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Katharina Schmidt,1 Kathrin Damm,1 Arndt Vogel,2 Heiko Golpon,3,4 Michael P Manns,2 Tobias Welte,3,4 J-Matthias Graf von der Schulenburg1,4 1Leibniz University of Hannover, Center for Health Economics Research (CHERH), Hannover, Germany; 2Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Hannover, Germany; 3Department of Pneumology, Hannover Medical School, Hannover, Germany; 4Biomedical Research in Endstage and Obstructive Lung Disease Hannover (BREATH), Member of the German Center for Lung Research (DZL), Hannover, Germany 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). Methods: Six discrete choice experiment (DCE) attributes were established on the basis of a systematic literature review and qualitative interviews. The DCE analyses comprised generalized linear mixed-effects model and latent class mixed logit model. Results: The study cohort comprised 310 patients (194 with LC, 108 with CRC, 8 with both types of cancer) with a median age of 63 (SD =10.66) years. The generalized linear mixed-effects model showed a significant (P

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. Moreover, the level of internal financing and litigation risks also seem to be high in the pharmaceutical industry. These facts could also affect the payout policy of the firms. Employing techniques of time series analysis, there is no evidence for dividend signaling and clear evidence for dividend smoothing in the European pharmaceutical industry. Given that dividend increases under certain assumptions can negatively affect the firms' ability to finance new investments in general and research and development projects in particular, these results of our empirical investigations could be described as highly plausible., (© 2022. The Author(s).)

Background: Electrically assisted bicycles (e-bikes) have become increasingly popular and may facilitate active commuting and recreational cycling., Objective: To evaluate the physical activity levels and usage characteristics of e-bikers and conventional cyclists under real-world conditions., Methods: We conducted a prospective observational study in Germany to examine the effects of e-biking compared with conventional cycling on reaching the World Health Organization (WHO) target for physical activity-at least 150 min of moderate-to-vigorous physical activity (MVPA) per week. Study participants (1250 e-bikers and 629 conventional bike users) were equipped with activity trackers to assess the time, distance and heart rate during cycling over four consecutive weeks. Questionnaires were used to assess any traffic accidents incurred over 12 months., Results: The proportion of participants reaching 150 min of MVPA per week was higher for conventional bike users than for e-bike users (35.0% vs 22.4%, p<0.001). In a multiple regression model, the odds of reaching the physical activity target were lower for e-biking than for conventional biking (OR=0.56; 95% CI 0.43 to 0.72) with age, sex, comorbidities and bike usage patterns as confounding factors. No significant differences were observed between bike groups for traffic accidents, yet when controlled for cycling time and frequency of cycling e-bikers had a higher risk of a traffic accident (OR=1.63; 95% CI 1.02 to 2.58)., Conclusion: E-bikes are associated with a lower probability of reaching WHO targets for MVPA due to reduced duration and a reduced cardiovascular effort during riding. However, e-bikes might facilitate active transportation, particularly in older individuals or those with pre-existing conditions., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: To ensure specialized care of patients with rare diseases, numerous centres for rare diseases were funded over the past few years. The reimbursement of patients' ambulatory care in hospitals, however, is characterized by a plurality of forms of care and payment. There is some evidence of deficits in the reimbursement of care of patients suffering from a rare disease from studies on individual rare diseases., Objectives: To investigate current forms of care provision and reimbursement of centres for rare diseases and to develop future approaches for sustainable compensation., Materials and Methods: Initially, centres for rare diseases in Germany were asked to provide information about their forms of care and reimbursement using questionnaires. Subsequently, two focus group interviews and one expert interview with representatives from centres for rare diseases, health insurance, health politics and patients were conducted to discuss current and future meritocratic forms of care provision and reimbursement. The data were evaluated using content analysis., Results and Conclusions: Thirty-nine centres for rare diseases participated in the questionnaire survey. Of those, 38% receive a flat fee/allowance for university outpatient departments, the amount of which varies notably, and 41% obtain a mixed payment comprising an allowance for university outpatient departments and other forms of reimbursement. An under-recovery of costs in centres for rare diseases and its impact on patient care were mentioned in the interviews. In this context, a need to further develop forms of care provision and reimbursement has been identified. Participants prefer a special flat fee/allowance for rare diseases that covers the time-consuming care for patients with rare diseases., (© 2022. The Author(s).)

