[Sustainable reimbursement of the B­centres for rare diseases in Germany-status quo and solution approaches]. / Nachhaltige Vergütung der B­Zentren für Seltene Erkrankungen in Deutschland ­ Status quo und Lösungsansätze.

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.

Rare Diseases on the Internet: An Assessment of the Quality of Online Information.

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.

Regional differences of outpatient physician supply as a theoretical economic and empirical generalized linear model.

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.

Age- and gender-based comorbidity categories in general practitioner and pulmonology patients with COPD.

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.

Amortization of gene replacement therapies: A health policy analysis exploring a mechanism for mitigating budget impact of high-cost treatments.

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.

Impact of electrically assisted bicycles on physical activity and traffic accident risk: a prospective observational study.

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.

HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies.

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.

Health-Related Genetic Direct-to-Consumer Tests in the German Setting: The Available Offer and the Potential Implications for a Solidarily Financed Health-Care System.

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.

Shaping an Effective Health Information Website on Rare Diseases Using a Group Decision-Making Tool: Inclusion of the Perspectives of Patients, Their Family Members, and Physicians.

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.

Adopting Quality Criteria for Websites Providing Medical Information About Rare Diseases.

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.

Cost effectiveness of pneumococcal vaccination for infants and children with the conjugate vaccine PnC-7 in Germany.

BACKGROUND: The introduction of the conjugate vaccine PnC-7 implies that a pneumococcal vaccine is available, for the first time, which also gives children under the age of 2 years reliable protection against invasive pneumococcal infections and offers some protection against non-invasive pneumococcal infections. OBJECTIVE AND PERSPECTIVE: In the context of a multiple-period Markov model, a cost-effectiveness analysis of a recommendation for general pneumococcal vaccination in Germany for infants and children under the age of 2 years was performed from the healthcare payer, public authority and societal perspectives. DESIGN: Various published data on age-specific incidence rates, mortality rates, efficacy of the conjugate vaccine PnC-7 and treatment costs of pneumococcal infections were incorporated into a Markov model to quantify the consequences of vaccinating versus not vaccinating. RESULTS: From a German healthcare payers' perspective, general vaccination with the conjugate vaccine would redeem 51.1% of the vaccination costs due to avoidable treatment costs, whereas, from a broader point of view, the benefits, expressed in monetary terms, would exceed the cost of vaccination. The conjugate vaccine would require an investment of euro72 866 per life-year saved (discounted, healthcare payers' viewpoint). Besides this benchmark, there are further outcome measures which cannot be ignored by those deciding on a general vaccination recommendation: 450 000 preventable episodes of illness and 134 cases of sequelae which can be prevented. CONCLUSIONS: The vaccination with the conjugate vaccine PnC-7 is cost saving from a broader perspective and the results should not be ignored by policy makers in regard to a general vaccination recommendation.

Sonographic diagnosis of "acute abdomen" in children and adults.

INTRODUCTION: The acute abdomen is the main term for an at first unclear emergency situation of the abdominal cavity. The acute abdomen belongs to the three most important reasons for the admission of patients into the emergency room. Further, this illness ranks 40% of all consultations in the ambulant care sector. The acute abdomen requires an early and direct diagnosis because of its potential of having a life threatening differential diagnosis. This HTA report aimed to assess the ultrasound diagnosis of the acute abdomen considering children and adults. This will be done from a medical and economic perspective. The differential diagnosis respectively the cause of the acute abdomen binds high direct treatment costs, especially in the stationary sector. Ultrasound diagnosis is a procedure that plays a big part in the differential diagnosis process and it is widely used in practise. Other research methods of diagnosing acute abdominal illness are: clinical examinations with inspection and palpation, surgical exploration and laparocopy as well as computer tomography and x-ray examination. OBJECTIVES: The main objective of this HTA report is to assess what significance sonography should have within the examination strategy of the acute abdomen from the medical and economical view. Second, this HTA report will evaluate under which circumstances the ultrasonographic diagnosis of the acute abdomen, considering medical and economical quality classifications, is the alternative of choice to comparable diagnostic measures. METHODS: The target population this HTA report is aimed at are children and adults with acute abdomen or embedded differential diagnosis. A systematic literature search was conducted covering all relevant medical and HTA-databases. Furthermore, handsearch was conducted inside of the known data bases of HTA-institutions as well as from medical and economical journals. The following databases were searched in cooperation with DIMDI to identify relevant literature: Biosis-Previews (BA93), Biotechnobase (ET80), Cab-Health (AZ72), Elsevier Biobase (EB94), EMBASE (EM74), Embase Alert (EA08), Ethmed (ED93), Euroethics (EU93), Gerolit (GE79), Heclinet (HN69), IHTA ( HT83), IPA (IA70), MEDLINE (ME90, Medline Alert (MEOA) and SciSearch (IS74). The search parameters were orientated by the embedded differential diagnosis's as well as diagnostic measures considering the acute abdomen. The bibliographic search covered the period from 1990 to 2003. The relevant languages are English, German in connection to French. The evaluation of the information has been graduated. It must be clear that the abstract follows the topic of the acute abdomen. The as such identified literature will be structured by its methodological quality and relevance. This will be analysed and later valued after it is clear that the minimum requirements are met. RESULTS: The identified and assessed primary studies, reviews and metaanalytic studies demonstrated the medical effectiveness as well as the economical efficiency of the sonographical diagnosis for individual questions or indications. The evaluated papers of the target population 'children' demonstrated that sonography resp. ultrasound as the basic examination is recommended for children with acute abdominal pain. DISCUSSION: The identified studies have not used an extensive setting of the ultrasound diagnosis of the acute abdomen. Instead they focus only on small sections, orientated on certain indications. Blinded medical studies have not been identified. Also, there is no health-economics study that evaluated the diagnostic measures by comparing all available technologies. Altogether, the identified and assessed publications show that sonography is an alternative of choice considering particular questions in comparison to other diagnostic measures of analyzing the acute abdomen. A general deflected dominance from the sonography facing the computer tomography for instance can not be seen as the result of this structured review of the published literature. CONCLUSION: During a structured examination strategy, e.g. guidelines, sonography of children has become the diagnostic method of choice. Taking up sonography as a primary diagnostic evaluation into the guidelines of the analysis of the considered literature is recommended. The usage of already existing ultrasound systems is also recommended. Seeing that existing publications of this subject are scarce, it is not possible to prognose the economical effects of an obligatory primary sonographical diagnostic.

Cost-effectiveness of managing HIV infection.

The worldwide spread of HIV infection continues, particularly in economically vulnerable, developing and emerging countries. There is a lack of resources to supply effective prevention measures and apply highly active antiretroviral therapies. In the regions concerned, economical effects of morbidity- and mortality-associated productivity losses in those of a working age remain serious. Thus, the United Nations Program on HIV/AIDS and the World Health Organization have predicted a dramatic decrease of South Africa's and other African states' domestic product in the coming years. Therefore, with the world economical consequences from the HIV pandemic, a large political challenge in the next 10 years will arise.