Life sciences Policy, research + analysis

Transforming lives: Making haemophilia B gene therapy a reality in Australia

October 2025

Transforming lives: Making haemophilia B gene therapy a reality in Australia

Haemophilia B is a rare, genetic disorder that turns even minor injuries into serious health risks. [1] Due to the absence of clotting factor IX, haemophilia B makes it difficult for the body to stop bleeding once it starts, resulting in prolonged bleeding episodes. [1, 2] In more severe cases, spontaneous internal bleeding can occur. [1]

In 2025, 646 Australians are living with haemophilia B, equal to around 2 people for every 100,000. [3]

Despite major medical progress, people with haemophilia B live with more complexity, limitations and uncertainty than most Australians. They face a constant burden of treatment, yet still suffer with breakthrough bleeds and compounding pain, mobility restrictions and disability. [5] The constant need for treatment imposes a mental and emotional toll, requiring endless planning, adjustment, consideration and care. [5]

“You have to make sure the treatment stays at the right temperature, so I always use carry on, never check-in. If I’m going for a four-week holiday, I’ll bring six to eight weeks of treatment, just in case.”

– Morgan, lives with severe haemophilia B

This treatment is a heavy economic burden for the healthcare system. Treatment costs for severe disease average $220,000 per person each year, 20x higher than those with mild disease. [3] In aggregate, these costs are too high to ignore.

These figures highlight the cumulative impact of this disease, and the potential opportunity from unlocking a more effective and equitable approach to care.

Fortunately, there is hope on the horizon. Gene therapy represents a turning point, a one-time treatment with the power to substantially reduce or eliminate the need for ongoing treatment, lessen disease burden, and improve long-term health and wellbeing. [6]

“It is a ‘treatment-free haemophilia’ – the therapy is so effective that the patient is free of both the disease and their treatment.”

– Professor Huyen Tran, Haematologist

More than just symptomatic relief, gene therapy can offer freedom from the constant anticipation of bleeding, treatment routines, and medical limitations. [5] This treatment can also deliver a long-term opportunity to ease the burden on Australia’s healthcare and disability systems.

Evohealth modelling estimates that if all adult Australians living with severe haemophilia B were treated with gene therapy, the national cost of haemophilia B could be reduced by $27.1 million annually. [3]

With a recent funding recommendation made for gene therapy and more undergoing clinical trials, this is the moment to redefine haemophilia B care in Australia. [7]

Swift and effective implementation is needed to translate this recommendation into equitable patient access. We need to meet the needs of people with haemophilia B and offer them a future not defined by infusions, pain, or limitations.

Our report Transforming lives: Making haemophilia B gene therapy a reality in Australia proposes three recommendations to pave the path forward for Australia’s haemophilia B community, and give people with this disease the chance to move beyond it.

Fast-track implementation of Recommendation 16 from the HTA Review to ensure innovative therapies are accessible as soon as possible.

Implement sustainable data systems to enable gene therapy monitoring and support evidence-based care.

Increase funding to deliver wraparound care for haemophilia B.

References

Haemophilia Foundation Australia. Haemophilia. 2024; Available from: https://www.haemophilia.org.au/bleeding-disorders/faqs/haemophilia/.
Haemophilia Foundation Australia. Haemophilia. 2023; Available from: https://www.haemophilia.org.au/HFA/media/Documents/Haemophilia/Haemophilia-information-booklet.pdf.
Evohealth. Haemophilia B economic model. 2025.
Haemophilia Foundation Australia. Haemophilia treatment. 2023; Available from: https://www.haemophilia.org.au/bleeding-disorders/haemophilia/haemophilia-treatment/.
Berntorp, E., et al., Quality of life in a large multinational haemophilia B cohort (The B-Natural study) – Unmet needs remain. Haemophilia, 2022. 28(3): p. 453-461.
Haemophilia Foundation Australia. How gene therapy works. 2019; Available from: https://www.haemophilia.org.au/ bleeding-disorders/living-with-a-bleeding-disorder/gene-emerging-therapies-hub/gene-therapy/how-gene-therapy-works/.
Department of Health, Disability and Ageing Medical Services Advisory Committee, 1728.1 – Etranacogene dezaparvovec for the treatment of Haemophilia B. 2025; Available from: https://www.msac.gov.au/applications/1728-1