Chronic obstructive pulmonary disease (COPD) is a debilitating medical condition often accompanied by multiple chronic conditions. COPD is more frequent among older adults and affects both genders. The aim of the current cross-sectional survey was to characterize chronic comorbidities stratified by gender and age among patients with COPD under the care of general practitioners (GP) and pulmonologists, using real-world patient data. A total of 7966 COPD patients (women: 45%) with more than 5 years of the observation period in the practice were examined using 60 different Chronic comorbid conditions (CCC) and Elixhauser measures. More than 9 in 10 patients had at least one, and 51.7% had more than three comorbidities. No gender difference was found in the number of comorbidities. However, men had higher Elixhauser-van Walraven index scores than women, and the types of comorbidities differed by gender. An increasing number of comorbidities was seen with aging but the patients in their 30s and 40s also had a high number of comorbidities. Moreover, GP patients had a higher number and a wider array of documented comorbidities than pulmonology patients did. Psychological comorbidities were common in all patients, but particularly among younger patients. These findings around gender- and age-stratified comorbidities under the care of GPs and pulmonologists have implications for the choice of data provenience for decision-making analysis and treatment selection and success., (© 2022. The Author(s).)

With gene replacement therapies (GRTs) increasingly and rapidly reaching the healthcare marketplace, the vast potential for improving patient health is matched by the potential budgetary impact for healthcare payers. GRTs are highly valuable given their potential life-extending or even curative benefits and may provide significant cost-offsets compared with standard of care. Current healthcare systems are, however, struggling to fund such valuable but costly therapies. Some payers have already implemented specific financing models to account for the new treatment paradigms, but these do not address the budget impact in the year of acquisition or administration of these costly technologies. This health policy analysis aimed to assess the rationale and feasibility of amortization, within the context of financing healthcare technologies, and specifically GRTs. Amortization is an accounting concept applied to intangible assets that allows for spreading the cost an intangible asset over time, allowing for repayment to occur via interest and principal payments sufficient to repay the intangible asset in full by its maturity. Our systematic scoping review on the amortization of healthcare technologies found a very small literature base with even that being unclear and inconsistent in its understanding of the issues. Where amortization was proposed as a solution for funding costly, but highly valuable GRTs, the concept was not fully investigated in detail, nor was the feasibility of the approach fully challenged. However, by providing clear definitions of relevant concepts along with an example of amortization models applied to some example GRTs, we propose that amortization can offer a promising method for funding of extraordinarily high-value healthcare technologies, thereby increasing market and patient access for these technologies. Nonetheless, healthcare accounting principles and financing guidelines must evolve to apply amortization to the rapidly developing GRTs., Competing Interests: Declaration of Competing Interest We declare no conflicts of interest in the development of this research and manuscript., (Copyright © 2021 Elsevier B.V. All rights reserved.)

This last decade has been marked by significant advances in the development of cell and gene (C&G) therapies, such as gene targeting or stem cell-based therapies. C&G therapies offer transformative benefits to patients but present a challenge to current health technology decision-making systems because they are typically reviewed when clinical efficacy data are very limited and when there is uncertainty about the long-term durability of outcomes. These challenges are not unique to C&G therapies, but they face more of these barriers, reflecting the need for adapting existing value assessment frameworks. Still, C&G therapies have the potential to be cost-effective even at very high price points. The impact on healthcare budgets will depend on the success rate of pipeline assets and on the extent to which C&G therapies will expand to wider pathologies beyond rare or ultra-rare diseases. Getting pricing and reimbursement models right is important for incentivising research and development investment while not jeopardising the sustainability of healthcare systems. Payers and manufacturers therefore need to acknowledge each other's constraints-limitations in the evidence generation on the manufacturer side, budget considerations on the payer side-and embrace innovative thinking and approaches to ensure timely delivery of therapies to patients. Several experts in health technology assessment and clinical experts have worked together to produce this publication and identify methodological and policy options to improve the assessment of C&G therapies, and make it happen better, faster and sustainably in the coming years.

