Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 01 Mar 2018
Four new rare disease treatments awaiting NICE review
1 March 2018 - Following yesterday’s international Rare Disease Day, here is a snapshot of four new rare disease treatments ...
Posted by Michael Wonder on 20 Feb 2018
Vertex offers ‘portfolio approach’ to secure access to CF meds
19 February 2018 - Vertex has presented NHS England with an “innovative long-term framework proposal” that hopes to secure access ...
Posted by Michael Wonder on 19 Feb 2018
Should the NHS fund a new drug costing £1million for Ollie and Amelia?
19 February 2018 - They have a deadly genetic disease their parents say is being halted by Brineura, but officials ...
Posted by Michael Wonder on 12 Feb 2018
Drug company says it will negotiate terms to allow children to access new treatment for rare genetic disorder
12 February 2018 - NICE has published draft guidance that does not recommend the drug cerliponase alfa (Brineura; Biomarin) for children ...
Posted by Michael Wonder on 12 Jan 2018
Pharma collaborates to improve chances of NICE backing Duchenne drugs
11 January 2018 - A group of pharma companies and Duchenne UK are collaborating to improve the chances of new ...
Posted by Michael Wonder on 20 Dec 2017
NICE turns down rare skin disease implant
20 December 2017 - The National Institute for Health and Care Excellence has rejected Clinuvel UK’s afamelanotide for treating erythropoietic ...
Posted by Michael Wonder on 05 Jul 2017
Strensiq new deal paves way for NICE approval of life saving drug
5 July 2017 - An agreement has been reached that means more people with a rare life threatening disease can be ...
Posted by Michael Wonder on 29 Jun 2017
Final NICE nod for Sanofi’s Gaucher therapy
29 June 2017 - Sanofi’s Cerdelga can now be funded on the NHS in England and Wales to treat type ...
Posted by Michael Wonder on 14 Jun 2017
Why do health technology assessment coverage recommendations for the same drugs differ across settings? Applying a mixed methods framework to systematically compare orphan drug decisions in four European countries
14 June 2017 - Health technology assessment coverage recommendations differ across countries for the same drugs. Unlike previous studies, this study ...
Posted by Michael Wonder on 12 Jun 2017
NICE refuses to expand use of Roche’s IPF drug Esbriet
12 June 2017 - The NICE it remaining steadfast in its position that Roche’s Esbriet should only be funded by ...
Posted by Michael Wonder on 01 Jun 2017
NICE backs new drug for Gaucher disease
31 May 2017 - The NICE has issued a positive final appraisal determination recommending Cerdelga (eliglustat) as a first-line treatment ...
Posted by Michael Wonder on 12 Apr 2017
Why drug appraisal methods need to take into account treatments for rare diseases
12 April 2017 - The UK government needs to adapt its drug and technology appraisal methods to prevent patients with rare ...