The DOI matches its Crossref record; 40 of the 40 references it cites were found in a registry or an index.
A predictive approach to selecting the size of a clinical trial, based on subjective clinical opinion
Abstract
The ‘textbook’ approach to determining sample size in a clinical trial has some fundamental weaknesses which we discuss. We describe a new predictive method which takes account of prior clinical opinion about the treatment difference. The method adopts the point of clinical equivalence (determined by interviewing the clinical participants) as the null hypothesis. Decision rules at the end of the study are based on whether the interval estimate of the treatment difference (classical or Bayesian) includes the null hypothesis. The prior distribution is used to predict the probabilities of making the decisions to use one or other treatment or to reserve final judgement. It is recommended that sample size be chosen to control the predicted probability of the last of these decisions. An example is given from a multi‐centre trial of superficial bladder cancer.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Statistical Methods for Rates and Proportions
Fleiss, J. L., Levin, B., & Paik, M. C. (2003) · Wiley Series in Probability and Statistics · doi:10.1002/0471445428 · Cited by 18,646
Judgment under Uncertainty: Heuristics and Biases
(1982) · doi:10.1017/cbo9780511809477 · Cited by 8,240
Judgment under uncertainty: List of contributors
Kahneman, D. J., Slovic, P., & Tversky, A. (1982) · Cited by 4,670
Judgment under uncertainty: Causality and attribution
Kahneman, D. J., Slovic, P., & Tversky, A. (1982) · Cited by 4,670
Statistical Methods in Medical Research.
Armitage, P. (1972) · Biometrics · doi:10.2307/2556182 · Cited by 4,029
Judgement under Uncertainty: Heuristics and Biases
Hirschman, E. C., Kahneman, D., Slovic, P., & Tversky, A. (1983) · Journal of Marketing Research · doi:10.2307/3151689 · Cited by 3,813
Clinical Trials: A Practical Approach.
Hill, I. D., & Pocock, S. J. (1984) · Biometrics · doi:10.2307/2531186 · Cited by 2,930
Peto, R., Pike, M. C., Armitage, P., Breslow, N. E., Cox, D. R., Howard, S. V., Mantel, N., McPherson, K., Peto, J., & Smith, P. G. (1976) · British Journal of Cancer · doi:10.1038/bjc.1976.220 · Cited by 2,294
Applied Statistical Decision Theory
Lindley, D. V., Raiffa, H., & Schlaifer, R. (1962) · Econometrica · doi:10.2307/1909900 · Cited by 2,172
Statistical Methods for Rates and Proportions.
Plewis, I., & Fleiss, J. L. (1982) · Journal of the Royal Statistical Society. Series A (General) · doi:10.2307/2982107 · Cited by 1,731
Applied Statistical Decision Theory.
Roberts, H. V., Raiffa, H., & Schlaifer, R. (1962) · Journal of the American Statistical Association · doi:10.2307/2282452 · Cited by 1,443
Introduction to sample size determination and power analysis for clinical trials
Lachin, J. M. (1981) · Controlled Clinical Trials · doi:10.1016/0197-2456(81)90001-5 · Cited by 1,012
“Proving the null hypothesis” in clinical trials
Blackwelder, W. C. (1982) · Controlled Clinical Trials · doi:10.1016/0197-2456(82)90024-1 · Cited by 887
The combination of randomized and historical controls in clinical trials
Pocock, S. J. (1976) · Journal of Chronic Diseases · doi:10.1016/0021-9681(76)90044-8 · Cited by 514
Cognitive Processes and the Assessment of Subjective Probability Distributions
Hogarth, R. M. (1975) · Journal of the American Statistical Association · doi:10.1080/01621459.1975.10479858 · Cited by 479
Controversy in Counting and Attributing Events in Clinical Trials
Sackett, D. L., & Gent, M. (1979) · New England Journal of Medicine · doi:10.1056/nejm197912273012602 · Cited by 413
Dunnett, C. W., & Gent, M. (1977) · Biometrics · doi:10.2307/2529457 · Cited by 389
Reliability of Subjective Probability Forecasts of Precipitation and Temperature
Murphy, A. H., & Winkler, R. L. (1977) · Applied Statistics · doi:10.2307/2346866 · Cited by 386
Monitoring clinical trials: Conditional or predictive power?
Spiegelhalter, D. J., Freedman, L. S., & Blackburn, P. R. (1986) · Controlled Clinical Trials · doi:10.1016/0197-2456(86)90003-6 · Cited by 307
Self-citationApproaches to sample size estimation in the design of clinical trials—a review
Donner, A. (1984) · Statistics in Medicine · doi:10.1002/sim.4780030302 · Cited by 250
An aid to data monitoring in long-term clinical trials
Halperin, M., Gordon Lan, K. K., Ware, J. H., Johnson, N. J., & DeMets, D. L. (1982) · Controlled Clinical Trials · doi:10.1016/0197-2456(82)90022-8 · Cited by 195
The Design and Analysis of Sequential Clinical Trials.