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. Even though various quantitative studies have analyzed the use of different information sources for specific rare diseases, little is known about the use of information sources for different rare diseases, how users rate these information sources based on their experiences, and how the use and importance of these information sources change over time., Methods: Fifty-five patients with a variety of rare diseases and 13 close relatives participated in qualitative interviews. For these interviews, a semi-structured guideline was developed, piloted, and revised. Data analysis involved a qualitative content analysis developed by Philipp Mayring., Results: The participants considered internet as the most important and widespread information source, especially for early information. Although patients have difficulty dealing with information obtained online, they consider online searching a quick and practical option to gather information. During the course of the disease, personal contact partners, especially self-help associations and specialized doctors, become more important. This is also because information provided online is sometimes insufficiently detailed to answer their information needs, which can be complemented by information from doctors and self-help., Conclusions: People rarely use just one type of source, but rather refer to different sources and informants. The source used depends on the type of information sought as well as other person-related factors such as preexisting knowledge and the disease stage. To improve people's information searching and connect them with medical specialists in rare diseases, a central information portal on rare diseases might be a suitable access point to provide free and quality assured information for patients, caregivers, and physicians. This would allow not only patients but also doctors to find quality assured information on symptoms and therapies as well as patient associations and specialized doctors.

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. We also strive to examine how patient perspectives are methodologically elicited and presented., Methods: Our empirical evidence comes from a systematic review of orphan drug value dossiers submitted to the German Federal Joint Committee as well as the corresponding evaluations conducted between January 1, 2011 and March 1, 2019 (n = 81). Data on patient perspective integration were extracted using the following patient-reported outcome subcategories: clinical patient-reported outcomes, health-related quality of life, patient preferences, and patient satisfaction., Results: The analysis demonstrates the specific relevance of patient-reported outcomes raised as part of the medical data set and presented during the early benefit assessment process. They are predominantly presented in the form of health-related quality of life data (n = 75%) and clinical outcomes (n = 49%). Preferences (n = 2%) and satisfaction (n = 1%) are still rarely presented, although the heated methodological discussion in Germany would suggest otherwise. While various methodologies for the integration of clinical outcomes and quality of life data were found, presenting data on satisfaction and preferences still lacks methodological rigor. The German Federal Joint Committee has not yet integrated these data in their decision text. Clinical outcomes and quality of life have been included in 46% and 73% of the cases, respectively., Conclusions: The underlying analysis demonstrates that there is still a relative high potential for the regular and systematic inclusion of patient perspectives within the early benefit assessment process for rare diseases. In particular, patient preferences and patient satisfaction are still rarely included suggesting the need for a clear-cut methodological foundation and incentives.

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. Therefore, we examine the implementation of the shared decision-making concept thus far, and explore whether efficiency potentials exist-which are particularly relevant within the rare disease field-and how they can be triggered., Methods: Our empirical evidence comes from 101 interviews conducted from March to September 2014 in Germany; 55 patients, 13 family members, and 33 health professionals participated in a qualitative interview study. Transcripts were analyzed using a directed qualitative content analysis., Results: The interviews indicate that the decision-making process is increasingly relevant in practice. In comparison, however, the shared decision-making agreement itself was rarely reported. A majority of interactions are dominated by individual, informed decision-making, followed by paternalistic approaches. The patient-physician relationship was characterized by a distorted trust-building process, which is affected by not only dependencies due to the diseases' severity and chronic course, but an often-reported stigmatization of patients as stimulants. Moreover, participation was high due to a pronounced engagement of those affected, diminishing as patients' strength vanish during their odyssey through health care systems. The particular roles of "expert patients" or "lay experts" in the rare disease field were revealed, with further potential in integrating the gathered information., Conclusions: The study reveals the named efficiency potentials, which are unique for rare diseases and make the further integration of shared decision-making very attractive, facilitating diagnostics and disease management. It is noteworthy that integrating shared decision-making in the rare disease field does not only require strengthening the position of patients but also that of physicians. Efforts can be made to further integrate the concept within political frameworks to trigger the identified potential and assess the health-economic impact.

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. We extracted the key characteristics of the studies and performed a study quality assessment. We identified eight studies on the costs of illness, of which five focused on occupational lung diseases and three on occupational skin diseases. Further, eight studies calculated loss of income/compensation payments. Out of the 16 identified articles, only two reported costs directly attributable to isocyanate-induced diseases (asthma). Studies were hardly comparable because they differed substantially in their methodological approaches. Moreover, the quality assessment of the studies revealed substantial limitations. While a wide range of isocyanate-related costs was identified, consequences of isocyanate-related occupational diseases were considerable in terms of societal costs and loss of income. In most studies, indirect costs were the main cost driver. There is a need for high-quality cost of illness studies on isocyanate-induced diseases stratified by degree of severity and sex. Such studies provide valuable information to develop preventive strategies and set priorities for measures to lower the burden of professional health risks.

