Optimizing expert and patient input in pediatric trial design

Detalhes bibliográficos
Autor(a) principal: Dhaenens, Britt A.E.
Data de Publicação: 2023
Outros Autores: Mahler, Fenna, Batchelor, Hannah, Dicks, Pamela, Gaillard, Segolene, Nafria, Begonya, Kopp-Schneider, Annette, Ribeiro, Maria Alexandra, Schwab, Matthias, Sparber-Sauer, Monika, Leubner, Jonas, de Wildt, Saskia N., Oostenbrink, Rianne
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10362/155491
Resumo: Funding Information: The conect4children (c4c) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement no. 777389. The Joint Undertaking receives support from the European Union's Horizon 2020 Research and Innovation Program and EFPIA. The publication reflects the authors' views and neither IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained therein. EU‐PEARL has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement no. 853966. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation program and EFPIA and Children's Tumor Foundation, Global Alliance for TB Drug Development Non‐profit Organization, and Springworks Therapeutics Inc. This publication reflects the authors' views. Neither the IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained herein. M.S. was supported in part by the Robert Bosch Stiftung, Stuttgart, Germany. Publisher Copyright: © 2023 The Authors. Clinical and Translational Science published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.
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spelling Optimizing expert and patient input in pediatric trial designLessons learned and recommendations from a collaboration between conect4children and European Patient-CEntric ClinicAl TRial PLatformsNeuroscience(all)Biochemistry, Genetics and Molecular Biology(all)Pharmacology, Toxicology and Pharmaceutics(all)Funding Information: The conect4children (c4c) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement no. 777389. The Joint Undertaking receives support from the European Union's Horizon 2020 Research and Innovation Program and EFPIA. The publication reflects the authors' views and neither IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained therein. EU‐PEARL has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement no. 853966. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation program and EFPIA and Children's Tumor Foundation, Global Alliance for TB Drug Development Non‐profit Organization, and Springworks Therapeutics Inc. This publication reflects the authors' views. Neither the IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained herein. M.S. was supported in part by the Robert Bosch Stiftung, Stuttgart, Germany. Publisher Copyright: © 2023 The Authors. Clinical and Translational Science published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.Advice from multiple stakeholders is required to design the optimal pediatric clinical trial. We present recommendations for acquiring advice from trial experts and patients/caregivers, derived from advice meetings that were performed through a collaboration of the Collaborative Network for European Clinical Trials for Children (c4c) and the European Patient-CEntric ClinicAl TRial PLatforms (EU-PEARL). Three advice meetings were performed: (1) an advice meeting for clinical and methodology experts, (2) an advice meeting for patients/caregivers, and (3) a combined meeting with both experts and patients/caregivers. Trial experts were recruited from c4c database. Patients/caregivers were recruited through a patient organization. Participants were asked to provide input on a trial protocol, including endpoints, outcomes, and the assessment schedule. Ten experts, 10 patients, and 13 caregivers participated. The advice meetings resulted in modification of eligibility criteria and outcome measures. We have provided recommendations for the most effective meeting type per protocol topic. Topics with limited options for patient input were most efficiently discussed in expert advice meetings. Other topics benefit from patient/caregiver input, either through a combined meeting with experts or a patients/caregivers-only advice meeting. Some topics, such as endpoints and outcome measures, are suitable for all meeting types. Combined sessions profit from synergy between experts and patients/caregivers, balancing input on protocol scientific feasibility and acceptability. Both experts and patients/caregivers provided critical input on the presented protocol. The combined meeting was the most effective methodology for most protocol topics. The presented methodology can be used effectively to acquire expert and patient feedback.NOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)RUNDhaenens, Britt A.E.Mahler, FennaBatchelor, HannahDicks, PamelaGaillard, SegoleneNafria, BegonyaKopp-Schneider, AnnetteRibeiro, Maria AlexandraSchwab, MatthiasSparber-Sauer, MonikaLeubner, Jonasde Wildt, Saskia N.Oostenbrink, Rianne2023-07-18T22:16:01Z20232023-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10362/155491eng1752-8054PURE: 66652176https://doi.org/10.1111/cts.13547info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2024-03-11T05:38:05Zoai:run.unl.pt:10362/155491Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T03:56:05.645383Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Optimizing expert and patient input in pediatric trial design
Lessons learned and recommendations from a collaboration between conect4children and European Patient-CEntric ClinicAl TRial PLatforms
title Optimizing expert and patient input in pediatric trial design
spellingShingle Optimizing expert and patient input in pediatric trial design
Dhaenens, Britt A.E.
Neuroscience(all)
Biochemistry, Genetics and Molecular Biology(all)
Pharmacology, Toxicology and Pharmaceutics(all)
title_short Optimizing expert and patient input in pediatric trial design
title_full Optimizing expert and patient input in pediatric trial design
title_fullStr Optimizing expert and patient input in pediatric trial design
title_full_unstemmed Optimizing expert and patient input in pediatric trial design
title_sort Optimizing expert and patient input in pediatric trial design
author Dhaenens, Britt A.E.
author_facet Dhaenens, Britt A.E.
Mahler, Fenna
Batchelor, Hannah
Dicks, Pamela
Gaillard, Segolene
Nafria, Begonya
Kopp-Schneider, Annette
Ribeiro, Maria Alexandra
Schwab, Matthias
Sparber-Sauer, Monika
Leubner, Jonas
de Wildt, Saskia N.
Oostenbrink, Rianne
author_role author
author2 Mahler, Fenna
Batchelor, Hannah
Dicks, Pamela
Gaillard, Segolene
Nafria, Begonya
Kopp-Schneider, Annette
Ribeiro, Maria Alexandra
Schwab, Matthias
Sparber-Sauer, Monika
Leubner, Jonas
de Wildt, Saskia N.
Oostenbrink, Rianne
author2_role author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv NOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)
RUN
dc.contributor.author.fl_str_mv Dhaenens, Britt A.E.
Mahler, Fenna
Batchelor, Hannah
Dicks, Pamela
Gaillard, Segolene
Nafria, Begonya
Kopp-Schneider, Annette
Ribeiro, Maria Alexandra
Schwab, Matthias
Sparber-Sauer, Monika
Leubner, Jonas
de Wildt, Saskia N.
Oostenbrink, Rianne
dc.subject.por.fl_str_mv Neuroscience(all)
Biochemistry, Genetics and Molecular Biology(all)
Pharmacology, Toxicology and Pharmaceutics(all)
topic Neuroscience(all)
Biochemistry, Genetics and Molecular Biology(all)
Pharmacology, Toxicology and Pharmaceutics(all)
description Funding Information: The conect4children (c4c) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement no. 777389. The Joint Undertaking receives support from the European Union's Horizon 2020 Research and Innovation Program and EFPIA. The publication reflects the authors' views and neither IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained therein. EU‐PEARL has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement no. 853966. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation program and EFPIA and Children's Tumor Foundation, Global Alliance for TB Drug Development Non‐profit Organization, and Springworks Therapeutics Inc. This publication reflects the authors' views. Neither the IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained herein. M.S. was supported in part by the Robert Bosch Stiftung, Stuttgart, Germany. Publisher Copyright: © 2023 The Authors. Clinical and Translational Science published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.
publishDate 2023
dc.date.none.fl_str_mv 2023-07-18T22:16:01Z
2023
2023-01-01T00:00:00Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10362/155491
url http://hdl.handle.net/10362/155491
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 1752-8054
PURE: 66652176
https://doi.org/10.1111/cts.13547
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron:RCAAP
instname_str Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
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reponame_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
collection Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository.name.fl_str_mv Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
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