Health care utilization and excess costs after pelvic fractures among older people in Germany.

Our study demonstrates a strong increase in utilization of inpatient health care and clear excess costs in older people in the first year after pelvic fracture, the latter even after adjustment for several confounders. Excess costs were particularly high in the first few months and mainly attributable to inpatient treatment. INTRODUCTION: We aimed to estimate health care utilization and excess costs in patients aged minimum 60 years up to 1 year after pelvic fracture compared to a population without pelvic fracture. METHODS: In this retrospective population-based observational study, we used routine data from a large statutory health insurance (SHI) in Germany. Patients with a first pelvic fracture between 2008 and 2010 (n=5685, 82% female, mean age 80±9 years) were frequency matched with controls (n=193,159) by sex, age at index date, and index month. We estimated health care utilization and mean total direct costs (SHI perspective) with 95% confidence intervals (CIs) using BCA bootstrap procedures for 52 weeks before and after the index date. We calculated cost ratios (CRs) in 4-week intervals after the index date by fitting mixed two-part models including adjustment for possible confounders and repeated measurement. All analyses were further stratified for men/women, in-/outpatient-treated, and major/minor pelvic fractures. RESULTS: Health care utilization and mean costs in the year after the index date were higher for cases than for controls, with inpatient treatment being particularly pronounced. CRs (95% CIs) decreased from 10.7 (10.2-11.1) within the first 4 weeks to 1.3 (1.2-1.4) within week 49-52. Excess costs were higher for inpatient than for outpatient-treated persons (CRs of 13.4 (12.9-13.9) and 2.3 (2.0-2.6) in week 1-4). In the first few months, high excess costs were detected for both persons with major and minor pelvic fracture. CONCLUSION: Pelvic fractures come along with high excess costs and should be considered when planning and allocating health care resources.

The impact of additive or substitutive clinical study design on the negotiated reimbursement for oncology pharmaceuticals after early benefit assessment in Germany.

BACKGROUND: We analysed the impact of clinical study design for oncological pharmaceuticals on the subsequent price negotiations after early benefit assessment between pharmaceutical companies and the German National Association of Statutory Health Insurance Funds. The analysis was conducted for all oncology pharmaceuticals that underwent the early benefit assessment in Germany since its introduction in 2011 up to September 2016. METHODS: It was differentiated between additive (new therapy in addition to baseline therapy) and substitutive study designs (baseline therapy to be replaced). The study design was derived from the dossiers of the pharmaceutical companies submitted to the Federal Joint Committee. Subgroup specific costs in case of granted added benefit were calculated as annual therapy costs and compared with the costs of the appropriate comparators to quantify price premiums. Further price influencing factors were analysed in univariate and multivariate regression analysis considering the budget impact for the statutory health insurance as well. RESULTS: The mean and the median of the additive premiums for substitutive designs (€50,477.68 and €49,841.24) were higher than for additive designs, if the comparator was different to best supportive care (€48,750.00 and €42,820.44). The mean multiplicative premium for the substitutive designs was 15.07 versus 2.29 for the additive designs. EU-Prices and target population size had a significant effect on the reimbursement. The adjusted R-square in the log Premium OLS-regressions reached 0.708 when including all explanatory variables and considering interaction between target population and annual costs of the comparator. CONCLUSIONS: Study design as an additional important influencing factor of the negotiations next to those stated in the framework agreement was identified and verified. Therefore, study design should be considered by pharmaceutical companies and by decision makers and payers within strategic price planning as a potential predictor. For some specific categories the number of cases was small. Further analyses should be performed when more oncology pharmaceuticals have passed the early benefit assessment.

Different interpretation of additional evidence for HTA by the commissioned HTA body and the commissioning decision maker in Germany: whenever IQWiG and Federal Joint Committee disagree.

