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Why system change is critical to harness the potential of gene therapies

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Pfizer UK

6 min read Partner content

Gene therapies and other advanced therapy medicinal products (ATMPs) offer potential advancements in the way we treat certain conditions – but our current approaches to medicine assessment and reimbursement need to evolve to ensure they can reach eligible patients

This content has been developed and paid for by Pfizer UK.

Dr Gillian Ellsbury, Vaccines & Antivirals Medical Director , Pfizer
Article authored by Lynn Clay, 
Head of Specialty Care at Pfizer UK

Gene therapies and other ATMPs represent a significant leap forward in medical science. These next-generation treatments could have the potential to transform the lives of people living with rare diseases and genetic conditions across the UK.

There are approximately 3.5 million people living with rare diseases in the UKwhose conditions severely impact their quality of life. With 95 per cent of rare diseases having no approved treatments,2 patients often require lifelong medical support and care. This places a considerable burden on health systems as those living with conditions face multiple hospital appointments and complex management.1,3  However, gene therapies and other ATMPs may offer potential hope to people suffering from rare diseases and help to alleviate the long-term burden on the health and care system.

The UK’s unique position

Initiatives, such as the 100,000 Genomes Project, have put the UK on the map as a world-class research hub, revolutionising our understanding of rare genetic conditions and helping to diagnose patients faster.4

For the patients that could potentially benefit, a diagnosis is a positive step forward but it’s important to ensure we improve all aspects of patient care.

We must also nurture the bright future of scientific research. The UK has advanced research and clinical delivery capabilities – but ongoing investment is essential to maintain this.5

Through advanced genetic research, scientists are finding ways to target the underlying cause of diseases in ways not previously thought possible. Gene and cell therapies are different to traditional medicines. They are highly personalised, aiming to target the root cause of conditions by augmenting, repairing, replacing, or regenerating organs, tissues, cells, genes, and metabolic processes in the body.6 A gene or cell therapy is usually a ‘one-off’ treatment that may deliver potential benefits over the course of a patient’s whole lifetime.7

Health systems need to keep pace with scientific innovation

While this represents an exciting change in how we treat diseases, hurdles exist across the patient pathway, from assessment and reimbursement right through to provision of care. While a small number of gene and cell therapies have been launched in the UK to date, there are a significant number of potential ATMPs expected to be launched in the coming years, meaning it’s vital to ensure that our health systems can support the routine use of these treatments.6

For example, the existing approach for assessing and reimbursing medicines was designed for medicines used to treat more common conditions rather than personalised treatments such as gene therapies and other ATMPs.

To better capture the value delivered by ATMPs to patients, the NHS and wider society, work needs to be done to evolve our health technology assessment process for new personalised technologies. In England, new medicines for rare diseases are currently assessed by the National Institute for Health and Care Excellence (NICE) through the Highly Specialised Technology (HST) or Single Technology Appraisal (STA) pathway but the eligibility criteria mean there isn’t always a clear assessment route, and many new therapies could fall through the gap. For instance, the STA pathway is designed for common conditions with larger population sizes but the entry criteria for HST evaluation is very narrow, meaning that the majority of new ATMPs are unlikely to qualify.8,9

The reality is that while gene therapies can offer significant savings to the health and care system and the economy more widely,7 the upfront cost presents short-term challenges for the NHS. Although the NHS has managed to address these challenges for a small number of approved ATMPs through bespoke commercial agreements, this approach may not be sustainable in the face of a rapidly growing ATMP pipeline.8

To reflect the potential long-term value that gene and cell therapies provide for patients, innovative payment models have the potential to help patients access new medicines sooner. For example, outcome-based agreements (OBAs) link payments to real-time clinical outcomes. Another option could be where the treatment cost is spread over a number of smaller payments over a predetermined period. Innovative payment methods should be implemented as part of a broader package of measures to limit financial risk for the NHS, encourage the adoption of medical innovation and ultimately support patient access.

Close collaboration for the greater good

The time for collective action is now. Health system change is needed at pace and scale to benefit patients, their carers and loved ones.

Close collaboration among policymakers, healthcare professionals, and the broader healthcare community is essential. As shown by the multi-stakeholder response to the COVID-19 pandemic, a ‘mission-led’ approach can help transform outcomes. The creation of a government-led Gene Therapy Taskforce would provide an opportunity for healthcare professionals, key government departments and agencies, academia and industry to come together with a ‘mission- led’ approach to tackle the barriers ahead. It would also drive the system-wide reform that patients need, from research and development through to equitable availability.

By coming together and championing change, we can set the health system up for success and ensure that potentially life-changing treatments are accessible to the people who need them the most.

February 2024


Department of Health and Social Care. England Rare Diseases Action Plan 2023 Report. Available at:  England Rare Diseases Action Plan 2023: main report - GOV.UK ( Last accessed: February 2024.

2  EFPIA. We Won’t Rest. Available at: Last accessed: January 2024.

3 Department of Health and Social Care. The UK Rare Disease Framework 2021. UK Rare Diseases Framework - GOV.UK ( Last accessed: January 2024.

4 Genomics Education England.  100,000 Genomes Project. Available at: Last accessed: January 2024.

5 Medicine Manufacturing Industry Partnership. Advanced Therapies Manufacturing Action Plan. Available at: Last accessed: January 2024.

6 ABPI. Transformative treatments. Available at: Last accessed: January 2024.

7 UK Bioindustry Association. Ensuring patient access to cell and gene therapies: the case for an innovative payment model. Available at: Last accessed: January 2024.

8 Hale G et al. Flexibility in assessment of rare disease technologies via NICE's single technology appraisal route: a thematic analysis. Journal of Comparative Effectiveness Research. 2023; 12 (11)

9 Clarke S et al. The impact of rarity in NICE’S health technology appraisals. Orphanet Journal of Rare Diseases. 2012 16 (218)

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