Evidence map›Paper›PMID 40930552›Full record

ArticleBMJ open2025

Modelling US health equity impacts of emicizumab for severe haemophilia A: aggregate distributional cost-effectiveness analysis.

Thomas Majda, Janet S Lee, Randall Curtis, Stacey L Kowal

Abstract read
In one paragraph

Article in BMJ open, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Thomas MajdaGenentech, South San Francisco, California, USA majdat@gene.com.ORCID http://orcid.org/0009-0008-6331-0416
Janet S LeeGenentech, South San Francisco, California, USA.
Randall CurtisFactor VIII Computing, Berkeley, California, USA.
Stacey L KowalGenentech, South San Francisco, California, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesEmicizumab 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.

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

interventionEmicizumab versus other haemophilia A prophylaxis treatments. PRIMARY OUTCOME MEASURES: Quality-adjusted life-years (QALYs) gained and change in Atkinson index of inequality in quality-adjusted life expectancy.

resultsWhen 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.

conclusionFunding 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.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedHealth EquityHemophilia ACost-Benefit AnalysisCost-Effectiveness AnalysisHumansQuality-Adjusted Life YearsUnited StatesAntibodies, BispecificAntibodies, Monoclonal, HumanizedemicizumabHealth Care CostsHealth EquityQuality of Life

Identifiers

PMID40930552
PMCPMC12421612

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.