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. 2013 Feb 15;177(4):285-9.
doi: 10.1093/aje/kws408. Epub 2013 Jan 7.

New Models for Large Prospective Studies: Is There a Risk of Throwing Out the Baby With the Bathwater?


New Models for Large Prospective Studies: Is There a Risk of Throwing Out the Baby With the Bathwater?

Michael B Bracken et al. Am J Epidemiol. .


Manolio et al. (Am J Epidemiol. 2012;175:859-866) proposed that large cohort studies adopt novel models using "temporary assessment centers" to enroll up to a million participants to answer research questions about rare diseases and "harmonize" clinical endpoints collected from administrative records. Extreme selection bias, we are told, will not harm internal validity, and "process expertise to maximize efficiency of high-throughput operations is as important as scientific rigor" (p. 861). In this article, we describe serious deficiencies in this model as applied to the United States. Key points include: 1) the need for more, not less, specification of disease endpoints; 2) the limited utility of data collected from existing administrative and clinical databases; and 3) the value of university-based centers in providing scientific expertise and achieving high recruitment and retention rates through community and healthcare provider engagement. Careful definition of sampling frames and high response rates are crucial to avoid bias and ensure inclusion of important subpopulations, especially the medically underserved. Prospective hypotheses are essential to refine study design, determine sample size, develop pertinent data collection protocols, and achieve alliances with participants and communities. It is premature to reject the strengths of large national cohort studies in favor of a new model for which evidence of efficiency is insufficient.

Comment in

  • Vehement agreement on new models?
    Manolio TA, Collins R; National Institutes of Health New Models Workshop Participants. Manolio TA, et al. Am J Epidemiol. 2013 Feb 15;177(4):290-1. doi: 10.1093/aje/kws410. Epub 2013 Jan 7. Am J Epidemiol. 2013. PMID: 23296352 Free PMC article. No abstract available.

Comment on

  • New models for large prospective studies: is there a better way?
    Manolio TA, Weis BK, Cowie CC, Hoover RN, Hudson K, Kramer BS, Berg C, Collins R, Ewart W, Gaziano JM, Hirschfeld S, Marcus PM, Masys D, McCarty CA, McLaughlin J, Patel AV, Peakman T, Pedersen NL, Schaefer C, Scott JA, Sprosen T, Walport M, Collins FS. Manolio TA, et al. Am J Epidemiol. 2012 May 1;175(9):859-66. doi: 10.1093/aje/kwr453. Epub 2012 Mar 12. Am J Epidemiol. 2012. PMID: 22411865 Free PMC article.

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