Stranraer teenager's fight for life-enhancing medicine

Katie Milby, pictured here with her close friends, is battling to have the drug Vimizim available on the Scottish NHS. Credit: ITV Border

A teenager's fight to get her life enhancing medication paid for on the NHS will go before the Scottish parliament.

Thirteen-year-old Katie Milby from Stranraer has a rare degenerative syndrome called Morquio A.

The condition affects her mobility general health and her stamina.

She's been using the drug Vimizim, which had been supplied free by the drug company, but now it's stopped paying believing the NHS should pay.

However the Scottish Medicines Consortium did not recommend Vimizin for routine use so the drug isn't being paid for by NHS Scotland.

"SMC was unable to recommend elosulfase alfa (Vimizim) for routine use by NHS Scotland because the case presented by the company was not robust enough to convince us about its longer-term benefits when balanced against its extremely high cost.

While our processes are designed to increase access to medicines for these kinds of conditions, our responsibility is to consider whether individual medicines offer value for money to the NHS in Scotland, and take account of all those patients who need treatment, not just those affected by the medicine under consideration.

– Scottish Medicines Consortium spokesperson

Fortunately, the local NHS board in Dumfries & Galloway has agreed it will pay for Katie's treatment.

Local MSP Alex Fergusson is due to raise the issue at Holyrood tomorrow, when he will ask the Scottish Government whether it will review the decision-making processes of the Scottish Medicines Consortium, following its decision not to reimburse Vimizim for people with Morquio A syndrome.

Morquio Syndrome has no known cure but the drug improves the life of young people with the condition by replacing a missing enzyme that allows growth to continue.

It also improves stamina and walking ability as well as relieving pain.

A spokesperson for BioMarin said:

BioMarin is disappointed that the Scottish Medicines Consortium has concluded that that Vimizim (elosulfase alfa) is not recommended for use within NHS Scotland.VIMIZIM® is the only enzyme replacement therapy for people with Morquio A Syndrome, a rare genetic condition that is progressively degenerative and life limiting.

“BioMarin continues to work closely with all stakeholders to ensure that a long term funding solution for Vimizim is found for all patients with Morquio A Syndrome, so that they are able to access treatment through NHS Scotland.

– BioMarin Spokesperson.

Katie's friend and fellow Stranraer Academy student Kyle Pirrie has started a petition to get Vimizim available on the NHS, which has so far gathered over 2,0000 signatures.

It is thought that only five people in Scotland have Morquio Syndrome, with a hundred and five people across the UK reportedly suffering from the condition.