Evaluation of two family-based intervention programs for children affected by rare disease and their families - research network (CARE-FAM-NET): study protocol for a rater-blinded, randomized, controlled, multicenter trial in a 2x2 factorial design.

Background: Families of children with rare diseases (i.e., not more than 5 out of 10,000 people are affected) are often highly burdened with fears, insecurities and concerns regarding the affected child and its siblings.

Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare.

Background: Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them.

Objectives: To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology-specifically economic evaluation methods-in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges.

Methods: An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs.

[Health Science Research with Primary Care Routine Data From Electronic Patient Records: the BeoNet Registry].

Reliable current information is needed for daily patient care and the health system, but comprehensive data, especially from primary care, are scarce and/or hard to access. The BeoNet Register-Database (BNR) aims to depict quality of medical care in Germany using routine data and with no additional documentation or costs for GPs. Registry design, data structure and database use will be demonstrated using chronic obstructive lung disease (COPD) as an example.

[Evaluation of family doctor-centred health care in Germany based on AOK Rheinland/Hamburg claims data].

Background: Health insurance funds in Germany are obliged to offer family doctor-centred health care models (Hausarztzentrierte Versorgung, "HzV"). The participation is voluntary for the insured persons. Participants agree to utilise outpatient specialist care only if their family doctor or general practitioner ("gatekeeper") refers them to a specialist.

Valuation of the EQ-5D-5L with composite time trade-off for the German population - an exploratory study.

Background: The EuroQol Group has extended the severity levels of the EQ-5D from three to five (EQ-5D-5L). There are valuation studies worldwide planned in order to convert the EQ-5D-5L health states into a single preference-based summary score based on country-specific value sets of social health status preference valuations. The EuroQol Group developed an internationally standardised EQ-5D-5L valuation protocol.

Measuring patients' priorities using the Analytic Hierarchy Process in comparison with Best-Worst-Scaling and rating cards: methodological aspects and ranking tasks.

Background: Identifying patient priorities and preference measurements have gained importance as patients claim a more active role in health care decision making. Due to the variety of existing methods, it is challenging to define an appropriate method for each decision problem. This study demonstrates the impact of the non-standardized Analytic Hierarchy Process (AHP) method on priorities, and compares it with Best-Worst-Scaling (BWS) and ranking card methods.

Comparison of different approaches applied in Analytic Hierarchy Process - an example of information needs of patients with rare diseases.

Background: The Analytic Hierarchy Process (AHP) is increasingly used to measure patient priorities. Studies have shown that there are several different approaches to data acquisition and data aggregation. The aim of this study was to measure the information needs of patients having a rare disease and to analyze the effects of these different AHP approaches.

Benefit assessment in Germany: implications for price discounts.

Background: The AMNOG regulation, introduced in 2011 in Germany, changed the game for new drugs. Now, the industry is required to submit a dossier to the GBA (the central decision body in the German sickness fund system) to show additional benefit. After granting the magnitude of the additional benefit by the GBA, the manufacturer is entitled to negotiate the reimbursement price with the GKV-SV (National Association of Statutory Health Insurance Funds).

Cost analysis of whole genome sequencing in German clinical practice.

Objectives: Whole genome sequencing (WGS) is an emerging tool in clinical diagnostics. However, little has been said about its procedure costs, owing to a dearth of related cost studies. This study helps fill this research gap by analyzing the execution costs of WGS within the setting of German clinical practice.

Modeling the cost-effectiveness of infant vaccination with pneumococcal conjugate vaccines in Germany.

Background: In 2009, the European Medicines Agency granted approval for two higher-valent pneumococcal conjugate vaccines. This study aims to evaluate the cost-effectiveness of universal infant (<2 years old) vaccination with a 13-valent pneumococcal conjugate vaccine (PCV13) in comparison with a 10-valent pneumococcal conjugate vaccine (PCV10) for the prevention of pneumococcal disease in Germany.

Methods: A population-based Markov model was developed to estimate the impact of PCV13 and PCV10 on invasive pneumococcal disease (IPD), non-invasive pneumonia (PNE), and acute otitis media (AOM) over a time horizon of 50 years.

Applying the Analytic Hierarchy Process in healthcare research: A systematic literature review and evaluation of reporting.

Background: The Analytic Hierarchy Process (AHP), developed by Saaty in the late 1970s, is one of the methods for multi-criteria decision making. The AHP disaggregates a complex decision problem into different hierarchical levels. The weight for each criterion and alternative are judged in pairwise comparisons and priorities are calculated by the Eigenvector method.

Costs and treatment patterns of incident ADHD patients - a comparative analysis before and after the initial diagnosis.

