Real‐world resource use and costs of haemophilia A‐related bleeding

Shrestha, Archana; Eldar-Lissai, Adi; Hou, Nova; Lakdawalla, Darius N.; Batt, Katharine

doi:10.1111/hae.13220

Cited by 37 publications

(40 citation statements)

References 22 publications

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“…In a recent analysis, the projected annual drug cost of prophylactic treatment in the US ranged from nearly $700,000 to $750,000 for standard and extended halflife FVIII products, respectively 9 . Separate studies have shown patients with hemophilia A incur higher costs due to greater rates of hospitalization, ER visits, office visits, medical procedures, and laboratory tests compared with the general population 7,14,15 . Greater indirect costs for these patients are driven by absenteeism and productivity loss 7,15 .…”

Section: Introductionmentioning

confidence: 99%

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Cook

Forbes

Adamski

et al. 2020

Journal of Medical Economics

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Aim: Hemophilia A is a genetic, chronic disorder classified by deficient or defective coagulation factor VIII (FVIII) that puts those affected at risk for spontaneous bleeding episodes, which lead to joint damage and chronic pain over time. Currently, most severe hemophilia A patients are treated with prophylactic FVIII, which requires costly and frequent infusions and lifelong adherence to medication. A gene therapy (valoctocogene roxaparvovec) is currently in development for the treatment of severe hemophilia A. This model assessed the potential cost-effectiveness of treating patients with valoctocogene roxaparvovec rather than prophylactic therapy. Materials and methods: We developed an individual-based, state-transition microsimulation model for assessing the likely cost-effectiveness of valoctocogene roxaparvovec compared to prophylactic FVIII. Men aged 30 with severe hemophilia A were modeled over a lifetime horizon, and costs were reported from the perspective of the United States health care system. Through microsimulation, patient-level heterogeneity was captured in starting weight, starting Pettersson score (PS), durability of valoctocogene roxaparvovec, and annual bleed rate (ABR). Results: The model projects that treatment with single-administration valoctocogene roxaparvovec would be cost-saving to people with hemophilia A at a price point comparable to other currently available gene therapy products due to its potential to reduce FVIII utilization, direct medical costs, lifetime bleeds, and accumulated joint damage. Limitations: The model relies upon evidence-based assumptions for clinical inputs due to limited data availability. Such uncertainty was mitigated by modeling heterogeneity across the population, specifically with regards to long-term gene therapy durability, lifetime bleed rates, and joint damage progression. Conclusion: Valoctocogene roxaparvovec was found to be cost-saving-on average by about $6.8 million per patient-and more effective than prophylactic therapy for treatment of hemophilia A. The comparative benefit of gene therapy was observed across a broad range of simulated patients that were representative of the real-world severe hemophilia A population.

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Section: Introductionmentioning

confidence: 99%

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Cook

Forbes

Adamski

et al. 2020

Journal of Medical Economics

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“…The GEPHARD contains most of this information for paediatric patients. In secondary databases, bleeding events treated at home are not captured . Information on bleeds, treatment patterns, treatment of inhibitors, joint health and target joints is partially available, or not at all.…”

Section: Resultsmentioning

confidence: 99%

“…In secondary databases, bleeding events treated at home are not captured. 22 Information on bleeds, treatment patterns, treatment of inhibitors, joint health and target joints is partially available, or not at all. Examples are severe bleeds necessitating out-or inpatient treatment and treatment patterns based upon prescriptions.…”

Section: As Shown Inmentioning

confidence: 99%

Prospects for research in haemophilia with real‐world data—An analysis of German registry and secondary data

Schopohl

Bidlingmaier²,

Herzig

et al. 2018

Haemophilia

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Rigorous observational analysis of German haemophilia RWD shows that there is potential to supplement current knowledge and begin to address selected policy goals. To improve the value of existing RWD, the following efforts are proposed: ethical, legal and methodological discussions on data linkage across different sources, formulation of transparent governance rules for data access, redefinition of the ICD-coding, standardized collection of outcome data and implementation of incentives for treatment centres to improve data collection.

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“…Small sample size in the current study prevented analysis of costs by any characteristic; in fact, simple comparison of costs by sex may be misleading at this time. The mean total costs were $112,090 for an 18‐month period in the current study, compared with $96 918 for 1 year, not including prophylaxis, from one study including similar‐aged males 11 . The diagnosis confusion observed among females with HA, relative to males, can delay appropriate non‐emergent (ie, prophylactic) treatment.…”

Section: Discussionmentioning

confidence: 99%

Characterizing female patients with haemophilia A: Administrative claims analysis and medical chart review

et al. 2020

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Aim Haemophilia A (HA) is a male‐predominant disorder, yet women and girls can have factor VIII (FVIII) deficiency with bleeding events requiring treatment. This study aimed to identify and characterize female patients with HA. Methods Administrative claims dated 01 January 2012‐31 July 2016 were accessed for patients with 18 months’ coverage by commercial or Medicare Advantage with Part D insurance. Patients were included by HA diagnoses or treatments and/or bleeding‐related diagnoses or procedures, and excluded by haemophilia B or qualitative platelet disorder diagnoses. A sample of charts was examined for bleeding history, HA therapies and bleeding treatments. All‐cause healthcare utilization and costs were also described. Results Among 353 patients meeting initial inclusion criteria, 86 charts were procured, with 8 patients identified as having HA. Their mean age was 60 ± 17 years and most were Medicare‐insured. The mean Charlson Comorbidity Index score was 2.50 ± 2.56; the most prevalent comorbid conditions involved coagulation/haemorrhage, fluid/electrolyte balance and non‐traumatic joint disorders. Over 18 months, a mean of 54 ambulatory visits and 120 pharmacy fills were observed; mean medical costs were $86 694 and pharmacy costs were $25 396. Conclusions Identifying females with HA is challenging using healthcare claims, because diagnostic nomenclature is unclear for female patients treated for bleeding events. Although chart abstraction enhanced claims data, very few female patients were identified with HA. Nevertheless, even in a small sample, sizeable burden in comorbidity and healthcare use was observed. Improved nomenclature and coding for HA diagnoses for women and girls is key to improving research and treatment.

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Real‐world resource use and costs of haemophilia A‐related bleeding

Cited by 37 publications

References 22 publications

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Prospects for research in haemophilia with real‐world data—An analysis of German registry and secondary data

Characterizing female patients with haemophilia A: Administrative claims analysis and medical chart review

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