We are disappointed to learn of the Scottish Medicines Consortium’s (SMC) announcement today that it is not recommending Duchenne drug, Translarna, be approved for funding by the NHS in Scotland.
The SMC’s decision affects access to Translarna in Scotland, for children like nine-year-old Michael (pictured, meeting the First Minister Nicola Sturgeon).
The SMC has highlighted its reasons for rejection as being:
“Because the submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.”
Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said:
We are bitterly disappointed by today’s announcement. Our thoughts are with the Scottish families whose every hope has been pinned on this decision. They have endured an 18-month wait for news about a drug that could transform their sons’ childhoods, only to receive this blow today.
Translarna could give eligible boys precious time to be able to walk for longer, helping to delay the progression of the condition. For a young person with a shorter life-expectancy, facing the acute psychological, physical and practical challenges of a progressive condition, this is of immeasurable value. It is now left to parents, in a postcode lottery, to approach their regional NHS board, in the hope funding for this drug can be agreed for their child. We will continue to fight with the families.
We are now calling on Translarna’s producers to negotiate a sustainable price and to work with the SMC towards a solution, to ensure Scottish families don’t miss out on a drug that could transform lives. No one should ever underestimate that Duchenne muscular dystrophy is a devastating condition, and we should take every opportunity to slow or halt the condition while we search for a cure.
How does this affect the rest of the UK?
This does not influence the decision by NICE – which is likely to determine access to Translarna in England, Wales, Northern Ireland and the Isle of Man. An announcement is expected on Monday 18 April.
This will undoubtedly be a very difficult time for families in Scotland on hearing this announcement. We’d like to remind you that we are here to support you. Please call us on our freephone helpline – 0800 652 6352 – or alternatively you can call Samaritans on freephone 116123, if you would like to talk.
What happens now?
Muscular Dystrophy UK strongly believes the SMC has made the wrong decision. They have failed to recognise the severity of Duchenne muscular dystrophy and the potential for Translarna to keep boys walking for longer.
We also have serious concerns about the restrictions the SMC placed on participation from families. Robert Meadowcroft, our Chief Executive, wrote to the SMC to raise these issues in February and March 2016.
We understand that the SMC and the drug company PTC Therapeutics are set to meet to discuss next steps. We’re clear that they both have a duty to find a way forward in the best interests of the boys and their families.
Calls to action
We are calling for:
- the SMC and the Scottish Government to introduce more flexible approaches to approving new drugs, including Managed Access Agreements. This type of agreement has been used in England, for example for the drug, Vimizim, for Morquio syndrome. This should be applied to Translarna and would enable access to the drug at a reduced cost while further evidence is gathered on its use.
- the First Minister of Scotland, Nicola Sturgeon, to intervene and promote a Managed Access Agreement on the drug
- all families whose children are eligible for the drug to speak to their child’s clinician and request that they submit an Individual Patient Treatment Request (IPTR) to their local Health Board. Although the SMC is not recommending the drug for use, it can still be prescribed on an individual basis. We are also in contact with our partner law firms to help take this forward.
How you can get involved
You can get involved by:
- signing a petition to the First Minister from boys in Scotland who are affected by Duchenne muscular dystrophy
- writing to the First Minister, Nicola Sturgeon and Chairman of SMC, Jonathan Fox, to ask them to intervene and promote a Managed Access Agreement on the drug
- tweeting the First Minister @NicolaSturgeon asking her to ensure access to Translarna on the NHS in Scotland
- writing to your MP to ask them to write to the First Minister
- asking your Scottish Parliamentary election candidates to pledge to campaign for access to Translarna, as well as other emerging treatments for Duchenne muscular dystrophy, by signing up to our Manifesto for Muscle.
For more information, please contact Jonathan Kingsley on 020 7803 4839 or email firstname.lastname@example.org
If you would like to speak to a member of our helpline team please call us on freephone 0800 652 6352, or alternatively you can call Samaritans on freephone 116123, if you would like to talk.