Experience With Direct-to-Patient Recruitment for Enrollment Into a Clinical Trial in a Rare Disease: A Web-Based Study

Abstract

BackgroundThe target sample size for clinical trials often necessitates a multicenter (center of excellence, CoE) approach with associated added complexity, cost, and regulatory requirements. Alternative recruitment strategies need to be tested against this standard model.ObjectivesThe aim of our study was to test whether a Web-based direct recruitment approach (patient-centric, PC) using social marketing strategies provides a viable option to the CoE recruitment method.MethodsPC recruitment and Web-based informed consent was compared with CoE recruitment for a randomized controlled trial (RCT) of continuing versus stopping low-dose prednisone for maintenance of remission of patients with granulomatosis with polyangiitis (GPA).ResultsThe PC approach was not as successful as the CoE approach. Enrollment of those confirmed eligible by their physician was 10 of 13 (77%) and 49 of 51 (96%) in the PC and CoE arms, respectively (P=.05). The two approaches were not significantly different in terms of eligibility with 34% of potential participants in the CoE found to be ineligible as compared with 22% in the PC arm (P=.11) nor in provider acceptance, 22% versus 26% (P=.78). There was no difference in the understanding of the trial as reflected in the knowledge surveys of individuals in the PC and CoE arms.ConclusionsPC recruitment was substantially less successful than that achieved by the CoE approach. However, the PC approach was good at confirming eligibility and was as acceptable to providers and as understandable to patients as the CoE approach. The PC approach should be evaluated in other clinical settings to get a better sense of its potential.

Attitudes towards mental health, mental health research and digital interventions by young adults with type 1 diabetes: A qualitative analysis

“…For researchers targeting young people with type 1 diabetes, low disease prevalence in the 15‐ to 24‐year‐age group (approximately 0.2%) is an additional recruitment barrier . Direct referral to psychosocial studies by health practitioners can be a valuable and cost‐effective method for recruiting people with medical conditions . However, low rates of attendance at diabetes clinics and medical appointments by young people with type 1 diabetes mean that even this targeted recruitment approach is likely to yield only a small group of eligible candidates.…”

“…6 Direct referral to psychosocial studies by health practitioners can be a valuable and cost-effective method for recruiting people with medical conditions. 13 However, low rates of attendance at diabetes clinics and medical appointments by young people with type 1 diabetes 14 mean that even this targeted recruitment approach is likely to yield only a small group of eligible candidates.…”

A novel approach to conducting clinical trials in the community setting: utilizing patient-driven platforms and social media to drive web-based patient recruitment

“…Results of the TAPIR trial included 49 patients in the traditional clinical center recruitment arm, with 10 in the online recruitment arm [33].. Enrollment goals for each arm was 3.3 participants per month, with actual enrollment rates of 0.4 (online recruitment) and 1.8 (traditional recruitment) participants per month. Social media recruitment efforts utilized for the online-recruitment arm resulted in 16,094 individuals visiting the public TAPIR website over an approximate two-year period.…”

“…The TAPIR trial also required online recruitment arm patients to provide their doctors with a Physician packet to complete for confirmation that they were eligible for the trial. Only 35 out of 47 physicians provided additional information about their patients, signifying a loss of patients at this stage, illustrating the importance of seeking ways to engaging clinicians in the recruitment of patients for research [33].…”

“…In The Assessment of Prednisone in Remission (TAPIR) trial, online recruitment strategies via (Webbased and social media strategies) were tested for comparison with traditional center of excellence recruitment strategies. This clinical trial tested whether patients with granulomatosis with polyangiitis (GPA) had better outcomes after their GPA was well-controlled if they stayed on a dose of 5 mg/day of prednisone or fully came off prednisone [33]. The online recruitment arm of the study utilized a Patient Advocacy Group (PAG) website and social media platforms (Facebook, Twitter, and Google+) to direct potential participants to a public study website.…”