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AGSD-UK attend NICE scoping workshop.

Scoping Workshop

Last Friday Kempton Rees and Luke Fraser were reps for the UK Pompe community regarding the proposal to appraise Neo GAA. The scoping workshop was called Glycogen storage disease type II (late-onset) – avalglucosidase alfa ID3737”.

Muscular Dystrophy UK and Metabolic Support UK were very helpful indeed too, they gave some guidance and background information and had reps attending.

Full appraisal questions

The NICE meeting included senior medical staff from some NHS Highly Specialised Centres for Metabolic conditions and staff from Sanofi Genzyme Ltd, the developers of Avalglucosidase alfa (also known as NEO GAA).

Topics focused on ensuring all the right questions would be raised in a full appraisal. This includes possible dose, potential benefits for patients and how it differs from current treatment available. It is likely that further discussion would include start-stop guidelines and a Managed Access Agreement as well as pricing.

We now wait between one to six months to find out whether there will be a full appraisal.

Will you get involved?

NICE, the National Institute for Health and Care Excellence, also offered training for those interested in participating in the future. We encourage anyone to take part in this training because if this new drug goes through for full appraisal AGSD UK will need as many people as possible supporting the campaign. 

 “We offer an induction open to all patient organisations regardless of previous involvement.  We intend this to be an informative introduction and / or reminder on how to get involved in technology appraisals and an opportunity to ask questions.  If you are interested in attending a 1 hour zoom session please contact for scheduled dates.