Resetting the model

Improving the way that decisions on rare disease medicines are made.

There are more than 6,000 rare diseases, but only 200 of these have a licensed innovative medicine in Europe

Genetic Alliance UK are currently working on an ambitious and innovative policy project: Resetting the Model. It’s aimed at improving the way that decisions on rare disease medicines are made. The project will deliver a flexible new vision for access to rare disease medicines for the UK. Around 3.5 million people in the UK will be affected by a rare disease at some point in their lives.

There are more than 6,000 rare diseases, but only 200 of these have a licensed innovative medicine in Europe. For this small fraction of a small patient group in the UK, there are sixteen pathways to assess treatments. All the pathways work in different ways, and sometimes we have four separate decisions being made on the same treatment – not all of them reaching the same conclusion.

This is causing an inequality of available medicines for UK patients compared to other large European nations. Research conducted by the Office of Health Economics shows a big disparity in the number of funded treatments for rare diseases between the home nations and high income European nations:

NUMBER OF FUNDED TREATMENTS FOR RARE DISEASES IN UK HOME NATIONS AND OTHER HIGH INCOME NATIONS IN EUROPE.

(ZAMORRA, MAIGNEN, GARAU, O’NEILL, MESTRE-FERRANDIZ, 2017)

Through conducting comprehensive research, engaging key stakeholders and unifying the patient voice behind the new model – our aim is to drive a new vision for access to medicines to the heart of government. The project started in January 2018 and has so far involved a discrete choice experiment survey in April 2019. We have been updating members throughout the project through webinars, newsletters and face to face meetings.