Competing Interests: Die vorliegende Studie wurde durch das Bundesministerium für Bildung und Forschung (BMBF) gefördert. Das Wissenschaftliche Institut der TK für Nutzen und Effizienz im Gesundheitswesen (WINEG) hat zur Aufgabe, die Wertigkeit von Innovationen und neuen programmatischen Ansätzen innerhalb der GKV zu hinterfragen. Die Autoren Prof. Dr. Roland Linder und Dr. Frank Verheyen erklären, dass aufgrund ihrer Zugehörigkeit zur Techniker Krankenkasse ein potenzieller Interessenkonflikt im Sinne der Richtlinien des International Committee of Medical Journal Editors besteht.

[This corrects the article DOI: 10.2196/resprot.7425.]., (©Svenja Litzkendorf, Tobias Hartz, Jens Göbel, Holger Storf, Frédéric Pauer, Ana Babac, Verena Lührs, Leena Bruckner-Tuderman, Franziska Schauer, Jörg Schmidtke, Lisa Biehl, TOF Wagner, J-Matthias Graf von der Schulenburg, Martin Frank. Originally published in JMIR Research Protocols (http://www.researchprotocols.org), 19.09.2018.)

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., Methods: Medications withdrawn from the German market between January 2011 and June 2016 following benefit assessment were categorized as opt-outs (max. 2 weeks after start of price negotiations) or supply terminations (during or after further price negotiations). Related guidelines were systematically analyzed. For all withdrawals, therapeutic area, additional benefit rating and recommendation status in relevant clinical guidelines were assessed., Results: Among 139 medications, 10 opt-outs and 12 supply terminations were identified. Twenty-one out of 22 withdrawn medicines (95%) received 'no additional benefit' appraisal by the G-BA (average 'no additional benefit' rating for all AMNOG products: 47%). Of the 22 medicines, 15 (68%) were recommended by at least one guideline at the time of benefit assessment and 18 (82%) on 1 June 2016. Heterogeneity among guidelines was high. Acceptance of clinical trial endpoints was different between G-BA appraisals and clinical guidelines., Conclusion: Our analysis revealed considerable differences across clinical guidelines as well as between clinical guidelines and HTA appraisals of the medicines that were withdrawn from the German market. Better alignment of the clinical perspective and close collaboration between all involved parties is required to achieve and maintain optimization of patient care.

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., Methods: A representative sample of the general German population (n = 1158) was interviewed using a composite time trade-off and a discrete choice experiment under close quality control. Econometric modeling was used to estimate values for all 3125 possible health states described by EQ-5D-5L. The value set was based on a hybrid model including all available information from the composite time trade-off and discrete choice experiment valuations without any exclusions due to data issues., Results: The final German value set was constructed from a combination of a conditional logit model for the discrete choice experiment data and a censored at -1 Tobit model for the composite time trade-off data, correcting for heteroskedasticity. The value set had logically consistent parameter estimates (p < 0.001 for all coefficients). The predicted EQ-5D-5L index values ranged from -0.661 to 1., Conclusions: This study provided values for the health states of the German version of EQ-5D-5L representing the preferences of the German population. The study successfully employed for the first time worldwide the improved protocol 2.0. The value set enables the use of the EQ-5D-5L instrument in economic evaluations and in clinical studies.

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)., Objective: The objective of this study, therefore, was to conduct scientific research on how such a portal must be designed to meet the needs of patients, their families, and medical professionals, and to provide high-quality information for information seekers., Methods: We chose a 3-step procedure to develop a needs-based prototype of a central information portal. In the first step, we determined the information needs of patients with rare diseases, their relatives, and health care professionals by means of qualitative interviews and their content-analytical evaluation. On the basis of this, we developed the basic structure of the portal. In the second step, we identified quality criteria for websites on rare diseases to ensure that the information linked with ZIPSE meets the quality demands. Therefore, we gathered existing criteria catalogs and discussed them in an expert workshop. In the third step, we implemented and tested the developed prototypical information portal., Results: A portal page was configured and made accessible on the Web. The structure of ZIPSE was based on the findings from 108 qualitative interviews with patients, their relatives, and health care professionals, through which numerous information needs were identified. We placed particularly important areas of information, such as symptoms, therapy, research, and advisory services, on the start page. Moreover, we defined 13 quality criteria, referring to factors such as author information, creation date, and privacy, enabling links with high-quality information. Moreover, 19 users tested all the developed routines based on usability and comprehensibility. Subsequently, we improved the visual presentation of search results and other important search functions., Conclusions: The implemented information portal, ZIPSE, provides high-quality information on rare diseases from a central point of access. By integrating the targeted groups as well as different experts on medical information during the construction, the website can assure an improved search for information for users. ZIPSE can also serve as a model for other Web-based information systems in the field of rare diseases., Registered Report Identifier: RR1-10.2196/7425., (©Svenja Litzkendorf, Tobias Hartz, Jens Göbel, Holger Storf, Frédéric Pauer, Ana Babac, Verena Lührs, Leena Bruckner-Tuderman, Franziska Schauer, Jörg Schmidtke, Lisa Biehl, TOF Wagner, J-Matthias Graf von der Schulenburg, Martin Frank. Originally published in JMIR Research Protocols (http://www.researchprotocols.org), 11.05.2018.)

