Rare Disease Treatment Prize

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Mission Possible: The role of challenge prizes in a revitalised UK Innovation Strategy

Rare Disease Treatment Prize A £25m prize to trial Phase I therapeutics in UK patients for conditions in Genomics England’s 20 priority therapeutic areas Why a prize? The Rare Disease Treatment Prize would incentivise UK biotech companies to trial their treatments in the UK rather than going abroad, through a combination of

Technology leadership for the UK The prize would focus support on UK biotechs, including startups – firms that are currently often testing and marketing their products abroad, particularly in the USA. The prize’s focus on support with clinical trials closely tied to NHS commissioning would help make the UK the go-to location in the world for developing and testing new drugs.

the prize’s financial incentive, provision of data, support with clinical trials and support with NHS procurement – kickstarting a socially and commercially beneficial sector in the UK.

Alignment with the seven technology families of UK strength and opportunity Advanced Materials and Manufacturing AI, Digital and Advanced Computing Bioinformatics and Genomics Engineering Biology Electronics, Photonics and Quantum Energy and Environment Technologies Robotics and Smart Machines

The problem Despite Genomic England’s world class database of individual’s genomes and the potential of NHS to match patients with treatments, UK biotechs tend to prefer doing their trials in the US because of market pull. This means that both children and adults are missing out on access to treatments that could have a dramatic impact on their health. The emerging field of personalised medicine, where drugs are tailored to our genomics is still an emerging field and the UK risks not being an attractive location to trial these drugs.

The impact The prize would signal government support for UK leadership in the genomic medicine sector. It would

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give UK biotech a chance to shape and become a dominant player in the market. If the UK can establish itself as a leader in testing genomic medicines, and support the pipeline from testing to adoption in the NHS, UK patients, in particular children with rare genetic diseases, will benefit from quicker access to lifesaving drugs.

The mission The Rare Disease Treatment Prize could be part of an innovation mission to halve the number of qualityadjusted life years lost to rare diseases in the UK by 2035.More rare disease clinical trials in the UK will support British-based start-ups and established biotechs, as well as expand a promising new driver of the clinical research sector.


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