Equity in the age of advanced therapies: insights from haemophilia

Laurence Woollard is Director of On The Pulse Consultancy and co-chair of ATMP Engage, a multi-stakeholder forum focused on patient involvement in advanced therapies. In his latest blog for Rare Disease Day, he reflects on how advanced therapies are being introduced within the NHS, using haemophilia to explore questions of equity and benefit-risk – highlighting the need for a Patient Charter.

Rare Disease Day this year focuses on equity. Within a healthcare context, equity is achieved, according to the World Health Organization, ‘when everyone can attain their full potential for health and wellbeing’ [1]. In practical terms, this means people living with rare conditions should have the same opportunity for timely diagnosis, effective treatment, and meaningful participation in society as those with more common disorders.

Advanced therapy medicinal products (ATMPs) – such as gene and cell therapies – can play an important role in narrowing this divide. By targeting or modifying the underlying genetic cause, these therapies may offer a meaningful alternative to symptom management and, in some cases, provide curative potential.

Since my last contribution to the ABPI in 2022 [2], the cell and gene therapy landscape has continued to expand and mature, building on decades of scientific progress, with increasing clinical trial activity and regulatory approvals worldwide. In the UK, data from the Cell and Gene Therapy Catapult [3] highlight sustained domestic clinical trial capability and a supportive research infrastructure. This broader sector momentum is reflected in reimbursement decisions – for example, 15 ATMPs are currently commissioned in England across 14 indications, with more than 2,500 patients treated to date [4].

Even with this momentum, moving ATMPs from development to adoption within the NHS is a complex undertaking. As the ABPI recently highlighted [5], there are several key considerations. These include affordability (i.e. high upfront costs for one-off treatments), uncertainty around the durability of benefit (central to both clinical and cost-effectiveness assessments), along with the specialist infrastructure required to deliver and monitor these therapies safely. Even where a regulatory route to market exists, availability does not guarantee sustained uptake; commercial and wider social factors can further shape how knowledge gaps are interpreted and how service readiness is managed in practice.

Haemophilia provides a timely example. NICE’s recommendation of the first gene therapy approved for haemophilia B – and the first product to receive managed access through the Innovative Medicines Fund (IMF) [6] – marked a significant milestone. Yet its rollout has also illustrated the nuances of introducing a novel, single-administration treatment into a condition where therapeutic advances over recent decades has been substantial and where the bar for subsequent innovation has incrementally risen alongside improvements in safety, efficacy, and quality of life [7].

At the time of writing, a small number of patients – understood to be fewer than ten – have received NHS-funded haemophilia B gene therapy, with additional centres preparing to assess and treat suitable individuals. For those infused, the potential personal health benefits associated with near-normal levels of bleed protection may be accompanied by wider social benefits, such as improved participation in work and education as well as reduced reliance on health and social care services (a marker of equity if I ever saw one).

Put simply, the very availability of an NHS-funded gene therapy for haemophilia is a success story in itself. That it is positioned as one option among established alternatives reflects pharmaceutical innovation converging with a national payer prepared to manage evidential uncertainty through a dedicated access pathway. In many rare diseases, comparable choices do not yet exist, which places a particular responsibility on how ringfenced funding is deployed in line with its intended purpose.

Notwithstanding, the cautious early adoption points to a confluence of real-world influences unique to this modality: (i) intrinsic constraints, such as eligibility criteria that restrict who can receive treatment; (ii) extrinsic variables, including the relative immaturity of long-term safety data and the operational aspects of delivering a first-in-class therapy within routine NHS care; and (iii) patient-specific considerations, particularly the logistical demands of pre- and post-monitoring [8]. Overly simplistic narratives – whether driven by unsubstantiated hype or by portraying smaller patient populations as a disproportionate social cost – can obscure the careful benefit–risk discussions taking place in clinical practice, a pattern also observed in UK media coverage of haemophilia gene therapy [9].

All this underscores the need to articulate a shared set of expectations – or principles – capable of guiding how ATMPs are communicated and delivered within the NHS, particularly for those living with rare conditions. Through ATMP Engage – a UK multi-stakeholder forum I co-chair alongside the Cell and Gene Therapy Catapult – we are developing a Patient Charter, shaped with input from patient advocacy groups and partners across the system, which seeks to reflect an ethos of equity while embedding transparency and shared decision making in how these technologies are introduced and used.

There is no getting away from the fact that acceptable levels of risk will differ across therapeutic areas. Many people living with haemophilia in the UK can now anticipate near-normal life expectancy. This is not to suggest unmet needs have been overcome – far from it. Nor does it imply that those additional years are necessarily lived in good health. But in other rare conditions, where treatment options remain limited, the trade-offs of an ATMP may look very different.

The common emerging thread, however, as reflected in the Charter initiative, is a collective imperative to prevent avoidable harm and foster learning between patient communities – whether closer to, or further from, clinical implementation – so that the value of ATMPs can be realised responsibly.

Acknowledgements:

The author is a person living with severe haemophilia A and did not receive an honorarium for writing this article. He wishes to thank Will Horsley, Lead Commissioner for NHS England’s Specialised Blood Disorders Clinical Reference Group; Professor Charlie Hay, Director of the UK National Haemophilia Database; and Dr Rich Gorman, Assistant Professor in Ethics and Social Science at Brighton and Sussex Medical School, for their input and thoughtful comments.

This article is a guest contribution. The views and opinions expressed are those of the author and do not necessarily reflect the views of the ABPI.

References:

1.    Health Equity. World Health Organization. https://www.who.int/health-topics/health-equity#tab=tab_1 [Accessed 24 February 2026].

2.    Woollard L. Moving beyond box-ticking and lip service – why patient involvement matters in a new era of ATMPs for rare diseases. ABPI. 2022. https://www.abpi.org.uk/media/blogs/2022/february/moving-beyond-box-ticking-and-lip-service-why-patient-involvement-matters-in-a-new-era-of-atmps-for-rare-diseases/ [Accessed 24 February 2026].

3.    Cell and Gene Therapy Catapult. UK 2025 ATMP Clinical Trials Database. 2026. Available at: https://ct.catapult.org.uk/resources/articles/clinical-trials-database-2025 [Accessed 24 February 2026].

4.    Doak B. Getting commissioned ATMPs to patients: key insights. [Presentation] Festival of Genomics and Biodata. 28 January 2026.

5.    ABPI. Unlocking access to future ATMPs in the UK: Comparing international approaches. 2024. Available from: https://www.abpi.org.uk/publications/unlocking-access-to-future-atmps-in-the-uk/ [Accessed 24 February 2026].

6.    NHS England. Innovative Medicines Fund. https://www.england.nhs.uk/medicines-2/innovative-medicines-fund/ [Accessed 24 February 2026].

7.    Henderson N, Hofer M, Bray G, et al. Investing in Innovation: A Spotlight on Haemophilia Therapies. London: Office of Health Economics. 2024. Available at: https://www.ohe.org/publications/spotlight-on-haemophilia-therapies/ [Accessed 24 February 2026].

8.    Pierce GF, Skinner M, O'Mahony B, et al. Why is the uptake of gene therapy in haemophilia less than expected? Res Pract Thromb Haemost 2025;9(5):102948. https://doi.org/10.1016/j.rpth.2025.102948

9.   Woollard L, Gorman R. The emerging need to manage patient expectations of haemophilia gene therapy amidst media hype in the UK. Ther Adv Rare Dis 2025;6. https://doi.org/10.1177/26330040251330317