Evolution of cost structures in rheumatoid arthritis over the past decade.

OBJECTIVE: To estimate the changes in direct and indirect costs induced by patients with rheumatoid arthritis (RA) in German rheumatology, between 2002 and 2011. To examine the impact of functional status on various cost domains. To compare the direct costs incurred by patients at working age (18-64 years) to patients at an age of retirement (≥65 years). METHODS: We analysed data from the National Database of the German Collaborative Arthritis Centres with about 3400 patients each year. Costs were calculated using fixed prices as well as annually updated cost factors. Indirect costs were calculated using the human capital as well as the friction cost approaches. RESULTS: There was a considerable increase in direct costs: from €4914 to €8206 in patients aged 18-64, and from €4100 to €6221 in those aged ≥65, attributable to increasing prescription of biologic agents (18-64 years from 5.6% to 31.2%, ≥65 years from 2.8% to 19.2%). This was accompanied by decreasing inpatient treatment expenses and indirect costs due to sick leave and work disability. The total growth of cost, on average, was €2437-2981 for patients at working age, and €2121 for patients at retirement age. CONCLUSIONS: The increase in treatment costs for RA over the last decade was associated with lower hospitalisation rates, better functional status and a lower incidence of work disability, offsetting a large proportion of risen drug costs. Since the rise in drug costs has manifested a plateau from 2009 onwards, no relevant further increase in total costs for patients with RA treated in German rheumatology is expected.

The role of biosimilars in the treatment of rheumatic diseases.

The first biological therapeutics in rheumatology are approaching patent expiration, encouraging development of 'follow-on' versions, known as 'biosimilars'. Biological agents range from simple replacement hormones to complex monoclonal antibodies and soluble receptors: large, intricate proteins with unique tertiary and quaternary structures that are inherently difficult to replicate. Post-translational modifications, such as glycosylation, may occur from changes in cell lines and/or manufacturing processes, resulting in products that are highly similar, but not identical, to approved 'reference' agents, hence, the term 'biosimilar', rather than 'bioidentical'. Even minor modifications in manufacturing processes, which iteratively occur with reference products due to improvements in efficiency, scale up to meet commercial demands or changes in manufacturing sites, may alter biological function and/or immunogenicity, potentially changing their safety and efficacy profile. As biosimilars are now in randomised controlled trials for treatment of rheumatic diseases, rheumatologists face decisions regarding equipoise and will need to consider their clinical use versus reference products. A clear understanding of the inherent differences between reference antibodies and biosimilars, their clinical implications and the processes governing regulation, approval and clinical use of biosimilars, is paramount. A panel of international experts in the field of rheumatology recently convened to evaluate and discuss these issues.

Patient perspective on the management of atrial fibrillation in five European countries.

BACKGROUND: Long-term management of chronic conditions, such as atrial fibrillation (AF), require frequent interactions with the healthcare systems. The multinational EUropean Patient Survey in Atrial Fibrillation (EUPS-AF) was conducted to investigate patient satisfaction with AF management in different of five European healthcare systems at a time of changing treatment paradigms for stroke prophylaxis, prior to the advent of newer oral anticoagulants. METHODS: Adults (>18 years) were recruited at random from the total populations of France, Germany, Italy, Spain and the UK using a randomized telephone dialling system. At least 300 respondents per country reporting to have a diagnosis of AF or receiving oral anticoagulation therapy for suspected AF or to have a heart rhythm disturbance completed a structured telephone interview. RESULTS: Most respondents were satisfied with their treatment for AF over the previous 12 months, with 85.5% (n = 1289) rating their care as good or better. Suboptimal clinical practices, however, were identified in several key areas. Coordination of primary and secondary care and a lack of patient engagement and support were particular issues, especially for those patients likely to have extensive contact with their healthcare system. CONCLUSIONS: In the context of Europe-wide guidelines for management of AF, most patients with AF were satisfied with their care, but for a greater proportion of patients, some aspects are unsatisfactory. Patient-centred surveys, such as the EUPS-AF, are crucial for understanding the factors that contribute to patient satisfaction and compliance with long-term treatment for chronic conditions.

Cost of illness in rheumatoid arthritis in Germany in 1997-98 and 2002: cost drivers and cost savings.

