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. 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.

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. Although families caring for children with rare diseases are known to be at risk for mental disorders, the evaluation of special programs under high methodological standards has not been conducted so far. Moreover, the implementation of interventions for this group into regular care has not yet been accomplished in Germany. The efficacy and cost-effectiveness of a family-based intervention will be assessed. METHODS/DESIGN: The study is a 2x2 factorial randomized controlled multicenter trial conducted at 17 study centers throughout Germany. Participants are families with children and adolescents affected by a rare disease aged 0 to 21 years. Families in the face-to-face intervention CARE-FAM, online intervention WEP-CARE or the combination of both will be treated over a period of roughly 6 months. Topics discussed in the interventions include coping, family relations, and social support. Families in the control condition will receive treatment as usual. The primary efficacy outcome is parental mental health, measured by the Structured Clinical Interview for DSM-IV (SCID-I) by blinded external raters. Further outcomes will be assessed from the parents' as well as the children's perspective. Participants are investigated at baseline, 6, 12 and 18 months after randomization. In addition to the assessment of various psychosocial outcomes, a comprehensive health-economic evaluation will be performed. DISCUSSION: This paper describes the implementation and evaluation of two family-based intervention programs for Children Affected by Rare Disease and their Family's Network (CARE-FAM-NET) in German standard care. A methodologically challenging study design is used to reflect the complexity of the actual medical care situation. This trial could be an important contribution to the improvement of care for this highly burdened group. TRIAL REGISTRATION: German Clinical Trials Register: DRKS00015859 (registered 18 December 2018) and ClinicalTrials.gov : NCT04339465 (registered 8 April 2020). Protocol Version: 15 August 2020 (Version 6.1). Trial status: Recruitment started on 1 January 2019 and will be completed on 31 March 2021.

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. 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.

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. 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.

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. 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.

[Health Science Research with Primary Care Routine Data From Electronic Patient Records: the BeoNet Registry]. / Gesundheitswissenschaftliche Forschung mit primärärztlichen Routinedaten der elektronischen Patientenakte: das BeoNet-Register.

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. The BNR is moving through pilot- and roll-out phases. Participating practices provide standardized information about (1) master data (2) practice profile (3) infrastructure and documentation systems. The routinely documented data will be pseudonymized before transmission via standardized secure interfaces to the registry database. Practices can participate in studies in cooperation with BNR if informed consent is obtained from the patients. The registry data will be linked with questionnaires on health care utilization, quality of life and disease-specific parameters. Researchers and physicians can access quasi anonymized data in specific datasets extracted from the registry. Regular automatic data quality checks and feedback from GPs, patients and researchers enhance the quality of the registry. Performance and data analysis are continuously optimized. The data security concept has been approved and ethical approval is on hand. GPs, pediatricians and pneumologists from Hanover, Munich and Heidelberg participated in the pilot phase. The registry database currently holds 98497 patient IDs and related information. BNR contains more than the standard billing data collected by health insurers. The data are applicable to a wide range of health practices, health care and economics and disease development questions. Questionnaires have been generated, tested and are being distributed. First descriptive analyses of prevalence, age and gender have been performed. Quality and validity checks have been implemented. 2016 marks the roll-out phase of the BNR. Further recruitment of practices throughout Germany, data linkage with additional questionnaires, recruitment of patients for health care research projects, quality checks and data analysis of disease-specific costs and health-related quality of life in COPD are progressing with the overall aim of improving pulmonary healthcare strategies. The floor is now open for retro- and prospective cross-sectional and longitudinal studies.

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. Based on the experiences of the first wave of valuation studies applying the protocol, a number of modifications to the implementation of composite time trade-off (cTTO) were proposed and tested in an exploratory study in Germany. METHODS: The aim of the study is to test the improved EQ-5D-5L valuation protocol 1.1 and the implementation of three modifications: (1) introduction of ranking task, (2) separating time trade-off (TTO) tasks for health states "Better Than Dead"(BTD)/"Worse Than Dead" (WTD), (3) allow for removal of problematic valuations from the cTTO data (the feedback module). Data were collected in computer assisted personal interviews with 200 members of the German general public. RESULTS: In comparison to the first wave of valuation studies a higher data quality can be observed in both study arms: increasing number of WTD valuations, reduced inconsistencies for health state 55555 as well as higher values for mild health states. Comparing both study arms, mean observed cTTO value for severity 6 is higher in the test arm. The proportion of inconsistent cTTO responses is lower in the test arm than in the control arm and is further reduced by the feedback module. The ranking task prolongs the interview without the desired effect. CONCLUSIONS: Both study arms yielded higher data quality in comparison to the first wave of EQ-5D-5L valuation studies. The valuation protocol combined with an intensive interviewer training and close data monitoring showed a high feasibility and acceptability to the respondents of the general population as well as the interviewers in Germany. Based on the results of this study and other countries, the separation of TTO tasks for health states BTD/WTD and the feedback module will be implemented in the valuation study for the EQ-5D-5L for Germany.