BACKGROUND: The purpose of this study was to analyse the impact of commissioned addenda by the Federal Joint Committee (FJC) to the HTA body (IQWiG) and their agreement with FJC decisions and to identify potential additional decisive factors of FJC. METHODS: All available relevant documents up to end of 2017 were screened and essential content extracted. Next to descriptive statistics, differences between IQWiG and FJC were tested and explored by agreement statistics (Cohen's kappa and Fleiss' kappa) and ordinal logistic regression. RESULTS: Most of the 90 addenda concerned oncological products. In all contingent comparisons, positive changes in added benefit or evidence level on a subpopulation basis (n = 124) prevailed negative ones. Fleiss' ordinal kappa for agreement of assessments, addenda, and appraisals reached a moderate strength for added benefit (0.474, 95%-CI, 0.408-0.540). Overall agreement between addenda and appraisals on a binary nominal basis is poor for added benefit (Cohen's kappa 0.183; 95%-CI: 0.010-0.357) ranging from "less than by chance" (respiratory diseases) to "perfect" (neurological diseases). The OR of the selected regression model showed that i) mortality, ii) unmet need, the positions of iii) the physicians' drug commission and iv) medical societies, and v) the annual therapeutic costs of the appropriate comparative therapy had a high influence on FJC's appraisals deviating from IQWiG's addenda recommendation. CONCLUSIONS: IQWiG's addenda have a high impact on decision-maker's appraisals offering additional analyses of supplementary evidence submitted by the manufacturers. Nevertheless, the agreement between addenda and appraisals varies, highlighting different decisive factors between IQWiG and FJC.

[Health-Related Quality of Life in the Context of Early Benefit Assessment of Drugs According to § 35a of the German Social Code Book V: A Challenging Endpoint for all the Involved Stakeholders]. / Gesundheitsbezogene Lebensqualität im Rahmen der frühen Nutzenbewertung von Arzneimitteln nach § 35a SGB V: Ein Endpunkt mit vielen Herausforderungen für alle beteiligten Akteure.

Considering repeated demands to present data on health-related quality of life (HRQoL) in the context of early benefit assessment of drugs in Germany, on the one hand, and the ongoing methodical debates on this outcome, on the other, the question about its significance in the evaluation procedure has been raised. Published documents of the first 90 early benefit assessments were extracted in order to estimate the importance of HRQoL in the assessment process for each involved stakeholder, and synoptically evaluated. The main challenge is to quantify a qualitative effect variable and to estimate its value. In several early benefit assessments, HRQoL had not been elicited a priori. In many cases where some data on HRQoL are presented, no added benefit can be derived. Finally, in most of the remaining assessments, no added benefit was recognized by IQWiG or the Federal Joint Committee. Even though the incorporation of HRQoL in clinical trial protocols is increasing, the endpoint is mostly conceptualized in an explorative manner. Hence, there is heterogeneity in the identified evidence. Beyond missing statistical significance, the assessment reports refer to a range of different methodological limitations, leading the responsible stakeholders to exclude HRQoL from their assessments, e. g. a high proportion of missing patient data or difficulties in the interpretation of the measurement results are mentioned. Regarding the implemented assessment approaches, inconsistencies in the applied conceptual and methodological standards were uncovered. To increase the importance of the endpoint HRQoL in the context of the early benefit assessment of drugs, establishing reliable measurement standards by consensus seems to be necessary. Furthermore, the valuation criteria should be made more transparent and attempts must be made to harmonize them with the approval process for at least the first early assessment of a new drug.

Direct costs in impaired glucose regulation: results from the population-based Heinz Nixdorf Recall study.

