Fund created for rare medicines

Jan 14 2013

A fund has been launched to help improve access to treatment for people with rare medical conditions in Scotland.

Health Secretary Alex Neil announced the £21 million fund at the same time as the Scottish Medicines Consortium (SMC) decided not to recommend cystic fibrosis drug Kalydeco for use in the NHS.

The drug, also known as ivacaftor, was made available on the NHS in England at the start of the year.

As he announced details of the new fund, which will only cover the cost of medicines whose routine use has been rejected by the SMC, Mr Neil said: "It is only right that Scottish patients with rare conditions have access to innovative medicines which are clinically justified and that they are not disadvantaged due to the very high cost of these treatments.

"I am therefore pleased to confirm today that the Scottish Government will establish a fund which will ensure that the cost of successful new individual patient treatment requests for orphan medicines are met."

So-called orphan medicines are those that treat illnesses affecting fewer than one in every 2,000 people.

MSPs heard last year that the Individual Patient Treatment Requests (IPTR) system, which patients can use to get drugs not yet approved for general use by the SMC, 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.

A separate review is examining if the IPTR arrangements can be improved, with the new fund established after interim advice from Professor Charles Swainson - a former medical director at NHS Lothian who is now an independent consultant.

The £21 million is available from March through to April next year and will help cover the cost of successful requests under the IPTR system.