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

Schopohl, Dorothee; Bidlingmaier, Christoph; Herzig, D; Klamroth, Robert; Kurnik, Karin; Rublee, Dale A.; Schramm, W.; Schwarzkopf, Larissa; Berger, Karin

doi:10.1111/hae.13443

Cited by 2 publications

(2 citation statements)

References 20 publications

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“…Appropriate governance is key to clearly define data ownership, to facilitate data collection, data access, data sharing and data linkage. All these aspects can be difficult to implement due to limited funding and resources available to registries, but also due to restrictions linked to national data protection requirements ( Schopohl et al, 2018 ; McGettigan et al, 2019 ). A clear sustainability plan laying down short and long terms strategies on the development and maintenance of the registries is critical to ensuring their continuous viability, adaptability and suitability to support regulators’ decision making ( European Medicines Agency, 2021b ).…”

Section: Challengesmentioning

confidence: 99%

Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe

et al. 2022

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Between 2000 and 2021, the European Medicines Agency (EMA) assigned the orphan designation to over 1,900 medicines. Due to their small target populations, leading to challenges regarding clinical trial recruitment, study design and little knowledge on the natural history of the disease, the overall clinical evidence submitted at the time of marketing authorisation application for these medicines is often limited. Patient registries have been recognised as important sources of data on healthcare practices, drug utilisation and clinical outcomes. They may help address these challenges by providing information on epidemiology, standards of care and treatment patterns of rare diseases. In this review, we illustrate the utility of patient registries across the different stages of development of medicinal products, including orphans, to provide evidence in the context of clinical studies and to generate post-authorisation long term data on their effectiveness and safety profiles. We present important initiatives leveraging the role of registries for orphan medicinal products’ development and monitoring to ultimately improve patients’ lives.

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Section: Challengesmentioning

confidence: 99%

Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe

et al. 2022

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“…Secondary data sources such as registries cannot be used, as the national German register of haemophilia patients does not contain comorbidity variables such as joint and cardiovascular disease (CVD). 13 Clinical medical records include only inpatient health services, and the dataset of the Association of Statutory Health Insurance Physicians (Kassenärztliche Vereinigung, KV) solely covers outpatient services. Only the claims data from the health insurance funds contain cross-sectoral information on inpatient and outpatient care of a patient.…”

Section: Introductionmentioning

confidence: 99%

Can German Health Insurance Claims Data Fill Information Gaps in Rare Chronic Diseases: Use Case of Haemophilia A

Kratzer,

Rölz,

Bidlingmaier

et al. 2024

Hamostaseologie

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Claims data are increasingly discussed to evaluate health care for rare diseases (resource consumption, outcomes and costs). Using haemophilia A (HA) as a use case, this analysis aimed to generate evidence for the aforementioned information using German Statutory Health Insurance (SHI) claims data. Claims data (2017–2019) from the German SHI ‘AOK Bayern - Die Gesundheitskasse’ were used. Patients with ICD-10-GM codes D66 and HA medication were included in descriptive analyses. Severity levels were categorized according to HA medication consumption. In total, 257 patients were identified: mild HA, 104 patients (mean age: 40.0 years; SD: 22.9); moderate HA, 17 patients, (51.2 years; SD: 24.5); severe HA, 128 patients, (34.2 years; SD: 18.5). There were eight patients categorized with inhibitors (37.8 years; SD: 29.6). Psychotherapy was reported among 28.8% (mild) to 32.8% (severe) of patients. Joint disease was documented for 46.2% (mild) to 61.7% (severe) of patients. Mean direct costs per patient per year were 1.34× for mild, 11× for moderate, 81× higher for severe HA patients and 223× higher for inhibitor patients than the mean annual expenditure per AOK Bayern insurant (2019). German SHI data provide comprehensive information. The patient burden in HA is significant with respect to joint disease and psychological stress regardless of the HA severity level. The cost of HA care for patients is high. Large cost ranges suggest that the individual situation of a patient must be considered when interpreting costs. The main limitation of SHI data analysis for HA was the lack of granularity of ICD codes.

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

Cited by 2 publications

References 20 publications

Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe

Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe

Can German Health Insurance Claims Data Fill Information Gaps in Rare Chronic Diseases: Use Case of Haemophilia A

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