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Getting Vertex Pharmaceuticals’ Orphan Drug Kalydeco (Ivacaftor) To The UK Will Continue (September 27, 2012)

September 27, 2012

       Getting Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) to the UK is a lengthy process and will continue.  This is an update to the Orphan Druganaut September 26, 2012 Blog Post.

        Per The Cystic Fibrosis (CF) Trust in the UK, the organization has been waiting for a response from the Clinical Priorities Advisory Group (CPAG) which met on September 25, 2012, to review & come up with a recommendation for Kalydeco.

        The CF Trust issues a September 27, 2012 Press Release, with the following updates as to the status of Kalydeco in the UK:

–    The CF Trust is asking for both NHS & Vertex Pharmaceuticals to quickly resolve all Kalydeco cost issues, so that all CF patients with the G551D mutation can benefit from this treatment as soon as possible

–    The CPAG says that Kalydeco provides clinical benefit

–    The CPAG though “deferred discussion of the drug’s cost effectiveness to a further meeting with Vertex in a month’s time. This meeting will then make recommendations to commissioners to take a final decision about funding in December”

–    There are different NHS commission arrangements in Scotland, Wales, & Northern Ireland. The CF Trust is continuing to push to get Kalydeco available in all parts of the UK.


Clinical Priorities Advisory Group (CPAG) September 26, 2012 Statement

Vertex Pharmaceuticals’ Statement to the CF Trust on the Outcome of the CPAG Meeting on September 25, 2012  

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

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