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Background Info

It is the very first medicine that treats the underlying cause of Cystic Fibrosis. It is clinically proven. This Groundbreaking Life saving medicine is readily available in the USA, It was approved by the US FDA 31st January 2012

Kalydeco was given European Marketing Approval (License), after a fast track process through the EMA on 23rd July 2012 receiving a 'first in its class' recommendation by them. Not withstanding this approval, Kalydeco still has to go through a long and laborious approval here in Scotland which has already delayed the availability for those who need this medicine. It is being prescribed in Holland, Italy & Germany. In England it has been given clinical approval by CPAG (Clinical Priorities Advisory Group). Minutes available here http://www.cftrust.org.uk/pressoffice/pressofficepo/kalydeco_updates/cpagmins 

Clinicians in Scotland are unable to prescribe Kalydeco to their patients due to lack of funding and current procedures. I have been advised by politicians and government that there is a "robust and transparent process " in place for approval through the SMC. I have been advised that there is in place an IPTR (Individual Patient Treatment Request) process to gain access to this drug. Unfortunately that is NOT  the case.  The CPAG (minutes of the meeting of 25th September 2012 link above) concluded in their report: quote..."When considering funding mechanisms, the view from commissioners was that it was very hard to identify any one section of this cohort of patients.As a result exceptionality would not apply.  And the Individual Funding Request Process was not an appropriate way to address funding "..... end quote. Therefore all the advice I have been given by the Health Secretary and other politicians would seem to be not appropriate for funding in this instance.  On this basis I urge the Scottish Government to put in place immediate funding which will halt lung damage and save lives among the CF G551D community, most of whom, by nature of this disease are young people.

The pharmaceutical company, Vertex have made a submission to the SMC and a decision will be made public in January 2013. It is understood that this decision will most likely be a refusal due to cost. There will then have to be a resubmission to the SMC. Even when there is a positive decision, it is understood that some sort of Risk Share agreement will need to be set up and put in place before clinicians can prescribe this medicine. The current process is delaying the prescribing of this drug.

There are approximately 70 with this mutation in Scotland, some of whom are under 6 years old for whom the drug, at present, is not recommended. Others await lung transplant.  An estimate of those requiring the drug could be 40-50. Since many do not live into their thirties, a disproportionate number are young. The medicine's full cost is £182000 per patient per year.

If this process follows the normal course, even if the recommendation is positive there will be a considerable delay before the drug is available for clinicians to prescribe. Lives will be lost and irreparable lung damage will be done. 

In a Medical News Today article, Professor Stuart Elborn, Professor of Respiratory Medicine at Queens University Belfast said “These data showing the consistent and sustained benefit of this medicine confirm that Ivacaftor has the potential to make a significant difference to the lives of children, young people and adults with Cystic Fibrosis G551D”

He added “The data don’t capture the full benefit for patients. It’s very noticeable in the patients I look after that they are able to do things they previously couldn’t after starting treatment with Ivacaftor. They feel better and more able to plan for the future” http://www.medicalnewstoday.com/articles/249902.php

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