Pompe disease
Rare disease · 2021–2021
1decision
1medicine
1carried consumer input
25individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| avalglucosidase alfa Long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid alpha-glucosidase deficiency) — specifically infantile-onset Pompe… | Nov 2021 | Not recommended | 25 individuals Australian Pompe Association · Rare Voices Australia |
Who spoke
Australian Pompe Association · Rare Voices Australia
Appraised elsewhere
NICE, in England and Wales, has appraised 1 technology for this condition, recommending 1 . A different committee, a different population and a different price: this is what has been looked at there, not a verdict on what happened here.
| Technology | Appraisal | Outcome | Date |
|---|---|---|---|
| avalglucosidase alfaAvalglucosidase alfa for treating Pompe disease | TA821 | recommended restricted | 2022-08 |
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 1 of 1 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.