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

Nature Medicine volume 31, pages 1379–1380 (2025)Cite this article

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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.

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Fig. 1: The Rare Therapies Launchpad: from research to treatment with iterative learning.

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Acknowledgements

Disclaimer: The authors are all members of the RTLP. The views expressed, however, are those of the authors and not necessarily the organizations they represent, nor of the RTLP as a body.

Author information

Authors and Affiliations

  1. The Association of the British Pharmaceutical Industry, London, UK

    Daniel J. O’Connor

  2. Cancer Research UK, London, UK

    Parker Moss

  3. Oxford-Harrington Rare Disease Centre, Department of Paediatrics, University of Oxford, Oxford, UK

    Matthew Wood

  4. Rosa Genomic Medicines, London, UK

    Martin Murphy

  5. University of Oxford, Oxford, UK

    Michael Parker

  6. Genomics England, London, UK

    Nicola Blackwood, Matthew A. Brown, Deb Lancaster & Vanessa Newman

  7. Centre for Human Genetics and Oxford NIHR Biomedical Research Centre, University of Oxford, Oxford, UK

    Jenny Taylor

  8. Boston Children’s Hospital, Boston, MA, USA

    Tim Yu

  9. Mila’s Miracle Foundation, Boulder, CO, USA

    Julia Vitarello

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  1. Daniel J. O’Connor
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Corresponding author

Correspondence to Julia Vitarello.

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Competing interests

D.J.O.C. is employed by a pharmaceutical industry trade association. M.W. is a co-founder of and consultant for Pepgen Limited. M.M. is founder and chair of Rosa Genomic Medicines, non-executive director of Autolus Therapeutics PLC, chair of investment committee L&G UK Universities Ventures LP and non-executive director of Synairgen PLC. N.B. is chair, Genomics England; chair, Oxford University Innovation; director, supervisory board, BioNTech SE; and senior independent non-executive director, RTW Biotech Opportunities Ltd (a full list of her interests can be accessed here: https://members.parliament.uk/member/4019/registeredinterests). M.A.B. is a consultant for Altis Medicines, Grey Wolf Therapeutics, Incyte and Ipsen and has research funding from UCB. D.L. is a trustee of Myeloma UK. T.Y. has received research support for an N=1 trial in ataxia telangiectasia from EveryONE Medicines and is named as an inventor on three patents pertaining to antisense oligonucleotide treatments: for transposon-associated diseases (WO2019055460A1), ataxia telangiectasia (US20230174979A1) and progranulin (US11359199B2); he has also received scientific consulting fees as pertains to ASO therapies from SynaptixBio, RegUp, Servier Pharmaceuticals and Lilly, serves as an unpaid board member for the N=1 Collaborative, the Oligonucleotide Therapeutics Society and the Society for RNA Therapeutics, and is an unpaid scientific advisor to several rare disease foundations. J.V. is a co-founder and unpaid board member of the N=1 Collaborative, a co-founder and consultant for EveryONE Medicines and an unpaid board member for several rare disease foundations.

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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 31, 1379–1380 (2025). https://doi.org/10.1038/s41591-025-03547-4

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