The topic of this article is pre-posterior distributions of success or failure. These distributions, determined before a study is run and based on all our assumptions, are what we should believe about the treatment effect if we are told only that the study has been successful, or unsuccessful. I show how the pre-posterior distributions of success and failure can be used during the planning phase of a study to investigate whether the study is able to discriminate between effective and ineffective treatments. I show how these distributions are linked to the probability of success (PoS), or failure, and how they can be determined from simulations if standard asymptotic normality assumptions are inappropriate. I show the link to the concept of the conditional introduced by Temple and Robertson in the context of the planning of multiple studies. Finally, I show that they can also be constructed regardless of whether the analysis of the study is frequentist or fully Bayesian.
Download full-text PDF |
Source |
---|---|
http://dx.doi.org/10.1002/pst.2450 | DOI Listing |
Pharm Stat
November 2024
Fairholme, Upper Street, Tilmanstone, Kent, UK.
The topic of this article is pre-posterior distributions of success or failure. These distributions, determined before a study is run and based on all our assumptions, are what we should believe about the treatment effect if we are told only that the study has been successful, or unsuccessful. I show how the pre-posterior distributions of success and failure can be used during the planning phase of a study to investigate whether the study is able to discriminate between effective and ineffective treatments.
View Article and Find Full Text PDFPharm Stat
July 2021
Statistical Sciences and Innovation, UCB Pharma, Slough, UK.
For any decision-making study, there are two sorts of errors that can be made, declaring a positive result when the truth is negative, and declaring a negative result when the truth is positive. Traditionally, the primary analysis of a study is a two-sided hypothesis test, the type I error rate will be set to 5% and the study is designed to give suitably low type II error - typically 10 or 20% - to detect a given effect size. These values are standard, arbitrary and, other than the choice between 10 and 20%, do not reflect the context of the study, such as the relative costs of making type I and II errors and the prior belief the drug will be placebo-like.
View Article and Find Full Text PDFPharm Stat
April 2016
Pfizer, Neusentis, The Portway Building, Granta Park, Cambridge, CB21 6GS, UK.
This paper illustrates how the design and statistical analysis of the primary endpoint of a proof-of-concept study can be formulated within a Bayesian framework and is motivated by and illustrated with a Pfizer case study in chronic kidney disease. It is shown how decision criteria for success can be formulated, and how the study design can be assessed in relation to these, both using the traditional approach of probability of success conditional on the true treatment difference and also using Bayesian assurance and pre-posterior probabilities. The case study illustrates how an informative prior on placebo response can have a dramatic effect in reducing sample size, saving time and resource, and we argue that in some cases, it can be considered unethical not to include relevant literature data in this way.
View Article and Find Full Text PDFJ Biopharm Stat
September 2012
School of Pharmacy, University of Otago, Dunedin, New Zealand.
Information theoretic methods are often used to design studies that aim to learn about pharmacokinetic and linked pharmacokinetic-pharmacodynamic systems. These design techniques, such as D-optimality, provide the optimum experimental conditions. The performance of the optimum design will depend on the ability of the investigator to comply with the proposed study conditions.
View Article and Find Full Text PDFBiom J
February 2010
Department of Biostatistics, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
Simulation-based assessment is a popular and frequently necessary approach for evaluating statistical procedures. Sometimes overlooked is the ability to take advantage of underlying mathematical relations and we focus on this aspect. We show how to take advantage of large-sample theory when conducting a simulation using the analysis of genomic data as a motivating example.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!