OBJECTIVE: For the first time, this population-based study sought to analyze healthcare utilization and associated costs in people with normal fasting glycemia (NFG), impaired fasting glycemia (IFG), as well as previously undetected diabetes and previously diagnosed diabetes linking data from the prospective German Heinz Nixdorf Recall (HNR) study with individual claims data from German statutory health insurances. RESEARCH DESIGN AND METHODS: A total of 1709 participants of the HNR 5-year follow-up (mean age (SD) 64.9 (7.5) years, 44.5% men) were included in the study. Age-standardized and sex-standardized healthcare utilization and associated costs (reported as € for the year 2008, perspective of the statutory health insurance) were stratified by diabetes stage defined by the participants' self-report and fasting plasma glucose values. Cost ratios (CRs) were estimated using two-part regression models, adjusting for age, sex, sociodemographic variables and comorbidity. RESULTS: The mean total direct healthcare costs for previously diagnosed diabetes, previously undetected diabetes, IFG, and NFG were €2761 (95% CI 2378 to 3268), €2210 (1483 to 4279), €2035 (1732 to 2486) and €1810 (1634 to 2035), respectively. Corresponding age-adjusted and sex-adjusted CRs were 1.53 (1.30 to 1.80), 1.16 (0.91 to 1.47), and 1.09 (0.95 to 1.25) (reference: NFG). Inpatient, outpatient and medication costs varied in order between people with IFG and those with previously undetected diabetes. CONCLUSIONS: The study provides claims-based detailed cost data in well-defined glucose metabolism subgroups. CRs of individuals with IFG and previously undetected diabetes were surprisingly low. Data are important for the model-based evaluation of screening programs and interventions that are aimed either to prevent diabetes onset or to improve diabetes therapy as well.

[Methods of health economic evaluation for health services research]. / Methoden der gesundheitsökonomischen Evaluation in der Versorgungsforschung.

On August 30, 2010, the German Network for Health Services Research [Deutsches Netzwerk Versorgungsforschung e. V. (DNVF e. V.)] approved the Memorandum III "Methods for Health Services Research", supported by the member societies mentioned as authors and published in this Journal [Gesundheitswesen 2010; 72: 739-748]. The present paper focuses on methodological issues of economic evaluation of health care technologies. It complements the Memorandum III "Methods for Health Services Research", part 2. First, general methodological principles of the economic evaluations of health care technologies are outlined. In order to adequately reflect costs and outcomes of health care interventions in the routine health care, data from different sources are required (e. g., comparative efficacy or effectiveness studies, registers, administrative data, etc.). Therefore, various data sources, which might be used for economic evaluations, are presented, and their strengths and limitations are stated. Finally, the need for methodological advancement with regard to data collection and analysis and issues pertaining to communication and dissemination of results of health economic evaluations are discussed.

[IQWiG's methods for the cost-benefit assessment : Comparison with an international reference scenario]. / Wo steht die Kosten-Nutzen-Bewertung des IQWiG : Abgleich mit einem internationalen Referenzszenario?

Standardization of international health economic guidelines has been repeatedly requested. In this context, an international reference case was proposed, which constitutes an agreed approach for the key elements of health economic evaluation including study perspective, comparators, source of effectiveness data, role of modeling, main (economic) outcome, source of utilities, characterizing uncertainty. It is, however, questionable whether such a reference scenario can reasonably be applied across all health care systems. Our analysis pursues the question to which degree the Institute for Quality and Efficiency in Health Care's (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG) "General methods for evaluating the relation between cost and benefit" comply with the key elements of the reference case. In case of divergences, they will be described and discussed in light of the German social legislation and in consideration of current scientific evidence. In conclusion, the analysis revealed that IQWiG complied with the reference case in almost all aspects. Differences were found only with respect to the choice of main (economic) outcome and the source of utilities. These differences seem justified and well explained in the context of the German social legislation as well as in view of the weaknesses of the quality-adjusted life year (QALY) concept.

[Patient-reported and patient-weighted outcomes in ophthalmology]. / Patientenberichtete und patientengewichtete Endpunkte in der Augenheilkunde.

Considering patients' values and preferences in comparative effectiveness research (CER) is one of the main challenges in ophthalmology (value-based medicine). This article defines core terms in CER. The concept of patient-relevant (or patient-important) outcomes is distinguished from patient-reported outcomes (PRO) by means of examples in the field of ophthalmology. In order to be able to give a consistant recommendation if an intervention leads to conflicting results for different outcomes (trade-off), a ranking of outcomes will be necessary. Examples of studies in glaucoma patients are provided that demonstrate the possibilities of ranking of outcomes based on patient preferences.