title: New models for large prospective studies: is there a better way?
abstract: Large prospective cohort studies are critical for identifying etiologic factors for disease, but they require substantial long-term research investment. Such studies can be conducted as multisite consortia of academic medical centers, combinations of smaller ongoing studies, or a single large site such as a dominant regional health-care provider. Still another strategy relies upon centralized conduct of most or all aspects, recruiting through multiple temporary assessment centers. This is the approach used by a large-scale national resource in the United Kingdom known as the "UK Biobank," which completed recruitment/examination of 503,000 participants between 2007 and 2010 within budget and ahead of schedule. A key lesson from UK Biobank and similar studies is that large studies are not simply small studies made large but, rather, require fundamentally different approaches in which "process" expertise is as important as scientific rigor. Embedding recruitment in a structure that facilitates outcome determination, utilizing comprehensive and flexible information technology, automating biospecimen processing, ensuring broad consent, and establishing essentially autonomous leadership with appropriate oversight are all critical to success. Whether and how these approaches may be transportable to the United States remain to be explored, but their success in studies such as UK Biobank makes a compelling case for such explorations to begin.
authors.affiliation: Office of Population Genomics, National Human Genome Research Institute, Bethesda, Maryland, USA. manolio@nih.gov
authors.forename: Teri A
authors.initials: TA
authors.lastname: Manolio
authors.forename: Brenda K
authors.initials: BK
authors.lastname: Weis
authors.forename: Catherine C
authors.initials: CC
authors.lastname: Cowie
authors.forename: Robert N
authors.initials: RN
authors.lastname: Hoover
authors.forename: Kathy
authors.initials: K
authors.lastname: Hudson
authors.forename: Barnett S
authors.initials: BS
authors.lastname: Kramer
authors.forename: Chris
authors.initials: C
authors.lastname: Berg
authors.forename: Rory
authors.initials: R
authors.lastname: Collins
authors.forename: Wendy
authors.initials: W
authors.lastname: Ewart
authors.forename: J Michael
authors.initials: JM
authors.lastname: Gaziano
authors.forename: Steven
authors.initials: S
authors.lastname: Hirschfeld
authors.forename: Pamela M
authors.initials: PM
authors.lastname: Marcus
authors.forename: Daniel
authors.initials: D
authors.lastname: Masys
authors.forename: Catherine A
authors.initials: CA
authors.lastname: McCarty
authors.forename: John
authors.initials: J
authors.lastname: McLaughlin
authors.forename: Alpa V
authors.initials: AV
authors.lastname: Patel
authors.forename: Tim
authors.initials: T
authors.lastname: Peakman
authors.forename: Nancy L
authors.initials: NL
authors.lastname: Pedersen
authors.forename: Catherine
authors.initials: C
authors.lastname: Schaefer
authors.forename: Joan A
authors.initials: JA
authors.lastname: Scott
authors.forename: Timothy
authors.initials: T
authors.lastname: Sprosen
authors.forename: Mark
authors.initials: M
authors.lastname: Walport
authors.forename: Francis S
authors.initials: FS
authors.lastname: Collins
mesh_terms: D006801:Humans; D007258:Informed Consent; D018579:Patient Selection; D011446:Prospective Studies; D012107:Research Design