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Getting Vertex Pharmaceuticals’ Orphan Drug Kalydeco (Ivacaftor) To The UK Continues (October 22, 2012 Update)

October 22, 2012

       The Cystic Fibrosis (CF) Trust in the UK issues on October 22, 2012, a Press Release with an update on the signing of the e-Petition. The e-Petition has over 11,000 signatures & has been sent to both Vertex Pharmaceuticals and the Clinical Priorities Advisory Group (CPAG) ahead of their meeting to discuss the funding of Kalydeco.   The meeting is scheduled for next week, where cost-effectiveness will lead to a recommendation to the Specialist Commissioning Groups.  Final funding decisions is expected in December 2012.   

        An October 21, 2012 article titled,” ‘Too expensive’ cystic fibrosis drug is blocked due to expense” in The Independent (UK), discusses the latest in the long journey of bringing Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) to the UK for Cystic Fibrosis (CF) patients. 

       Per the article : 

1)  Cost of Kalydeco/patient/year is $182,000 British Pounds Sterling = approximately $291,418 US dollars 

2)  Kalydeco is being blocked from use in England because of a battle over cost 

3)  National Health Service (NHS) has not given the okay for Kalydeco due to the high cost per patient 

4)  A CF Trust petition signed by 12,000 is handed over to Vertex Pharmaceuticals on Friday, October 19, 2012, urging the company to decrease the price 

5)  Vertex Pharmaceuticals’ representatives & NHS officials from Clinical Priorities Advisory Group (CPAG) is to meet this week to try to solve this pricing problem 

6)  > 9,000 CF patients in the UK; Kalydeco works on patients with a specific G551D gene mutation – about 440 CF patients in the UK. 

       Please reference Orphan Druganaut October 22, 2012 Blog Post titled,”What is the status of patient access to orphan drugs in the UK ?” for additional information.    

Copyright  © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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