How to ask haemophilia patients about their preferences for gene therapies


Recent developments in gene therapy offer haemophilia patients the promise of permanent benefits or even a cure. But because these treatments are new, there are uncertainties about long-term efficacy and safety. This is a challenge for agencies that decide if a drug should be approved and what it should cost. PREFER researchers designed the very first patient preference study about what people with haemophilia think about gene therapies being brought to market.

Gene therapies are one of the emerging, complex biological therapies that are currently making their way from research and development to market. But health technology assessment and payer decision-making struggle to fit this new technology into their current evaluation schemes. Haemophilia is a genetic disorder that impairs the body’s ability to stop bleeding. PREFER researchers are hoping that patient preferences will help bridge the gaps.

In a recent Fontiers in Medicine publication, PREFER researchers outline their transparent approach to identifying the most suitable method to collect patient preferences to answer their research question regarding haemophilia patients’ preferences for gene therapy. They also involved patients and other stakeholders in the process to ensure they were asking the right questions. And an educational tool was also prepared and provided to survey participants at the launch of the preference study. This educational tool ensured that the haemophilia patients that took part had the information they needed to make informed decisions when going through the survey questions. Recruitment for the survey was completed in 2020. Soon, the researchers hope to share the results.

“With new and innovative treatments emerging, there is an urgent need among decision-makers to know what patients prefer, what benefits they are after, and what risks they are willing to take. In addition, there’s a lack of systematic integration of patient preferences and perspectives in decision-making today. The PREFER project is looking to find out when and how to listen to the patient voice. Looking at what patients prefer can help guide decision-making in a patient-centric direction” says Eline van Overbeeke, one of the authors.

By Anna Holm

van Overbeeke E, Hauber B et al, Patient Preferences to Assess Value IN Gene Therapies: Protocol Development for the PAVING Study in Hemophilia, Frontiers in Medicine, 9 March 2021

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Last modified: 2021-11-10