title: Experience With Direct-to-Patient Recruitment for Enrollment Into a Clinical Trial in a Rare Disease: A Web-Based Study.
abstract: The 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.
  
  
  
  The 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.
  
  
  
  PC 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).
  
  
  
  The 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.
  
  
  
  PC 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.
authors.affiliation: Rare Diseases Clinical Research Network Data Coordinating Center, Health Informatics Institute, University of South Florida, Tampa, FL, United States.
authors.forename: Jeffrey
authors.identifier: 0000-0003-4526-888X
authors.initials: J
authors.lastname: Krischer
authors.affiliation: University of Pennsylvania, Philadelphia, PA, United States.
authors.forename: Peter F
authors.identifier: 0000-0003-2570-7056
authors.initials: PF
authors.lastname: Cronholm
authors.affiliation: Rare Diseases Clinical Research Network Data Coordinating Center, Health Informatics Institute, University of South Florida, Tampa, FL, United States.
authors.forename: Cristina
authors.identifier: 0000-0002-0660-8127
authors.initials: C
authors.lastname: Burroughs
authors.affiliation: University of Pennsylvania, Philadelphia, PA, United States.
authors.forename: Carol A
authors.identifier: 0000-0002-0016-6866
authors.initials: CA
authors.lastname: McAlear
authors.affiliation: Rare Diseases Clinical Research Network Data Coordinating Center, Health Informatics Institute, University of South Florida, Tampa, FL, United States.
authors.forename: Renee
authors.identifier: 0000-0001-5262-0082
authors.initials: R
authors.lastname: Borchin
authors.affiliation: University of Pennsylvania, Philadelphia, PA, United States.
authors.forename: Ebony
authors.identifier: 0000-0002-9188-2373
authors.initials: E
authors.lastname: Easley
authors.affiliation: University of Pennsylvania, Philadelphia, PA, United States.
authors.forename: Trocon
authors.identifier: 0000-0002-8416-6432
authors.initials: T
authors.lastname: Davis
authors.affiliation: Vasculitis Foundation, Kansas City, MO, United States.
authors.forename: Joyce
authors.identifier: 0000-0003-0383-0926
authors.initials: J
authors.lastname: Kullman
authors.affiliation: Mount Sinai Hospital, Toronto, ON, Canada.
authors.forename: Simon
authors.identifier: 0000-0001-5793-6262
authors.initials: S
authors.lastname: Carette
authors.affiliation: St. Joseph's Healthcare Hamilton, Hamilton, ON, Canada.
authors.forename: Nader
authors.identifier: 0000-0002-8270-2617
authors.initials: N
authors.lastname: Khalidi
authors.affiliation: University of Utah, Salt Lake City, UT, United States.
authors.forename: Curry
authors.identifier: 0000-0002-4494-7672
authors.initials: C
authors.lastname: Koening
authors.affiliation: Cleveland Clinic Foundation, Cleveland, OH, United States.
authors.forename: Carol A
authors.identifier: 0000-0001-9498-321X
authors.initials: CA
authors.lastname: Langford
authors.affiliation: Boston University School of Medicine, Boston, MA, United States.
authors.forename: Paul
authors.identifier: 0000-0003-4937-0515
authors.initials: P
authors.lastname: Monach
authors.affiliation: University of Pittsburgh, Pittsburgh, PA, United States.
authors.forename: Larry
authors.identifier: 0000-0003-4483-0359
authors.initials: L
authors.lastname: Moreland
authors.affiliation: Mount Sinai Hospital, Toronto, ON, Canada.
authors.forename: Christian
authors.identifier: 0000-0001-6287-9549
authors.initials: C
authors.lastname: Pagnoux
authors.affiliation: Mayo Clinic, Rochester, MN, United States.
authors.forename: Ulrich
authors.identifier: 0000-0002-6559-1206
authors.initials: U
authors.lastname: Specks
authors.affiliation: University of Pennsylvania, Philadelphia, PA, United States.
authors.forename: Antoine G
authors.identifier: 0000-0002-7864-0185
authors.initials: AG
authors.lastname: Sreih
authors.affiliation: Mayo Clinic, Rochester, MN, United States.
authors.forename: Steven
authors.identifier: 0000-0001-5215-1263
authors.initials: S
authors.lastname: Ytterberg
authors.affiliation: University of Pennsylvania, Philadelphia, PA, United States.
authors.forename: Peter A
authors.identifier: 0000-0001-9284-7345
authors.initials: PA
authors.lastname: Merkel
authors.affiliation: Vasculitis Clinical Research Consortium, Philadelphia, PA, United States.
mesh_terms: D000328:Adult; D000368:Aged; D005260:Female; D014890:Granulomatosis with Polyangiitis / Q000175:diagnosis / Q000188:drug therapy; D006801:Humans; D020407:Internet; D008297:Male; D008875:Middle Aged; D018579:Patient Selection; D011241:Prednisone / Q000008:administration & dosage; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D035583:Rare Diseases / Q000175:diagnosis* / Q000188:drug therapy*; D011795:Surveys and Questionnaires
chemical_list: D011241:Prednisone
keywords: clinical trial; direct-to-consumer advertising; granulomatosis with polyangiitis; research subject recruitment; social media