An aid to data monitoring in long-term clinical trials

Halperin, Max; Lan, K. K. Gordon; Ware, James H.; Johnson, Norman J.; DeMets, David L.

doi:10.1016/0197-2456(82)90022-8

Cited by 179 publications

(55 citation statements)

References 7 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In 1982 Lan et al [63] and Halperin et al [64] proposed the idea of stochastic curtailment. The idea here is to stop a trial as soon as its outcome is determined with high probability.…”

Section: Other Approachesmentioning

confidence: 99%

A 25‐year review of sequential methodology in clinical studies

Todd

2006

Statistics in Medicine

View full text Add to dashboard Cite

SUMMARYThis paper explores the theoretical developments and subsequent uptake of sequential methodology in clinical studies in the 25 years since Statistics in Medicine was launched. The review examines the contributions which have been made to all four phases into which clinical trials are traditionally classified and highlights major statistical advancements, together with assessing application of the techniques. The vast majority of work has been in the setting of phase III clinical trials and so emphasis will be placed here. Finally, comments are given indicating how the subject area may develop in the future.

show abstract

“…In 1982 Lan et al [63] and Halperin et al [64] proposed the idea of stochastic curtailment. The idea here is to stop a trial as soon as its outcome is determined with high probability.…”

Section: Other Approachesmentioning

confidence: 99%

A 25‐year review of sequential methodology in clinical studies

Todd

2006

Statistics in Medicine

View full text Add to dashboard Cite

show abstract

“…Therefore, 2 * estimated by conditional power criteria may be variable. (3) Bayesian predictive power criteria with a non-informative prior Spiegelhalter, Freedman, and Blackburn (1986) proposed a Bayesian predictive power approach for clinical trial monitoring as an alternative to the conditional power approach proposed by Halperin et al (1982). Predictive power can be considered an unconditional prediction of conditional power that can take into account the uncertainty of the conditional power approach, which is assessed by two points of the hypothesis 2 = 0, .…”

Section: Sample Size Re-estimation Criteriamentioning

confidence: 99%

Utility of Adaptive Sample Size Designs and A Review Example

Uemura¹,

Ando²,

Matsuyama³

2017

JSSA

View full text Add to dashboard Cite

Sample size can be a key design feature that not only affects the probability of a trial's success but also determines the duration and feasibility of a trial. If an investigational drug is expected to be effective and address unmet medical needs of an orphan disease, where the accrual period may require many years with a large sample size to detect a minimal clinically relevant treatment effect, a minimum sample size may be set to maintain nominal power.In limited situations such as this, there may be a need for flexibility in the initial and final sample sizes; thus, it is useful to consider the utility of adaptive sample size designs that use sample size re-estimation or group sequential design. In this paper, we propose a new adaptive performance measure to consider the utility of an adaptive sample size design in a trial simulation. Considering that previously proposed sample size re-estimation methods do not take into account errors in estimation based on interim results, we propose Bayesian sample size re-estimation criteria that take into account prior information on treatment effect, and then, we assess its operating characteristics in a simulation study. We also present a review example of sample size re-estimation mainly based on published paper and review report in Pharmaceuticals and Medical Devices Agency (PMDA).

show abstract

“…Such decisions are based on the appearance of unantici pated and unacceptable side effects (121), and on the appearance of a presumed winner in a treatment comparison (61). As in other areas of clinical research, ethical, scientifi c, and statistical controversies accompany this early stopping.…”

Section: Stoppingmentioning

confidence: 99%