Policy and campaigning

Our campaign for the Early Access Programme for givinostat

Thank you to the incredible families who are using their voices to help our campaign for early access: please read on to find out what some of you have been doing and how you can still help!

The situation has been changing fast from when we first told you all about the EAP two weeks ago:

1. We’ve moved from a handful of sites starting the EAP process, to 19 (we can’t disclose the exact sites yet).
2. We’ve written to all 24 NHS Trusts which are part of the NorthStar clinical network, in collaboration with MDUK, urging them to give clinics the resources they need to take part in the EAP. Read the letter here.
3. We’ve opened conversations with MPs about political engagement.
4. Big thanks to Jill and Richard Binns who have already written to their MP and had them submit a question to the Health Secretary. It’s not been answered yet, but when it is the response will be here.

We still need your help!

But we still need you to contact your hospital doctor, your hospital Trust, the regulator (the MHRA), and your MP.

Find guidance on writing to your hospital doctor, your Trust, and the regulator here.

Please read on to find out about writing to your MP.

Today we’re highlighting the stories of two families who are taking action, Tony Levene, and Lisa Kuhwald

Tony Levene and his son Joey went to meet their local MP, Christian Wakeford, to lobby for access to givinostat. When Joey explained the situation to him, and why this treatment matters, their MP said he couldn’t argue with that! Tony is working with us and his MP to hopefully hold an event in Parliament soon, so we can bring Joey’s convincing arguments straight to politicians! Watch this space – we’ll let you know as soon as we get a date in Parliament secured.

Lisa’s son Felix is non-ambulant, so he isn’t eligible for the EAP. But she too has written to her local MP, Connor Rand, to fight for givinostat. Following our call to action last week, Lisa also wrote to the MHRA about givinostat. She gave us permission to share the letter she wrote to them, which you can read here. Please consider personalising it for your child and using it as a template to write to the CEO of the MHRA, Dr June Raine. You can contact the MHRA at [email protected]. If you want to see the template we shared in our last update, you can find it here.

We’re making important progress – Please keep going!

So many of you have been writing to hospital Trusts, the MHRA and your MPs. Please keep going: Today’s call to action is two-fold;

1. Use our template to write to your MP like Lisa, Tony, Richard and Jill, and tell them about the givinostat EAP. Ask them for their help in making this free medicine available in the NHS. You can find a template you can personalise here, with information included on how to contact your MP.
2. We are in discussions with MPs about holding an event as soon as we can in Parliament on givinostat and the EAP. Once we have a date we’ll let you know – when we do please ask your MPs to come, and let our Senior Policy Manager, Will Pender, ([email protected]) know when you have done so, and their reply.

If you’ve not seen the news about givinostat, and why it matters, read our first message to the community about it here. Givinostat is not a cure; but it could help boys to keep using their muscles for longer. Neither is it approved it the UK; the MHRA and NICE are assessing givinostat but are yet to make a decision.

But givinostat could be available, now. Until the regulators have both decided, the company behind givinostat is giving the NHS access to the treatment for free through an Early Access Programme (EAP). You read the eligibility criteria for the EAP here.

The MHRA give medicines a ‘label’, which defines which groups of people the medicine is licensed for. In the USA, the FDA has approved a label for everyone with DMD who is aged 6 years and over, regardless of whether they can walk. We want the MHRA to quickly grant the same broad label for givinostat. This is for two main reasons.

On the one hand, it will help get the drug into the NHS as quickly as possible, as NICE will only recommend a medicine which is licenced by the MHRA. Also, NICE can only approve it within the label set by the MHRA. In theory, the broader the label, the more people who NICE could let benefit.

On the other hand, and more urgently, this could directly affect the EAP. Doctors which have struggled to secure approval in their Trusts to take part in the EAP may find it easier to do so if givinostat is a licenced medicine approved for DMD. In addition, we hope some of the criteria for the EAP will be broadened if the MHRA grants a licence.

Thank you so much to everyone who has been helping us campaign on this so far!