Evidence mapPaperPMID 41904313Full record

SynthesisPharmacoEconomics2026

The Economic Burden of Duchenne Muscular Dystrophy: A Systematic Review.

Erik Landfeldt, Maria Åberg, Luca Bello, Valérie Deroo, Alfred Peter Born, Silene Giusti, Anne-Berit Ekström

Abstract readSystematic Review
In one paragraph

Synthesis in PharmacoEconomics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

7 authors.

Erik LandfeldtIQVIA, Hagaplan 4, 113 68, Stockholm, Sweden. erik.landfeldt@iqvia.com.ORCID http://orcid.org/0000-0002-5758-7151
Maria ÅbergEquilib Services, Umeå, Sweden.
Luca BelloDepartment of Neurosciences, DNS, University of Padova, Padua, Italy.
Valérie DerooItalfarmco S.p.A., Milan, Italy.
Alfred Peter BornDepartment of Pediatric and Adolescent Medicine, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark.
Silene GiustiItalfarmco S.p.A., Milan, Italy.
Anne-Berit EkströmDepartment of Pediatrics, Institute of Clinical Sciences, Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveDuchenne muscular dystrophy (DMD) is a rare, progressive, severely disabling and ultimately fatal genetic neuromuscular disease requiring lifelong multidisciplinary clinical care and support. The objective of this study was to conduct a systematic review and synthesis of estimates of costs of illness of DMD.

methodsIn this systematic review (International Prospective Register of Systematic Reviews [PROSPERO] identifier: CRD420251153340), we searched PubMed, MEDLINE, Embase, the Health Technology Assessment Database, and the National Health Service Economic Evaluation Database for studies reporting costs of DMD. Risk of bias was assessed using the Newcastle-Ottawa scale.

resultsWe identified 19 publications involving 6993 children and adults with DMD from 14 countries (Australia, Brazil, Bulgaria, Denmark, Egypt, France, Germany, Hungary, Italy, Portugal, Spain, Sweden, the UK, and the USA). Across studies and strata, the mean per-patient annual direct medical cost of illness (in 2025 international US dollars) was estimated at between $2620 and $209,980, the mean per-patient annual direct nonmedical cost between $5670 and $103,800, and the mean per-patient annual indirect (productivity) cost between $400 and $48,390. Most studies exhibited some risk of bias.

conclusionsDMD is associated with a substantial economic burden related to the formal disease management, as well as extensive informal care and indirect (productivity) costs shouldered by parents and other nonprofessional caregivers. Although relatively well-captured for a few countries, comprehensive cost data are lacking for many geographical settings. Our portfolio of costs of DMD will help inform assessments of burden of illness, health technology evaluations of new therapies, and data gap analyses for future research.

Indexed as

Cost of IllnessHealth Care CostsMuscular Dystrophy, DuchenneHumans

Identifiers

PMID41904313
PMCPMC13291079

What Socratic holds

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LicenceCC BY-NC
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Registered trials

None linked

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 Socratic graph.