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Excellence in Rare Diseases and Orphan Drugs

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Highly Commended

Delivering Innovation in a Rare Disease Launch

By Amicus Therapeutics UK Ltd


Summary of work

Galafold® (migalastat) is the first commercialised product from US-biotech Amicus which began development of its UK operation in May 2015. With a marketing authorisation for Fabry disease expected mid-2016 and no footprint in, or experience of, the UK healthcare system, there was an urgent need to rapidly secure funding from NHS England (NHSE), the sole payer for specialised services in England and to get a positive recommendation in Scotland. Without ‘funding approval’ access to the UK market is blocked.

A value proposition was developed which maximised the chances of patients being able to receive treatment while establishing Amicus as a sustainable business in the UK.

In just under 18 months Amicus, working closely with UK treatment centres and patient groups, undertook original research to map the treatment pathway and costs, develop an in-depth understanding of patient numbers and their needs, and built a compelling value proposition. With their support Amicus then successfully negotiated the challenging Scottish HTA requirements, resulting in a positive recommendation from the SMC on 07.11.16; and in England with a positive recommendation in the fastest ever Highly Specialised Technology (HST) assessment, with a ‘minded to approve’ by NICE end 11.16 and Final Evidence Determination and recommendation to fund published in 02.17.

Judges' comments

We thought that Amicus did a very good job for a small outfit in the start-up phase. They showed an extraordinary understanding of payers and we really liked the working with clinicians to make their case and building insight into acceptance.



Highly commended

Delivering Innovation in a Rare Disease Launch - By Amicus Therapeutics UK Ltd


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with support from Health IQ, Republic M! and Havas Life Medicom