Mum of cystic fibrosis sufferer hits out at Kalydeco ruling

Jan 17 2013 by Leona Greenan, Hamilton Advertiser

A Hamilton mum, whose son suffers the “Celtic” form of cystic fibrosis, has welcomed news of a new fund that could help pay for a wonderdrug to treat the condition.

Marion Ferguson, who runs a support group for families of CF sufferers, was speaking after the Scottish Government agreed to set up a £21million Rare Conditions Medicines Fund.

News of the fund followed the announcement by the Scottish Medicines Consortium (SMC) not to recommend Kalydeco for use on the NHS, primarily on the grounds of affordability.

It was, however, approved for use in England back in December. Kalydeco costs £180,000 per patient a year and helps patients with rare form of cystic fibrosis known as G551D.

Marion, whose 15-year-old son Thomas has type G551D, said: “This medicine really is amazing and is giving hope not only to those with G551D but all of those with Cystic Fibrosis.”

Marion, on behalf of the Cystic Fibrosis G551D patients in Scotland, had asked Scottish Government Health Minister Alex Neil to allow the drug to be prescribed in Scotland.

She added: “It is simply not good enough to allow the lives of children and young people in Scotland to deteriorate whilst their fellow suffers in England lives are being transformed by receiving this remarkable new medicine.

“However, now with the welcomed announcement from Alex Neil, we are cautiously optimistic that we will get access to Kalydeco.”

She called on Mr Neil to clarify the process by which sufferers could apply for the medicine.”

For Marion’s son Thomas, who attends Holy Cross High School, Cystic Fibrosis means hours of treatment and numerous medications every day.

Marion, who is the retail director of Ink Spot and Dazzle in Hamilton’s Castle Street, said: “When Thomas is ill he is admitted to hospital for a minimum of two weeks.

“The people being prescribed this medicine in other countries are managing to stay hospital-free. Surely there are considerable savings to be had with fewer hospital admissions.” The Cystic Fibrosis Trust are urging all parties, including NHS Scotland, Vertex, the makers of the drug, and others to work together effectively to ensure funding for Kalydeco is agreed and available from March 1.Ed Owen, the Cystic Fibrosis Trust’s chief executive, said: “We will be looking for further clarification about the process going forward, including how soon a consultant can make an individual treatment request so that patients have access to Kalydeco as soon as possible.”

The Scottish Government said: “The SMC operate independently of Ministers. There is a review underway to consider how new medicines are appraised and accessed in Scotland.

“The new fund will cover the cost of successful individual treatment requests for medicines for patients with rare conditions, which are not otherwise available for routine prescription.”

MSP for Hamilton, Larkhall and Stonehouse, Christine McKelvie, said: “Getting hold of expensive but effective drugs such as Kalydeco will make a massive difference to the lives of young people who are trying to cope with the debilitating effect of rare conditions.”