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. Therefore, the improvement of health information provision has been integrated in many national plans for rare diseases, discussing the telephone as one access option. In this context, this study examines the need for a telephone service offering information for people affected by rare diseases, their relatives, and physicians., Methods: In total, 107 individuals participated in a qualitative interview study conducted in Germany. Sixty-eight individuals suffering from a rare disease or related to somebody with rare diseases and 39 health care professionals took part. Individual interviews were conducted using a standardized semi-structured questionnaire. Interviews were analysed using the qualitative content analysis, triangulating patients, relatives, and health care professionals. The fulfilment of qualitative data processing standards has been controlled for., Results: Out of 68 patients and relatives and 39 physicians, 52 and 18, respectively, advocated for the establishment of a rare diseases telephone service. Interviewees expected a helpline to include expert staffing, personal contact, good availability, low technical barriers, medical and psychosocial topics of counselling, guidance in reducing information chaos, and referrals. Health care professionals highlighted the importance of medical topics of counselling-in particular, differential diagnostics-and referrals., Conclusions: Therefore, the need for a national rare diseases helpline was confirmed in this study. Due to limited financial resources, existing offers should be adapted in a stepwise procedure in accordance with the identified attributes.

Background: Despite diverging definitions on rare conditions, people suffering from rare diseases share similar difficulties. A lack of experience by health professionals, a long wait from first symptoms to diagnosis, scarce medical and scientific knowledge, and unsatisfactory treatment options all trigger the search for health information by patients, family members, and physicians. Examining and systematically integrating stakeholder needs can help design information platforms that effectively support this search., Objective: The aim of this study was to innovate on the group decision-making process involving patients, family members, and physicians for the establishment of a national rare disease Internet platform. We determined differences in the relevance of health information-especially examining quantifiable preference weights-between these subgroups and elucidated the structure and distribution of these differences in people suffering from rare diseases, their family members, and physicians, thus providing information crucial to their collaboration., Methods: The included items were identified using a systematic Internet research and verified through a qualitative interview study. The identified major information needs included medical issues, research, social help offers, and current events. These categories further comprised sublevels of diagnosis, therapy, general disease pattern, current studies, study results, registers, psychosocial counseling, self-help, and sociolegal advice. The analytic hierarchy process was selected as the group decision-making tool. A sensitivity analysis was used to determine the stability and distribution of results. t tests were utilized to examine the results' significance., Results: A total of 176 questionnaires were collected; we excluded some questionnaires in line with our chosen consistency level of 0.2. Ultimately, 120 patients, 24 family members, and 32 physicians participated in the study (48 men and 128 women, mean age=48 years, age range=17-87 years). Rankings and preference weights were highly heterogeneous. Global ranking positions of patients, family members, and physicians are shown in parentheses, as follows: medical issues (3/4, 4, 4), research (3/4, 2/3, 3), social help offers (1, 2/3, 2), and current events (2, 1, 1); diagnosis (6, 8, 9), therapy (5, 9, 7), general disease pattern (9, 4/5/6, 6), current studies (7, 4/5/6, 3), study results (8, 7, 8), registers (4, 1, 5), psychosocial counseling (1, 2, 4), self-help (3, 3, 2), and sociolegal advice (2, 4/5/6, 1). Differences were verified for patients for 5 information categories (P=.03), physicians for 6 information categories (P=.03), and family members for 4 information categories (P=.04)., Conclusions: Our results offer a clear-cut information structure that can transparently translate group decisions into practice. Furthermore, we found different preference structures for rare disease information among patients, family members, and physicians. Some websites already address differences in comprehension between those subgroups. Similar to pharmaceutical companies, health information providers on rare diseases should also acknowledge different information needs to improve the accessibility of information., (©Ana Babac, Svenja Litzkendorf, Katharina Schmidt, Frédéric Pauer, Kathrin Damm, Martin Frank, Johann-Matthias Graf von der Schulenburg. Originally published in the Interactive Journal of Medical Research (http://www.i-jmr.org/), 20.11.2017.)

