Cell and gene therapies – where to next?

Stem cells | Credit: Stock photo, AdobeStock AI Concepts

Kerry Kriel, Early Asset Strategist | Bayer

This article has been initiated and funded by Bayer plc who are fully responsible for the content. This article will be reviewed in December 2025.

The medical and scientific communities are still enthralled by the potential of cell and gene therapies (CGTs), though as a concept, it’s been around for some time^{1, 2}. Since their inception, we’ve seen the approval and use of CAR-T cell treatments for cancers³ and a gradual increase in the numbers of gene therapies for rare, mainly paediatric diseases^4,5,6.

Whilst the focus to date has been on developing CGTs for cancers, rare and ultra-rare diseases, we mustn’t forget patients living with some common but severe chronic diseases who have a reduced quality of life because of their condition. They too will have much to gain from CGTs because of their high unmet needs living with their debilitating conditions.       

In the UK, through the formation of the Cell and Gene Therapy Catapult and the Advanced Therapy Treatment Centres Network to support and promote innovation, we’re recognised as a hub for CGT discovery, research and manufacturing in Europe⁷ and many companies have chosen to locate their CGT operations here⁸.  

However, the O’Shaughnessy review⁹ which reported earlier in 2023, was conducted in response to the ABPI’s concerns¹⁰ that the UK was losing its status as a destination for commercial clinical trials with knock-on effects on patient access, NHS services and the economy. It is therefore important that we aim to reverse this trend and continue to invest in R&D partnership working with the NHS, especially in the case of CGTs.

We know that the number of CGTs which will gain a license is expected to grow significantly over the next decade^11, 12. And while this may see an increase in capacity and highly skilled jobs in the UK, we need to think very carefully about how the demand for these therapies will need to be met by the NHS. This is all the more pressing since many such treatments will be highly specialised and will most likely need to be conducted in tertiary settings at centres of excellence where the resources and expertise are located.

Another issue is that of reimbursement. Following NICE approval, CGTs are currently offered via a patient access scheme or a commercial access agreement which has worked to date with the small number of CGTs that have been approved¹³. However, in the medium to longer term, a pragmatic approach is needed and we need to think of payment methods that spread out the high upfront costs associated with CGTs. By spreading the payments over the years that the NHS derives value from the CGT, we can ensure that these products are funded and available so that patients can get the care they need, industry is appropriately compensated and, more crucially, the NHS is better able to plan and manage its finances.

We know that other countries are currently looking at a range of payment options like outcomes-based models and annuity pricing and we should be running similar pilots to determine which ones work best for us so that the UK can remain ahead in the delivery of CGT treatments on the NHS.   

This is why Bayer, working closely with others, has been campaigning to have CGTs included in a ministerial portfolio. CGTs cover a range of disease areas and include a wide range of considerations including workforce and skills and it is hard to know where to situate it. This is probably why there is currently no minister with overall responsibility for CGTs and parliamentary questions on the subject have been dealt with by junior ministers from both the Department of Health and Social Care and Department for Science, Innovation and Technology.

What is needed for CGTs to be at the forefront of innovation in our life sciences industry from discovery through to commercialisation. Having a named minister in charge of CGTs would not only raise its profile within government but also recognises that CGTs are a national priority.

Job bag number: PP-CGTP-GB-0014. Date of publication: December 2023

1. https://www.frontiersin.org/articles/10.3389/fmed.2021.756029/full  

2. https://www.researchgate.net/figure/Gene-Therapy-Timeline_fig2_303890127   

3. https://www.england.nhs.uk/cancer/cdf/car-t-therapy/ 

4. https://www.england.nhs.uk/2022/02/nhs-to-roll-out-life-saving-gene-therapy-for-rare-disease-affecting-babies/ 

5. https://www.england.nhs.uk/2021/03/nhs-england-strikes-deal-on-life-saving-gene-therapy-drug-that-can-help-babies-with-rare-genetic-disease-move-and-walk/ 

6. https://www.genomicseducation.hee.nhs.uk/blog/nice-approves-new-sma-gene-therapy/ 

7. https://www.europeanpharmaceuticalreview.com/article/161176/uk-at-the-forefront-of-advanced-therapies/   

9. https://www.gov.uk/government/publications/commercial-clinical-trials-in-the-uk-the-lord-oshaughnessy-review/commercial-clinical-trials-in-the-uk-the-lord-oshaughnessy-review-final-report 

10. https://www.abpi.org.uk/r-d-manufacturing/clinical-research/an-opportunity-for-growth-clinical-research-in-the-uk/  

11. https://cgt.ams3.cdn.digitaloceanspaces.com/Catapult-Annual-Review-2022.pdf  

12. https://www.nature.com/articles/s41467-020-19505-2  

13. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8864053/ 

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