Background: Over the last 50 years, clinical research investigating new treatments has been transforming the care of patients with haemophilia but we still have a long way to go and most clinical investigators are facing difficulties in recruiting appropriate candidates. A survey was conducted to evaluate what motivates people with haemophilia to participate in clinical research and to identify factors that might influence their willingness to participate.
Material and methods: A specific questionnaire concerning motivation and barriers to participation in clinical trials was sent to 135 adults with haemophilia. A classification tree was used to identify predictors of willingness to participate.
Results: Sixty-two patients returned the completed questionnaire, of whom 51 declared a potential willingness to participate in a clinical trial, although many were concerned about the new treatments' possible side effects or about time away from work. Predictors of willingness to participate were evaluated using a classification tree and four groups were established. Group 1 comprised patients aged ≤45 years old. Group 2 comprised patients>45 years old who reported having no knowledge of clinical research modalities. The two other groups comprised patients>45 years old who reported having some knowledge of clinical research modalities, with group 3 being ≤59 years old and group 4 being >59 years old. The rate of willingness to participate was 96.6%, 28.6%, 70.6% and 100.0%, respectively.
Discussion: The rate of willingness to participate in clinical research was significantly lower in patients who reported having no knowledge of clinical trial modalities, highlighting the relevance of providing improved knowledge about the modalities, risks, and benefits of clinical research to enhance participation in haemophilia trials.