Gene therapy preferences and informed decision-making: Results from a National Hemophilia Foundation Community Voices in research survey

IntroductionTo inform education and treatment discussions, it is important to understand how persons with haemophilia prefer to learn about and discuss new therapies and to identify variables that influence decision-making. AimThe aim of this study was to evaluate preferences and variables which influence decision-making related to gene therapy and other novel haemophilia therapies. MethodsAn online survey was sent to men with severe haemophilia enrolled in the National Hemophilia Foundation Community Voices in Research online platform for patient-powered research. ResultsOne hundred four men completed the survey including 33% Hispanics, 96 who had had not gene therapy and 71/96 (74%) who were on prophylaxis. Ninety-five percent were somewhat or very familiar with gene therapy. Men with haemophilia obtain information about new therapies from several sources, most commonly their haemophilia treatment team, patient advocacy groups and self-study. Participants identified safety and efficacy as well as other educational needs to inform decision-making. Of those without prior gene therapy, 73% indicated a high likelihood of considering gene therapy. Hispanic ethnicity and government-issued insurance were associated with a higher likelihood of considering gene therapy as a treatment option. ConclusionHaemophilia Treatment Centers and patient advocacy groups must be able to educate persons with haemophilia about aspects of novel therapies which are important to the individual, especially short- and long-term safety and efficacy. Further research is needed to determine how patient activation and health literacy influence decision-making and how to achieve equitable access and valid informed consent for novel therapies.