Currently, there are two approved treatments for SMA: risdiplam and nusinersen. These treatments were initially approved temporarily under Managed Access Agreements while the NHS and NICE gathered data to assess their cost-effectiveness. Now, as these agreements come to an end, both treatments face a crucial review.
While these treatments may not have worked for everyone, they have undeniably transformed the lives of the majority of SMA patients. However, many of these life-changing improvements often go unnoticed by traditional measurement scales and clinical assessments. That’s where we need your support!
We cannot afford to be complacent. There is a very real risk that one or both of these treatments may not receive further approval. We mustn’t take anything for granted. That’s why it’s absolutely vital to gather as much data as possible to strengthen our submission to NICE and ensure that these treatments remain available.
We urgently need you to complete the following surveys dependent on your circumstances. You can complete this on your own behalf or someone can complete it for you, but either way, please do complete it!
This may be our one and only opportunity to explain to NICE and the NHS and what treatment means to us, families and loved ones living with SMA, and more importantly, the impact it would have one removing such treatment. Please note that we are not overestimated the risks, we cannot take anything for granted. These surveys must be completed by 17 March 2024.
We also invite anybody to send a written case studies of their experiences with treatment, how has it improved your loved ones health? This could be physical, emotional, what impact would it have losing the treatment? You are welcome to send videos of things that you can do now that perhaps you could do previously, or even things that you can do now that perhaps wouldn’t have been able to add you not being on treatment due to progression. Remember treatment is not all about improvements, stability is a significant improvement for many due to the undeniable natural progression of the condition.
Join us in this important endeavor. Together, let’s make sure that the voice of SMA patients is heard loud and clear. Your input can make a significant difference in securing these treatments for the future. If you would like to submit a case study then please do so by emailing us at treatsma@treatsma.uk or office@smauk.org.uk
If you’d like to know more about the approval process then we jointly took part in a webinar which can be viewed below:
