Translarna Lobby

Alex’s Wish took part in a lobby at Westminster on Wednesday, 14th January with the aim to ensure early availability and full funding of a pioneering new treatment called Translarna. Translarna is the first drug to address the underlying cause of Duchenne. We were welcomed by Dave Anderson MP, Chair of the All Party Parlimentary Group for Muscular Dystrophy.

About Translarna

Translarna is only applicable to 10-15% of the Duchenne population with a nonsense mutation, unfortunately not applicable to Alex. It’s also the first treatment to have received conditional approval by the European Medicines Agency (EMA). It is the first licensed drug treating an underlying genetic cause of Duchenne ever to have been approved.  It was a landmark decision for the Duchenne community. Clinical trials of Translarna indicate that it could slow the progression of the condition and keep boys walking for longer. It has already been made available in European countries, including Spain, Germany, France and Italy.

Delays in patient access

Translarna was being assessed by NHS England, who will decide on whether it approve and fund it for boys in the England (around 200 patients).  Families had understood a decision would be taken in December 2014 and if positive the treatment would be available from April 2015.  However, due to the threat of a legal challenge from a non muscular dystrophy charity over the criteria used to assess treatments, NHS England announced out of the blue in December 2014 that it was putting the assessment on hold whilst it reconsiders its processes.

Families of boys who could benefit from the treatment were devastated to learn this news. Delays on even a few months could be the difference between accessing the treatment or not, as in that time a child may loss the ability to walk. Many MPs attended the lobby and Andy Burnham said, “when it is an issue between walking and not walking the NHS cannot hide behind processes”.  We were very disappointed that no one from NHS England attended the lobby, despite being invited.

Following this successful lobby, the Muscular Dystrophy Campaign, Duchenne charities and families submitted a petition of 24,000 signatures to No 10 Downing Street to get things moving as quickly as possible. If you haven’t yet signed the petition please do it only takes a few seconds. 

Next steps

The Duchenne community will continue to push NHS England and NICE to make a decision and various meetings are planned in the near future. Watch this space!

Follow the latest developments on Twitter by searching for #everydaycounts #duchenne #translarna #NHSEngland

Posted in Lobbying