Great Ormond Street Hospital (GOSH), supported by LifeArc and Great Ormond Street Hospital Children’s Charity (GOSH Charity), has announced plans to revolutionise how children living with a rare disease can gain access to life-changing treatments that have been proven to work.
A big challenge with developing treatments for rare diseases is that due to small patient numbers they are often not commercially viable for companies to invest in and keep on the market, even if the treatment has been proven to cure a condition.
GOSH, LifeArc and GOSH Charity hope to address this through the plan which would be the first of its kind in the UK and the first time an NHS Trust would hold the market authorisation - or licence – for a gene therapy drug, meaning patients can directly access and benefit from the treatment.