[Whole-genome sequencing in German clinical practice : Economic impacts of its use in selected areas of application]. / Ganzgenomsequenzierung in der deutschen Versorgung : Ökonomische Auswirkungen eines Einsatzes in ausgewählten Anwendungsgebieten.

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.

[Central information portal on rare diseases : Implementation of quality- and needs-oriented information management]. / Zentrales Informationsportal über seltene Erkrankungen : Umsetzung eines qualitäts- und bedarfsorientierten Informationsmanagements.

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.

Treatment-related experiences and preferences of patients with lung cancer: a qualitative analysis.

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.

[Impact of guideline adherence on mortality in treatment of left heart failure]. / Auswirkung einer leitliniengerechten Behandlung auf die Mortalität bei Linksherzinsuffizienz.

BACKGROUND: The German national guidelines on chronic heart failure provide treatment recommendations to physicians and reflect the current level of evidence; however, it is questionable to what extent these recommendations are applied in the routine practice and what the effect of guideline adherence on mortality is. METHODS: In this study the claims data of a major German health insurance fund collected over a period of 4 years were analyzed. Using binary logistic regression and Cox regression analyses the influence of drug prescriptions, diagnostic measures, influenza vaccination, the New York Heart Association (NYHA) status, the age and gender on mortality were examined. RESULTS: The study population consisted of 85,465 heart failure patients. Approximately 60 % of the drugs were prescribed according to the guidelines. There was a positive correlation between a higher NYHA status and mortality with an odds ratio (OR) of 3.264. Especially pharmacotherapy with angiotensin-converting enzyme (ACE) inhibitors and beta blockers according to the guidelines was associated with a lower mortality rate (OR 0.448 resp. 0.444). Also patients diagnosed using echocardiography at regular intervals showed a lower risk of dying (OR 0.314). CONCLUSION: The results of this large sample could confirm the results of clinical trials that a therapy according to the guidelines has a significant impact on mortality. By analyzing the claims data evidence was found that in the treatment of heart failure patients the medical results could be improved by adherence to guideline recommendations.

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. The ranking of information needs is then used to display information categories on a web-based information portal about rare diseases according to the patient's priorities. METHODS: The information needs of patients suffering from rare diseases were identified by an Internet research study and a preliminary qualitative study. Hence, we designed a three-level hierarchy containing 13 criteria. For data acquisition, the differences in outcomes were investigated using individual versus group judgements separately. Furthermore, we analyzed the different effects when using the median and arithmetic and geometric means for data aggregation. A consistency ratio ≤0.2 was determined to represent an acceptable consistency level. RESULTS: Forty individual and three group judgements were collected from patients suffering from a rare disease and their close relatives. The consistency ratio of 31 individual and three group judgements was acceptable and thus these judgements were included in the study. To a large extent, the local ranks for individual and group judgements were similar. Interestingly, group judgements were in a significantly smaller range than individual judgements. According to our data, the ranks of the criteria differed slightly according to the data aggregation method used. CONCLUSIONS: It is important to explain and justify the choice of an appropriate method for data acquisition because response behaviors differ according to the method. We conclude that researchers should select a suitable method based on the thematic perspective or investigated topics in the study. Because the arithmetic mean is very vulnerable to outliers, the geometric mean and the median seem to be acceptable alternatives for data aggregation. Overall, using the AHP to identify patient priorities and enhance the user-friendliness of information websites offers an important contribution to medical informatics.

[Cost-Effectiveness of Pharmacological Smoking Cessation Therapies - A Systematic Literature Review]. / Kosteneffektivität von pharmakologischen Raucherentwöhnungsmaßnahmen ­ ein systematisches Literaturreview.

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.

[Age- and Gender-specific Costs as Well as Drug Therapies of ADHD Patients]. / Alters- und geschlechtsspezifische Kosten sowie die Versorgung mit medikamentösen Therapien von ADHS-Patienten.

