PMS8 National Burden of Achondroplasia in Adults and Children: An Analysis of the National Inpatient Sample

Chen, E.; Yan, Tingjian; Chang, Eunice; Broder, Michael S.; Tarbox, Marian H.; Abrahamson, Larkin A; Pan, Wayne

doi:10.1016/j.jval.2021.04.685

Cited by 1 publication

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“…Three studies published as congress abstracts reported costs or HCRU data in achondroplasia [ 54 – 56 ] (Table 3 ). One study was US-based and reported hospital-related costs and length of stay [ 55 ]; the other two studies were international and reported HCRU.…”

Section: Resultsmentioning

confidence: 99%

“…The US-based study used 2017 data from the National Inpatient Sample and estimated total hospitalisation-associated costs for all patients with achondroplasia at approximately $40 million [ 55 ]. Notably, costs were equally contributed by adults and children ($19.7 million for adults, $19.9 million for children).…”

Section: Resultsmentioning

confidence: 99%

“…Notably, costs were equally contributed by adults and children ($19.7 million for adults, $19.9 million for children). Total mean per-patient inpatients costs were $19,959 (95% confidence interval [CI] $16,801–$23,118), an increase of $7789 for people with achondroplasia compared to the general population [ 55 ]. Average hospital length of stay was 6.8 days (5.7–8.0), an increase of 2.2 days compared to the general population.…”

Section: Resultsmentioning

confidence: 99%

“…In addition to burden on HRQoL, achondroplasia has a significant economic burden on healthcare systems, with one US study finding all achondroplasia hospitalisations cost approximately $40 million in 2017 and that individuals with achondroplasia spent 2.2 days longer in hospital than patients without the condition [ 55 ]. The same study reported that costs were contributed to equally by children and adults, highlighting the impact of the condition beyond childhood and over the course of the lifetime.…”

Section: Discussionmentioning

confidence: 99%

“…Furthermore, while total height may be increased by all identified interventions, based on current evidence it is unclear whether they confer benefits to individuals’ QoL or functioning. HCRU/cost data are also very limited, with only one study reporting cost data [ 55 ] and three studies reporting resource use specifically for achondroplasia [ 54 – 56 ]. In addition, the identified study considered costs from a healthcare payer perspective and did not provide a breakdown of individual cost components.…”

Section: Discussionmentioning

confidence: 99%

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Burden and Treatment of Achondroplasia: A Systematic Literature Review

et al. 2023

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Background Achondroplasia is the most common form of skeletal dysplasia. Recent advances in therapeutic options have highlighted the need for understanding the burden and treatment landscape of the condition. This systematic literature review (SLR) aimed to identify health-related quality of life (HRQoL)/utilities, healthcare resource use (HCRU), costs, efficacy, safety and economic evaluation data in achondroplasia and to identify gaps in the research. Methods Searches of MEDLINE, Embase, the University of York Centre for Reviews and Dissemination (CRD), the Cochrane Library and grey literature were performed. Articles were screened against pre-specified eligibility criteria by two individuals and study quality was assessed using published checklists. Additional targeted searches were conducted to identify management guidelines. Results Fifty-nine unique studies were included. Results demonstrated a substantial HRQoL and HCRU/cost-related burden of achondroplasia on affected individuals and their families throughout their lifetimes, particularly in emotional wellbeing and hospitalisation costs and resource use. Vosoritide, growth hormone (GH) and limb lengthening all conferred benefits for height or growth velocity; however, the long-term effects of GH therapy were unclear, data for vosoritide were from a limited number of studies, and limb lengthening was associated with complications. Included management guidelines varied widely in their scope, with the first global effort to standardise achondroplasia management represented by the International Achondroplasia Consensus Statement published at the end of 2021. Current evidence gaps include a lack of utility and cost-effectiveness data for achondroplasia and its treatments. Conclusions This SLR provides a comprehensive overview of the current burden and treatment landscape for achondroplasia, along with areas where evidence is lacking. This review should be updated as new evidence becomes available on emerging therapies. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-023-02549-3.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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