Economic burden of growth hormone deficiency in a US pediatric population

Kaplowitz, Paul B.; Manjelievskaia, Janna; López-González, Lorena; Morrow, Cynthia D; Pitukcheewanont, Pisit; Smith, Alden

doi:10.18553/jmcp.2021.21030

Cited by 7 publications

(3 citation statements)

References 24 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…A study from the United States [ 17 ] based on claims data aimed to estimate the health care costs among children with GHD who had either Medicaid ( n = 6820) or commercial health insurance ( n = 14,070). This study reported an average unadjusted annual GHD-related costs of $ 18,069 (somatropin outpatient pharmacy costs: $ 11,951; inpatient costs: $ 5471; outpatient costs: $ 647) for Medicaid-insured patients and $27,893 (somatropin outpatient pharmacy costs: $ 24,130; inpatient costs: $ 1934; outpatient costs: $ 1829) for commercially insured patients.…”

Section: Discussionmentioning

confidence: 99%

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

Cappa,

Pozzobon,

Orso

et al. 2024

J Endocrinol Invest

View full text Add to dashboard Cite

Purpose Growth 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. Methods A 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. Results 143 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. Conclusions The 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.

show abstract

Section: Discussionmentioning

confidence: 99%

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

Cappa,

Pozzobon,

Orso

et al. 2024

J Endocrinol Invest

View full text Add to dashboard Cite

show abstract

“…Frequent dosing causes a heavy mental and economic burden on the patients and leads to poor compliance, thus obtaining suboptimal outcomes 7 , 8 , 9 , 10 , 11 . According to a research survey in the United States, only 18.4% of medicaid patients and 32.3% of commercial patients persisted in the proportion of days covered was more than 80%, while more than 40% of patients stopped treatment before the age of 13 12 . More importantly, the follow-up healthcare costs of untreated GHD patients are approximately twice than that of treated GHD patients.…”

Section: Introductionmentioning

confidence: 99%

Actively separated microneedle patch for sustained-release of growth hormone to treat growth hormone deficiency

Yang

Liu

Wang

et al. 2023

Acta Pharmaceutica Sinica B

View full text Add to dashboard Cite

“…With early diagnosis and treatment initiation, children with GHD often achieve a height within the normal range for their family; research further indicates beneficial impacts on their social and emotional well-being [5,7,[23][24][25]. In the US, a retrospective review of two claims databases for children diagnosed with GHD between 2007 and 2018 who were insured by either Medicaid (n = 6820) or commercial health insurance (n = 14,070) found 63.2% (Medicaid) and 68.4% (commercial) GHD patients were treated at some point with somatropin [26].…”

Section: Introductionmentioning

confidence: 99%

Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB)

Brod

Rasmussen

Alolga

et al. 2022

PharmacoEconomics Open

View full text Add to dashboard Cite

PurposeThe aim was to evaluate the measurement properties of the Growth Hormone Deficiency-Child Treatment Burden Measure-Child (GHD-CTB-Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency-Child Treatment Burden Measure-Observer (GHD-CTB-Observer), an observer-reported outcome (ObsRO) version completed by parents/guardians of children with growth hormone deficiency (GHD) aged 4 to < 9 years; and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB), a PRO that assesses the treatment burden of parents/guardians living with children with GHD aged 4 to < 13 years. Methods A non-interventional, multi-center, clinic-based study across 30 private practice and large institutional sites in the United States and the United Kingdom was conducted. The sample consisted of 145 pre-pubertal children aged 9 to < 13 years at enrollment with a physician confirmed GHD diagnosis as well as 98 parents/guardians of pre-pubertal younger children aged 4 to < 9 years at enrollment with a physician confirmed GHD diagnosis. The child sample consisted of 59 treatment-naïve children (no prior exposure to growth hormone [GH] therapy; were starting GH treatment at study start per standard of care) and 184 children already maintained on treatment for at least 6 months. At baseline, all study participants completed a paper validation battery including all measures needed to conduct the validation analyses. Follow-up assessments with children in the maintenance group and their caregiver/parent were conducted approximately 2 weeks post-baseline to evaluate test-retest reproducibility. To evaluate sensitivity to change and meaningful change thresholds, treatment-naïve participants in both child and parent/guardian populations were assessed within 1 week of report of minimal improvement between week 3 and week 11 and at week 12. Psychometric analyses were implemented following an a priori statistical analysis plan. Results Factor analyses confirmed the a priori conceptual domains and Overall score for each measure (GHD-CTB-Child and GHD-CTB-Observer domains: Physical, Emotional Well-being, and Interference; GHD-PTB domains: Emotional Wellbeing and Interference). Internal consistency was acceptable for all measures (Cronbach's alpha > 0.70). Test-retest reliability was acceptable for the Physical, Emotional, and Overall domains of the GHD-CTB versions, and the Emotional and Overall domains of the GHD-PTB (intraclass correlation coefficient above 0.70). All but one of the convergent validity hypotheses for the GHD-CTB versions and all hypotheses for the GHD-PTB were proven (r > 0.40). Known-groups validity hypotheses were significant for length of time to administer the injections in the GHD-CTB versions (p < 0.001 for Physical, Emotional, and Overall, and p < 0.01 for Interference) and whether parents/guardians versus child gave the injections more often for the Emotional domain of the GHD-PTB (p < 0.05). Associated effect sizes ranged from −0.27 to −0.57 for GHD-CTB versions an...

show abstract

Economic burden of growth hormone deficiency in a US pediatric population

Cited by 7 publications

References 24 publications

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

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

Actively separated microneedle patch for sustained-release of growth hormone to treat growth hormone deficiency

Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB)

Contact Info

Product

Resources

About