Performance measures in dose-finding experiments
| dc.contributor.author | Flournoy, Nancy | |
| dc.contributor.author | Moler Cuiral, José Antonio | |
| dc.contributor.author | Plo, Fernando | |
| dc.contributor.department | Estadística, Informática y Matemáticas | es_ES |
| dc.contributor.department | Estatistika, Informatika eta Matematika | eu |
| dc.date.accessioned | 2020-07-03T12:54:10Z | |
| dc.date.available | 2020-07-03T12:54:10Z | |
| dc.date.issued | 2020 | |
| dc.description.abstract | In the first phase of pharmaceutical development, and assuming that the probability of positive response increases with dose, the main statistical goal is to estimate a percentile of the dose–response function for a given target Г. We compare the Maximum Likelihood and centred isotonic regression estimators of the target dose and we discuss several performance criteria to assess inferential precision, the amount of toxicity exposure and the trade-off between them for a set of some exemplary adaptive designs. We compare these designs using graphical tools. Several scenarios are considered using simulation, including the use of several start-up rules, the change of slope of the dose-toxicity function at the target dose and also different theoretical models, as logistic, normal or skew-normal distribution functions. | en |
| dc.description.sponsorship | Fernando Plo and Jose Moler acknowledge the financial support received from the projects Ministerio de Economía y Competitividad (MTM2014‐53340‐P) and Secretaría de Estado de Investigación, Desarrollo e Innovación (MTM2016‐77015‐R). | en |
| dc.format.extent | 24 p. | |
| dc.format.mimetype | application/pdf | en |
| dc.identifier.doi | 10.1111/insr.12363 | |
| dc.identifier.issn | 0306-7734 | |
| dc.identifier.uri | https://academica-e.unavarra.es/handle/2454/37296 | |
| dc.language.iso | eng | en |
| dc.publisher | John Wiley & Sons | en |
| dc.relation.ispartof | International Statistical Review, 2020, 1-24 | en |
| dc.relation.projectID | info:eu-repo/grantAgreement/MINECO//MTM2014-53340-P/ES/ | |
| dc.relation.projectID | info:eu-repo/grantAgreement/ES/1PE/MTM2016-77015-R/ | |
| dc.relation.publisherversion | https://doi.org/10.1111/insr.12363 | |
| dc.rights | © 2020 The Authors. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. | en |
| dc.rights.accessRights | info:eu-repo/semantics/openAccess | |
| dc.subject | Adaptive designs | en |
| dc.subject | Optimal designs | en |
| dc.subject | Maximum tolerated dose | en |
| dc.subject | Performance criteria | en |
| dc.title | Performance measures in dose-finding experiments | en |
| dc.type | info:eu-repo/semantics/article | |
| dc.type.version | info:eu-repo/semantics/publishedVersion | |
| dspace.entity.type | Publication | |
| relation.isAuthorOfPublication | ec10e36c-8a72-4ba6-b481-289a596f1c4a | |
| relation.isAuthorOfPublication.latestForDiscovery | ec10e36c-8a72-4ba6-b481-289a596f1c4a |