Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design
No inferioridad; Fase II; Brazo único
| Autores: | , , , , , , |
|---|---|
| Formato: | artículo |
| Estado: | Versión publicada |
| Fecha de publicación: | 2020 |
| País: | España |
| Recursos: | Departament de Salut de la Generalitat de Catalunya (DS) |
| Repositorio: | Scientia. Dipòsit d'Informació Digital del Departament de Salut |
| OAI Identifier: | oai:scientiasalut.gencat.cat:11351/6486 |
| Acesso em linha: | https://hdl.handle.net/11351/6486 |
| Access Level: | acceso abierto |
| Palavra-chave: | Càncer - Tractament Medicina - Investigació Assaigs clínics ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics::Clinical Studies as Topic::Clinical Trials as Topic::Clinical Trials, Phase II as Topic Other subheadings::Other subheadings::/methods ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Methods::Research Design DISEASES::Neoplasms TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::características de los estudios epidemiológicos::estudios clínicos como asunto::ensayos clínicos como asunto::ensayos clínicos en fase II como asunto Otros calificadores::Otros calificadores::/métodos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos::diseño de la investigación ENFERMEDADES::neoplasias |
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| dc.title.none.fl_str_mv |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design |
| title |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design |
| spellingShingle |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design Miguel-Huguet, Bernat Càncer - Tractament Medicina - Investigació Assaigs clínics ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics::Clinical Studies as Topic::Clinical Trials as Topic::Clinical Trials, Phase II as Topic Other subheadings::Other subheadings::/methods ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Methods::Research Design DISEASES::Neoplasms TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::características de los estudios epidemiológicos::estudios clínicos como asunto::ensayos clínicos como asunto::ensayos clínicos en fase II como asunto Otros calificadores::Otros calificadores::/métodos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos::diseño de la investigación ENFERMEDADES::neoplasias |
| title_short |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design |
| title_full |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design |
| title_fullStr |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design |
| title_full_unstemmed |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design |
| title_sort |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design |
| dc.creator.none.fl_str_mv |
Miguel-Huguet, Bernat Perez-Garcia, Jose Manuel Páez, David Guerrero‑Zotano, Angel Cortés Castan, Javier Sampayo-Cordero, Miguel Garde-Noguera, Javier |
| author |
Miguel-Huguet, Bernat |
| author_facet |
Miguel-Huguet, Bernat Perez-Garcia, Jose Manuel Páez, David Guerrero‑Zotano, Angel Cortés Castan, Javier Sampayo-Cordero, Miguel Garde-Noguera, Javier |
| author_role |
author |
| author2 |
Perez-Garcia, Jose Manuel Páez, David Guerrero‑Zotano, Angel Cortés Castan, Javier Sampayo-Cordero, Miguel Garde-Noguera, Javier |
| author2_role |
author author author author author author |
| dc.contributor.none.fl_str_mv |
Institut Català de la Salut [Sampayo-Cordero M] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. Medica Scientia Innovation Research (MedSIR), Ridgewood, NJ, USA. [Miguel-Huguet B] Hospital Universitari de Bellvitge, L’Hospitalet, Barcelona, Spain. [Pérez-García J] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. IOB, Institute of Oncology, QuironSalud Group, Barcelona and Madrid, Spain. [Páez D] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. Hospital de la Santa Creu i Sant Pau, Barcelona, Spain. Universitat Autònoma de Barcelona (UAB), Bellaterra, Spain. [Guerrero-Zotano ÁL] Vanderbilt-Ingram Cancer Center; Vanderbilt University Medical Center, Nashville, TN, USA. [Garde-Noguera J] Hospital Arnau de Vilanova, Valencia, Spain. [Cortés J] IOB, Institute of Oncology, QuironSalud Group, Barcelona and Madrid, Spain. Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain. Medica Scientia Innovation Research (MedSIR), Barcelona, Spain Vall d'Hebron Barcelona Hospital Campus |
| dc.subject.none.fl_str_mv |
Càncer - Tractament Medicina - Investigació Assaigs clínics ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics::Clinical Studies as Topic::Clinical Trials as Topic::Clinical Trials, Phase II as Topic Other subheadings::Other subheadings::/methods ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Methods::Research Design DISEASES::Neoplasms TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::características de los estudios epidemiológicos::estudios clínicos como asunto::ensayos clínicos como asunto::ensayos clínicos en fase II como asunto Otros calificadores::Otros calificadores::/métodos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos::diseño de la investigación ENFERMEDADES::neoplasias |
| topic |
Càncer - Tractament Medicina - Investigació Assaigs clínics ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics::Clinical Studies as Topic::Clinical Trials as Topic::Clinical Trials, Phase II as Topic Other subheadings::Other subheadings::/methods ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Methods::Research Design DISEASES::Neoplasms TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::características de los estudios epidemiológicos::estudios clínicos como asunto::ensayos clínicos como asunto::ensayos clínicos en fase II como asunto Otros calificadores::Otros calificadores::/métodos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos::diseño de la investigación ENFERMEDADES::neoplasias |
| description |
No inferioridad; Fase II; Brazo único |
| publishDate |
2020 |
| dc.date.none.fl_str_mv |
2020 2021 2021 |
| dc.type.none.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion |
| format |
article |
| status_str |
publishedVersion |
| dc.identifier.none.fl_str_mv |
https://hdl.handle.net/11351/6486 |
