Urgent Call to Action – Translarna

January 30, 2015 11:13 am

Translarna Lobby

Patients with Duchenne Muscular Dystrophy have been specifically disadvantaged by the failure of the NHS England system to consider funding drugs for rare diseases. England was a leader in the trials for Translarna, in keeping with the government strategy to promote innovation and inward investment in trials. Just 80 patients might benefit from this drug if it is approved now, but every month a funding decision is delayed boys lose eligibility for the drug to be prescribed. Meanwhile in other countries where patients and hospitals did not invest so much in the trial process, the drug is now funded. Not only are patients suffering, but the reputation of England as a place to develop innovative therapies is under challenge. Patients with not only Duchenne but also other rare diseases are demoralized that the research and development to which they and the NHS contribute threatens to come to nothing due to a series of unnecessary bureaucratic hurdles.

Can the government show its commitment to patients with rare diseases by making an exceptional decision for the 80 boys for whom this treatment may otherwise not be available until too late? While guaranteeing that this situation will not arise again and that other rare disease indications will be speedily addressed in a streamlined and fair process in the future. 

There are four things we urgently need to draw your attention to and need your help with!

Number One

Action Duchenne in co-operation with the broader Duchenne community will be hosting a public vigil outside Skipton House, the offices of NHS England, in Elephant and Castle, London on February 11th. This event will utilise the momentum and public awareness raised from the recent parliamentary lobby to keep the pressure on NHS England to take emergency steps to ensure Translarna is made available now to all children who could benefit. For more information visit: http://www.actionduchenne.org/vigil-for-translarna-nhs-england-11th-february/

Number Two

NICE will be organising a scoping workshop in March to agree procedures and terms of reference to be able to evaluate the longer term funding and patient access for the drug Translarna. Alex’s Wish and all other Duchenne patient organisations will be registering and taking part in this consultation.

Number 3

Following this scoping meeting and NHS approval, NICE will be creating a website for patients and their families to be able to make submissions that will be considered for a full scale evaluation of Translarna. It is vital that all Duchenne families and those living with Duchenne contribute to this NICE consultation as this will determine long term access to Translarna and set a precedent for other Duchenne drugs coming to market in the UK. We’ll send you the link as soon as this website goes live.

Number 4

NHS England are consulting over their procedures for commissioning drugs like Translarna for the next 90 days.


Can we try to get them to prioritize novel drugs for Duchenne? Please email england.boffice_speccom@nhs.net for more information.

Lets make sure our community is heard.

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