Studies without internal controls

J. C. Bailar, Thomas Louis, P. W. Lavori, M. Polansky

Research output: Contribution to journalArticle

Abstract

Sometimes question of clinical interest can be addressed only by investigators without concurrent controls that are under the supervision of the investigator (internal controls). Such studies nearly always make use of other types of comparisons (external controls), such as historical controls. In this paper we examine studies of clinical treatments that have weak internal controls or lack internal controls, as illustrated by recent examples from the Journal. These studies have a small but important and unique role in clinical investigation. Five interrelated features can add to the strength of such studies: (1) an intent by the investigator, expressed before the study, that the treatment will affect the outcomes reported; (2) planning of the analysis before the data are generated; (3) articulation of a plausible hypothesis before the results are observed; (4) a likelihood that the results would still have been of interest if they had been 'opposite' in some sense; and (5) reasonable grounds for generalizing the results from the study subjects to a substantially broader group of patients. In spite of potential pitfalls, carefully selected and reported studies without internal controls can play a substantial part in the aquisition of scientific knowledge.

Original languageEnglish (US)
Pages (from-to)156-162
Number of pages7
JournalNew England Journal of Medicine
Volume311
Issue number3
StatePublished - 1984
Externally publishedYes

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Research Personnel
Therapeutics
Clinical Studies

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Bailar, J. C., Louis, T., Lavori, P. W., & Polansky, M. (1984). Studies without internal controls. New England Journal of Medicine, 311(3), 156-162.

Studies without internal controls. / Bailar, J. C.; Louis, Thomas; Lavori, P. W.; Polansky, M.

In: New England Journal of Medicine, Vol. 311, No. 3, 1984, p. 156-162.

Research output: Contribution to journalArticle

Bailar, JC, Louis, T, Lavori, PW & Polansky, M 1984, 'Studies without internal controls', New England Journal of Medicine, vol. 311, no. 3, pp. 156-162.
Bailar JC, Louis T, Lavori PW, Polansky M. Studies without internal controls. New England Journal of Medicine. 1984;311(3):156-162.
Bailar, J. C. ; Louis, Thomas ; Lavori, P. W. ; Polansky, M. / Studies without internal controls. In: New England Journal of Medicine. 1984 ; Vol. 311, No. 3. pp. 156-162.
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