UNLABELLED: Backround and Objectives: Attention deficit hyperactivity disorder (ADHD) is one of the most commonly diagnosed mental disorders in children and adolescents. The rate of persistence into adulthood varies up to 60% and shows the importance of the disease. Here we present age-stratified cost information on adult patients, as well as data on occupational therapy, medication and multimodal treatment. Furthermore, we also investigated retrospectively if methylphenidate was prescribed for adults already before its approval in 2011. METHODS: Claims data of a major German insurance fund (Techniker Krankenkasse) was available. Inclusion criteria were patients with ADHD diagnosis, either hospitalized or treated on an outpatient basis in 2006, 2007 and 2008 and insured over this period. This enabled identifying this disease as chronic. The data were analyzed as part of a control group design (1:3). Cost differences were examined as also the odds ratios for the burden of comorbidities and use of atomoxetine and methylphenidate. RESULTS: 77.9% of the identified ADHD patients were male (mean age: 16.5 years ±11.1). The mean total costs of patient treatment were € 2,032 (±4,112). The odds ratio was highest for the indication developmental disorders of scholastic skills (15.4) and differed between the sexes (female: 24.0 vs. male: 14.2). Drug prescription was higher in male than in female patients (atomoxetine: 7.2 vs. 5.9% and methylphenidate 59.1 vs. 48.4%). CONCLUSIONS: This study provides important insights into the importance of the adult ADHD collective. There were increasing resource consumption identified in adult ADHD patients. In addition, methylphenidate was used off-label for treating adults already before 2011 and its approval in 2011 provided increased certainty for physicians regarding prescription of this drug.

Suitability and Usability of Claims Data for Review of Guidelines for the Treatment of Chronic Heart Failure.

Background: Guidelines have special importance in medicine, however, it is questionable to what extent these recommendations are applied in daily care, and under which conditions claims data can be used for verification of guideline adherence. Method: Advantages and limitations of claims data for verification of guidelines compliance in the therapeutic area as well as the guidelines themselves were analysed and critically assessed. To substantiate these results, claims data of a major German health insurance fund (Techniker Krankenkasse) were analysed. Results: 104 236 patients were identified. With certain limitations, claims data are useful for verifying guideline adherence; it could be shown that in pharmacotherapy the beta-adrenergic receptor blocker was used to the highest extent (70.5%). In contrast, only 56.4% of patients were treated with pure ACE inhibitors and ACE combined preparation. Conclusion: In order to validate guideline adherence by means of claims data analyses, a number of conditions relating to the database, the therapeutic area and the guidelines themselves have to be considered. Guideline recommendations, which, for example, are based on clinical data, cannot be reviewed by using claims data. Despite these limitations, claims data provide a suitable tool for reviewing selected guideline recommendations. They show that the current use of pharmacotherapy as well as clinical and diagnostic interventions might be increased in accordance with the guideline recommendations.

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. The slowly increasing application of the AHP was the motivation for this study to explore the current state of its methodology in the healthcare context. METHODS: A systematic literature review was conducted by searching the Pubmed and Web of Science databases for articles with the following keywords in their titles or abstracts: "Analytic Hierarchy Process," "Analytical Hierarchy Process," "multi-criteria decision analysis," "multiple criteria decision," "stated preference," and "pairwise comparison." In addition, we developed reporting criteria to indicate whether the authors reported important aspects and evaluated the resulting studies' reporting. RESULTS: The systematic review resulted in 121 articles. The number of studies applying AHP has increased since 2005. Most studies were from Asia (almost 30%), followed by the US (25.6%). On average, the studies used 19.64 criteria throughout their hierarchical levels. Furthermore, we restricted a detailed analysis to those articles published within the last 5 years (n = 69). The mean of participants in these studies were 109, whereas we identified major differences in how the surveys were conducted. The evaluation of reporting showed that the mean of reported elements was about 6.75 out of 10. Thus, 12 out of 69 studies reported less than half of the criteria. CONCLUSION: The AHP has been applied inconsistently in healthcare research. A minority of studies described all the relevant aspects. Thus, the statements in this review may be biased, as they are restricted to the information available in the papers. Hence, further research is required to discover who should be interviewed and how, how inconsistent answers should be dealt with, and how the outcome and stability of the results should be presented. In addition, we need new insights to determine which target group can best handle the challenges of the AHP.

