Blog
RSS FeedPosted 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12 November 2021 - NICE has today published draft guidance for public consultation which does not recommend elosulfase alfa (also called ...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NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy
2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...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 ...
Posted by Michael Wonder on 29 Sep 2020
The estimation of health state utility values in rare diseases: overview of existing techniques
25 September 2020 - There are several techniques for estimating health state utility values, each of which presents pros and cons ...
Posted by Michael Wonder on 18 Sep 2020
NICE U-turn sees Akcea's Waylivra win NHS funding
18 September 2020 - Akcea Therapeutics UK's Waylivra (volanesorsen), the first and only therapy for Familial Chylomicronaemia Syndrome (FCS), will ...
Posted by Michael Wonder on 06 Mar 2020
Girl loses High Court battle over NICE review of BioMarin’s phenylketonuria drug
5 March 2020 - An 11-year-old girl with the rare disease phenylketonuria has lost a legal battle in the High ...
Posted by Michael Wonder on 24 Oct 2019
NHSE signs ‘definitive’ agreement for Vertex CF drugs
24 October 2019 - NHS England has announced a definitive agreement with Vertex Pharmaceuticals to make all three of their ...
Posted by Michael Wonder on 17 Sep 2019
News NHS England explores funding options for cystic fibrosis drug
17 September 2019 - NHS England is “committed to exploring and developing alternative routes” to enable people with cystic fibrosis ...
Posted by Michael Wonder on 12 Sep 2019
Batten disease treatment: parents win battle ahead of court date
11 September 2019 - Families of children with a rare degenerative disease have won their fight for funding for NHS ...
Posted by Michael Wonder on 11 Sep 2019
Children with rare inherited condition to benefit from drug through managed access agreement
11 September 2019 - It is estimated that in the UK there are around 30 to 50 children living with the ...
Posted by Michael Wonder on 18 Jun 2019
Buyers’ Club wants government support on Orkambi imports
17 June 2019 - Proposes three point plan to work round impasse. ...
Posted by Michael Wonder on 11 Jun 2019
MPs to debate cystic fibrosis drug Orkambi in Parliament
10 June 2019 - A debate is due to take place in Westminster about a drug for people living with ...
Posted by Michael Wonder on 11 Jun 2019
Orkambi row: government now considering Crown Use licensing
11 June 2019 - MPs call for radical solutions as deadlock remains. ...
Posted by Michael Wonder on 05 Jun 2019
Families create buyers club for cut-price cystic fibrosis drug
5 June 2019 - Stalemate in NHS talks over Orkambi leads parents to buy generic version from Argentina. ...
Posted by Michael Wonder on 27 May 2019
NHS England wants money back guarantee on Orkambi
24 May 2019 - Vertex resisting real-world data proposal. ...