Most haemophilia patients are positive towards gene therapies


Structured patient input can support decision-making across the medical product life cycle. This is where patient preference studies are useful. In a recent Haemophilia publication, PREFER researchers share their findings regarding haemophilia patients’ preferences for gene therapies. Despite the uncertainties about long-term efficacy and safety of the treatments, it turns out most of the patients are positive. 

Haemophilia is a genetic disorder that affects the body’s ability to stop bleeding. Severe cases are treated by regular intravenous treatment. Gene therapy could potentially offer a cure for haemophilia. Most haemophilia patients are positive towards gene therapies. Their willingness to receive gene therapy was motivated by the promise of a reduction in bleeds, a high and stable level of blood clotting factors, an easier day-to-day life, and the chance of stopping their prevention therapy.

“Knowing what trade-offs patients make can help support decision-making processes. In this case especially in regulatory, health technology assessment and payer bodies. Patients were more concerned about uncertainty regarding long-term safety than uncertainty regarding long-term efficacy, for example. This is information that will be useful,” says Eline van Overbeeke, one of the authors. 

Gene therapies for haemophilia are in the late stages of development, but are not yet available to patients. These new treatments could potentially cure type A and B haemophilia with one infusion. In addition to supporting regulatory, HTA and payer decision-making, the PREFER researchers also hope their findings can inform clinical trial design, pay-for-performance schemes and real-world evidence studies in the future. 

By Anna Holm

van Overbeeke E, Michelsen S, Hauber B, Peerlinck K, Harmans C, Lambert C, Goldman M, Simoens S, Huys I, Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study, Haemophilia, 2020;00:1–8. 

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Last modified: 2021-11-10