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., Objectives: The needs of patients and relatives regarding online-based information are analyzed. Based on this, we examined to what extent the information basis, which is available according to the ZIPSE quality criteria, can cover these needs. If necessary, measures have to be developed to ensure quality- as well as needs-oriented information management., Materials and Methods: Qualitative interviews with patients and relatives were conducted, which were then evaluated using content analysis. Subsequently, a quantitative evaluation of the information on rare diseases in the portal was made. The research addresses how many websites do not fulfil the quality criteria, from which group of provider these websites originate and which criteria are not fulfilled. This is followed by a comparison of the quantitative and qualitative results., Results and Conclusions: When looking for information on the Internet, the websites of self-help groups represent a significant source. These are perceived as very trustworthy and in the later course of the disease, offer detailed information on important information areas. Information websites from self-help groups, however, often do not meet quality requirements. Therefore, a transparent representation is made regarding the quality of the ZIPSE information pages. Pages that are not quality-assured can be actively requested, but will be clearly identified.

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., Methods: A cost analysis of WGS based on a quality-assured process chart for WGS at the German Cancer Research Center (DKFZ), Heidelberg, constitutes the basis for this evaluation. Data from the National Association of Statutory Health Insurance Funds and the Robert-Koch-Institute, Berlin, were used for calculations of specific clinical applications., Results and Discussion: WGS in newborn screening leads to costs of € 2.85 bn and to an increase of total expenditure by 1.41%. Sequencing of all tumor patients would cost approximately € 0.84 bn, which corresponds to 0.42% of total expenditures. In all scenarios, the sole consideration of procedure costs results in increasing costs. However, in cost discussions potential savings (reduction of disease-related follow-up-costs, improved cost-effectiveness of medical measures etc.) should be considered. Such considerations are the subject of economic indication-specific evaluations. WGS has the potential to generate a large number of deterministic findings for which treatment options are limited. Hence, it is necessary to limit indications, in which WGS has proven medical evidence.

Background: The importance of the Internet as a medium for publishing and sharing health and medical information has increased considerably during the last decade. Nonetheless, comprehensive knowledge and information are scarce and difficult to find, especially for rare diseases. Additionally, the quality of health or medical information about rare diseases is frequently difficult to assess for the patients and their family members., Objective: The aim of this study is to assess the quality of information on the Internet about rare diseases. Additionally, the study aims to evaluate if the quality of information on rare diseases varies between different information supplier categories., Methods: A total of 13 quality criteria for websites providing medical information about rare diseases were transferred to a self-disclosure questionnaire. Identified providers of information on the Internet about rare diseases were invited to fill out the questionnaire. The questionnaire contained questions about the information provider in general (eg, supplier category, information category, language, use of quality certificates, and target group) and about quality aspects that reflect the 13 quality criteria. Differences in subgroup analyses were performed using t tests., Results: We identified 693 websites containing information about rare diseases. A total of 123 questionnaires (17.7%) were completely filled out by the information suppliers. For the remaining identified suppliers (570/693, 82.3%), the questionnaires were filled out by the authors based on the information available on their website. In many cases, the quality of websites was proportionally low. Furthermore, subgroup analysis showed no statistically significant differences between the quality of information provided by support group/patient organization compared to medical institution (P=.19). The quality of information by individuals (patient/relative) was significantly lower compared to information provided by support group/patient organization (P=.001), medical institution (P=.009), and other associations and sponsoring bodies (P=.001) as well., Conclusions: Overall, the quality of information on the Internet about rare diseases is low. Quality certificates are rarely used and important quality criteria are often not fulfilled completely. Additionally, some information categories are underrepresented (eg, information about psychosocial counseling, social-legal advice, and family planning). Nevertheless, due to the high amount of information provided by support groups, this study shows that these are extremely valuable sources of information for patients suffering from a rare disease and their relatives., Competing Interests: Conflicts of Interest: None declared., (©Frédéric Pauer, Svenja Litzkendorf, Jens Göbel, Holger Storf, Jan Zeidler, Johann-Matthias Graf von der Schulenburg. Originally published in the Journal of Medical Internet Research (http://www.jmir.org), 18.01.2017.)

