Searching for a cure, Improving lives

Clarity required from the Prime Minister over Translarna

Posted on: September 8th, 2015 | 0 comments

Action Duchenne have written to Prime Minister, David Cameron, requesting clarity over his correspondence with Luca Fernandes, who lives with Duchenne muscular dystrophy.


On June 9, Action Duchenne and MDUK submitted letters to Number 10 Downing St appealing for an intervention on Translarna. Nevertheless, despite first hand accounts from patients and families detailing the intransigence of NHS England, the life changing benefits of Translarna, and the gross injustice to which they’d been submitted, Mr Cameron refused to intervene, stating that, “decisions about whether the NHS should fund new treatments and drugs for rare diseases are very complex […] and they are rightly made by clinical experts at NHS England, not politicians”.

However, in addition to this comprehensive reply, the Prime Minister also responded to hand written letters from six boys, living with Duchenne. One of these, sent to Luca Fernandes, contains a disquieting statement in which Mr Cameron affirms, “the NHS should not use Translarna until further information becomes available on how well the drug works”.


We are concerned that this advice seems to contradict the expectations of the Duchenne community about the scope and considerations of NICE’s ongoing Highly Specialised Technology appraisal of Translarna, which is set to render a funding decision independent of any ‘further information’ on November 17.

Subsequently, we have requested further clarification that the Prime Minister’s statement is not prescient of an impending and premeditated refusal, by NICE, to grant funding approval for Translarna.


To read our full letter, click the following link: PM David Cameron Letter


Action Duchenne are also investigating the use of Pharmaceutical Price Regulation Scheme (PPRS) payments by NHS England  and the implications that the direction of these resources had upon Translarna’s negative funding decision in July.


For more information on the ongoing campaign for access to Translarna, and the broader campaigns work of Action Duchenne, please contact Aaron Revel (Campaigns Officer) on 020 8556 9955 or via

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