Prescott, R. J., & Whitehead, J. (1983) · Biometrics · doi:10.2307/2531365 · Cited by 182
Statistics and Ethics in Surgery and Anesthesia
Gilbert, J., McPeek, B., & Mosteller, F. C. (1977) · Science · doi:10.1126/science.333585 · Cited by 152
Repeated confidence intervals for group sequential clinical trials
Jennison, C., & Turnbull, B. W. (1984) · Controlled Clinical Trials · doi:10.1016/0197-2456(84)90148-x · Cited by 150
Predictive Probability Early Termination Plans For Phase II Clinical Trials
Herson, J. (1979) · Biometrics · doi:10.2307/2530109 · Cited by 140
Statistics and Medical Experimentation
Meier, P. (1975) · Biometrics · doi:10.2307/2529434 · Cited by 119
Freedman, L. S., & Spiegelhalter, D. J. (1983) · Journal of the Royal Statistical Society Series D (The Statistician) · doi:10.2307/2987606 · Cited by 111
Self-citationFeinstein, A. R. (1975) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt1975184491 · Cited by 105
Randomised Controlled Clinical Trials
Bulpitt, C. J. (1996) · Springer eBooks · doi:10.1007/978-1-4615-6347-1 · Cited by 87
Stopping Rules for Clinical Trials Incorporating Clinical Opinion
Freedman, L. S., Lowe, D., & Macaskill, P. (1984) · Biometrics · doi:10.2307/2530902 · Cited by 87
Self-citationA Bayesian Approach to Bioequivalence for the 2 x 2 Changeover Design
Selwyn, M. R., Dempster, A. P., & Hall, N. (1981) · Biometrics · doi:10.2307/2530518 · Cited by 83
Statistical controversies in the design of clinical trials—Some personal views
Brown, B. W. (1980) · Controlled Clinical Trials · doi:10.1016/s0197-2456(80)80004-3 · Cited by 80
On choosing the number of interim analyses in clinical trials
McPherson, K. (1982) · Statistics in Medicine · doi:10.1002/sim.4780010105 · Cited by 67
Terminating a trial—the ethical problem
Meier, P. (1979) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt1979255part2633 · Cited by 60
Randomised Controlled Clinical Trials
Bulpitt, C. J. (1983) · Springer eBooks · doi:10.1007/978-1-4757-6358-4 · Cited by 47
A Bayesian approach to the interpretation of subgroup results in clinical trials
Donner, A. P. (1982) · Journal of Chronic Diseases · doi:10.1016/0021-9681(82)90057-1 · Cited by 41
Controversies and achievements in clinical trials
Armitage, P. (1984) · Controlled Clinical Trials · doi:10.1016/0197-2456(84)90151-x · Cited by 38
Statistics in question. Assessing clinical trials--trial size.
Gore, S. M. (1981) · BMJ · doi:10.1136/bmj.282.6277.1687 · Cited by 38
Decision-Analytic Determination of Study Size
Thompson, M. S. (1981) · Medical Decision Making · doi:10.1177/0272989x8100100206 · Cited by 33
Stopping rules for clinical trials
Freedman, L. S., Lowe, D., & Macaskill, P. (1983) · Statistics in Medicine · doi:10.1002/sim.4780020210 · Cited by 25
Self-citation
Cited by
Bayesian approaches to randomized trials
Spiegelhalter, D. J., Freedman, L. S., & Parmar, M. K. B. (1994) · Journal of the Royal Statistical Society. Series A (Statistics in Society) · doi:10.2307/2983527 · Cited by 720
Self-citationGoodman, S. N., & Berlin, J. A. (1994) · Annals of Internal Medicine · doi:10.7326/0003-4819-121-3-199408010-00008 · Cited by 654
Noninvasive ventilation to prevent respiratory failure after extubation in high-risk patients*
Nava, S., Gregoretti, C., Fanfulla, F., Squadrone, E., Grassi, M., Carlucci, A., Beltrame, F., & Navalesi, P. (2005) · Critical Care Medicine · doi:10.1097/01.ccm.0000186416.44752.72 · Cited by 552
Probabilistic prediction in patient management and clinical trials
Spiegelhalter, D. J. (1986) · Statistics in Medicine · doi:10.1002/sim.4780050506 · Cited by 358
Self-citationMonitoring clinical trials: Conditional or predictive power?
Spiegelhalter, D. J., Freedman, L. S., & Blackburn, P. R. (1986) · Controlled Clinical Trials · doi:10.1016/0197-2456(86)90003-6 · Cited by 307
Self-citation
Metadata
- Authors
- Spiegelhalter, D. J., & Freedman, L. S.
- Venue
- Statistics in Medicine
- Publisher
- Wiley
- Published
- 1986-01
- Volume
- 5
- Issue
- 1
- Pages
- 1-13
- ISSN
- 0277-6715 (ISSN-L)
- 1097-0258
- OpenAlex
- W1971051509
- Cited by
- 276
- References
- 40
- FWCI
- 9.84
- Type
- journal article
- Language
- en
- Open access
- closed
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
