Within Avalglucosidase alfa’s approval process, MDUK took part in a NICE Scoping Workshop in September 2020, liaising with other patient groups and clinicians in advance. In November, we continued by partnering with the Association of Glycogen Storage Diseases to submit a written response to the NICE appraisal. We successfully nominated MDUK Trustee Baroness Thomas of Winchester who lives with Pompe disease, as a patient expert and she gave evidence to the NICE committee that approved it.
Over the past three years, we have seen several other treatments either become available or take significant steps to becoming approved for use by the NHS in the future.
In recent years, we have also worked to progress other treatments for a number of muscle-wasting conditions including three treatments for SMA (Spinraza, Risdiplam and Zolgensma), two for Duchenne muscular dystrophy (Translana and Fordadistrogene movaparvovec), one for myotonic dystrophy (Mexiletine), two for myasthenia gravis (Vyvgart and Ravlizumab) and a further treatment for Pompe disease (Cipaglucosidase alfa with miglustat).
MDUK is proud to be working closely with NICE and the SMC throughout the approval process, working hard in our FastTrack to treatments campaign to identify and remove the barriers that cause delays to ensure that patients can access and benefit from new, safe and effective treatments as quickly as possible. We do this by providing the patient perspective in NICE and SMC appraisals by attending committee meetings, putting forward patient representatives and submitting written responses to appraisals on the impact of living with the condition and changes since receiving treatment. Indeed, a wealth of this progression and advancement is thanks to patient involvement. Without insight into patients’ experiences, this progression would be unobtainable. We are determined to reach a world in which there are treatments, and eventually cures, for every muscle-wasting condition quicker by working together.
Rob Burley, MDUK Director of Care, Communications and Support, said, "When I joined MDUK over three years ago there was only one treatment available for one group of people living with a muscle-wasting condition and another going through the treatment appraisal process. It’s amazing progress that there are now ten treatments for people living with a range of muscle-wasting conditions either available in some form or going through the appraisal process. This is a remarkable advancement in such a short amount of time, and we're eager to continue our collaboration to secure more treatments, and ultimately find cures for muscle-wasting conditions."