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Background Charcot-Marie-Tooth (CMT) neuropathies entail a large group of diseases with different gene mutation patterns, which produce heterogeneous phenotypes. Although health-related quality of life (HRQOL) is significantly impaired, a comprehensive assessment of HRQOL in CMT patients in Germany considering phenotypical heterogeneity represented a research gap. Objective The aim was to assess HRQOL and the satisfaction with health care in CMT patients in Germany. Methods CMT patients > 15 years with a genetically confirmed CMT subtype were recruited through a national CMT patient registry. HRQOL was assessed using the EQ-5D-5L questionnaire. Furthermore, subjective impairments in daily or work activities and satisfaction with health care were assessed using 4-point scales. Results HRQOL in CMT patients (n = 385) was impaired compared to the German population. Most patients reported problems in the dimension mobility (89.6%), pain/discomfort (89.4%) and usual activities (81.0%). Except for patients with hereditary neuropathy with liability to pressure palsy (HNPP), we found no differences in HRQOL between the CMT subtypes. 72.0%of CMT patients were satisfied with available health care services. However, patients reported to expect more CMT-specific knowledge and support as well as easier prescription and cost coverage procedures from health professionals and insurances. Conclusions The patient-reported outcomes in the assessed CMT cohort elucidate the need for more specific health care services that also address the heterogeneous phenotypes. Although the assessment has been limited to the German health services setting, insights may be applicable to CMT-specific care in other national settings.

Zusammenfassung Ziel Die Studie analysiert die Wahrnehmung und Präferenzen der Bevölkerung zu Umfang, Zugang und Finanzierung der Gesundheitsversorgung von Asylsuchenden (AS). Methodik Standardisierte schriftliche Befragung der Bevölkerung >18 Jahre, kombiniertes Quota-Verfahren, deskriptive und inferenzstatistische Auswertung. Ergebnisse Die Stichprobe (n=419) war eher schlecht über die Thematik informiert. Es zeigten sich heterogene Präferenzen. 47,0% favorisierten eine Beibehaltung der Regelungen zum Versorgungsumfang, 22,7% eine Ausweitung. Für eine Gesundheitskarte sprachen sich 56,1% aus. Die EU, Steuerzahler, Herkunftsländer und AS selbst wurden als Finanziers der Versorgung präferiert. Schlussfolgerung Eine Berücksichtigung von Präferenzen kann die Akzeptanz und Legitimation von Entscheidungen stärken. Die vorliegenden Ergebnisse legen nahe, dass dies zu moderaten Anpassungen der Gesundheitsversorgung für AS führen würde. Aufgrund der mangelnden subjektiven Informiertheit der Befragten ist qualitative Forschung angezeigt.

This study analyses public perceptions and preferences regarding scope, access and funding of health care for asylum seekers (AS).Standardized survey addressing the population18 years, combined quota plan, descriptive and inferential statistics.The sample (n=419) was rather badly informed about this topic. We found heterogeneous preferences. 47.0% preferred to maintain current regulations of the scope of health care, 22.7% wanted an expansion. 56.1% favored a health card. The EU, tax payers, countries of origin and AS were preferred as funders.A consideration of preferences might strengthen the acceptance and legitimation of decisions. Our results indicate that this would lead to moderate changes in health care for AS. Since participants were not well informed subjectively, we suggest qualitative research.ZIEL: Die Studie analysiert die Wahrnehmung und Präferenzen der Bevölkerung zu Umfang, Zugang und Finanzierung der Gesundheitsversorgung von Asylsuchenden (AS).Standardisierte schriftliche Befragung der Bevölkerung18 Jahre, kombiniertes Quota-Verfahren, deskriptive und inferenzstatistische Auswertung.Die Stichprobe (n=419) war eher schlecht über die Thematik informiert. Es zeigten sich heterogene Präferenzen. 47,0% favorisierten eine Beibehaltung der Regelungen zum Versorgungsumfang, 22,7% eine Ausweitung. Für eine Gesundheitskarte sprachen sich 56,1% aus. Die EU, Steuerzahler, Herkunftsländer und AS selbst wurden als Finanziers der Versorgung präferiert.Eine Berücksichtigung von Präferenzen kann die Akzeptanz und Legitimation von Entscheidungen stärken. Die vorliegenden Ergebnisse legen nahe, dass dies zu moderaten Anpassungen der Gesundheitsversorgung für AS führen würde. Aufgrund der mangelnden subjektiven Informiertheit der Befragten ist qualitative Forschung angezeigt.

ObjectiveTo assess cost associated with the disease-specific need of patients diagnosed with Charcot-Marie-Tooth neuropathies (CMT) in Germany.MethodsPatients with CMT were identified through the national patient registry and invited to complete a standardized questionnaire. The data collected include information about health care use, informal care, and other disease-related expenses as well as the working situation. Based on this information, we estimated the annual cost of CMT from the perspective of society.ResultsThis study included 397 patients with a genetically confirmed CMT diagnosis. We estimated total annual cost of illness (COI) of $22,362 (95% CI $19,464–$25,723) per patient, of which 67.3% were direct costs. The highest single cost factor was informal care cost. For Germany, we extrapolated total cost of CMT of $735.0 million ($639.8 million–$845.5 million). Multivariate regression analysis showed that total annual cost increased with disease severity (Charcot-Marie-Tooth Neuropathy Score). Age, CMT subtype, comorbidities, body mass index, and employment status were also predictors of a change in cost (p < 0.05). Moreover, we found differences in total cost depending on marital status, subjectively evaluated impairments, dependence on other persons, care level, educational level, and disease duration.ConclusionsCMT is associated with a substantial economic burden. For the first time, the COI of CMT has been assessed and will serve as important input to decision-making in health policy, especially regarding research and development of therapies. Moreover, our results indicate the importance of the patient-reported perception of disease severity related to the consumption of resources.

