Alder Hey Children’s NHS Foundation Trust is now participating in the Early Access Programme (EAP) for Givinostat. This is a new treatment being offered to children and young people with DMD (Duchenne Muscular Dystrophy).
Givinostat has shown positive results in clinical trials in ambulant DMD patients and is currently awaiting approval from the National Institute of Health and Care Excellence (NICE) for wider availability through the NHS in England. This is a process that every drug supplied through the NHS must go through, to ensure that the NHS only supplies drugs that are safe, effective, and value for money. If Givinostat is not approved by NICE, the NHS will not commission it. A decision by NICE on Givinostat is expected in the Autumn (2025).
Prior to the NICE review of Givinostat the manufacturer of the drug, ITF Pharma UK, has made this drug available to eligible patients through an Early Access Programme (EAP).
The EAP means that Givinostat is available to patients free of charge, through the NHS, but does not include the costs required to deliver this treatment safely, including any extra staff related costs. We have therefore had to carefully take some time to secure the necessary funding to deliver this new treatment safely. We are now in the process of recruiting these new staff to ensure that our children and young people eligible for treatment can continue to be safely monitored and assessed while receiving the medication. We have also considered the best way we can safely and fairly provide this medication to children within our service.
The Neuromuscular team is delighted that Alder Hey can now offer Givinostat to eligible ambulant patients within our service (those who can stand or take a few steps independently).
We have been working to put in place the necessary support in order to provide this service to our patients safely and effectively. We greatly appreciate the patience and understanding from our families whilst we set up this service.
At this time we are unfortunately unable to offer Givinostat to non-ambulatory patients within the current EAP. We are also unable to accept referrals for children from other hospitals but we will continue to explore how we can expand our offering in the future.
We have carefully reviewed our patient list to prioritise children within our service who need the treatment most urgently or are at higher risk of losing movement (ambulation) soon. We have already started to see our most urgent patients and will be contacting other eligible patients with details of their appointments in the future months.
We appreciate the urgency and importance of providing timely access to treatments like Givinostat for eligible patients, particularly given the progressive nature of Duchenne Muscular Dystrophy. We are fully committed to providing all our children and young people with the best possible care. We remain grateful to our children, young people and their families for their continued feedback and support.
Frequently Asked Questions
What is Givinostat?
Givinostat is a potential treatment for Duchenne Muscular Dystrophy (DMD) that has been developed by the pharmaceutical company ITF Pharma UK.
Why does it matter that Givinostat has not been approved by NICE?
This is a process that every drug supplied through the NHS must go through, to ensure that the NHS only supplies drugs that are safe, effective, and value for money.
NICE has the knowledge and resources to determine the clinical and cost effectiveness of new treatments. Individual hospitals cannot assess a drug in this way.
NICE is currently assessing the clinical effectiveness of Givinostat, alongside its cost effectiveness and safety profile. A decision by NICE on Givinostat is expected in the autumn.
If Givinostat is not approved by NICE, the NHS will not commission Givinostat and hospitals will be unable to supply Givinostat to our patients.
What is the Early Access Programme (EAP)?
Until NICE gives its opinion on Givinostat, ITF Pharma UK has made the drug available to eligible patients through an EAP, where the manufacturer funds the supply of the drug to eligible patients.
ITF Pharma UK does not fund the other costs associated with giving Givinostat and does not provide any extra funding for staff to support this service. It is left up to individual hospitals to try to find the extra funding and the extra staff that are needed to support the Early Access Programme.
What are we hearing from families and charities?
We know that our patients and their families want Givinostat to be given as soon as possible. We know that waiting to hear if Givinostat will be supplied by Alder Hey prior to the NICE decision has been frustrating. We understand that this situation is distressing for our patients and families who are impacted by the effects of DMD every day.
We are committed to listening to you, to communicating with you and to updating you on our plans to make Givinostat available to all eligible patients.
Why aren’t all hospitals providing Givinostat under the Early Access Programme?
Givinostat has not yet been approved by NHSE and is therefore not a commissioned drug. It is up to each individual hospital to review the available clinical evidence and their resources to decide if they want to supply Givinostat to their patients.
Transition
Any patient who has currently commenced their transition pathway to adult services will need to continue this route and may not be offered Givinostat at this time. We will discuss this with those young people individually as we continue to support them with their transition to adult services. We may not be able to commence Givinostat but we are working with our colleagues at adult trusts to support a timely and smooth transition.
Non-ambulant patients
We are currently not able to commence Givinostat for non-ambulant patients via the current EAP. We will continue to explore options for expanding our offering in the future.
Why are you unable to accept referrals from other hospitals?
Alder Hey has a significant number of patients within our Neuromuscular Service who are eligible for Givinostat. We also have an average of 10-15 new referrals for DMD annually. Givinostat is very complicated and expensive to deliver and requires very intensive input from our staff. We have had to secure funding and increase staffing to enable us to deliver Givinostat safely. At this time we only have the resources in place to provide Givinostat to those within our service. We will continue to explore any opportunities to expand this offering in the future.
Clinical effectiveness
It’s important to understand that the NICE review of the clinical effectiveness of the drug has not yet been completed and this won’t take place until later this year. If it is approved for use in the NHS by NICE, funding for Givinostat will follow around three months later.
Staffing
Making Givinostat available requires processes to be put in place to monitor patients with regular blood tests. This requires extra staffing at a significant cost. Patients require regular blood tests to ensure they are responding well to the treatment and not experiencing any side effects. Patients also require regular appointments and close monitoring by nursing teams. These are important considerations in ensuring patient safety now and in the future.
Trusts need to consider the financial and staffing resources that this involves at a time when the NHS is under increasing pressure to manage finances, with demand for the NHS growing. Trusts are exploring how they can do this safely and what the impact will be on other patients.
What is happening across children’s hospitals now?
Most trusts across England are commencing patients on Givinostat, however the roll out of this is dependent on patient numbers and workforce capacity.
We are working together with other hospitals in the Children’s Hospital Alliance, whilst locally making decisions most appropriate for our communities, based on the situation in our own hospitals. Some Trusts will find it easier to supply Givinostat than others.
Each Trust must go through its own processes and assessments to ensure the safety of its patients – as with any drug not yet approved by NICE.
What will happen if NICE approve and fund Givinostat?
If Givinostat is approved by NICE this means that the supply of the drug will be funded (commissioned) by the NHS and the team at Alder Hey will be required to provide this treatment to our patients. We are used to introducing new drug treatments approved by NICE into the hospital within the required timeframe.
What will happen if NICE do not approve and fund Givinostat?
If NICE do not approve Givinostat, the drug will not be commissioned by the NHS.
Under the EAP the supplier will fund the cost of the drug until it is no longer clinically appropriate to give Givinostat. This means that even if NICE doesn’t approve Givinostat patients already started on treatment will remain on Givinostat until the Neuromuscular Team decide to stop administering the drug for medical reasons.