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Pilot launched to support children with rare conditions to access personalised therapies

November 22, 2023


  • New programme announced to develop a pathway for children with rare conditions to access therapies made specifically for them.

  • First project will focus on the use of antisense oligonucleotides (ASOs) in children with ultra-rare and life-threatening brain conditions.

  • Longer term the Rare Therapies Launch Pad will support access to other types of individualised therapies across a wider range of rare conditions.

Today, Wednesday 22 November, the government announced its support for the Rare Therapies Launch Pad, a new programme that will develop a pathway for children with rare conditions to access individualised therapies.


The programme’s first project will explore the use of individualised therapies known as antisense oligonucleotides (ASOs) to treat children with ultra-rare and life-threatening brain conditions.


The pilot is developed by a consortium including Genomics England, the Medicines and Healthcare products Regulatory Agency (MHRA), Oxford-Harrington Rare Disease Centre, Mila’s Miracle Foundation and the Association of the British Pharmaceutical Industry (ABPI). It will help identify a sustainable and scalable approach to delivering individualised therapies for children across a wider range of rare conditions, including establishing a proportionate regulatory pathway. This end-to-end pathway would cover diagnosis, the design and rapid manufacturing of these therapies, and treatment. The project also aims to generate evidence to help establish potential reimbursement for individualised therapies beyond the pilot.


Following years of significant investment in life sciences, including across genomics and innovative medicines regulation, the UK is uniquely placed to deliver the Rare Therapies Launch Pad.


Tens of millions of children globally and hundreds of thousands in the UK have rare and potentially life-threatening genetic conditions but less than 10% of rare conditions have a treatment. New therapies can now be created for use by a single patient but currently there is no route to access these individualised medicines for most patients.




Pilot launched to support children with rare conditions to access... Genomics England. (n.d.). https://www.genomicsengland.co.uk/news/pilot-launched-to-support-children-with-rare-conditions-to-access-personalised-therapies

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