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. The objective of this article is to review the economic evidence of targeted therapies in metastatic NSCLC. METHODS: A systematic literature review was conducted to identify cost-effectiveness (CE) as well as cost-utility studies. Medline, Embase, SciSearch, Cochrane, and 9 other databases were searched from 2000 through April 2013 (including update) for full-text publications. The quality of the studies was assessed via the validated Quality of Health Economic Studies (QHES) instrument. RESULTS: Nineteen studies (including update) involving the MoAb bevacizumab and the Tyrosine-kinase inhibitors erlotinib and gefitinib met all inclusion criteria. The majority of studies analyzed the CE of first-line maintenance and second-line treatment with erlotinib. Five studies dealt with bevacizumab in first-line regimes. Gefitinib and pharmacogenomic profiling were each covered by only two studies. Furthermore, the available evidence was of only fair quality. CONCLUSION: First-line maintenance treatment with erlotinib compared to Best Supportive Care (BSC) can be considered cost-effective. In comparison to docetaxel, erlotinib is likely to be cost-effective in subsequent treatment regimens as well. The insights for bevacizumab are miscellaneous. There are findings that gefitinib is cost-effective in first- and second-line treatment, however, based on only two studies. The role of pharmacogenomic testing needs to be evaluated. Therefore, future research should improve the available evidence and consider pharmacogenomic profiling as specified by the European Medicines Agency. Upcoming agents like crizotinib and afatinib need to be analyzed as well.

[Measures to improve the health situation of patients with rare diseases in Germany. A comparison with the National Action Plan]. / Maßnahmen zur Verbesserung der gesundheitlichen Situation von Menschen mit seltenen Erkrankungen in Deutschland. Ein Vergleich mit dem Nationalen Aktionsplan.

BACKGROUND: Approximately 4 million patients with a rare disease live in Germany. The medical care of these patients is problematic because of the rarity and heterogeneity of different clinical pictures. The Federal Ministry of Health has therefore published a research report on "Measures to improve the health situation of people with rare diseases in Germany" in 2009. OBJECTIVE: The aim of this paper is to present the main recommendations of this research report and relate it to current developments in the field of medical care for people with rare diseases. METHODOLOGY: The care situation of patients with rare diseases was determined using questionnaires, expert interviews and focus group discussions with representatives of patients, service providers and stakeholders from the health institutions. RESULTS: The main range of actions that have been identified in the research report were centre and network formation, specialized forms of medical care, diagnosis and treatment, information and experience exchange, performance fees and reimbursement of the costs, guidelines and patient pathways, the research, the implementation of a National Action Alliance and the development of a National Action Plan. DISCUSSION: In March 2010 a National Action League for People with Rare Diseases (NAMSE) was founded. The NAMSE created a national plan of action for people with rare diseases for improving medical care in the field of rare diseases which was approved by the Federal Government in August 2013. Thus, two important areas of the research report have already been implemented. In a comparison of the areas of activity of the research report with those of the National Action Plan it becomes clear that priorities will be in the context of health services research in rare diseases, for example the introduction of centres of reference for rare diseases, measures to accelerate the diagnostic process and the promotion of research and information management in the future.

Cost-effectiveness of tiotropium versus salmeterol: the POET-COPD trial.

The aim of this study was to perform a 1-yr trial-based cost-effectiveness analysis (CEA) of tiotropium versus salmeterol followed by a 5-yr model-based CEA. The within-trial CEA, including 7,250 patients with moderate to very severe chronic obstructive pulmonary disease (COPD), was performed alongside the 1-yr international randomised controlled Prevention of Exacerbations with Tiotropium (POET)-COPD trial comparing tiotropium with salmeterol regarding the effect on exacerbations. Main end-points of the trial-based analysis were costs, number of exacerbations and exacerbation days. The model-based analysis was conducted to extrapolate results to 5 yrs and to calculate quality-adjusted life years (QALYs). 1-yr costs per patient from the German statutory health insurance (SHI) perspective and the societal perspective were €126 (95% uncertainty interval (UI) €55-195) and €170 (95% UI €77-260) higher for tiotropium, respectively. The annual number of exacerbations was 0.064 (95% UI 0.010-0.118) lower for tiotropium, leading to a reduction in exacerbation-related costs of €87 (95% UI €19-157). The incremental cost-effectiveness ratio was €1,961 per exacerbation avoided from the SHI perspective and €2,647 from the societal perspective. In the model-based analyses, the 5-yr costs per QALY were €3,488 from the SHI perspective and €8,141 from the societal perspective. Tiotropium reduced exacerbations and exacerbation-related costs, but increased total costs. Tiotropium can be considered cost-effective as the resulting cost-effectiveness ratios were below commonly accepted willingness-to-pay thresholds.