Scotland promises faster access to new treatments for rare diseases

PATIENTS in Scotland with very rare diseases will get faster access to new treatments following a revised decision-making process on approving clinical effectiveness.

Scottish Government is introducing a new definition of 'ultra-orphan medicines' used to treat very rare conditions affecting fewer than 1 in 50,000 people - around 100 people or less in Scotland. The changes means that any medicine meeting the new definition of an ultra-orphan medicine and approved by the Scottish Medicines Consortium (SMC) can be made available on the NHS for at least three years while further information on its clinical effectiveness is gathered. The SMC will then review the evidence and may make a final decision on its routine use in NHS Scotland.

Medicines that fall under the new definition and have been recently reviewed by SMC but not recommended for routine use will be admitted to the new pathway.

Former Health Secretary Shona Robison said: "Changes introduced this month through the new PACS Tier Two system already give doctors the right to seek access to licensed treatments not generally available in the NHS in Scotland.

"These new rules for medicines that can treat those with the rarest of diseases will give faster access to new treatments. The process has been designed to be consistent and quick to implement for patients, and it will strengthen Scotland’s reputation as an international life sciences hub.

"Given ultra-orphan drugs are often very expensive, it is also vital that pharmaceutical companies play their part and bring a fair price, first time, to the process."

The changes, which will come into effect from 1 October 2018, follow on from Dr Brian Montgomery’s Review of Access to New Medicines, which was published in December 2016.