Blog
RSS FeedPosted by Michael Wonder on 04 Feb 2019
UK patient activist group takes aim at Orkambi patent
4 February 2019 - Calls to bypass patent adds to pressure on Vertex. ...
Posted by Michael Wonder on 04 Feb 2019
Calls for action on patients denied £100,000 cystic fibrosis drug
4 February 2019 - Ministers urged to consider revoking patent on life-extending Orkambi, which NHS cannot afford. ...
Posted by Michael Wonder on 31 Jan 2019
Vertex an “extreme outlier” in pricing and behaviour, says NHS England
31 January 2019 - Members of parliament aim to break three year deadlock. ...
Posted by Michael Wonder on 10 Oct 2018
XLH patients to get routine access to first new therapy in 30 years
10 October 2018 - For the first time in more than 30 years children and young adults in England and ...
Posted by Michael Wonder on 23 Aug 2018
Sobi secures NHS reimbursement for Ravicti
23 August 2018 - Rare disease group Swedish Orphan Biovitrum (Sobi) says it has gained NHS England reimbursement for Ravicti ...
Posted by Michael Wonder on 15 Aug 2018
'You have the medicine that can make me feel much better': boy, 8, pens letter asking drug company to lower prices
14 August 2018 - Watch eight-year-old Luis Walker, who has cystic fibrosis, read his letter to a pharmaceutical company urging ...
Posted by Michael Wonder on 09 Jul 2018
Vertex, NHS England no closer to Orkambi settlement
9 July 2018 - Vertex and NHS England remain locked in a battle over the provision of cystic fibrosis drug ...
Posted by Michael Wonder on 05 Jul 2018
Vertex calls NHS England ‘outrageous’ as Orkambi talks stall again
5 July 2018 - Public row about confidential pricing talks drags on. ...
Posted by Michael Wonder on 27 Jun 2018
Improving access to new treatments in rare diseases
21 June 2018 - Emily Crossley, co-founder and co-chief executive of Duchenne UK, tells us how a ground-breaking HERCULES project ...
Posted by Michael Wonder on 19 Jun 2018
Patients call for end to two-year row over cystic fibrosis drug funding
19 June 2018 - UK patients are stepping up efforts to resolve the “heartbreaking” row that is denying their families ...
Posted by Michael Wonder on 16 Jun 2018
Rare disease therapy Crysvita to be barred from NHS
15 June 2018 - Children and young people with X-linked hypophosphataemia are unlikely to get routine access to Kyowa Kirin’s ...
Posted by Michael Wonder on 23 May 2018
NICE says no to Chiesi’s rare disease drug
23 May 2018 - England’s cost effectiveness watchdog NICE has published draft guidance not recommending Chiesi’s enzyme replacement therapy Lamzede ...
Posted by Michael Wonder on 16 May 2018
PM urged to back access to ‘life-saving’ drug for children with cystic fibrosis
16 May 2018 - Hundreds of children plagued by a life-shortening disease have written to the Prime Minister begging her ...
Posted by Michael Wonder on 09 May 2018
Rare diseases: addressing the challenges in diagnosis, drug approval, and patient access
9 May 2018 - Rare diseases represent a wide range of disorders and constellations of clinical signs and symptoms. ...
Posted by Michael Wonder on 23 Apr 2018
Company urged to lower cost of life-changing drug for cystic fibrosis
21 April 2018 - Health ministers have urged a pharmaceutical company to drop the price of a life-changing cystic fibrosis ...