Today is a landmark day for all families with the news that 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.
The decision is based on a Managed Access Agreement and now needs to be implemented by NHS England. We’re pleased that the Managed Access Agreement – which we called for last year and have worked to help develop – has led to the approval of the drug.
A Managed Access Agreement is when a drug is made available for a limited period of time (in this case for 5 years) often at a discounted price, to allow further evidence to be gathered on its use whilst also ensuring that patients receive access to the drug.
What happens now?
There is now a legally required consultation period on the implementation of this positive guidance before the assessment process is formally completed on 25 May.
It is fantastic news that we are close to boys being able to access Translarna on the NHS in England. The priority now is to make sure that NHS England implements the decision as soon as possible.
While NHS England has 3 months to implement NICE’s guidance from 25 May, we believe that this would be an unacceptably long period of time for families, who have already waited over 18 months for a decision.
For the last drug that went through a Managed Access Agreement – Vimizim to treat Morquio disease – the decision was implemented in 28 days. We’re calling for Translarna to be implemented by NHS England with the same speed within 28 days.
You can get involved by: Writing to your MP asking them to contact James Palmer, Clinical Director for Specialised Services at NHS England, who has previously said that he could push through a streamlining of the process after the final guidance
- Writing to your MP asking them to sign up to a Parliamentary motion put forward by Mary Glindon MP and to contact James Palmer, Clinical Director for Specialised Services at NHS England, who has previously said that he could push through a streamlining of the process after the final guidance
- Tweeting @NHS England urging them to implement the decision without delay
Louisa Hill, whose son Archie (10) met David Cameron last year to appeal for support for the campaign, said:
This is amazing, life-changing news. We can’t quite believe it, and just want to thank everyone who has supported us. We are immensely proud of Archie.
Duchenne muscular dystrophy brings so many challenges into his life, and this will become even greater in the future. Yet now, for the first time, there is something in our corner. We can have real hope that when, one day, there may be a drug to not only to slow down the condition, but completely stop it, our son will be strong enough to benefit.
Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said:
Parents of children eligible for Translarna have fought courageously for this outcome, and to give their children the chance to keep walking for longer. This announcement comes as wonderful news and a true victory for the families.
A Managed Access Agreement means that within a matter of months, NHS England will be supplying eligible children with the first genetic therapy available to treat the condition. Such an agreement will allow us to gain a clearer picture of the full potential of Translarna, and, crucially, to buy precious time for other promising potential treatments to reach licensing stage. It is a chance to transform childhoods.
However, we are concerned now that it could takes months for NHS England to implement the agreement and get the drug to clinic. Having waited 18 months for a decision, this is a delay boys and their families can ill afford.
We call on NHS England to act with the urgency and resolve that these children and their families deserve.
Professor Dame Kay Davies commented:
This is an historic moment in the development of therapies for Duchenne muscular dystrophy and shows that drugs can move all the way from early stage clinical trials to NHS clinics.
I am delighted at NICE’s decision and pay tribute to Muscular Dystrophy UK and fellow charities for their tireless efforts, which have led to NICE’s announcement today.
Muscular Dystrophy UK Ambassador and England football, Jack Wilshere, said:
As an ambassador for Muscular Dystrophy UK, I am delighted at the news that Translana will be funded in England. It will make a life-changing difference to boys like my friends Archie and Harry.
Thank you to everyone for helping to get this amazing result, and continuing the fight against muscle-wasting conditions.
Campaigning pays off
The tireless campaigning of families has brought us to this approval from NICE. With you, we’ve worked to bring together clinicians, NHS decision-makers and politicians to fight for access to Translarna. That has meant:
- two visits to Downing Street (including the five boys delivering their letters to the Prime Minister, pictured);
- a meeting with the PM;
- six meetings with Health Ministers
- working with other charities for a joint parliamentary lobby event
- over 90 questions and speeches from MPs and Peers across the political parties on access to Translarna.
- nearly 800 pieces of press coverage across the country, including BBC News, ITV News, Good Morning Britain, the Daily Mail, the Sun, the Guardian and the Daily Mirror.
- Over 2.5 million people worldwide hearing about the campaign through our Thunderclap
- support from Muscular Dystrophy UK Ambassador Jack Wilshere
Dave Anderson MP, Chair of the All Party Parliamentary Group for Muscular Dystrophy, said:
All the campaigning from the families and charities over the last 18 months has finally paid off. This is an historic decision and I know that all my parliamentary colleagues from across the party spectrum who have taken a keen interest in the progress of the assessment for Translarna will join me in welcoming this excellent news for the boys who can benefit from the treatment. However, the lengthy frustrating delays which we have seen must not be allowed to happen again for future treatments which are put forward for assessment. The approval system for new treatments must be urgently improved.
Cheryl Gillan MP, MP for Chesham and Amersham whose constituent Archie Hill will benefit from access to Translarna, commented:
This is wonderful news for Archie and all the boys who can benefit from Translarna. The long wait for NICE’s verdict is finally over and it is the outcome we have all been hoping for. I would like to pay tribute to the Hill family and to all the families involved in the campaign for their tenacious and tireless efforts to reach this point.
What does this mean for future treatments for Duchenne muscular dystrophy?
Translarna will be the first drug dealing with the underlying genetic cause of Duchenne muscular dystrophy available on the NHS in England.
This is an historic moment and a major step forward in the fight for potential treatments for muscular dystrophy: proof that new drugs can make it all the way from early experiments in the laboratory to the clinic for the benefit of patients.
With other treatments currently in clinical trials, we hope that Translarna is the first of many treatments to come through and be approved.
However, the assessment that has finally led to the approval of Translarna has been far too slow and bureaucratic, with many consultation stages failure to negotiate on price at an early stage.
We’re campaigning to fix the UK’s drug approval system. Find out more.
As part of our ‘Duchenne dialogue’ series over the coming weeks we will also have a series of webinars and Facebook chats with leading research and clinical experts in the Duchenne field to talk about potential future treatments.
What about the rest of the UK?
We expect NICE’s decision to lead to implementation in Wales, Northern Ireland and the Isle of Man. Today we are writing to Health Ministers in Northern Ireland, Wales and the Isle of Man to ensure that there is no delay to accessing Translarna.
However, in Scotland, the process has been entirely separate. Last week we heard of the bitterly disappointing decision by the Scottish Medicines Consortium to recommend that Translarna not be approved for funding by the NHS in Scotland.
As a result of the NICE decision, today we are writing to Scottish Government First Minister Nicola Sturgeon the Scottish Affairs Select Committee in the House of Commons to seek a review of how families in Scotland are not able to access to Translarna, while it is made available in England.
The fight goes on for access to Translarna in Scotland and you can find out more about how you can get involved in the campaign here.
For more information, please contact Peter Sutton on 020 7803 4838 or email firstname.lastname@example.org
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