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Faster route to access in Scotland for rare disease patients requiring ultra-orphan drugs

By Alison Kneen, Vice President International Operations

Fantastic news for Scottish patients who suffer from very rare diseases. As of October 1, 2018, patients will receive faster access to new treatments as a result of changes being made to the Scottish Medicine Consortium (SMC), the health technology assessment (HTA) body pathway which is used to evaluate “ultra-orphan medicines.”

So, what’s to be implemented; if a medicine meets the definition of “ultra-orphan”, and the SMC considers it to be clinically effective, it will automatically be made available on the National Health Service in Scotland for at least three years while more information on its effectiveness is gathered.  This is regardless of cost considerations at this point. At the end of the period, the SMC will review the evidence and take a final decision on its routine NHS Scotland use.

The definition of an “ultra-orphan” medicine has now been revised to make it clearer and will now be defined as a product for a condition that extends beyond the 1 in 50,000 criteria to include multiple factors i.e. whether the condition is chronic and severely disabling, requires highly specialized management, and whether there are any other therapeutic options of proven benefit.

In addition, there is good news for products which have recently been reviewed by SMC and that fall under the revised definition of ultra-orphan but “not recommended for routine use.” These will now be admitted to the new pathway, meaning they will be immediately available to patients.

It is also important to note that in Scotland a rare disease drug that does not meet all the newly proposed criteria may still be granted ultra-orphan status which will be considered on a case-by-case basis which opens more opportunity and flexibility for making treatments accessible to patients for these ultra-rare diseases.

Affordability however, is still a major issue and the new pathway will assist getting over that barrier while more evidence is generated allowing negotiations on price to continue behind the scenes.

The new changes are a follow up to the “Review of Access to New Medicines,” report by Dr Brian Montgomery, which was published in December 2016. In his report, he found that access to end-of-life (EOL) and orphan medicines through SMC assessment had increased since new procedures were introduced in 2014, but that the SMC’s rate of acceptance of ultra-orphan medicines had not risen to the same extent, meaning that Scottish patients were being denied access to valuable treatment.

The report also advised the Scottish government to develop, agree and implement a new definition of ultra-orphan medicine, which was previously defined as a product for a condition that affects fewer than 1 in 50,000 people, which would equate to approximately 100 people or less in Scotland.

To find out more about how to access the Scottish rare disease market, get in touch with Partners4Access, specialists in access for orphan drugs, cell and gene therapy. Email P4A at contact@partners4access or visit our website www.partners4access.com

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