Shared decision-making and haemophilia gene therapy


Shared decision-making is a catchy piece of language – and it increasingly feels like it’s everywhere when it comes to haemophilia care! So, what is it all about? Simon Fletcher and his colleagues, Kate Khair and Kathryn Jenner, discuss the details in their paper ‘Shared decision-making for gene therapy in haemophilia care‘, recently published in The Journal of Haemophilia Practice.

Shared decision-making is about recognising that healthcare professionals and patients bring different, yet equally important, forms of expertise to discussions and choices about healthcare. This is increasingly important with the growing range – and complexity – of different options for haemophilia treatment.

The shared decision-making process provides a way to account for people’s individual preferences, values, and goals when thinking about what therapies might be best for them. Rather than being told about or simply given a product, it involves creating space for open discussion about what is important to individuals – a road towards making care more ‘personalised’.


Practical challenges

Shared decision-making sounds great on paper! But there are a lot of challenges to implementing it in practice, as Simon and his colleagues explain. They observe that although we increasingly see shared decision-making being recommended in healthcare policy and scientific articles, the extent to which it is truly practiced is unclear.

Shared decision-making requires training, skills, time and space. It’s questionable whether healthcare systems are investing in the support and resources needed to truly make shared decision-making a success and not just a buzzword.

Additionally, shared decision-making involves a big culture change. It requires making space for listening to the lived experience of people with haemophilia and recognising that they may have differing opinions about treatment to their healthcare providers.


Gene therapy

As Simon and his co-authors explain, shared decision-making is particularly important in the context of gene therapy. Based on the current technologies available, gene therapy for haemophilia is likely to be a one-time-only treatment, and it’s irreversible. This makes it absolutely vital that people with haemophilia are well informed about the treatment and the uncertainties it involves, in terms of both risk and benefit.

For shared decision-making about gene therapy to work properly, people with haemophilia need support to understand the range of the care, treatment and support options available. Having this understanding is essential to make informed decisions based on evidence, quality information and personal preference. After all, can you really ‘share’ in a decision about gene therapy treatment if you don’t quite understand what some of the downsides might be?


Patient understanding

How to ensure people with haemophilia are equipped and engaged with the knowledge and information they need to understand a complex treatment like gene therapy is a particular challenge. Simon and his colleagues argue that there is a need for tools that can support people with haemophilia to learn about gene therapy’s benefits and risks – tools that can explore the differences between expectations and realities of these complex and new treatments.

At the same time, should we, as people with haemophilia, all be expected to become experts in our condition, just to make the most of new treatments? Proper support would be needed here to avoid the risk of creating further health inequalities – without this, it may be that only people who have the time to ‘study up’ are enabled to successfully participate in shared decision-making pathways.

As Simon and his co-authors reflect, patient organisations have a vital role here, in supporting their communities to understand new treatments like gene therapy – as well as empowering people to expect and take advantage of practices like shared decision-making. The recent ‘Self-advocacy – What’s it all about?’ by Local Families with Bleeding Disorders is a great example of this.


Supporting both sides

There’s (quite rightly) a huge area of work going on to develop checklists, prompts, discussion guides and educational materials to support ‘patients’ grappling with gene therapy and shared decision making – but healthcare professionals need support and training too. Holding a space open, encouraging people to speak freely and instilling confidence is not an easy task, particularly in the context of healthcare systems which for years have centred clinician’s voices and expertise.

Shared decision-making is described as a two-sided intervention that aids clinical decision making. We need to ensure that both ‘sides’ of this novel form of dialogue are supported to put it into practice. As Simon and his colleagues conclude, gene therapy for haemophilia will only be a success if people with haemophilia ‘are fully educated and informed by fully educated and informed healthcare teams’.


Further reading

Fletcher S, Jenner K, Khair K. Shared decision-making for gene therapy in haemophilia care. J Haem Pract 2023; 10(1): 62-67. doi: 10.2478/jhp-2023-0009.

Fletcher S. Tuning in on gene therapy. Haemnet blog. 14 November 2022.

Noone D. Visualising haemophilia gene therapy. Haemnet blog. 13 March 2023.


About the author

Rich Gorman lives with severe haemophilia A. He works as a researcher at Brighton and Sussex Medical School.


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