ObjectiveTo analyse and compare the cost-effectiveness of different interventions to reduce salt consumption.DesignA systematic review of published cost-effectiveness analyses (CEA) and cost-utility analyses (CUA) was undertaken in the databases EMBASE, MEDLINE (PubMed), Cochrane and others until July 2016. Study selection was limited to CEA and CUA conducted in member countries of the Organisation for Economic Co-operation and Development (OECD) in English, German or French, without time limit. Outcomes measures were life years gained (LYG), disability-adjusted life years (DALY) and quality-adjusted life years (QALY). Relevant aspects in modelling were analysed and compared. Quality assessments were conducted using the Drummond and Jefferson/British Medical Journalchecklist.SettingOECD member countries.SubjectsMainly adults.ResultsFourteen CEA and CUA were included in the review which analysed different strategies: salt reduction or substitution in processed foods, taxes, labelling, awareness campaigns and targeted dietary advice. Fifty-nine out of sixty-two scenarios were cost-saving. The incremental cost-effectiveness ratio in international dollars (Intl.$; 2015) was particularly low for taxes, a salt reduction by food manufacturers and labelling (303 900 Intl.$/DALY). However, only six studies analysed cost-effectiveness from a societal perspective and quality assessments showed flaws in conducting and a lack of transparency in reporting.ConclusionsA population-wide salt reduction could be cost-effective in prevention of hypertension and CVD in OECD member countries. However, comparability between study results is limited due to differences in modelling, applied perspectives and considered data.

ZusammenfassungStudienziel war die Untersuchung, inwieweit Ernährungsverhalten und Ernährungszustand von Kindern durch das Ernährungsmuster der Eltern beeinflusst werden. Bei 345 neun-bis elfjährigen Kindern, die im Rahmen der Kieler Adipositas-Präventionsstudie (KOPS) erfasst wurden, konnten nur schwach signifikante Zusammenhänge zwischen dem Ernährungsmuster bzw. dem Fast Food-Konsum von Kindern und deren Eltern nachgewiesen werden. Allerdings bestand eine stark positive Korrelation zwischen dem Obst-und Gemüseverzehr von Eltern und Kindern, wobei die Korrelationen für den Obstverzehr schwächer waren als für den Gemüseverzehr. Das Ernährungsverhalten der Mutter stand in einem stärkeren Zusammenhang zur Lebensmittelaufnahme der Kinder als das des Vaters. Eine Beziehung zwischen dem Ernährungsverhalten der Eltern und dem Ernährungszustand der Kinder konnte nicht aufgezeigt werden. Diese Ergebnisse legen nahe, dass eine Verbesserung des Ernährungsverhaltens von Eltern über die Vorbildfunktion zu einer gesunden Ernährungsweise von Kindern vor allem in Bezug auf den Obst-und Gemüseverzehr beitragen könnte.

Background This study aimed at analyzing the economic burden and disease-specific health-related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in Germany. SMA is a so far non-curable neuromuscular disease of the anterior nerve cells that causes high rates of morbidity and mortality. Methods In a cross-sectional study we analyzed the cost of illness (COI) and factors that influence the direct, indirect and informal care costs of affected patients and their families by using standardized, self-developed questionnaires. We used the PedsQL™© Measurement Model to analyze the disease-specific HRQOL of patients. Results One hundred eighty nine patients with SMA types I to III aged

Background Our study aimed to determine the burden of illness in dystrophinopathy type Duchenne (DMD) and Becker (BMD), both leading to progressive disability, reduced working capacity and high health care utilization. Methods A micro-costing method was used to examine the direct, indirect and informal care costs measuring the economic burden of DMD in comparison to BMD on patients, relatives, payers and society in Germany and to determine the health care burden of these diseases. Standardized questionnaires were developed based on predefined structured interview guidelines to obtain data directly from patients and caregivers using the German dystrophinopathy patient registry. The health-related quality of life (HRQOL) was analyzed using PedsQL™ Measurement Model. Results In total, 363 patients with genetically confirmed dystrophinopathies were enrolled. Estimated annual disease burden including direct medical/non-medical, indirect and informal care costs of DMD added up to € 78,913 while total costs in BMD were € 39,060. Informal care costs, indirect costs caused by loss of productivity and absenteeism of patients and caregivers as well as medical costs of rehabilitation services and medical aids were identified as the most important cost drivers. Total costs notably increased with disease progression and were consistent with the clinical severity; however, patients’ HRQOL declined with disease progression. Conclusion In conclusion, early assessments of economic aspects and the disease burden are essential to gain extensive knowledge of a distinct disease and above all play an important role in funding drug development programs for rare diseases. Therefore, our results may help to accelerate payer negotiations such as the pricing and reimbursement of new therapies, and will hopefully contribute to facilitating the efficient translation of innovations from clinical research over marketing authorization to patient access to a causative treatment. Electronic supplementary material The online version of this article (doi:10.1186/s13023-014-0210-9) contains supplementary material, which is available to authorized users.