Advantages of a wholly Bayesian approach to assessing efficacy in early drug development: a case study
| Autor: | Jeremy D. Gale, Claire Smith, Phil Woodward, Rosalind Walley |
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| Rok vydání: | 2014 |
| Předmět: |
Pharmacology
Statistics and Probability Models Statistical Relation (database) Clinical Trials Phase I as Topic Computer science Bayesian probability Statistics as Topic Bayes Theorem Bayesian inference Interim analysis Multiple-criteria decision analysis Placebo Effect Resource (project management) Treatment Outcome Sample size determination Research Design Creatinine Clinical endpoint Econometrics Albuminuria Humans Pharmacology (medical) Renal Insufficiency Chronic Probability |
| Zdroj: | Pharmaceutical statistics. 14(3) |
| ISSN: | 1539-1612 |
| Popis: | 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. Copyright © 2015 John Wiley & Sons, Ltd. |
| Databáze: | OpenAIRE |
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