The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK

Abstract

Twenty-first century technologies increasingly allow the identification and treatment of the underlying genetic causes of disease¹. Millions of children around the world are affected by rare diseases, some of which are caused by ‘private mutations’ that are so rare that they can often only be treated with individualized therapies². Disappointingly, these scientific breakthroughs have not yet led to an increase in the use of individualized medicines owing to regulatory, commercial and economic barriers to adoption^3,4,5,6. The Rare Therapies Launchpad (RTLP) is a UK pilot program set up to help identify sustainable and scalable approaches to address these barriers, to allow the establishment of an equitable and sustainable national infrastructure to ensure that UK children will benefit from the life-saving potential of cutting-edge science.