Background: The global genetic direct-to-consumer (DTC) market will reach a volume of USD 230 billion in 2018. The expenditures for this genetic analysis are borne by the customer, whereas consequential costs may arise for a solidarily financed system. In a first step, it is essential to gain an overview of the currently available offer in the German setting., Methods: In April 2016, we conducted a systematic internet search in the Google search engine. In November 2016, we updated the information of the webpages in terms of country, language, types of health-related tests, additional offer of non-health-related DTC test, information about sensitivity and specificity, certification and accreditation, costs as well as reference to German Act on Genetic Testing., Results: Thirty-five webpages were included in the final overview. A plurality of different predictive analysis options was identified. Price information was not available for all offered genetic analyses. Costs for predictive analysis in one disease vary between EUR 90 and 990, for predictive package analysis between EUR 232.18 and 375, and for genetic lifestyle analysis between EUR 84.55 and 570.20., Conclusions: Genetic results may lead to uncertainty and anxiety; therefore, subsequent costs for a solidarily financed system may arise. Genetic DTC tests may have an influence on different players on the micro-, meso- and macro-levels, which may have a cost-cutting or cost-increasing effect on health-care expenditures. The increased interest in genetic analysis as well as the possibility of worldwide internet-based access to genetic tests requires population-wide education., (© 2017 S. Karger AG, Basel.)

Background: Lung cancer is one of the most common types of cancer worldwide, and it causes significant challenges for patients due to the poor survival rate and treatment-related side-effects. Because of lung cancer's great burden, identification and use of the patients' preferences can help to improve patients' quality of life., Objective: Interviews with patients who have lung cancer were used to ascertain a range of experiences and to make recommendations regarding the improvement of treatment based on these patients' preferences. Because chemotherapy is the common treatment option for lung cancer, we focused on this treatment. The interviews were audio-taped, verbally transcribed and evaluated via content analysis., Setting and Participants: A total of 18 participants (11 men and 7 women) with small or non-small-cell lung cancer who were receiving chemotherapy in one clinic were interviewed between June and July 2013., Results: Two main aspects with different subthemes were identified during the interviews. One main aspect focused on organizational context, such as the treatment day process, or experiences with different stakeholders, such as with the health insurance company or physicians. The other category referred to experiences that influenced psychosocial factors, including physical and mental experiences., Discussion and Conclusion: Patients reported different experiences concerning physical, psychological and organizational areas during chemotherapy. Nevertheless, some potential areas for improving care, and therefore the quality of life of patients with lung cancer, could be identified. These improvement measures highlighted that with small, non-time-consuming and inexpensive changes, the treatment for patients with lung cancer can be improved., (© 2015 The Authors. Health Expectations Published by John Wiley & Sons Ltd.)

Aim of the Study: Through the promotion of smoking cessation premature mortality can be prevented. Therefore it is necessary to provide effective and cost-effective smoking cessation interventions. In Germany the cost of pharmacological smoking cessation measures are not yet reimbursed by the statutory health insurance. The aim of this study is to present the evidence on the cost-effectiveness of already approved pharmacological smoking cessation therapies and to evaluate their quality. Method: A systematic literature research was conducted in the databases by DIMDI (Medline, Embase, etc.) in April 2013 (update April 2014). The study research was focused on studies for cost effectiveness of nicotine replacement therapy (NRT), varenicline and bupropion. The assessment of study quality was performed using the "Quality of Health Economic Studies" (QHES) instrument. Results: 33 Of the original 10 340 identified studies were finally included. The majority of the studies show that an additional prescription of NRT or bupropion to a medical consultation is a cost-effective strategy. In addition, in most studies varenicline is a dominant strategy compared to bupropion. Overall, the study quality was found to be very heterogeneous between 45 and 80 points (Ø 63.7 points). Conclusion: The studies show that treatment with varenicline is the most cost effective strategy followed by bupropion and the NRT. However, the studies can only be compared inadequately due to different levels of age and country-specific intervention costs., (© Georg Thieme Verlag KG Stuttgart · New York.)

