Doubts over future of rare drug fund in Scotland

There are doubts about the sustainability of Scotland’s funding for rare and end-of-life medicines, which has been instrumental in increasing access to these treatments, according to a new report.

Scotland’s New Medicines Fund (NMF), a ring-fenced money pot worth £90 million introduced in 2013-14, funds these drugs.

In a review of access to medicines commissioned by the Scottish government, NHS Fife medical director Dr Brian Montgomery said that, so far, the NMF has met the costs of patients requiring end-of-life, orphan and ultra-orphan medicines.

Like England’s Cancer Drugs Fund (CDF), the NMF has increased in size since its launch, when it was worth only £21 million, to keep pace with demand.

In England, this eventually led to a complete rethink of the CDF to prevent it from overspending, and it now provides interim reimbursement for some cancer drugs while further cost-effectiveness data are gathered following NICE's assessments.

Montgomery said: “It is not clear if the NMF will be sustained and, if so, whether it will be increased to meet the anticipated growth in demand.”

But Montgomery said that if NHSScotland wants to maintain this increased level of access, there should be an alternative assessment pathway for ultra-orphan medicines.

This should “preserve the integrity” of the Scottish Medicines Consortium (SMC) cost-effectiveness body, while also maintaining access to these medicines.

According to the report, the SMC has increased its rate of acceptance for end-of-life and orphan medicines, but its rate of acceptance for ultra-orphan medicines remains low.

This left patients resorting to Individual Patient Treatment Requests, and its successor the Peer Approved Clinical System.

Montgomery said that some people felt that these systems did not reflect the principles laid down by the country’s chief medical officer, Dr Catherine Calderwood, in her annual review entitled 'Realistic Medicine'.

A key principle of this report is to reduce unnecessary variation in practice and outcomes.

NHSScotland should also engage at an early stage with pharma to ensure medicines reach patients, according to the review.

Montgomery said: “Difficult choices will have to be made as NHSScotland seeks to optimise its use of a finite resource subject to ever-increasing demands while at the same time the pharmaceutical industry operates in a competitive commercial environment.”

In a response, the Association of the British Pharmaceutical Industry’s director for Scotland, Sandra Auld, said: “The recommendation that we need an alternative assessment pathway for ultra-orphan medicines is one that we feel could make a real difference to patients accessing these medicines.”