Vigil for Translarna

February 17, 2015 10:54 am

On Wednesday, 11th February, Alex’s Wish took part in a vigil at the door of the NHS England alongside a number of Duchenne charities, families and supporters to demand access to a new ground-breaking drug Translarna.  Translarna is the first drug to be approved that offers the prospect of delaying disease progression and keeping boys on their feet for longer.  Whilst it will only help 13% of the Duchenne population (unfortunately Alex is not eligible as he has a different mutation type) the fight to get this drug to those that need it is felt across the Duchenne population. 

Supporters raised placards, distributed impact statements and demanded a meeting with a representative of NHS England, which was denied to the Duchenne community at last month’s parliamentary lobby.

After just over half an hour, Neil Churchill the Director of Patient Experience came to meet with us.  He stated that NHS England’s delay in accessing Translarna and the current 90 consultation was part of a broader agenda to getting ‘things right’ when it comes to funding treatments for rare conditions.  ‘Last year the process was wrong and it needed to be put right’ he argued and encouraged everyone to influence the new process through the medium of public consultation.

Neil was unable to clarify why the process was unfit for purpose in the first place. Nor was he able to comment about other EU countries that are already offering this treatment to patients, stating that England has its own processes and we have to follow them regardless of what’s going on elsewhere.

What was disappointing was that he had not fully considered the significant impact this delay would have on those boys who are eligible and their families.  As Duchenne is a degenerative condition, boys could lose their ability to walk at any time, and whilst this delay continues they are in fact costing some boys the ability to walk and access this treatment altogether.

It’s absolutely shocking that they are taking so long, this is the first ever drug to be approved for Duchenne that will delay symptoms and keep boys on their feet for longer. Whilst the drug will only help 13% of the Duchenne population, this does not give us much hope for future drugs coming to market. Charities like Alex’s Wish are raising huge sums of money to fund the science, but until NHS England can sort out their bureaucratic processes and decide how to fund new treatments for rare conditions, we have little hope of helping our boys.

The work continues. #Translarna

Posted in