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Lookup NU author(s): Dr Gill NormanORCiD, Ross FairbairnORCiD, Chizoba OparahORCiD, Ayomikun OgunyemiORCiD, Professor Niina KolehmainenORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
Objective: To provide intelligence and foresight about persistent evidence gaps in medicines for children and young people experiencing gender incongruence or dysphoria. Design: A horizon scan, with foresight analysis, of clinical trial registries. Setting: Global Patients: Children and young people (0-17 years) experiencing gender incongruence or dysphoria. Interventions: Any medicine under evaluation in an ongoing study registered on a clinical trial registry. Main outcome measures: Any. Results: We identified fifteen ongoing studies across twenty-three registries, with total actual or planned sample size of n=1073. Twelve studies were based in the United States. The medicine classes under evaluation were limited to gonadotrophin-releasing hormone (GnRH) analogues/agonists, cross sex/gender-affirming hormones, anti-androgen therapy and progesterone therapy. Target populations were primarily older adolescents. Outcomes most commonly assessed were body structure and functions related to puberty/reproduction (n=6 studies), and body structures related to brain, blood vessels or heart (n=5); followed by bone structures (n=4), fat mass/BMI (n=4), metabolic functions (n=4), and gender identity, gender/body image or gender dysphoria (n=3). The studies’ contributions on effectiveness and safety will be restricted because many do not use a control group (8/15 studies), random allocation (11/15), or blinding (12/15). Conclusions: Ongoing studies are unlikely to provide substantial new evidence to address evidence gaps in effectiveness or safety of medicines for children and young people experiencing gender incongruence or dysphoria. It will depend on new studies, including NHS England’s trial of puberty supressing treatments, with adequate power, rigorous designs, and inclusion of all relevant outcomes, to provide such evidence
Author(s): Norman G, Fairbairn R, Dobson E, Oparah C, Ogunyemi A, Kolehmainen N
Publication type: Article
Publication status: Published
Journal: Archives of Disease in Childhood
Year: 2026
Pages: Epub ahead of print
Online publication date: 24/08/2026
Acceptance date: 14/03/2026
Date deposited: 07/04/2026
ISSN (print): 0003-9888
ISSN (electronic): 1468-2044
Publisher: BMJ Publishing Group
URL: https://doi.org/10.1136/archdischild-2025-329435
DOI: 10.1136/archdischild-2025-329435
Data Access Statement: Data are available upon reasonable request. All data relevant to the study are included in the article or uploaded as supplementary information.
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