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(1) Background: Achieving inactive disease decreases long-term joint damage in patients with polyarticular juvenile idiopathic arthritis (polyJIA). The aim of our study was to describe average time to treatment and medication changes over time. (2) Methods: Incident polyJIA patients were retrospectively identified in the InGef and WIG2 longitudinal health claims databases. Drug escalation level changes were evaluated longitudinally and cross-sectionally across three years, as follows: no treatment, glucocorticoids (GCs) and/or non-steroidal anti-inflammatory drugs (NSAIDs), conventional synthetic disease-modifying antirheumatic drugs (csDMARDs), and biological disease-modifying antirheumatic drugs (bDMARDs). (3) Results: On average, newly diagnosed polyJIA patients received their first csDMARD prescription after 128 days and their first bDMARD prescription after 327 days. More patients were treated with csDMARDs than with bDMARDs at diagnosis; however, 24% and 12% (InGef and WIG2 databases, respectively) had no JIA treatment. After three years, 45% and 31% were not taking any treatments, while 18% and 36% were prescribed bDMARDs. Among patients initiating bDMARDs, most continued treatment for three years, with some switching to csDMARDs or discontinuing treatment. Patients treated only with csDMARDs took them longer, compared to those additionally taking other DMARDs. Patients treated with bDMARDs took them about twice as long as the csDMARDs they took prior. (4) Conclusion: A substantial number of patients with polyJIA are not treated as intensively as guidelines recommend.
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The effectiveness of digital care applications in people with need of home care: A systematic review Abstract: Background: The growing need for long-term care poses challenges for healthcare systems. In June 2021, digital care applications (DiPA) were introduced as a new service in the statutory long-term care insurance in Germany. Their aim is to counteract the increased care need and/or support independence in the home. Aim: This project systematically identified and reported on national and international evidence on DiPA effectiveness, as described by the Federal Institute for Drugs and Medical Devices (BfArM). Methods: The systematic review was conducted in accordance with the PRISMA statement by means of systematic searches in the databases Medline and Cinahl, and supplemented by an extensive manual search (08/2022). Study quality was assessed using the Risk of Bias Tool (RoB2). The results were synthesized narratively. Results: Eight randomized controlled studies were included; improvements in cognition, activities of daily living, and mobility in home care patients, were reported. Study quality was low to moderate, due to mostly unblinded study designs and low case numbers. Conclusions: The currently available evidence fails to provide a reliable basis for assessing the benefits of DiPA, to neither DiPA manufacturers, nor to responsible parties in the healthcare system. Further research on DiPA effectiveness, particularly with high-quality studies, are necessary to assess its potential in the German care sector.
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Background: Lysosomal storage diseases (LSDs), metabolic disorders resulting in build-up of endogenous waste and progressive organ damage, can be treated with intravenous enzyme replacement therapy (ERT). ERT can be administered either in specialized clinics, at a physician's office or in the home care setting. Legislative goals in Germany strive to shift to more outpatient care while maintaining treatment objectives. This study investigates the patient perspective on home-based ERT in terms of acceptance, safety, and treatment satisfaction in LSD patients. Methods: This was a longitudinal observational study, carried out under real-world conditions in patients' home environment, covering 30 months from January 2019 to June 2021. Patients with LSDs who were deemed suitable for home-based ERT by their physicians were recruited to the study. Patients were interviewed before the start of the first home-based ERT and then at regular intervals thereafter using standardized questionnaires. Results: Data from 30 patients were analyzed: 18 with Fabry disease, 5 with Gaucher disease, 6 with Pompe disease and 1 with Mucopolysaccharidosis type I (MPS I). Age ranged from 8 to 77 years (mean age 40). The reported average waiting time prior to infusion of more than half an hour decreased from 30% of the patients affected at baseline to 5% across all follow-up time points. All patients felt adequately informed about home-based ERT throughout follow-ups and reported that they would choose home-based ERT again. At almost each time point, patients indicated that home-based ERT had improved their ability to cope with the disease. All but one patient indicated feeling safe at each follow-up time point. Compared to 36.7% at baseline, only 6.9% of patients reported a need for improvement in their care after 6 months of home-based ERT. Mean treatment satisfaction increased by approximately 1.6 scale points after 6 months of home-based ERT compared to baseline, and by another 0.2 scale points after 18 months. In terms of quality of care, all but one patient rated home-based ERT as an equivalent alternative throughout follow-ups. Patients would recommend home-based ERT to other suitable LSD-patients. Conclusion: Home-based ERT increases patients' treatment satisfaction, and patients perceive the quality of care as an equivalent alternative, compared to ERT in a center, clinic, or at a physician's office.
