Scottish government announces £21m fund to help patients with rare conditions


  The Scottish Government is launching a fund to cover the cost of medicines for individual patients with rare conditions, which are not available for routine prescription.

£21 million will be invested to pay for the cost of medicines known as “orphan drugs”. These are medicines for illnesses which affect fewer than 1 in 2,000 people.

Only those medicines not recommended for routine use by the Scottish Medicines Consortium will be covered by the fund.

The new fund has been put in place following interim advice from Professor Charles Swainson who is examining the current Individual Patient Treatment Request (IPTR) arrangements in the NHS in Scotland, as part of the ongoing access to new medicines review.

The fund will be available from March this year through to April 2014 and will help to cover successful new Individual Patient Treatment Requests.

Health Secretary Alex 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.

“This fund bridges the period to the establishment of next year’s value based pricing for medicines and any changes that are made following the completion of the ongoing access to new medicines review.

“We recognise that the Scottish Medicines Consortium is globally respected and has the fastest and most efficient medicine review process anywhere in the UK, but that for some individual patients with rare conditions there is a need for further support. This new fund will complement it by making the IPTR processes fairer.”

Jim Hume MSP, the Scottish Liberal Democrat health spokesperson, said: “While this extra money may help people access the medicines they need, ministers must make sure that the underlying problems around access to new drugs are tackled so the system is fair for patients across Scotland, regardless of background.”

Requests to the fund will be assessed on a case by case basis, based on the clinical circumstances of individual patients. Detailed operational arrangements will be developed and announced in due course. The fund will be managed by NHS National Services Scotland.

The EU’s definition of an “orphan medicine” is defined as one for which the frequency of the disease is less than 5 per 10,000 of the EU population.

The £21 million is in addition to the current board funding allocations.

The Scottish Medicines Consortium (SMC) appraises all newly licensed medicines and provides advice to NHS Boards on their clinical and cost-effectiveness.

Currently, if a medicine is accepted for use by the SMC then individual health boards set the criteria for prescribing it. If the medicine is not accepted then health boards do not make it routinely available. However, clinicians can prescribe medicines that are not accepted for routine use by the SMC for individual patients in certain circumstances by special request through Individual Patient Treatment Requests.

A review is underway to assess the current systems for making new medicines available across NHS Scotland. This review is being led by Professor Philip Routledge.

Separate to this work the Chief Pharmaceutical Officer Professor Bill Scott, with Professor Charles Swainson, has been reviewing the Individual Patient Treatment Request arrangements to establish whether any further improvements can be made. This new fund is being made as a result of Professor Swainson’s interim advice.