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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. email@example.com.
This article was published in the following journal.
Name: Statistics in medicine
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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.
Studies performed to evaluate the safety of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques in healthy subjects and to determine the safe dosage range (if appropriate). These tests also are used to determine pharmacologic and pharmacokinetic properties (toxicity, metabolism, absorption, elimination, and preferred route of administration). They involve a small number of persons and usually last about 1 year. This concept includes phase I studies conducted both in the U.S. and in other countries.
Human experimentation that is not intended to benefit the subjects on whom it is performed. Phase I drug studies (CLINICAL TRIALS, PHASE I AS TOPIC) and research involving healthy volunteers are examples of nontherapeutic human experimentation.
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