NICE has recommended funding on the NHS in England for Translarna.

NICE has recommended funding on the NHS in England for Translarna.

This historic decision would make available on the NHS the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy.

Translarna would treat boys whose Duchenne is caused by a nonsense mutation, who are aged five years and over and who are still able to walk.

In order to give time for NHS England and the drug company, PTC Therapeutics, to agree an final acceptable price, NICE has set July as the date where its recommendation will become final. We’ll then be pushing NHS England to implement this decision in order to get the drug to boys in clinic as quickly as possible.

Translarna for Duchenne Muscular Dustrophy

Duchenne Muscular Dystrophy drug campaign

A new drug is giving hope to boys in Europe with Duchenne Muscular Dystrophy, but it's not yet been made available on the NHS in the UK. A petition to change that has been signed by thousands of people.

 Read the full story here.

Letter from Robert Meadowcroft, Chief Executive Muscular Dystrophy Campaign

9-year-old campaigner makes personal plea to the Prime Minister

A nine-year-old boy with a rare muscle wasting disease has made a personal plea to the Prime Minister to make the drug he needs available on the NHS. Archie Hill handed in a petition at Number 10. He was joined in Downing Street by families from across the South who have youngsters suffering from the same condition. Phil Hornby reports (video courtesy of Meridian News )

(Regrettably this will not play on Apple devices)

Campaigners to hand in petition to Prime Minister

Nine year-old Archie Hill, of Gerrards Cross, Buckinghamshire, who has muscular dystrophy is handing in his personal plea to Prime Minister David Cameron to help him.

A petition of 22,000 signatures will be presented at 10 Downing Street by families waiting for access to Translarna, the first ever drug tackling the causes of Duchenne muscular dystrophy.

In a statement NHS England says:“Patient groups have asked the NHS to consult with the public about priorities for new services and treatments to be funded from April, and that consultation begins next week.

"Meanwhile patients whose doctors say they have an urgent need for a new treatment can ask medical experts to fast track their case.”

 

NHS England 'considering' how it makes decisions about drugs like Translarna

A petition signed by more than 23,000 people is to be presented to David Cameron, urging him to get the drug Translarna approved for boys who have a certain kind of Duchenne Muscular Dystrophy.

The drug was approved in Europe in August.

Families of children with the condition claim they are being left to suffer due to NHS administrative delays.

We are considering how we make decisions about specialised services treatments, such as Translarna, and how these decisions will be prioritised.

The NHS England Board decided that the consultation on prioritisation for specialised services should be 90 days, and a response cannot be given consideration until this 90 days has passed.

Meanwhile any prioritisation which is urgent on clinical grounds will be dealt with quickly though our existing procedures.

It is important to remember that this process is looking at services we would routinely commission for groups of patients and the route of individual funding requests remains the same.

– NHS England

Driving forward the first treatment for Duchenne muscular dystrophy

Driving forward the first treatment for Duchenne muscular dystrophy

"I think you might be interested to see the update set out in my note (with a photo) which I sent to the staff team following the success of the Parliamentary work and the media push yesterday, including a meeting with David Cameron in Downing Street.  We had an exceptionally good day and the team is now completing the follow up actions to take this forward.

Further, we were advised yesterday that NICE will now undertake a ‘highly specialised technology’ (HST) review of the drug Translarna and this process is likely to take several months.  In the meantime, NHS England has issued a short statement to say that eligible patients may be able to access the drug through an individual funding request made with the support of their consultant and this looks like a holding position while the NICE review is undertaken."
 
"I (wish to) underline the impact the charity – our charity - has made today in pressing for urgent access to the new treatment called Translarna which will help a specific group of the boys with Duchenne muscular dystrophy.
 
We secured over 200 pieces of media coverage in national newspapers and regional TV/radio news programmes; reaching approximately 8 million people and we brought many families to London for a meeting with 30 MPs and members of the House of Lords this morning. 
 
After very helpful discussions at the packed meeting, a petition with 24,000 signatures calling for fast access was taken to 10 Downing Street where Prime Minister David Cameron then met with some of the families with Nic and Sal and a handful of MPs.   Mr Cameron committed the government to look at this issue and to removing any avoidable delays in assessing access to Translarna; he had been primed by a question we had planted which he had toanswer in Prime Minister’s Questions this morning.
 
We are now following up all this progress with further actions to keep the pressure up for fast access to new treatments – letters to Mr Cameron, to Simon Stevens as CEO of the NHS, to MPs – and making sure that families affected by Duchenne and indeed all forms of muscle wasting conditions know we are here to support them and continue to fight for fast access to treatments.
 
I have always said that the success we achieve and the progress we make is down to the efforts of everyone who works here as part of the staff team and this is true today.   We have all played a part in supporting today’s developments which have been expertly led by the Media, Campaigns and Digital teams and drawing on colleagues across the charity.
 
Finally, Archie Hill, who is aged nine with Duchenne muscular dystrophy, had his wish granted when he met the Prime Minister following his letter which was picked up far and wide on social media.   
 
Using the attached photo, please do feel free to tweet:  Today Archie's dream came true when he met @David_Cameron. Thanks to all who tweeted and made it happen #Translarna
 
It’s been a good day in which the work and impact of the Muscular Dystrophy Campaign has been widely recognised."