Cell and gene therapies have the potential to transform outcomes for patients with serious and rare diseases, but scientific success alone is not enough. Developers increasingly need to consider regulation, reimbursement, manufacturing and healthcare adoption as an integrated part of development from the outset.
That was a central message from Dr Jacqueline Barry, Chief Clinical Officer at the Cell and Gene Therapy Catapult, in a recent Oxford Global Cell 2026 thought-leadership interview exploring clinical translation, regulation, patient access and how the UK can scale advanced therapies.
Barry oversees activities spanning non-clinical development, regulation, clinical trials, health economics and market access. She also leads work connected to the UK’s Advanced Therapy Treatment Centre Network, which works with the NHS to improve access to advanced therapies.
Reimbursement cannot be an afterthought
One of the recurring challenges, Barry explained, is that developers may focus heavily on obtaining regulatory approval without considering early enough whether the evidence they generate will ultimately satisfy payers and health technology assessment bodies.
This is particularly difficult in advanced therapies, where clinical studies may involve small patient populations, rare diseases and, in some cases, single-arm trials. Such evidence may be appropriate scientifically but can create additional challenges when demonstrating value and securing reimbursement.
The implication is clear: clinical development, regulatory strategy and reimbursement planning need to evolve together rather than sequentially.
Patient and healthcare-system adoption are equally important. Unlike many conventional medicines, advanced therapies can require specialist treatment centres, new clinical pathways and complex delivery models. Developers therefore need to understand not only whether a therapy is safe and effective, but whether healthcare systems can realistically implement it.
Automation and data will be central to scale
Manufacturing remains another major barrier. Moving from small-scale production towards reliable global supply requires new approaches to scalability, process control and cost.
Barry highlighted automation, digitalisation and better use of manufacturing data as particularly important. Automated platforms and digital control systems have the potential to improve consistency while reducing the cost of goods — a critical consideration for personalised and complex biological products.
She also pointed to the growing opportunity to make better use of existing data across manufacturing, non-clinical development and clinical trial design. AI, in silico approaches, organoids and other new approach methodologies could help developers generate more relevant evidence while reducing dependence on traditional animal models.
A more joined-up pathway
The UK is also exploring regulatory approaches intended to accelerate development and access, particularly for rare diseases and areas of unmet medical need. Barry highlighted the importance of closer collaboration between organisations including the MHRA, NICE, the NHS and other stakeholders, alongside greater use of prior knowledge and more flexible clinical development pathways.
For developers, however, the broader lesson starts much earlier.
Advanced therapies need to be developed with the end pathway in mind — from manufacturing and clinical evidence through to reimbursement, healthcare delivery and patient adoption.
As increasingly sophisticated cell and gene therapies move towards the clinic, the organisations most likely to succeed may be those that stop treating access as a final-stage challenge and instead design for it from the beginning.
