Blog
RSS FeedPosted by Michael Wonder on 15 Jan 2024
Sebelipase alfa for the treatment of patients with Wolman disease (final guidance)
10 January 2023 - Sebelipase alfa is recommended as an option for the treatment of patients with Wolman disease only if ...
Posted by Michael Wonder on 13 Dec 2023
Velmanase alfa for the treatment of patients with alfa mannosidosis
13 December 2023 - NICE has published final evidence-based recommendations on the use of velmanase alfa (Lamzede) for treatment of ...
Posted by Michael Wonder on 04 Nov 2023
Elexacaftor with tezacaftor and ivacaftor, tezacaftor with ivacaftor and lumacaftor with ivacaftor for the treatment of patients with cystic fibrosis
3 November 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 26 Oct 2023
Olipudase alfa for the treatment of patients with acid sphingomyelinase deficiency (Niemann-Pick disease type B and AB)
26 October 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 15 Aug 2023
Cipaglucosidase alfa and miglustat for the treatment of patients with late-onset Pompe disease
15 August 2023 - NICE has published evidence-based recommendations on the use of cipaglucosidase alfa (Pombiliti) with miglustat (Opfolda) for ...
Posted by Michael Wonder on 04 Aug 2023
Setmelanotide acetate for the treatment of people with obesity and hyperphagia associated with Bardet-Biedl syndrome
3 August 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 02 Mar 2023
Asfotase alfa for the treatment of certain patients with paediatric-onset hypophosphatasia
1 March 2023 - This evaluation reviews the evidence for asfotase alfa for the treatment of patients with paediatric‑onset hypophosphatasia (HST6), ...
Posted by Michael Wonder on 18 Jul 2022
Velmanase alfa for the treatment of alfa mannosidosis
15 July 2022 - The Department of Health and Social Care has asked NICE to produce guidance on using velmanase alfa ...
Posted by Michael Wonder on 06 Jul 2022
Setmelanotide for the treatment of patients with obesity caused by LEPR or POMC deficiency
6 July 2022 - NICE has issued fvidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC ...
Posted by Michael Wonder on 05 May 2022
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
5 May 2022 - NICE has published evidence based recommendations on the use of selumetinib (Koselugo) for the treatment of children ...
Posted by Michael Wonder on 01 Mar 2022
ABPI response to England Rare Diseases Action Plan
28 February 2022 - England's Rare Disease Action plan is published today with details of how to improve diagnosis, care and ...
Posted by Michael Wonder on 22 Feb 2022
NICE recommends Albireo’s Bylvay (odevixibat) for all PFIC types
22 February 2022 - Positive NICE review completed in less than six months post MHRA approval. ...
Posted by Michael Wonder on 13 Nov 2021
NICE does not recommend elosulfase alfa for NHS use following evaluation of new evidence
12 November 2021 - NICE has today published draft guidance for public consultation which does not recommend elosulfase alfa (also called ...
Posted by Michael Wonder on 02 Jun 2021
NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy
2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...
Posted by Michael Wonder on 31 May 2021
Examining the impact of different country processes for appraising rare disease treatments: a case study analysis
31 May 2021 - Conventional appraisal and reimbursement processes are being challenged by the increasing number of rare disease treatments with ...