This survey is for carers of adults and children diagnosed with SMA.
The National Institute of Health and Care Excellence (NICE) approved Nusinersen (Spinraza) for funding by the NHS in England in July 2019 and for Risdiplam (Evrysdi) in August 2020.
This was on the condition that researchers would collect more information from patients and their clinicians on how well it works, how safe it is and whether or not it is good value for money. This was because, at that time, the NICE committee did not feel there was enough evidence from the clinical trials to answer these questions. SMA, being a rare disease meant the numbers of participants were not big enough to satisfy a final decision.
This is why both treatments have been funded via time limited Managed Access Agreements (MAA) which come to an end this year. This is also why, if the person you care for is prescribed either of these drugs, they will have been completing physio assessments, answering questions and completing patient reported outcomes.
With two treatments being assessed which treat both the paediatric and adult population living with all types of SMA, sharing all of these perspectives with NICE is a difficult task.
The clinical data already collected does not fully represent the real life impact these treatments have had on the everyday lives of those living with SMA. Therefore, it has never been more crucial for the three leading patient organisations to come together to collect important information from the SMA community across the UK.
This survey will only take approximately 15 minutes and your responses can make a difference.
If you have any questions about this research then please email portia.thorman@smauk.org.uk
