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. PURPOSE: 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). LIMITATIONS: 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. CONCLUSION: 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.
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. PURPOSE: 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). LIMITATIONS: 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. CONCLUSION: 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: Scott D Halpern; Jason H T Karlawish; David Casarett; Jesse A Berlin; Raymond R Townsend; David A Asch Journal: Am Heart J Date: 2003-12 Impact factor: 4.749
Authors: Ajay K Israni; Scott D Halpern; Christopher McFadden; Rubeen K Israni; Alan Wasserstein; Sidney Kobrin; Jeffrey S Berns Journal: Kidney Int Date: 2004-03 Impact factor: 10.612
Authors: R J Barst; L J Rubin; W A Long; M D McGoon; S Rich; D B Badesch; B M Groves; V F Tapson; R C Bourge; B H Brundage; S K Koerner; D Langleben; C A Keller; S Murali; B F Uretsky; L M Clayton; M M Jöbsis; S D Blackburn; D Shortino; J W Crow Journal: N Engl J Med Date: 1996-02-01 Impact factor: 91.245
Authors: Dustin C Krutsinger; Jacqueline McMahon; Alisa J Stephens-Shields; Brian Bayes; Steven Brooks; Brian L Hitsman; Su Fen Lubitz; Celine Reyes; Robert A Schnoll; S Ryan Greysen; Ashley Mercede; Mitesh S Patel; Catherine Reale; Fran Barg; Jason Karlawish; Daniel Polsky; Kevin G Volpp; Scott D Halpern Journal: Contemp Clin Trials Date: 2018-11-08 Impact factor: 2.226
Authors: Laura P Forsythe; Victoria Szydlowski; Mohammad Hassan Murad; Stanley Ip; Zhen Wang; Tarig A Elraiyah; Rachael Fleurence; David H Hickam Journal: J Gen Intern Med Date: 2014-08 Impact factor: 5.128
Authors: Dustin C Krutsinger; Kuldeep N Yadav; Elizabeth Cooney; Steven Brooks; Scott D Halpern; Katherine R Courtright Journal: Contemp Clin Trials Commun Date: 2019-06-04
Authors: Gregg Harry Rawlings; Nigel Beail; Iain Armstrong; Robin Condliffe; David G Kiely; Ian Sabroe; Andrew R Thompson Journal: BMJ Open Date: 2020-12-07 Impact factor: 2.692