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Scotland: new approval pathway for ultra-orphan medicines

People in Scotland with rare diseases may be able to access new treatments in a more efficient manner following the introduction of a new definition of ‘ultra-orphan medicines’. ‘Ultra-orphan medicines’ will include medicines that can treat people with very rare conditions affecting fewer than 1 in 50,000 people, or approximately 100 individuals in Scotland.

As Health Secretary Shona Robison said:

Patients with the rarest of conditions are often children and treatment choice can be limited, so we are acting to make access to specialised medicines easier across the NHS.

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.

The new decision-making process and definition will also grant the Scottish Medicines Consortium (SMC) the ability to decide whether certain medicines for rare orphan diseases should be treated as ultra-orphan medicines.

Under the new process, an ultra-orphan medicine that the SMC considers to be clinically effective will be made available on the NHS while information on its effectiveness is being collected. After this period, the SMC will review the evidence gathered and may then may make a final decision on its regular use in NHS Scotland. Medicines that qualify for the new definition and which have not been recommended for routine use by the SMC following a recent review will be included in the new pathway.

Shona Robison highlighted her hopes for the new process:

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 [that] 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 will be effective from 1 October 2018.

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