| url |
https://hdl.handle.net/11351/6486 |
| dc.language.none.fl_str_mv |
Inglés |
| language_invalid_str_mv |
Inglés |
| dc.relation.none.fl_str_mv |
Contemporary Clinical Trials Communications;20 https://doi.org/10.1016/j.conctc.2020.100678 |
| dc.rights.none.fl_str_mv |
Attribution 4.0 International http://creativecommons.org/licenses/by/4.0/ info:eu-repo/semantics/openAccess |
| rights_invalid_str_mv |
Attribution 4.0 International http://creativecommons.org/licenses/by/4.0/ |
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openAccess |
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application/pdf application/pdf application/pdf |
| dc.publisher.none.fl_str_mv |
Elsevier |
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Elsevier |
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Scientia reponame:Scientia. Dipòsit d'Informació Digital del Departament de Salut instname:Departament de Salut de la Generalitat de Catalunya (DS) |
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Departament de Salut de la Generalitat de Catalunya (DS) |
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Scientia. Dipòsit d'Informació Digital del Departament de Salut |
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Scientia. Dipòsit d'Informació Digital del Departament de Salut |
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| _version_ |
1869424512413466624 |
| spelling |
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's designMiguel-Huguet, BernatPerez-Garcia, Jose ManuelPáez, DavidGuerrero‑Zotano, AngelCortés Castan, JavierSampayo-Cordero, MiguelGarde-Noguera, JavierCàncer - TractamentMedicina - InvestigacióAssaigs clínicsANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics::Clinical Studies as Topic::Clinical Trials as Topic::Clinical Trials, Phase II as TopicOther subheadings::Other subheadings::/methodsANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Methods::Research DesignDISEASES::NeoplasmsTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::características de los estudios epidemiológicos::estudios clínicos como asunto::ensayos clínicos como asunto::ensayos clínicos en fase II como asuntoOtros calificadores::Otros calificadores::/métodosTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos::diseño de la investigaciónENFERMEDADES::neoplasiasNo inferioridad; Fase II; Brazo únicoNo inferioritat; Fase II; Braç únicNon-inferiority; Phase II; Single-armIntroduction: Non-inferiority (NI) analysis is not usually considered in the early phases of clinical development. In some negative phase II trials, a post-hoc NI analysis justified additional phase III trials that were successful. However, the risk of false positive achievements was not controlled in these early phase analyses. We propose to preplan NI analyses in superiority-based Simon’s two-stage designs to control type I and II error rates. Methods: Simulations have been proposed to assess the control of type I and II errors rates with this method. A total of 12,768 two-stage Simon’s design trials were constructed based on different assumptions of rejection response probability, desired response probability, type I and II errors, and NI margins. P-value and type II error were calculated with stochastic ordering using Uniformly Minimum Variance Unbiased Estimator. Type I and II errors were simulated using the Monte Carlo method. The agreement between calculated and simulated values was analyzed with Bland-Altman plots. Results: We observed the same level of agreement between calculated and simulated type I and II errors from both two-stage Simon’s superiority designs and designs in which NI analysis was allowed. Different examples has been proposed to explain the utility of this method. Conclusion: Inclusion of NI analysis in superiority-based single-arm clinical trials may be useful for weighing additional factors such as safety, pharmacokinetics, pharmacodynamic, and biomarker data while assessing early efficacy. Implementation of this strategy can be achieved through simple adaptations to existing designs for onearm phase II clinical trialsJavier Cortés has received consulting and advisor fees from: Roche, Celgene, Cellestia, AstraZeneca, Biothera Pharmaceutical, Merus, Seattle Genetics, Daiichi Sankyo and Erytech. In addition, Javier Cortés has received honorarias from: Roche, Novartis, Celgene, Eisai, Pfizer and Samsung. Add more, Javier Cortés has received research funding fees to the institution from Roche. Finally, Javier Cortés has stock options, patents and intellectual property from MedSIR.ElsevierInstitut Català de la Salut[Sampayo-Cordero M] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. Medica Scientia Innovation Research (MedSIR), Ridgewood, NJ, USA. [Miguel-Huguet B] Hospital Universitari de Bellvitge, L’Hospitalet, Barcelona, Spain. [Pérez-García J] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. IOB, Institute of Oncology, QuironSalud Group, Barcelona and Madrid, Spain. [Páez D] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. Hospital de la Santa Creu i Sant Pau, Barcelona, Spain. Universitat Autònoma de Barcelona (UAB), Bellaterra, Spain. [Guerrero-Zotano ÁL] Vanderbilt-Ingram Cancer Center; Vanderbilt University Medical Center, Nashville, TN, USA. [Garde-Noguera J] Hospital Arnau de Vilanova, Valencia, Spain. [Cortés J] IOB, Institute of Oncology, QuironSalud Group, Barcelona and Madrid, Spain. Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain. Medica Scientia Innovation Research (MedSIR), Barcelona, SpainVall d'Hebron Barcelona Hospital Campus202120212020info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfapplication/pdfapplication/pdfhttps://hdl.handle.net/11351/6486Scientiareponame:Scientia. Dipòsit d'Informació Digital del Departament de Salutinstname:Departament de Salut de la Generalitat de Catalunya (DS)InglésContemporary Clinical Trials Communications;20https://doi.org/10.1016/j.conctc.2020.100678Attribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:scientiasalut.gencat.cat:11351/64862026-06-12T09:38:37Z |
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15,811543 |