The prevalence of depression and mood disorders has been steadily rising in Australian youth, with a concomitant increase in antidepressant pharmacotherapy prescription rates. Yet, the tolerability and efficacy of antidepressant drugs in youth remain poor. Pharmacogenetic (PGx) testing, or the personalised and guided treatment of medication based on genetic data, has been suggested to improve the effectiveness and tolerability of antidepressants. However, limited studies have evaluated the utility of antidepressant PGx-guided treatment in adolescent and young adult populations. Thus, this pilot randomised controlled trial (RCT), the GENE-YD Study, will evaluate the feasibility for a large-scale RCT assessing the effect of PGx-guided antidepressant prescription vs treatment as usual in youth with major depressive disorder (MDD).

Eighty young people between 16 and 24 years of age and in the early stages of pharmacotherapy treatment for MDD will be recruited. Following initial screening, participants will be randomised on a 1:1 ratio to either the intervention or control study group. Participants in the intervention condition will have their treatment tailored based on their PGx profile. Participants randomised into the control group will have their prescription based on current best practice recommendations, or treatment as usual. Individuals will be assessed at drug prescription baseline and again 6 and 12 weeks following drug prescription. The primary outcome of the study will be to evaluate the feasibility and acceptability of the GENE-YD protocol. Specifically, this study will explore participation recruitment strategies and attrition to the study protocols to guide the recruitment processes of a large-scale RCT, along with participating satisfaction in overall study protocols. Secondary outcomes will inform the utility and variability of specific measures (eg, depression rating scales, quality of life measures and medication adherence scales) that may be scaled up for use in a future full-scale trial.

Ethics approval was granted by the Department of Health, Western Australia’s Human Research Ethics Committee (RGS0000006822) and recognised by the University of Western Australia’s Human Research Ethics Committee (2024/ET000685). All participants will be required to provide written informed consent. Results will be published in international peer-reviewed journals.

ACTRN12624000760572.

Emicizumab is the first bispecific antibody approved for prophylaxis in people with haemophilia A with or without factor VIII inhibitors. Aggregate distributional cost-effectiveness analysis assesses health equity impacts by evaluating how health effects and costs from funding an intervention are distributed among population subgroups. The objective was to evaluate how funding emicizumab for people with severe haemophilia A (PwSHA) impacts population health and health disparities in the USA.

Population-level model of PwSHA from the perspective of the US healthcare system, using published sources and considering a lifetime time horizon.

Emicizumab versus other haemophilia A prophylaxis treatments.

Quality-adjusted life-years (QALYs) gained and change in Atkinson index of inequality in quality-adjusted life expectancy.

When an estimated 6512 PwSHA in the USA were treated with emicizumab, the US healthcare system would save US$160 billion over those individuals’ lifetimes. If these cost savings fund additional healthcare interventions in the overall population, funding emicizumab would improve overall US population health (1 068 903 QALYs gained, using a threshold of US$150 000/QALY) and reduce existing overall US inequities (–0.01% on the Atkinson index).

In all scenarios tested for sensitivity, increased emicizumab and prophylaxis utilisation led to further reductions in health disparities and greater increases in population health. Results were robust to deterministic variations in the allocation of cost savings due to emicizumab use.

Funding emicizumab treatments for PwSHA improves overall population health and reduces overall health inequities in the USA. Cost savings from the use of emicizumab free up important resources that can be leveraged to support other healthcare interventions, but decisions on how these funds are used have large consequences for equity.