We are calling on our Health Service in Northern Ireland to authorise, approve and deliver Risdiplam under the Early Access to Medicines Scheme for Adults with Spinal Muscular Atrophy (SMA) living in NI.

Myself and my friends with have been fighting for our lives, our entire lives. We were born with the genetic condition Spinal Muscular Atrophy. It has robbed us of our ability to walk, stand, sit unaided, swallow, and even weakened our breathing muscles. For some of us, we even need to use non invasive ventilators when we sleep.

For me, and my family, we have born the most severe brunt of SMA. My sister Martina died on January 4 1997, just 20 days before her 15th birthday. 

Risdiplam HALTS all progression in SMA and allows some people to gain back some abilities. It prevents or lessens the serious chest infections some of us fight constantly. The same type of infection which took my sister, Martina.

I am 30 years old. Over the past 5 years, myself and my friends have watched with bittersweet happiness as actual treatments for SMA were approved. Due to our advanced disease, we were not eligible to receive these treatments.

On September 17 2020, that all changed. Risdiplam - a drug which helps our bodies produce the protein we are missing which causes our muscle weakness and complex health issues - was made available under an Early Access to Medicines Scheme (EAMS) by the Medicines and Healthcare products Regulatory Agency (MHRA).

An EAMS programme is used where there is a “severe unmet clinical need” for treatment so that those patients most in need of intervention can have treatment while the European Medicines Agency (EMA) license the drug.

EAMS means that Risdiplam is currently free of charge to the NHS. 

The EAMS programme for Risdiplam guarantees access for eligible patients now and for at least 5 years. This includes if the EMA decide to not license or restrict the license to certain groups of patients with SMA or if NHS decides not to fund Risdiplam for all patients under the license.

Essentially, EAMS guarantees access to adults with SMA whatever the outcome of the licensing and NHS commissioning processes.

We have been working with our specialist medical team to bring access in Northern Ireland since September 17. In October 2020 we were told there were pharmacy issues which were quickly resolved.

In November 2020 we were told that the “clinical governance leads” needed to sign off the EAMS, but that this was a “formality” and would take no longer than 2-3 weeks.

Ever since then, every 3 weeks we have been told it will take 2-3 weeks to sign off or that sign off was expected “imminently”.

On 7 January 2021 we had a clinic appointment to go through the paperwork associated with EAMS, and we agreed that treatment with Risdiplam was something we wanted to do.

At the end of January we were once again told sign off from the clinical governance leads was expected “imminently”. On 18 February 2021 a statement was provided to BBC Radio Ulster which said the EAMS programme had been approved and patients could expect to begin receiving Risdiplam in the next 2-3 weeks.

Then, thanks to the efforts of my MLA, I received an update dated 19 February 2021 which contradicted the statement given to BBC Radio Ulster just the day before. It stated that governance sign off was still being completed and that patients had not yet been assessed for their suitability.

Both statements said that children with SMA in Northern Ireland have been able to access EAMS since November 2020. We are happy for them, but feel like we are being treated differently because of our age.

As patients this left us with many questions.

Is EAMS for Risdiplam in adults signed off or not?

We have already signed consent forms and been deemed eligible. Why are statements saying we have not been?

Why has the adult EAMS not been sorted when children’s has?

What actually remains to be completed?

We are calling on our Health Minister Robin Swann MLA, the Secretary of State for Northern Ireland the Rt Hon Brandon Lewis CBE MP, the Northern Ireland Department of Health and the Belfast Health and Social Care Trust to approve this EAMS for Risdiplam for adults with SMA in Northern Ireland and give us a chance to live.

The European Medicines Agency is expected to make a decision on whether to license Risdiplam in March. The FDA in the USA has already licensed Risdiplam and there is no reason to suggest that the European Medicines Agency will not follow suit. Once that decision is made, our time is up.

Our ask is simple: provide to adults with SMA in Northern Ireland what children are receiving and what adults across the UK are receiving - hope for the future and no more progression from SMA.

There are less than 10 adults living with SMA who are eligible for Risdiplam under EAMS. But this would be life changing for every one of us.

Thank you for supporting us.