title: Motivations of patients with pulmonary arterial hypertension to participate in randomized clinical trials.
abstract: BACKGROUND
  There is substantial need to rigorously evaluate existing and new therapies for pulmonary arterial hypertension (PAH) and other severe and relatively rare conditions affecting younger patients. However, the ability to conduct meaningful randomized clinical trials (RCTs) in such contexts often is limited by difficulties obtaining adequate patient enrollment.
  
  
  OBJECTIVE
  To understand the motivations of patients with PAH for participating in RCTs so as to facilitate enrollment in future trials among patients with similar diseases.
  
  
  METHODS
  We conducted semistructured interviews of a diverse sample of patients with World Health Organization (WHO) Group I PAH. We purposefully recruited a diverse sample of participants until theoretical saturation was reached. We randomly assigned patients to review hypothetical RCTs that did or did not allow continuation of background PAH therapies and elicited their reasons for or against enrolling. Interviews were transcribed and analyzed using constant comparison techniques to code and sort data into discrete themes.
  
  
  RESULTS
  The 26 PAH patients enrolled before theoretical saturation was reached identified 24 factors that would influence their RCT enrollment decisions. These factors grouped naturally into four themes: (1) personal medical benefits, (2) personal medical risks/harms, (3) nonmedical benefits, and (4) nonmedical burdens. Personal benefits were cited as commonly as altruistic motives. One third of the patients (9/26) suggested that they would defer enrollment decisions to their treating clinicians. Seventy-nine percent of patients (11/14) assigned to consider trials without background therapies expressed concerns about clinical deterioration (vs. 17% (2/12) among patients assigned to consider trials allowing background therapies).
  
  
  CONCLUSIONS
  The sample was recruited from a single academic center. Furthermore, the use of hypothetical trials may not elicit identical decision-making processes as may be used among patients contemplating actual trial participation.
  
  
  CONCLUSIONS
  For PAH patients considering RCT enrollment, the potentials for personal benefit and risk are at least as important as altruistic motives. Minimizing the time demands of participating, financial remuneration, and allowing participants to continue current therapies are factors, which might enhance enrollment to trials in similar disease areas.
authors.affiliation: Department of Medicine, University of Pennsylvania, Philadelphia, 19104-6021, USA.
authors.forename: Ricki
authors.initials: R
authors.lastname: Carroll
authors.forename: Jules
authors.initials: J
authors.lastname: Antigua
authors.forename: Darren
authors.initials: D
authors.lastname: Taichman
authors.forename: Harold
authors.initials: H
authors.lastname: Palevsky
authors.forename: Paul
authors.initials: P
authors.lastname: Forfia
authors.forename: Steven
authors.initials: S
authors.lastname: Kawut
authors.forename: Scott D
authors.initials: SD
authors.lastname: Halpern
mesh_terms: D000328:Adult; D000368:Aged; D000533:Altruism; D065627:Familial Primary Pulmonary Hypertension; D005260:Female; D006801:Humans; D006976:Hypertension, Pulmonary / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D009042:Motivation; D010358:Patient Participation / Q000523:psychology*; D018579:Patient Selection; D016032:Randomized Controlled Trials as Topic / Q000523:psychology*; D060926:Remuneration; D035842:Research Subjects / Q000523:psychology*; D018570:Risk Assessment