Medicines for children and young people who seek support around gender: a horizon scanning study of the emerging medicines pipeline

Vollständiger Abstract

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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 (aged 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 15 ongoing studies across 23 registries with a total actual or planned sample size of 1073. Twelve studies were based in the USA. The medicine classes under evaluation were limited to gonadotrophin-releasing hormone 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/body mass index (n=4), metabolic functions (n=4) and gender identity, gender/body image or gender dysphoria (n=3). The contributions of the studies 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-suppressing treatments, with adequate power, rigorous designs and inclusion of all relevant outcomes, to provide such evidence.