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BACKGROUND: With its digital health application (DiGA)-system, Germany is considered one of Europe's pioneers in the field of evidence-based digital health. Incorporating DiGA into standard medical care must be based on evidence-based success factors; however, a comprehensive overview of the evidence required of scientific studies for their approval is lacking. OBJECTIVE: The study aims to, (1) identify specific requirements defined by the Federal Institute for Drugs and Medical Devices (German: Bundesinstitut für Arzneimittel- und Medizinprodukte; BfArM) to design adequate studies, proving a positive healthcare effect, and (2) to assess the evidence given for applications permanently listed in the DiGA directory. METHODS: A multi-step approach was used: (1) identification of the evidence requirements for applications permanently listed in the DiGA directory, (2) identification of the evidence available supporting them. RESULTS: All DiGA permanently listed in the DiGA directory (13 applications) are included in the formal analysis. Most DiGA addressed mental health (n = 7), and can be prescribed for one or two indications (n = 10). All permanently listed DiGA have demonstrated their positive healthcare effect through a medical benefit, and most of them provide evidence for one defined primary endpoint. All DiGA manufacturers conducted a randomized controlled trial. DISCUSSION: It is striking that- although patient-relevant structural and procedural improvements show high potential for improving care, especially in terms of processes - all DiGA have provided a positive care effect via a medical benefit. Although BfArM accepts study designs with a lower level of evidence for the proof of a positive healthcare effect, all manufacturers conducted a study with a high level of evidence. CONCLUSION: The results of this analysis indicate that permanently listed DiGA meet higher standards than required by the guideline.
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Proyectos de Investigación , Humanos , Alemania , Ensayos Clínicos Controlados Aleatorios como AsuntoRESUMEN
BACKGROUND: Juvenile idiopathic arthritis (JIA) describes heterogenous categories of chronic inflammatory rheumatic conditions of unknown origin in children and adolescents. Epidemiological data in the literature vary, depending on geographic location, ethnicity and the case definition used. We evaluated epidemiology, especially that of the categories defined by the International League of Associations for Rheumatology (ILAR). METHODS: Using data from two different longitudinal health claims databases (WIG2 and InGef) from January 1st, 2013 to December 31st, 2019, we looked at patients aged 2 to 15 years old with at least one main inpatient or two secondary inpatient/verified outpatient ICD-10 diagnoses in at least two different quarters within one calendar year. We calculated prevalence and incidence (per 100,000 patients) and extrapolated data to the entire German population, looking at differences in gender and age groups. Additionally, we collected data on "other" not necessary comorbidities in our JIA patient population. RESULTS: Of the 3-4 million patients in the databases (respectively) in 2018, we found a total of 546 (WIG2) and 849 (InGef) patients that met our JIA case definition, with an incidence of 34 (29-41) and 60 (53-67) and prevalence of 133 (122-145) and 168 (157-179). Both incidence and prevalence throughout the age range were mostly higher in females than males, however the difference between females and males increased with increasing age. Of the ILAR categories, oligoarthritis was the most prevalent (70 and 91 per 100,000), with about half of our JIA patients in this category, followed by undifferentiated arthritis (49 and 56 cases per 100,000) and rheumatoid factor negative (RF-) (31 and 39 per 100,000). Incidence in 2018 was the highest in these three categories. Atopic dermatitis, vasomotor and allergic rhinitis, and uveitis were the pre-defined comorbidities seen most often in both databases. CONCLUSION: This study provides current incidence and prevalence JIA data in Germany, contributing to knowledge on burden of disease and tools for healthcare planning.
