Electronic versions


  • 2020 Managing Access

    Accepted author manuscript, 271 KB, PDF document

    Embargo ends: 23/02/21


  • A.R. Champion
    Welsh Health Specialised Services Committee, Pontypridd
  • S. Lewis
    Welsh Health Specialised Services Committee, Pontypridd
  • S. Davies
    Welsh Health Specialised Services Committee, Pontypridd
  • Dyfrig Hughes
Advanced Therapy Medicinal Products (ATMPs) which include gene and somatic-cell therapies and tissue-engineered medicines, have the potential to transform current care pathways by offering durable and potentially curative outcomes. However, they are exceptionally expensive, with prices exceeding £1m per patient in some cases. With an expectation that a large number of ATMPs will soon gain marketing authorisation (global market is estimated to reach £9bn to £14bn by 2025), healthcare payers and providers face a number of challenges to facilitate patient access to this new category of medicines.
This viewpoint reflects on the experience of introducing ATMPs into the National Health Service in Wales where £1 in every £200 spent on medicines in Wales (2019/20) is expected to be on ATMPs for just 20 patients. Evidence to date makes it apparent that decisions regarding clinical and cost-effectiveness, and the scale of the budget impact of implementing ATMPs creates both financial and service risks. Consequently, there are significant policy implications. A critical examination is made of the approaches taken for the health technology assessment and appraisal of ATMPs, the methods of payment and service impacts of these medicines, and the approach taken to horizon scanning and subsequent modelling of the financial impact over the next 10 years.


  • advanced therapies, cell therapy, cost-effectiveness, gene therapy, regenerative medicine
Original languageEnglish
JournalBritish Journal of Clinical Pharmacology
Early online date3 Jun 2020
Publication statusE-pub ahead of print - 3 Jun 2020
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