Adaptive design in surveys and clinical trials: similarities, differences and opportunities for cross-fertilization

Michael Rosenblum, Peter V Miller, Benjamin Reist, Elizabeth A. Stuart, Michael Thieme, Thomas A. Louis*

*Corresponding author for this work

Research output: Contribution to journalArticle

Abstract

Adaptive designs involve preplanned rules for modifying an on-going study based on accruing data. We compare the goals and methods of adaptation for trials and surveys, identify similarities and differences, and make recommendations for what types of adaptive approaches from one domain have high potential to be useful in the other. For example, clinical trials could benefit from recently developed survey methods for monitoring which groups have low response rates and intervening to fix this. Clinical trials may also benefit from more formal identification of the target population, and from using paradata (contextual information collected before or during the collection of actual outcomes) to predict participant compliance and retention and then to intervene to improve these. Surveys could benefit from stopping rules based on information monitoring, applying techniques from sequential multiple-assignment randomized trial designs to improve response rates, prespecifying a formal adaptation protocol and including a data monitoring committee. We conclude with a discussion of the additional information, infrastructure and statistical analysis methods that are needed when conducting adaptive designs, as well as benefits and risks of adaptation.

Original languageEnglish (US)
Pages (from-to)963-982
Number of pages20
JournalJournal of the Royal Statistical Society. Series A: Statistics in Society
Volume182
Issue number3
DOIs
StatePublished - Jun 1 2019

Fingerprint

Adaptive Design
Fertilization
Clinical Trials
Monitoring
monitoring
Analysis and Statistical Methods
Randomized Trial
Stopping Rule
Compliance
statistical analysis
Recommendations
Assignment
Infrastructure
infrastructure
Predict
Target
Similarity
Clinical trials
Group
Response rate

Keywords

  • Adaptive design
  • Randomized trial
  • Sample survey

ASJC Scopus subject areas

  • Statistics and Probability
  • Social Sciences (miscellaneous)
  • Economics and Econometrics
  • Statistics, Probability and Uncertainty

Cite this

Rosenblum, Michael ; Miller, Peter V ; Reist, Benjamin ; Stuart, Elizabeth A. ; Thieme, Michael ; Louis, Thomas A. / Adaptive design in surveys and clinical trials : similarities, differences and opportunities for cross-fertilization. In: Journal of the Royal Statistical Society. Series A: Statistics in Society. 2019 ; Vol. 182, No. 3. pp. 963-982.
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Adaptive design in surveys and clinical trials : similarities, differences and opportunities for cross-fertilization. / Rosenblum, Michael; Miller, Peter V; Reist, Benjamin; Stuart, Elizabeth A.; Thieme, Michael; Louis, Thomas A.

In: Journal of the Royal Statistical Society. Series A: Statistics in Society, Vol. 182, No. 3, 01.06.2019, p. 963-982.

Research output: Contribution to journalArticle

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