The purpose of the meeting was to scope the remit of the actual Technology Appraisal (TA) process which will not start until the European Medicines Agency (EMA) gives Market Approval (MA) to Avalglucosidase Alpha. It will take about 12-15 months before the remit is prepared and the Highly Specialised Technology (HST) assessment will begin once Sanofi-Genzyme notify NICE that they have received Market Authorisation from EMA.
Allan Muir, Pompe Support Network chair, was able to provide historical experience of patients during the development of Myozyme and also the experience of patients receiving the commercial therapy. As Vice-chair of the International Pompe Association (IPA), Allan was also able to give a global perspective to therapies for Pompe. Other patient groups also attended the meeting with Metabolic Support UK offering information about one family’s clinical trial experience, and two adult Pompe patients offered their perspectives.
The meeting was chaired by Dr Peter Jackson (Clinical Pharmacologist, NICE) and clinical perspectives were provided by Prof Patrick Deegan (Addenbrookes), Dr Reena Sharma (BIMDG), and Dr Paul Gissen (GOSH). Other NHS and Sanofi representatives and observers completed the meeting attendance of about 21 people.
We don’t expect to receive minutes or feedback from the meeting; but hopefully we were able to ensure that the anticipated HST Assessment of Avalglucosidase alpha will take into account the views and experience of the UK Pompe community.
N.B.
If you are a UK resident and would like to share your experiences and perspectives at events such as this, or be more involved with supporting the Pompe community, please contact the Pompe Support Network at hello@pompe.uk.