Background: The European Union considers diseases to be rare when they affect less than 5 in 10,000 people. It is estimated that there are between 5000 and 8000 different rare diseases. Consistent with this diversity, the quality of information available on the Web varies considerably. Thus, quality criteria for websites about rare diseases are needed., Objective: The objective of this study was to generate a catalog of quality criteria suitable for rare diseases., Methods: First, relevant certificates and quality recommendations for health information websites were identified through a comprehensive Web search. Second, all considered quality criteria of each certification program and catalog were examined, extracted into an overview table, and analyzed by thematic content. Finally, an interdisciplinary expert group verified the relevant quality criteria., Results: We identified 9 quality certificates and criteria catalogs for health information websites with 304 single criteria items. Through this, we aggregated 163 various quality criteria, each assigned to one of the following categories: thematic, technical, service, content, and legal. Finally, a consensus about 13 quality criteria for websites offering medical information on rare diseases was determined. Of these categories, 4 (data protection concept, imprint, creation and updating date, and possibility to contact the website provider) were identified as being the most important for publishing medical information about rare diseases., Conclusions: The large number of different quality criteria appearing within a relatively small number of criteria catalogs shows that the opinion of what is important in the quality of health information differs. In addition, to define useful quality criteria for websites about rare diseases, which are an essential source of information for many patients, a trade-off is necessary between the high standard of quality criteria for health information websites in general and the limited provision of information about some rare diseases. Finally, transparently presented quality assessments can help people to find reliable information and to assess its quality., Competing Interests: Conflicts of Interest: None declared.

Background: In the European Union, about 30 million people are affected by one of the 7,000 to 8,000 diseases being defined as rare. In Germany alone, an estimated 4 million people suffer from a rare disease. In many cases, therapeutic options and knowledge of specific rare diseases are strongly limited., Objective: The aim of this study was to identify the deficits and challenges confronting healthcare services for people suffering from a rare disease from the medical professional's perspective., Method: As many as 530 medical professionals were invited to complete an online questionnaire, which was also available on the website of the General Medical Council of Lower Saxony. The questionnaire focused on questions in the following fields: structure of the medical care system; diagnosis and therapy; information sources and information exchange; and improvement of healthcare situation. Data were analyzed using IBM SPSS 22., Result: We received 65 completed questionnaires. The evaluation indicates deficits in the medical services provided for people with a rare disease and shortcomings in the communication between clinical disciplines. In addition, diagnostic and therapeutic options are limited, and quality-tested information is rare., Conclusion: Many of the identified deficits have already been addressed in the German national plan of action for people affected by rare diseases. Furthermore, newly discovered deficits have been evaluated. The German government implemented healthcare structures to improve healthcare services for people with rare diseases. However, budget deficits for specialized structures have occurred inhibiting the expansion of healthcare services. Moreover, many patients need systemic treatment requiring the further development of interdisciplinary care., (Copyright © 2016. Published by Elsevier GmbH.)

Background: Regional differences in physician supply can be found in many health care systems, regardless of their organizational and financial structure. A theoretical model is developed for the physicians' decision on office allocation, covering demand-side factors and a consumption time function., Methods: To test the propositions following the theoretical model, generalized linear models were estimated to explain differences in 412 German districts. Various factors found in the literature were included to control for physicians' regional preferences., Results: Evidence in favor of the first three propositions of the theoretical model could be found. Specialists show a stronger association to higher populated districts than GPs. Although indicators for regional preferences are significantly correlated with physician density, their coefficients are not as high as population density., Conclusions: If regional disparities should be addressed by political actions, the focus should be to counteract those parameters representing physicians' preferences in over- and undersupplied regions.

Background: Decision-analytic modelling (DAM) has become a widespread method in health technology assessments (HTA), but the extent to which modelling is used differs among international HTA institutions. In Germany, the use of DAM is optional within HTAs of the German Institute of Medical Documentation and Information (DIMDI). Our study examines the use of DAM in DIMDI HTA reports and its effect on the quality of information provided for health policies., Methods: A review of all DIMDI HTA reports (from 1998 to September 2012) incorporating an economic assessment was performed. All included reports were divided into two groups: HTAs with DAM and HTAs without DAM. In both groups, reports were categorized according to the quality of information provided for healthcare decision making., Results: Of the sample of 107 DIMDI HTA reports, 17 (15.9%) used DAM for economic assessment. In the group without DAM, conclusions were limited by the quality of economic information in 51.1% of the reports, whereas we did not find limited conclusions in the group with DAM. Furthermore, 24 reports without DAM (26.7%) stated that using DAM would likely improve the quality of information of the economic assessment., Conclusion: The use of DAM techniques can improve the quality of HTAs in Germany. When, after a systematic review of existing literature within a HTA, it is clear that DAM is likely to positively affect the quality of the economic assessment DAM should be used.