Evidence map›Paper›PMID 38198073›Full record

ArticleJournal of endocrinological investigation2024

The economic burden of pediatric growth hormone deficiency in Italy: a cost of illness study.

M Cappa, G Pozzobon, M Orso, M Maghnie, G Patti, F Spandonaro, S Granato, G Novelli, D La Torre, M Salerno and 1 more

Abstract read
In one paragraph

Article in Journal of endocrinological investigation, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. Article
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

11 authors.

M CappaResearch Area for Innovative Therapies in Endocrinopathies, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.ORCID http://orcid.org/0000-0001-5647-7519
G PozzobonIRCCS San Raffaele Hospital, Pediatric Unit-Università Vita-Salute, Milan, Italy.
M OrsoC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy. massi.orso@hotmail.it.ORCID http://orcid.org/0000-0003-0200-1202
M MaghnieDepartment of Pediatrics, IRCCS Istituto Giannina Gaslini, Genoa, Italy.ORCID http://orcid.org/0000-0002-7183-5238
G PattiDepartment of Pediatrics, IRCCS Istituto Giannina Gaslini, Genoa, Italy.ORCID http://orcid.org/0000-0002-6217-1919
F SpandonaroC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.ORCID http://orcid.org/0000-0003-1388-2850
S GranatoMedical Department, Pfizer Italia, Rome, Italy.
G NovelliHealth and Value, Pfizer Italia, Rome, Italy.
D La TorreGlobal Medical Affairs, Pfizer Rare Disease, Rome, Italy.
M SalernoDepartment of Translational Medical Sciences, Paediatric Endocrinology Unit, University of Naples 'Federico II', Naples, Italy.ORCID http://orcid.org/0000-0003-1310-3300
B PolistenaC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.ORCID http://orcid.org/0000-0002-0138-0223

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeGrowth hormone deficiency (GHD) is a rare condition with a worldwide prevalence of 1 patient in 4000 to 10,000 live births, placing a significant economic burden on healthcare systems. The aim of this study is to generate evidence on the economic burden of children and adolescents with GHD treated with rhGH and their parents in Italy.

methodsA cost of illness analysis, adopting the prevalence approach, has been developed, producing evidence on the total annual cost sustained by the Italian National Health System (NHS) and by the society. The study is based on original data collected from a survey conducted among Italian children and adolescents with GHD and their parents.

results143 children/adolescents with GHD and their parents participated to the survey, conducted from May to October 2021. Patients had a mean age of 12.2 years (SD: 3.1) and were mostly males (68.5%). The average direct healthcare cost sustained by the NHS was € 8,497.2 per patient/year; adding the out-of-pocket expenses (co-payments and expenses for private healthcare service), the total expense was € 8,568.6. The indirect costs, assessed with the human capital approach, were € 847.9 per patient/year. The total of direct and indirect cost is € 9,345.1 from the NHS perspective, and € 9,416.5 from a social perspective. The total cost incurred by the Italian NHS for children with GHD (range: 5,708-8,354) was estimated in € 48.5-71.0 million, corresponding to 0.04-0.06% of the total Italian public health expense in the year 2020.

conclusionsThe total annual cost for GHD children is close to € 10,000, and is mainly due to the cost of rhGH treatment. This cost is almost entirely sustained by the NHS, with negligible out-of-pocket expenses. The economic burden on the Italian NHS for the health care of established GHD children is fourfold higher than the prevalence of the disease in the overall Italian population.

Indexed as

Cost of IllnessHealth Care CostsHuman Growth HormoneAdolescentChildDwarfism, PituitaryFemaleGrowth DisordersHealth ExpendituresHumansItalyMaleParentsPrevalenceHuman Growth HormoneChildrenCost of illnessEconomic burdenGrowth hormone deficiencyItaly

Identifiers

PMID38198073
PMCPMC11196344

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Registered trials

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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.