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Meta-analysis is routinely performed in many scientific disciplines. This analysis is attractive since discoveries are possible even when all the individual studies are underpowered. However, the meta-analytic discoveries may be entirely…

Methodology · Statistics 2023-05-09 Marina Bogomolov , Ruth Heller

The principle of allocating an equal number of patients to each arm in a randomized controlled trial remains widely believed to be optimal for maximising statistical power. However, this long-held belief only holds true if the treatment…

Methodology · Statistics 2025-10-15 Lukas Pin , Stef Baas , David S. Robertson , Sofía S. Villar

In experimental design, aliasing of effects occurs in fractional factorial experiments, where certain low order factorial effects are indistinguishable from certain high order interactions: low order contrasts may be orthogonal to one…

Methodology · Statistics 2024-08-30 Paul R. Rosenbaum , Jose R. Zubizarreta

Like it or not, attempts to evaluate and monitor the quality of academic research have become increasingly prevalent worldwide. Performance reviews range from at the level of individuals, through research groups and departments, to entire…

Physics and Society · Physics 2017-03-31 R. Kenna , O. Mryglod , B. Berche

Clinical trials usually target average treatment effects, but treatment decisions are made for individuals. This tension motivates a common criticism of evidence-based medicine: a treatment that is beneficial on average may be inappropriate…

Applications · Statistics 2026-05-29 Zach Shahn , Mats Stensrud

Determining and measuring cause-effect relationships is fundamental to most scientific studies of natural phenomena. The notion of causation is distinctly different from correlation which only looks at association of trends or patterns in…

Methodology · Statistics 2019-10-22 Aditi Kathpalia , Nithin Nagaraj

Meta-analysis is commonly used to combine results from multiple clinical trials, but traditional meta-analysis methods do not refer explicitly to a population of individuals to whom the results apply and it is not clear how to use their…

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

Methodology · Statistics 2022-11-10 Wenqi Shi , Xi Lin

In this review, we present econometric and statistical methods for analyzing randomized experiments. For basic experiments we stress randomization-based inference as opposed to sampling-based inference. In randomization-based inference,…

Methodology · Statistics 2017-10-26 Susan Athey , Guido Imbens

Sample size calculation is crucial in biomedical in vivo research investigations mainly for two reasons: to design the most resource-efficient studies and to safeguard ethical issues when alive animals are subjects of testing. In this…

Applications · Statistics 2025-05-27 Hasan Al-Nashash , Jiajin Wei , Ke Yang , Ayman Alzaatreh , Mohsen Adeli , Tiejun Tong , Angelo All

Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…

Machine Learning · Computer Science 2023-12-06 Yash Chandak , Shiv Shankar , Vasilis Syrgkanis , Emma Brunskill

Many biomedical experiments are carried out by pooling individual biological samples. However, pooling samples can potentially hide biological variance and give false confidence concerning the data significance. In the context of microarray…

Quantitative Methods · Quantitative Biology 2008-06-02 Shu-Dong Zhang , Timothy W. Gant

There has been significant attention given to developing data-driven methods for tailoring patient care based on individual patient characteristics. Dynamic treatment regimes formalize this through a sequence of decision rules that map…

Methodology · Statistics 2022-02-22 Eric J. Rose , Erica E. M. Moodie , Susan Shortreed

Predictions under interventions are estimates of what a person's risk of an outcome would be if they were to follow a particular treatment strategy, given their individual characteristics. Such predictions can give important input to…

Methodology · Statistics 2025-06-17 Ruth H. Keogh , Nan van Geloven

Replication is complicated in psychological research because studies of a given psychological phenomenon can never be direct or exact replications of one another, and thus effect sizes vary from one study of the phenomenon to the next--an…

Methodology · Statistics 2021-07-21 Blakeley B. McShane , Jennifer L. Tackett , Ulf Bockenholt , Andrew Gelman

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

Methodology · Statistics 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Higher Education Institutions in the UK and elsewhere are under increasing pressure to measure the impact of their research, which can include how the research has increased scientific engagement amongst the general public. For various…

Physics and Society · Physics 2021-03-12 Chris D. White , Anthony Phillips , Beltran Sajonia-Coburgo-Gotha

Incentives in experimental design are often misaligned: experimenters design and finance experiments to seek regulatory approval, while regulators seek to maximize social-welfare. We propose a framework to resolve this conflict, wherein…

Econometrics · Economics 2026-05-19 Karun Adusumilli , Abhi Vemulapati

Sample size derivation is a crucial element of the planning phase of any confirmatory trial. A sample size is typically derived based on constraints on the maximal acceptable type I error rate and a minimal desired power. Here, power…

Typically, a randomized experiment is designed to test a hypothesis about the average treatment effect and sometimes hypotheses about treatment effect variation. The results of such a study may then be used to inform policy and practice for…

Methodology · Statistics 2026-05-01 Elizabeth Tipton , Michalis Mamakos
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