Dividend policy issues in the European pharmaceutical industry: new empirical evidence.

This paper examines dividend policy issues in the European pharmaceutical industry. This sector is of particular interest because of the high research and development expenditures and the associated risks characterizing the business models of many firms in this industry. In fact, from the perspective of corporate finance theory, this is a particular challenge for the managers of these corporations that may also have implications for the dividend policy implemented by the firms forming this sector.

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.

Use and importance of different information sources among patients with rare diseases and their relatives over time: a qualitative study.

Background: Finding reliable information on one of more than 7000 rare diseases is a major challenge for those affected. Since rare diseases are defined only by the prevalence criterion, a multitude of heterogeneous diseases are included. Common to all, however, are difficulties regarding information access.

Patient-reported data informing early benefit assessment of rare diseases in Germany: A systematic review.

Background: Since the implementation of the Regulation on Patient Integration (2003), the Act on the Reorganization of the Pharmaceutical Market (2011), and the Patient Rights Law (2013), the inclusion of patient perspectives has been further anchored in the German early benefit assessment process. During the assessment of rare disease interventions, patient perspectives are particularly important, as clinical studies are often designed acknowledging small samples and patients suffering from severe symptoms and the chronic course of the disease. Therefore, our research question is whether patient perspectives are considered as part of early benefit assessments for rare diseases.

Integrating patient perspectives in medical decision-making: a qualitative interview study examining potentials within the rare disease information exchange process in practice.

Background: Many European countries have recently implemented national rare disease plans. Although the network is strengthening, especially on the macro and meso levels, patients still go a long way through healthcare systems, with many health professionals involved and scarce evidence to gather. Specifically, patient involvement in the form of shared decision-making can offer further potential to increase healthcare systems' efficiency on a micro level.

Costs of isocyanate-related occupational diseases: A systematic review.

Although isocyanates are increasingly used in manufacturing and workplace exposure to isocyanates is widely recognized as one of the most frequent causes for occupational lung and skin diseases, little is known about the economic burden on the affected individual and the society. This study provides an overview on costs of occupational diseases related to isocyanates. We performed a systematic literature search of studies in the electronic databases of the German Institute of Medical Documentation and Information, and the Canadian Centre for Occupational Health and Safety.

Metadata Correction: Conceptualization and Implementation of the Central Information Portal on Rare Diseases: Protocol for a Qualitative Study.

[This corrects the article DOI: 10.2196/resprot.7425.

"Market withdrawals" of medicines in Germany after AMNOG: a comparison of HTA ratings and clinical guideline recommendations.

Background: According to the AMNOG act, the German Federal Joint Committee (G-BA) determines the additional benefit of new medicines as a basis for subsequent price negotiations. Pharmaceutical companies may withdraw their medications from the market at any time during the process. This analysis aims to compare recommendations in clinical guidelines and HTA appraisals of medicines that were withdrawn from the German market since the introduction of AMNOG in 2011.

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.

Conceptualization and Implementation of the Central Information Portal on Rare Diseases: Protocol for a Qualitative Study.

Background: Recently, public and political interest has focused on people living with rare diseases and their health concerns. Due to the large number of different types of rare diseases and the sizable number of patients, taking action to improve the life of those affected is gaining importance. In 2013, the federal government of Germany adopted a national action plan for rare diseases, including the call to establish a central information portal on rare diseases (Zentrales Informationsportal über seltene Erkrankungen, ZIPSE).

German Value Set for the EQ-5D-5L.

Objectives: The objective of this study was to develop a value set for EQ-5D-5L based on the societal preferences of the German population. As the first country to do so, the study design used the improved EQ-5D-5L valuation protocol 2.0 developed by the EuroQol Group, including a feedback module as internal validation and a quality control process that was missing in the first wave of EQ-5D-5L valuation studies.

Telephone health services in the field of rare diseases: a qualitative interview study examining the needs of patients, relatives, and health care professionals in Germany.

Background: Rare diseases are, by definition, very serious and chronic diseases with a high negative impact on quality of life. Approximately 350 million people worldwide live with rare diseases. The resulting high disease burden triggers health information search, but helpful, high-quality, and up-to-date information is often hard to find.

Therapy preferences of patients with lung and colon cancer: a discrete choice experiment.

Objectives: There is increasing interest in studies that examine patient preferences to measure health-related outcomes. Understanding patients' preferences can improve the treatment process and is particularly relevant for oncology. In this study, we aimed to identify the subgroup-specific treatment preferences of German patients with lung cancer (LC) or colorectal cancer (CRC).

[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.

[Priority setting and rationing of pharmaceuticals - an experimental analysis of discussion processes].

Background/objective: In the face of rising expenditure among statutory sickness funds in Germany it is necessary to start a discussion about priority setting in the healthcare system. For a long time this issue has been avoided in healthcare debates. As a result, normative directives are still missing, which can lead to priority setting among healthcare providers in daily healthcare practice.

[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.

[Central information portal on rare diseases : Implementation of quality- and needs-oriented information management].

Background: A central information portal on rare diseases (ZIPSE) has been conceptualized and implemented that allows patients, relatives and health care professionals to access quality-assured information. For this purpose, quality criteria have been developed specifically for rare diseases. At the same time, the information basis should take into account the specific needs of those interested.

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.

[Whole-genome sequencing in German clinical practice : Economic impacts of its use in selected areas of application].

Background: The diagnostic use of whole-genome sequencing (WGS) is a growing issue in medical care. Due to limited resources in public health service, budget-impact analyses are necessary prior to implementation.

Objective: A budget-impact analysis for WGS of all newborns and diagnostic investigation of tumor patients in different oncologic indications were evaluated.

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).