Givinostat – Early Access Scheme – the fight continues for patients to gain access

Alex has been taking part in clinical trials most of his life – 9 years in fact. We are so grateful this has been the case, when Alex was diagnosed with Duchenne at 3 years old – there were no trials available for us to even consider.

The purpose of taking part in clinical trials when your child is living with a rare condition, that has a significant unmet medical need, is because it is your only chance of getting a potential drug that might help slow down disease progression.

The trial that Alex has been on for the past 5 years is proving to be delaying disease progression. The drug is called Givinostat. We will be forever grateful we were one of the lucky ones to get on this trial.

There’s been a huge amount of focus on getting Givinostat approved here in the UK. And whilst it has now been approved by the MHRA in December for those patients who meet the criteria to access this drug under the Early Access Programme (EAP) in the UK. It now lies with each of the NHS trusts to decide whether it can support giving patients access under the EAP scheme.

Until NICE approves this drug, it cannot be given automatically on the NHS – and that is likely not to happen until the end of this year.

Despite the drug not costing the NHS under the EAP scheme, there are complex issues around admin capacity to process paperwork and undertake blood tests and monitoring for patients – and each NHS Trust is reviewing this and will decide whether it can support. You can find out more about these hurdles here.

This is the last thing families need to hear, they need this drug now – and will go to any length to make it happen.

Duchenne UK has been campaigning tirelessly, alongside an army of passionate families living with Duchenne over the past few months. They are moving mountains to drive their #timeismuscle campaign. They held an event in Parliament on 22nd Jan which saw 25 MPs attend, which they have agreed to support the campaign. You can find out more https://www.duchenneuk.org/timeismusclecampaign/.

Decision makers are now taking notice, but it has taken a huge amount of work by the Duchenne community and is ongoing. This is what rare disease populations have to do to get what their children need.

We hope all NHS trusts offer this drug to families as quickly as possible. Every day our boys are getting weaker, time is not on our side. For families affected by Duchenne in the UK who are not able to gain access, are being urged to reach write letters to their local NHS Trusts and MPs, and they can find the template letters on Duchenne UK’s page – https://www.duchenneuk.org/timeismusclecampaign/

Givinostat is not a cure, and it will only go so far to slow down muscle wasting.

We’re so grateful to all the boys that have taken part in this clinical trial. Without them we would never be in a position where new treatments like this are becoming available. When you first take part in a clinical trial, you have no idea if you are backing the right horse or if there will be serious side-effects. Luckily this wasn’t the case with Givinostat, and it makes it all worthwhile knowing the drug is now at this stage of development.

We just hope hope families can access it soon.

Posted in Drug approval process