Patients have been informed that the current lead consultant will be leaving at the end of March and there has been no communication about any arrangements in place for the continuation of care beyond this date.
The news raises major concerns about the access and availability of treatments including risdiplam, a treatment currently available to eligible adults living with Spinal Muscular Atrophy (SMA) through a Managed Access Agreement (MAA) for the Northern Ireland community.
The Trust is the sole provider of this treatment in Northern Ireland, and with the departure of the lead consultant, no prescriptions will be able to be signed for. Those patients currently taking the medication are on a ‘ticking time bomb’ with only weeks left of access to a life-changing drug.
The organisations are also concerned about the potential implications for access to other treatments, including Spinraza, a second SMA treatment, and the Duchenne muscular dystrophy treatment Translarna.
For patients with SMA or Duchenne muscular dystrophy, treatments aim to halt progression of the condition so that it does not get worse. A worsening of the condition could mean a loss of the use of muscles for swallowing or breathing, which represents a significant risk to the patients’ lives and their quality of life.
Michaela's Story
Michaela Hollywood MBE, 33, from Co-Down, who is Deputy Chief Executive of Pathfinders was diagnosed with SMA in 1991 at eight months old. She was first prescribed risdiplam in March 2021 and described it as ‘liquid gold’ and has seen a noticeable positive impact.
There was a 48-hour period where she was without access, which had a detrimental effect. In the first three hours she had a heavy sensation down her arms and fingers and within just 12 hours she couldn’t swallow.
With rapid changes evident in such a short space of time, Michaela is concerned that this could have dangerous results for her and many others currently using the drug if they were to stop, even temporarily.
Michaela predicts that she currently has around nine weeks of supplies left in what she describes as being ‘92 doses safe’. She said: “Too many people fought too hard to get this drug to be giving it up too easily. It’s a frustrating situation as there has been a lack of communication about the next steps, which could have a catastrophic impact on the lives of those living with SMA and their families too. Without access to treatment, and not because there’s a lack of drugs, this could see a rise in hospital admittance, so we’re hoping to get answers. At the moment patients are being kept in the dark.”
Our comments
Director of Campaign, Care and Support, Rob Burley, at Muscular Dystrophy UK, the leading charity for more than 110,000 children and adults in the UK living with one of over 60 muscle wasting and weakening conditions, said: “We’ve heard from a number of people and their families who are understandably extremely worried about the current situation, and we share their concerns. We’ll do all we can do help resolve this and can’t express enough the urgency to get some answers and clear information from the trust.”
Giles Lomax, CEO of SMA UK added: “It’s really important that everyone across the UK has equal access to treatment and we will advocate as strongly and as quickly as possible to ensure that there is a clear and immediate plan in place to continue with the Risdiplam service within the Belfast trust.”
Along with Spinal Muscular Atrophy UK (SMA UK) and Pathfinders Neuromuscular Alliance we have written to the Belfast Health and Social Care Trust expressing their concerns and as yet have not received a clear response on the future of the adult neuromuscular service.
If you are concerned about access to treatments in Northern Ireland please contact our support team. Call 0800 652 6352 (Monday-Thursday 10am-2pm) or email info@musculardystrophyuk.org.