In light of NHS England’s non-decision on Translarna last week, Action Duchenne shared a platform with the MPS Society in parliament yesterday to discuss the existing access barriers to medicines for patients with rare or ultra rare conditions. This event was chaired and supported by Greg Mulholland MP, who has been an inspirational and leading figure in the ongoing campaign.

Action Duchenne CEO, Paul Lenihan MBE contextualised the situation for those in attendance, outlining that 17 months after Translarna gained EMA approval from 28 member states, families and boys in Britain are still waiting to benefit from this groundbreaking treatment. In addition to failing to construct a fit and robust process to evaluate treatments, NHS England have moreover provided inconsistent and disingenuous advice to the community throughout this period.

After NHS England scrapped its specialised commissioning process Paul stated at the parliamentary lobby in January, “a decision not to make a decision, is cowardice”. After NHS England’s announcement to defer their decision on Translarna to NICE, it is clear that these words remain as germane as ever seven months down the line.

Action Duchenne trustee Mark Silverman also offered a parents’ perspective on Translarna’s ongoing unavailability. In a powerful and impassioned speech he accused NHS England of “a gross dereliction of duty”, and implored those MPs in attendance to speak to Health Minister Jeremy Hunt, and help hold him to account.


– As noted above, Translarna is still to be evaluated by NICE via their Highly Specialised Technology Route. We are expecting an interim decision on funding on November 17. Action Duchenne will continue to work within the NICE framework to help ensure Translarna receives long term funding approval.

– We will also seek to ensure interim funding for Translarna. NHS England has no fair and equitable means of funding treatments for orphan, rare and ultra rare conditions. We are therefore composing letters to Health Secretary Jeremy Hunt and Prime Minister David Cameron asking them to act upon their ministerial power to intervene and direct NHS England to offer funding approval.

– We will continue to support and advise families to make clinically critically urgent funding requests for Translarna. A treatment is clinically critically urgent when “the patient is as risk of imminent significant and irreversible clinical deterioration”. Owing to the degenerative nature of Duchenne and Translarna’s novel status, this is hugely pertinent.

– We are meeting with Director of Specialised Commissioning, Richard Jeavons tomorrow (8th July) to discuss NHS England’s decision to defer their decision to NICE.