儿科临床试验的未来——为儿童健康设定研究重点

The future of paediatric clinicaltrials – setting research priorities Technical report The future of paediatric clinical trials – setting research priorities for child health: technical report ISBN 978-92-4-011659-7 (electronic version)ISBN 978-92-4-011660-3 (print version) © World Health Organization 2025 Some rights reserved. This work is available under the Creative Commons Attribution-NonCommercial-ShareAlike 3.0 IGO licence (CC BY-NC-SA 3.0 IGO;https://creativecommons.org/licenses/by-nc-sa/3.0/igo). Under the terms of this licence, you may copy, redistribute and adapt the work for non-commercial purposes,provided the work is appropriately cited, as indicated below. In any use of this work, there should be nosuggestion that WHO endorses any specific organization, products or services. The use of the WHO logo isnot permitted. If you adapt the work, then you must license your work under the same or equivalent CreativeCommons licence. If you create a translation of this work, you should add the following disclaimer along with Any mediation relating to disputes arising under the licence shall be conducted in accordance with the mediationrules of the World Intellectual Property Organization (http://www.wipo.int/amc/en/mediation/rules/). Suggested citation. The future of paediatric clinical trials – setting research priorities for child health: technicalreport. Geneva: World Health Organization; 2025. Licence:CC BY-NC-SA 3.0 IGO. Cataloguing-in-Publication (CIP) data. CIP data are available athttps://iris.who.int/. Sales, rights and licensing. To purchase WHO publications, seehttps://www.who.int/publications/book-orders.To submit requests for commercial use and queries on rights and licensing, seehttps://www.who.int/copyright. Third-party materials. If you wish to reuse material from this work that is attributed to a third party, such astables, figures or images, it is your responsibility to determine whether permission is needed for that reuse andto obtain permission from the copyright holder. The risk of claims resulting from infringement of any third-party- General disclaimers. The designations employed and the presentation of the material in this publication do notimply the expression of any opinion whatsoever on the part of WHO concerning the legal status of any country,territory, city or area or of its authorities, or concerning the delimitation of its frontiers or boundaries. Dotted The mention of specific companies or of certain manufacturers’ products does not imply that they are endorsedor recommended by WHO in preference to others of a similar nature that are not mentioned. Errors and All reasonable precautions have been taken by WHO to verify the information contained in this publication.However, the published material is being distributed without warranty of any kind, either expressed or implied.The responsibility for the interpretation and use of the material lies with the reader. In no event shall WHO be Cover photo credits:© WHO / Loan Tran Design and layout by 400 Communications Limited. Contents Acknowledgements 1 2.What is the goal of thisresearchagenda?3 3.What is the scope of thisresearch agenda?4 5.Top research priorities overall and by thematic area8 References17 Annex 2.Ranked list of research questions22 Acknowledgements Witwatersrand, South Africa), Igor Rudan (University ofEdinburgh, United Kingdom), Fiona Russell (Universityof Melbourne, Australia), Judd Walson (Johns HopkinsUniversity, United States of America) and Jun Zhang The research priority-setting process and stakeholderconsultation on clinical trial priorities for children wereco-convened by the WHO Department of Research forHealth (Science Division) and Department of Maternal,Newborn, Child and Adolescent Health and Ageing.This technical report was drafted by Cadi Irvine (WHO WHO extends thanks to everyone who submittedresearch ideas and the members of the scoring group,who reviewed and assessed the final list. Special thanksgo to Igor Rudan (Centre for Global Health, University ofEdinburgh, United Kingdom) and Peige Song (School ofPublic Health, Zhejiang University School of Medicine,China) for analytical support, Cécile Claire Trottet WHO gratefully acknowledges the members of theexternal steering group for their support in designingand implementing the priority-setting exercise asfollows: Tahmeed Ahmed (International Centre forDiarrhoeal Disease Research, Bangladesh), GuillermoLuis Chantada (Hospital Sant Joan de Déu, Spain),Rebecca Grais (Pasteur Network, France), Fyezah 1.Background In November 2023, WHO convened the first GlobalClinical Trials Forum in Geneva(5)in response to aWorld Health Assembly resolution(6). The Forumbrought together more than 100 stakeholders –governments, researchers, funders, regulators andcommunities – who collectively recognized that thecurrent clinical research environment is not delivering Significant progress has been made in child andinfant health over r