NICE seeks additional information about givinostat
We are deeply disappointed by the decision by the National Institute for Health and Care Excellence (NICE) to seek a second committee meeting for Duchenne muscular dystrophy (DMD) medicine givinostat (Duvyzat). We will be doing everything we can to assist the committee in reaching a swift conclusion to this appraisal.
Seeking additional information
Following a NICE appraisal committee meeting on 10 July, the committee has chosen to seek additional information to be considered at a second committee meeting in October. This means yet more delays in the journey to secure routine access to givinostat through the NHS in England.
We know this is very difficult news and means further delays that our community cannot afford. We want to assure you that despite this setback, the process is still moving forward. NICE has taken the unusual step of not issuing draft guidance with a negative decision, which is what would usually happen at this stage. We are hopeful that a second meeting will deliver a positive outcome.
We recognise the strength it takes to keep advocating for progress and fighting for what’s right and we understand the toll it can take but you have never given up hope and neither will we.
Next steps for NICE’s appraisal of givinostat
We want to reassure you that while this further delay is hugely disappointing, it is rare for NICE to make a positive decision after the initial committee meeting, and many treatments are approved after a second meeting.
NICE has asked stakeholders such as Duchenne UK to help them answer questions the committee still has about givinostat and DMD.
We will be responding strongly urging NICE to approve givinostat, which has been proven to slow down the progression of the disease in clinical trials and could positively impact the lives of those living with DMD and their families.
We are in regular contact with the NICE stakeholder team for this appraisal. We have provided robust feedback on the process to date, which we hope will ensure that the second meeting accurately portrays the seriousness of DMD and the true impact that this devastating disease has on patients, families and carers, which is why givinostat which could delay its progression is so vital and must be made available routinely on the NHS.
What this decision means for current access to givinostat through the Early Access Programme (EAP)
The decision to seek further answers at a second committee meeting does not affect access to givinostat through the EAP, which provides the medicine to the NHS for free by the manufacturer ITF Pharma UK. The EAP will remain open until NICE reaches a final decision as to whether givinostat should be made available as a treatment in NHS England. Anyone already receiving the treatment at the point of a final decision will continue to do so, regardless of the outcome.
Parliamentary event to highlight issues with access to givinostat
This delay is very hard, particularly for people who haven’t been able to get access through the EAP. We will also be holding a drop-in event in parliament to mark World Duchenne Awareness Day 2025 to keep up our engagement with MPs, and seek their help in securing access to givinostat through the EAP. It is taking place from:
- 4-6pm on Wednesday 3 September in Room W2, Westminster Hall, Palace of Westminster
This year’s World Duchenne Awareness Day theme is ‘Family: The Heart of Care’ and we are inviting people with DMD and their families to come along and talk to MPs about our campaign. We also encourage you to invite your MP too. You can email [email protected] if you’d like to attend or know more about it.
If you have any questions about current and future access, NICE’s decision, you can email us at [email protected]. We are also building an information page about the NICE appraisal that we will add to when we know more. Here is a link to it.
NICE meeting
Thank you to everyone who took part in the first NICE committee meeting about givinostat as an observer. We found it a difficult experience and a lot of people who observed have got in contact with us to say that they felt the same. This was very disappointing, and we have been in contact with NICE about this. Since 10 July, we have had several high-level meetings to discuss how NICE engage with patient experts and observers and to ensure that they handle appraisals for devastating, paediatric, life-limiting conditions like DMD more sensitively and appropriately in future.