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Drug approved for cystic fibrosis

16:25, Jan 17 2013

 

Patients with a rare form of cystic fibrosis will be given access to a new drug which has not been approved for use on the NHS, the Health Secretary has announced.

Ivacaftor - or Kalydeco as it is also known - will be funded through the new £21 million fund to help improve access to drugs for people with rare medical conditions, said Alex Neil.

The fund was announced earlier this week. At the same time, the Scottish Medicines Consortium (SMC) decided not to recommend Ivacaftor for use in the NHS. The drug was made available on the health service in England at the start of the year.

Patients in Scotland will still have to apply for Ivacaftor using the Individual Patient Treatment Request (IPTR), which has been criticised by medics as an "inequitable process". But Mr Neil said the Government would take "swift action" to ensure there is no barrier for clinicians who want to prescribe it.

Following a meeting on Thursday with the Ivacaftor Patient Interest Group, Mr Neil and the group released a joint statement which said: "Access to these rare medicines is currently available through the Individual Patient Treatment Request (IPTR) process. However, we recognise the fact that as Ivacaftor benefits 100% of the patients with the 'Celtic gene' or G551D genetic mutation, it presents a challenge to the IPTR process as it stands.

"That is why the Scottish Government will take swift action to address how best to ensure that where specialist clinicians are supportive of prescribing Ivacaftor, the IPTR process does not present a barrier to accessing it. Guidance on the prescribing of Ivacaftor will be issued to all NHS Boards.

"The Cabinet secretary was clear that patients with the G551D gene mutation should not end up competing against each other for access to medicines."

Ed Owen, chief executive of the Cystic Fibrosis Trust, said: "We had a very positive meeting with Mr Neil and I am delighted that with his commitment all patients with the G551D mutation will get access to Kalydeco under the Individual Patient Treatment Request (IPTR) process in Scotland."

Holyrood's Health Committee heard last year that the IPTR system is an "inequitable process" that favours young, middle-class and articulate applicants over more suitable vulnerable people who cannot speak up for themselves.

In November, Mr Neil said a review would be carried out to look at current systems for making new medicines available across the NHS in Scotland. As well as the new fund, a separate review is currently examining if the IPTR arrangements can be improved.

 

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