Daniel J. O’Connor, Parker Moss, Matthew Wood, Martin Murphy, Michael Parker, Nicola Blackwood, Matthew A. Brown, Deb Lancaster, Vanessa Newman, Jenny Taylor, Tim Yu &
Julia Vitarello
March 10, 2025
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 adoption3,4,5,6. 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.
In 2018, a young girl called Mila in the USA became the first person in the world to be treated with a medicine designed for one person — this drug was called milasen7. Inspired by her example, a group of aligned stakeholders came together in the UK to build on Mila’s experience and to form the RTLP. The UK government used its 2023 Autumn Statement to endorse the RLTP, which has set out to mainstream Mila’s experience, enabling access to innovative medicines for children in the UK. It plans to harness the collective expertise and real-life experience from treating small cohorts of children with rare, fatal neurological conditions, and to model the critical diagnostic, development, regulatory and reimbursement change needed to support some of the UK’s most vulnerable children. To achieve this, the RTLP is focused on establishing the end-to-end steps of a new pathway, including resources, infrastructure and data requirements, which will be suitable for multiple conditions and different therapeutic modalities; mapping key stakeholder responsibilities to each step of the pathway, appropriately sharing risk and accountability between clinicians and families, industry, the National Health Service (NHS), the regulator and payer organizations; identifying gaps in policy, regulation and legislation and determining where precedents, amendments and new rules or legislation are needed; and fostering an ecosystem that will provide access to sustainable, reimbursable individualized medicines.
O’Connor, D.J., Moss, P., Wood, M. et al. The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK. Nat Med (2025). https://doi.org/10.1038/s41591-025-03547-4
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