title: Participation of people with haemophilia in clinical trials of new treatments: an investigation of patients' motivations and existing barriers.
abstract: 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.
  
  
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Institute of Health and Society (IRSS), Catholic University of Louvain (UCL), Brussels, Belgium.|Haemostasis and Thrombosis Unit, Division of Haematology, Saint-Luc University Hospital, Brussels, Belgium.
authors.forename: Séverine
authors.initials: S
authors.lastname: Henrard
authors.affiliation: Institute of Health and Society (IRSS), Catholic University of Louvain (UCL), Brussels, Belgium.
authors.forename: Niko
authors.initials: N
authors.lastname: Speybroeck
authors.affiliation: Haemostasis and Thrombosis Unit, Division of Haematology, Saint-Luc University Hospital, Brussels, Belgium.
authors.forename: Cedric
authors.initials: C
authors.lastname: Hermans
mesh_terms: D000328:Adult; D000367:Age Factors; D002986:Clinical Trials as Topic; D005260:Female; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009042:Motivation; D010358:Patient Participation; D011795:Surveys and Questionnaires