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Methodology for sample size calculation for phase III clinical trials is well established and widely used. In contrast, for earlier phase clinical trials or pilot studies, although there is an acceptance that the methods used for phase III trials are not appropriate, there is little consensus over methods that should be used. This paper explores this problem from a Bayesian decision-theoretic perspective. The aim is to obtain sample sizes that would be appropriate for studies funded by a large funder such as a public sector body or major pharmaceutical company. The sample sizes obtained are optimal in that they minimise the average number of patients required per successfully identified effective therapy or equivalently maximise the number of effective therapies successfully identified over a long period. It is indicated that the number of patients included in a phase II clinical trial should be approximately 0.03 times that planned to be included in the phase III study. This is similar to that proposed by other researchers in this area, though rather smaller than actually used for many phase II trials. Copyright © 2011 John Wiley & Sons, Ltd.
Warwick Medical School, University of Warwick, UK. firstname.lastname@example.org.
This article was published in the following journal.
Name: Statistics in medicine
Designing studies with an internal pilot phase may optimise the use of pilot work to inform more efficient randomised controlled trials (RCTs). Careful selection of preagreed decision or 'progression'...
Importance Use of expansion cohorts (EC) in phase I trials is increasing. However, the utility of phase I EC in aiding drug development is unclear. We sought to determine factors associated with the i...
Enhanced knowledge of the biological and genetic basis of cancer is re-defining target population for new treatments. In oncology, potential targets for a new therapeutic agent often include various s...
Prior phase III trials in advanced pancreatic cancer have been predominantly unsuccessful. In this review, we attempt to understand how past preclinical data were translated into phase III clinical tr...
This is the introductory paper in a series of 8 papers (see box). In this series we integrate the theoretical design options with the practice of conducting pragmatic trials. For most new market-appro...
Sample size definition provides important information, allowing the groundwork for transparent reporting. The sample predefinition allows the trial to be large enough to be able to addres...
The aim of the following pilot study is to examine boredom in a community sample of adolescents and a clinical sample of adolescents. This is important in order to examine differences amon...
This study aims to evaluate the effectiveness of a psychotherapeutic intervention model in nursing for the nursing diagnosis "Anxiety". A pilot randomised controlled trial will be carried ...
Background: In genetic studies, the quality of DNA samples is tested first. Samples that are low-quality are not used. Some studies involve minority ethnic groups. And example is admixed A...
Pilot study for assessing the effectiveness of a Navigator program to aid in clinical trial participation amongst the Chinese demographic
Comparative studies to verify the effectiveness of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques determined in phase II studies. During these trials, patients are monitored closely by physicians to identify any adverse reactions from long-term use. These studies are performed on groups of patients large enough to identify clinically significant responses and usually last about three years. This concept includes phase III studies conducted in both the U.S. and in other countries.
Studies that are usually controlled to assess the effectiveness and dosage (if appropriate) of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques. These studies are performed on several hundred volunteers, including a limited number of patients with the target disease or disorder, and last about two years. This concept includes phase II studies conducted in both the U.S. and in other countries.
Planned post-marketing studies of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques that have been approved for general sale. These studies are often conducted to obtain additional data about the safety and efficacy of a product. This concept includes phase IV studies conducted in both the U.S. and in other countries.
Works about research studies that evaluate interventions or exposures on biomedical or health-related outcomes. The two main types of clinical studies are interventional studies (clinical trials) and observational studies.
A work that reports on the results of a research study to evaluate interventions or exposures on biomedical or health-related outcomes. The two main types of clinical studies are interventional studies (clinical trials) and observational studies. While most clinical studies concern humans, this publication type may be used for clinical veterinary articles meeting the requisites for humans.
In a clinical trial or interventional study, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or change...