Participation for Innovation: Survey of Motivation for Clinical Studies Evaluating New Treatments for Hemophilia

Abstract 4778

Over the last 50 years, clinical research into new treatments has helped to transform the care of individuals with hemophilia. Life expectancy for severe hemophiliacs has indeed increased from 11 years in the 1960s to more than 60 years today and many patients with hemophilia are now able to lead an active and fulfilled life. All this has been possible largely through the commitment of people with hemophilia to active participation in clinical research. While much has been achieved, much remains to be accomplished such as the development and validation of longer acting clotting factor concentrates, new bypassing agents and the validation of treatment strategies aiming at preventing or reducing the risk of inhibitor development. Although many clinical trials are currently ongoing around the world, most of them are facing difficulties in recruiting candidate patients, mainly in developed countries. In this context, there is a major need to improve the patients' awareness of the importance of clinical research in hemophilia and highlight the opportunities of an active participation in clinical trials. A survey was launched to evaluate the motivation of patients with hemophilia for clinical studies and identify factors which might influence their willingness to participate. A specific questionnaire was sent to 189 patients with hemophilia (127 adults and 62 children) regularly attending the hemophilia treatment Center of the Cliniques Universitaires Saint-Luc, Brussels, Belgium. Different aspects such as socio-demographic status, familial situation, current hemophilia treatment, knowledge about principles and perceived benefits and risks of clinical studies, positive and negative factors for motivation were extensively addressed. Preliminary results show that most patients have a limited knowledge about the general principles of clinical research. Patients on prophylaxis show a higher motivation to participate than patients treated on demand. Patients are mainly concerned by possible side effects of new treatments. The time lost from school or work refrains many motivated patients from an active participation. Moreover, very few patients are ready to take part in phase 1 trial fearing unknown reactions or side-effects. This survey, which will soon be extended to several hemophilia centers in Europe, highlights the importance to raise awareness and provide better knowledge about the modalities, the risks and benefits of clinical research.