Patients’ preferences for gene therapy in haemophilia


For innovative treatments and treatments for rare diseases, finding a way to include the patient perspective in decision-making can be crucial. With answers from 117 people with haemophilia, PREFER researchers present their results from their clinical case study about haemophilia patients’ preferences for gene therapy. They found that patient preferences vary greatly. And that informing patients about gene therapy can facilitate acceptance. 

Haemophilia is a rare disease that prevents blood from clotting. Regular intravenous administration of coagulation factor is the current standard of care. From gene therapy, one infusion might be able to replace this lifelong commitment. But the duration of treatment effects are uncertain, which makes decision-making about whether or not this new therapy should become available to patients difficult. Considering patient perspectives can help HTA authorities, payers and regulators make those decisions.

A paper recently published in Haemophilia outlines how patients’ preferences for gene therapy in haemophilia differ. Key elements that could explain the variation in preferences were education on gene therapy and residence.

“Some patients would not hesitate to switch from their current treatment to gene therapy. Others would never under the conditions presented in our survey. In part, this can be explained by our survey respondents’ own annual bleeding rates, their age, whether they received education on gene therapies and where they live.,” says Eline van Overbeeke, who lead the study during her PhD at the University of Leuven.

The results from this clinical case study showed the importance of patient education on gene therapy, as the researchers included an educational tool on gene therapy at the start of their survey and the time that patients spent on this tool was found to have an impact on the results. The patients who took more time to take in the educational materials offered, tended to be more accepting of gene therapies. For innovative treatments, educational tools may be especially useful to collect the informed views of patients. This to make sure they have the information they need to make informed trade-offs between the different benefits and risks of current treatment options and gene therapy.

By Anna Holm

van Overbeeke, Eline; Hauber, Brett; Michelsen, Sissel; Peerlinck, Kathelijne et al. Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey. Haemophilia. 2021; 1-11. 

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Last modified: 2021-11-10