Background And Objectives: The costs and treatment patterns of attention deficit hyperactivity disorder (ADHD) are subjects of health services research in Germany and worldwide. Previous publications focused mainly on prevalent patients and thus research gaps were identified regarding costs and treatment patterns of incident patients before and after the first diagnosis.

Methods: Analyses were conducted using claims data obtained from a large German sickness fund (Techniker Krankenkasse).

Regional differences in health care of patients with inflammatory bowel disease in Germany.

Background: The regional availability of specialized physicians is an important aspect in healthcare of patients with IBD. The association between physician density and healthcare is not yet clear. Most studies did not consider district type, which reflects population density.

EGFR mutation status and first-line treatment in patients with stage III/IV non-small cell lung cancer in Germany: an observational study.

Introduction: EGFR mutations confer sensitivity to EGFR tyrosine kinase inhibitors (TKI) in advanced non-small cell lung cancer (NSCLC). We investigated the clinicopathologic characteristics associated with EGFR mutations and their impact on real-world treatment decisions and outcomes in Caucasian patients with advanced NSCLC.

Methods: REASON (NCT00997230) was a noninterventional multicenter study in patients (≥18 years) with stage IIIb/IV NSCLC, who were candidates for EGFR mutation testing and first-line systemic treatment, but not eligible for surgery or radiotherapy.

A systematic review of the cost-effectiveness of targeted therapies for metastatic non-small cell lung cancer (NSCLC).

Background: Non-small cell lung cancer (NSCLC) imposes a substantial burden on patients, health care systems and society due to increasing incidence and poor survival rates. In recent years, advances in the treatment of metastatic NSCLC have resulted from the introduction of targeted therapies. However, the application of these new agents increases treatment costs considerably.

Early benefit assessment (EBA) in Germany: analysing decisions 18 months after introducing the new AMNOG legislation.

Objectives: Since the introduction of the German health care reform in January 2011, an early benefit assessment (EBA) is required for all new medicines. Pharmaceutical manufacturers have to submit a benefit dossier for evaluation by the Institute for Quality and Efficiency in Health Care (IQWiG). A final decision is made by the Federal Joint Committee (G-BA).

Potential cost-effectiveness and benefit-cost ratios of adult pneumococcal vaccination in Germany.

Background: Invasive (IPD, defined as detection of pneumococci in sterile body fluids like meningitis or bacteremic pneumonia) and non-invasive Streptococcus pneumoniae infections (i.e. non-bacteremic pneumonia, otitis media) in adults are associated with substantial morbidity, mortality and costs.

Cost effectiveness of ulcerative colitis treatment in Germany: a comparison of two oral formulations of mesalazine.

Background: The treatment of ulcerative colitis (UC) can place a substantial financial burden on healthcare systems. The anti-inflammatory compound 5-aminosalicylic acid (5-ASA; mesalazine) is the recommended first-line treatment for patients with UC. In this analysis, the incremental cost effectiveness ratio (ICER) of two oral formulations of 5-ASA (Mezavant® and Asacol®) is examined in the treatment of patients with mild-to-moderate, active UC in Germany.

Resource usage in outpatient care and reimbursement for cystic fibrosis in Germany.

Objective: Objective of this study is to assess and evaluate resource use in outpatient treatment in Germany and to compare it with remuneration.

Methods: Outpatient treatment was evaluated in seven different centers for pediatric and adult CF patients. Data were recorded during one representative month in 2006.

Measuring economic consequences of preterm birth - Methodological recommendations for the evaluation of personal burden on children and their caregivers.

This study aims to identify the impact of a preterm birth on financial and emotional burden from the families' perspective. Additionally, a comprehensive schedule of recommendations for a sufficient evaluation of all aspects of burden is developed. Based on the results of a literature search relevant categories and sub-domains for a questionnaire covering multiple aspects of associated financial and emotional burden are identified and converted into a recommendation scheme.

Promoting vaccinations - an analysis of measures taken by German statutory health insurers.

Introduction: Prophylactic vaccinations play a significant role in health care. As a relatively cost-effective preventive measure they can help to avert transmissible diseases and thus protect not only the vaccinated individuals themselves but also those who have not been vaccinated. In order to achieve this, a high vaccination rate is necessary; for many prophylactic vaccinations this rate is not reached in Germany.

Health care costs and their predictors of inflammatory bowel diseases in Germany.

Objectives: Detailed cost studies of inflammatory bowel diseases (IBD) for Germany are limited. Aim of this study was to collect resource-use data related to IBD via a cross-sectional study, to quantify these from the perspective of the Statutory Health Insurance (SHI) and to identify cost-driving factors.

Methods: Patients with Crohn's disease (CD) or ulcerative colitis (UC) from 24 gastroenterological specialists' practices and two hospitals were enrolled in an internet-based database between March 2006 and July 2007.