Rare Diseases
(asked on 17th November 2014) - View Source
Question to the Department of Health and Social Care:
To ask Her Majesty’s Government how many medicines for the treatment of rare diseases the National Institute for Health and Care Excellence has considered; and how many it has approved since it took over responsibility for that function from the Advisory Group on National Specialised Services.
The National Institute for Health and Care Excellence (NICE) has been asked to evaluate the following treatments under its highly specialised technologies programme:
Assessment title | Anticipated publication date |
Atypical haemolytic uraemic syndrome - eculizumab | January 2015 |
Gaucher disease (type 1) - eliglustat | August 2015 |
Mucopolysaccharidosis (type IVA) - elosulfase alfa | October 2015 |
Paediatric-onset hypophosphatasia - asfotase alfa | To be confirmed |
NICE has not yet issued any final guidance through this programme.