaftrio. Unfortunately, in other parts of the UK access to F508del and any other mutation is not covered.

The EMA are currently evaluating Vertex’s application. If the EMA approve Vertex’s application, the MHRA should recognise the EMA decision and routine prescribing can begin.

We’d like anyone with one copy of F508del to be given Kaftrio today. However, for those in the greatest clinical need, clinicians may be able to apply for compassionate use.

Stay in touch!

We will not stop until everyone has the treatments and support they need. If you’d like to stay up to date on campaigning news like this, please subscribe to our Campaigner Newsletter.

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