After submitting a letter to No.10 Downing St on June 9, the Prime Minister David Cameron sent a host of replies to Action Duchenne yesterday to pass on to those patients and families who implored him to intervene on Translarna.

In one letter to parent and Action Duchenne supporter, Rob Baker, Mr Cameron empathised; “I understand your concern about access to a new and potentially beneficial treatment. I fully appreciate your frustration and realise that waiting to find out if Translarna will be provided has caused further anxiety and stress to you and your family at such a difficult time”.

However, despite these sentiments, the PM has refused to intervene and direct NHS England to approve funding for Translarna, stating rather, that parents should put their faith in NICE’s Highly Specialised Technology Appraisal process to yield a just, albeit, belated decision. “Decisions about whether the NHS should fund new treatments and drugs for rare diseases are very complex, as I hope you understand, and they are rightly made by clinical experts at NHS England, not politicians”.

(Read David Cameron’s full letter by to Rob Baker by clicking on  David Cameron Reply (part 1) David Cameron Reply (part 2) )

Whilst we appreciate the Prime Minister taking the time to write individually to a number of parents, patients and supporters, we are disappointed with his reply. It is clear that the clinical experts from NHS England he entrusts to make complex decisions have, in the instance of Translarna, wholly failed to do so. Indeed, as last weeks stance by NHS England’s Patient and Public Voice Assurance Group (PPVAG) proves, they still have no fair and equitable means of funding treatments for orphan, rare and ultra rare conditions.

Subsequently Action Duchenne will:

– Continue to challenge both the Prime Minister and the Secretary of State for Health, Jeremy Hunt, to make an intervention on Translarna by highlighting the inconsistencies and misguidance which have characterised NHS England’s communications and (non) decision making processes.

– We have further submitted  FOI Requests to the Treasury, Department of Health and NHS England, seeking to gain crucial information on existing funding arrangements for new drugs, and the minutes from last months CPAG meeting. We will use the information garnered from these inquiries to substantiate our Ministerial lobbying efforts and elevate our previous formal complaint to the Parliamentary and Health Service Ombudsman.

– 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, and are meeting with the manufacturer PTC Therapeutics today to discuss the NICE submission.

A big thanks to the Hill family, Mark Silverman, the Liquorishs, Jacqueline and Tony Lincoln, the Peggs, the Geary’s, Pedro Ferndandes and all the boys who submitted letters to No.10 in June.