title: Improving efficiency and reducing costs: Design of an adaptive, seamless, and enriched pragmatic efficacy trial of an online asthma management program.
abstract: Clinical trials are critical for medical decision-making, however, under the current paradigm, clinical trials are fraught with problems including low enrollment and high cost. Promising alternatives to increase trial efficiency and reduce costs include the use of (1) electronic initiatives that permit electronic remote data capture (EDC) for direct data collection at a site (2), electronic medical records (EMR) for patient identification and data collection, and (3) adaptive, enrichment designs with pragmatic approaches. We describe the design of a seamless, multi-site randomized Phase II/III trial to evaluate an asthma management intervention in urban adolescents with asthma. Patients are randomized, asked to access four online sessions of the intervention or control asthma management program, and are then followed for one year. The primary efficacy endpoint is self-reported asthma control as measured by the Asthma Control Test (ACT). Comparative effectiveness parametric approaches are utilized to conduct the trial in a real world setting with reduced costs. Escalated electronic initiatives are implemented for patient identification, assent, enrollment and tracking. Patient enrollment takes place during primary care visits. A centralized database with EDC is used for CRF data collection with integration of EMR data. This Phase II/III trial plans to have a total sample size of 500 patients with an interim look at the completion of Phase II (n=250), The interim analyses include an assessment of the intervention effect, marker(s) identification and the feasibility study of EMR data as the trial CRF data collection. Patient enrollment has begun and is ongoing.
authors.affiliation: Department of Public Health Sciences, Henry Ford Hospital, Detroit, MI, USA. Electronic address: mlu1@hfhs.org.
authors.forename: Mei
authors.initials: M
authors.lastname: Lu
authors.affiliation: Department of Pediatrics, Georgia Regents University, Augusta, GA, USA.
authors.forename: Dennis R
authors.initials: DR
authors.lastname: Ownby
authors.affiliation: Department of Internal Medicine, Division of Allergy, Henry Ford Hospital, Detroit, MI, USA.
authors.forename: Edward
authors.initials: E
authors.lastname: Zoratti
authors.affiliation: The Center for Health Research/Southeast, Kaiser Permanente Georgia, Atlanta, GA, USA.
authors.forename: Douglas
authors.initials: D
authors.lastname: Roblin
authors.affiliation: Department of Public Health Sciences, Henry Ford Hospital, Detroit, MI, USA.
authors.forename: Dayna
authors.initials: D
authors.lastname: Johnson
authors.affiliation: Department of Public Health Sciences, Henry Ford Hospital, Detroit, MI, USA.
authors.forename: Christine Cole
authors.initials: CC
authors.lastname: Johnson
authors.affiliation: Department of Public Health Sciences, Henry Ford Hospital, Detroit, MI, USA.
authors.forename: Christine L M
authors.initials: CL
authors.lastname: Joseph
mesh_terms: D000293:Adolescent; D001249:Asthma / Q000188:drug therapy* / Q000191:economics; D003362:Cost-Benefit Analysis; D003625:Data Collection / Q000379:methods*; D057286:Electronic Health Records; D005260:Female; D006801:Humans; D008297:Male; D062072:Patient Acuity; D012107:Research Design; D057566:Self Report; D055815:Young Adult
keywords: Adolescents; Asthma management intervention; Clinical trials; Comparative effectiveness research; Enrichment design; Seamless adaptive design