Harrisons Fund

Devastating Decision from SMC

11 Apr 2016

We are devastated to hear of the Scottish Medicines Consortium's decision on Translana in Scotland today, they said:

'The Committee was unable to recommend ataluren for routine use in NHS Scotland for patients with Duchenne Muscular Dystrophy (MD). The treatment is licensed for use in those patients with a rare genetic mutation that affects 13% of those with the condition. Duchenne MD is life limiting disease which causes muscle wasting. Following consideration through the PACE process, the Committee was unable to recommend ataluren as there was too much uncertainty about the overall clinical benefits it might bring in relation to its cost. Following a meeting between the company and SMC, the company has indicated their intention to resubmit.'

Gary Fegan, father of Cormac puts the heartache best below:

'I feel absolutely devastated at this decision, which at the end of the day came down to economics, not whether or not the treatment works. Two other families in Scotland have boys currently using Translarna and we know that it is effective and have tried to put this case across, which seems to be have just been dismissed. In the case of Cormac it has transformed his disruptive behaviour that led to his eventual diagnosis as well as helping make him physically stronger.

The SMC has not grasped the importance of allowing these children to remain walking and independent into their late teens and importantly post puberty rather than the natural progression of being confined to a wheelchair before their teens. With a life expectancy of Duchenne children being just their mid-twenties and children still succumbing to Duchenne before their tenth birthday, being able to keep a child walking for an additional 8 years is over a quarter of their entire life and through their most formative years at that!

With the extended trial about to close and with Cormac's IPTR coming up for renewal the future is uncertain for everyone lucky enough to have access to Translarna and utterly devastating for those families who have eligible children now or in the future; all of this against a background of NHS cuts.

We can only hope that PTC appeal and an agreeable deal is struck. Patients and families feel completely powerless in this whole decision, all we can do is beg the SMC and PTC to move away from a sealed bid approach to pricing and get into constructive dialogue; we aren't talking about buying a house, this is children's lives. It sickens me that it comes down to "what is the cost of the treatment?" when the SMC haven't truly understood the benefit!

Parents of children and young adults with Duchenne or any other life limiting disorder will know that for too long we've lived with no hope. Almost weekly now we see headlines about gene editing or "Duchenne cured in dogs" and after the initial flicker of hope, it is instantly extinguished by realisation that even if these treatments do work, it's likely to be 10 years before they hit trials. So it makes it even more heart-breaking that a drug like Translarna, again over 10 years in the making, has been rejected. Translarna isn't a cure, it doesn't work for everyone and it's not even 100% effective in those it should be able to treat, but for some, like Cormac it makes a huge difference.

Cormac has been described as a relatively lucky boy when it comes to Duchenne by other Duchenne parents because there's a treatment of sorts available. I'm now not sure whether it's better to live with next to no hope or know there's an effective treatment available within your grasp that you can't have. This journey is hard enough as it is; it doesn't need more obstacles and heartache thrown in the mix.

Today's decision could easily be a small ripple that quickly dissipates with attention within the community turning to the decision by NICE or the FDA Ad Comm for Eteplirsen. It's easy with all the stress of living with Duchenne to not worry about this, especially if it won't help your boy. But this is setting a precedent that effectively says, if you present an orphan drug for Duchenne that isn't a cure then it won't be approved. These drugs are looked at in isolation, what if Translarna + utrophin upregulation gives 15 years of ambulation but utrophin upregulation, like Translarna only gives 8 years? Then we deny both drugs and never have a chance to find out.

Until there's a cure then it will be a combination of drugs and treatments that will help keep our children alive and enjoying life until they finally get stabilised and then cured. Today's decision put the first nail in the coffin of this dream for this generation. So I'm asking you reading this to take 5 minutes to do something. You can share these words, you can start a petition, you can speak to a reporter – whatever you do, please don't sit back and wait for the day where a treatment that could help your child or loved one is in this position before taking action. In the way I'll be flying to Washington DC to support Eteplirsen in 10 days, a drug that won't help Cormac in a country where he doesn't live, I know that all these decisions are looked at and discussed in the approval process. The more negative decisions that are made, then the easier it will be to make them in the future.' Gary Fegan


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