OBJECTIVE: Comparison of overall RA-related costs and of relative contribution of single-cost domains before and after the introduction of TNF-blocking agents in Germany. METHODS: Two cohorts of RA outpatients (ACR '87 criteria) with long-standing disease are assessed in terms of disease-related costs and cost composition (n = 106 patients in 1997-98 and n = 180 patients in 2002 with similar patient characteristics). Full-cost analyses are performed including direct disease-related costs (medical and non-medical) and productivity costs as collected by patient questionnaires. Absolute costs (€/patient/year) are compared and the impact of single-cost domains on overall costing in RA is estimated (relative proportions of cost components within samples). RESULTS: Overall costs are comparable (1997-98: €4280; 2002: €3830; not significant). Differences can be observed in medication (1997-98: €550; 2002: €1580; P < 0.001) and hospitalization costs (1997-98: €1240; 2002: €500; P < 0.001). Productivity costs are significantly lower (€1480 vs €850; P < 0.05) in 2002. The impact of medication costs is outstanding in the 2002 sample (42 vs 12%), the proportion of hospitalization costs is substantially lower (29 vs 13%). Costs for DMARDs in 2002 are mostly driven by TNF blockers (37%). The number of DMARDs per patient is higher in 2002 as are costs for osteoporosis medication and gastroprotective treatment. CONCLUSION: Although overall costs before and after the introduction of TNF blockers are comparable, the decrease in hospitalization and productivity costs is promising in terms of future long-term cost savings. The development of these aspects and of the increasing medication costs will have to be evaluated with longer time frames.

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

BACKGROUND: The treatment of ulcerative colitis (UC) can place a substantial financial burden on healthcare systems. The anti-inflammatory compound 5-aminosalicylic acid (5-ASA; mesalazine) is the recommended first-line treatment for patients with UC. In this analysis, the incremental cost effectiveness ratio (ICER) of two oral formulations of 5-ASA (Mezavant® and Asacol®) is examined in the treatment of patients with mild-to-moderate, active UC in Germany. METHODS: A Markov cohort model was developed to assess the cost effectiveness of Mezavant compared with Asacol over a 5-year period in the German Statutory Health Insurance (SHI). Drug pricing details for 2009 were applied throughout the model, and overall resource use was determined and also fitted to 2009 from published results of a large cross sectional study of German SHI patients. Cost per quality adjusted life year (QALY) was the primary endpoint for this study. Remission rates were obtained using data from a randomised, phase III trial of Mezavant with an active Asacol reference arm and a long-term, open label, safety and tolerability trial of Mezavant. Uncertainty in the study model was assessed using one-way and probabilistic sensitivity analyses applying a Monte Carlo simulation. RESULTS: Over a 5-year period, healthcare costs for patients receiving Mezavant were 624 Euro lower than for patients receiving Asacol. Additionally, patients receiving Mezavant gained 0.011 QALYs or 18 more days in remission compared with Asacol. One-way sensitivity analyses suggest that these results are driven by both differences in the acquisition cost between mesalazine formulations and differences in treatment efficacy. Furthermore, sensitivity analyses suggest a probability of 76% for cost savings and higher QALYs with Mezavant compared with Asacol. If adherence and its influence on the remission rates and the risk of developing colorectal cancer were included in the model, the results might have even been more favorable to Mezavant due to its once daily dosing regimen. CONCLUSIONS: This model suggests that patients treated with Mezavant may achieve increased time in remission and higher QALYs, with lower direct costs to the SHI when compared with Asacol. Mezavant may therefore be a suitable first-line option for the induction and maintenance of remission in UC.

A structured review and guide through studies on health-related quality of life in kidney cancer, hepatocellular carcinoma, and leukemia.

Aim of this paper is to review and describe Health-Related Quality of Life (HRQoL) measures applied in kidney cancer, hepatocellular carcinoma, and leukemia patients under drug therapy. A comprehensive search in PubMed was conducted to identify studies assessing quality of life (QoL) in these indications. In total 32 studies, including four studies through reference list checking and 21 different HRQoL instruments, were identified. Six generic, five disease-specific, and 10 domain-specific instruments were identified. In conclusion no overall standards in HRQoL measurement could be observed in the respective indications.

Cost of transfusion-dependent myelodysplastic syndrome (MDS) from a German payer's perspective.

No curative treatment exists for patients with myelodysplastic syndrome (MDS) besides allogeneic stem cell transplantation. Hence, palliative treatment is provided for a life time accruing high health care cost. As no study in cost of MDS exists in Germany, the objective of this study was to assess and analyze costs of transfusion-dependent low/intermediate-1-risk MDS in Germany from a payers' perspective. From seven centers, 116 low/intermediate-1-risk transfusion-dependent MDS patients with and without isolated 5q-deletion were identified. Claims data and patient records of the previous 5 years were used to collect health care utilization data retrospectively. Publicly available tariff books and remuneration schemes were applied to evaluate mean costs per year in Euro with 2007 as base year. The annual cost of MDS patients was estimated at 14,883. Subgroup analyses showed differences in patient's characteristics and outcomes among patients treated at a hospital-based vs. an office-based setting. Patients treated at the hospital-based registry show higher cost, whereas the reasons for that still need to be detected. Overall, per annum direct costs range from 12,543 (SD 12,967) to 24,957 (SD 36,399) in different subgroups of patients. In both groups, patients with 5q-deletion use more medication than those without deletion. Mean costs for medication in the office-based setting are 5,902 for patients with isolated 5q-deletion vs. 3,932 for patients with no deletion, respectively. MDS leads to a high health care utilization and resulting costs for the health care system which requires a detailed analysis of underlying services.

[Aspects of outpatient palliative care and assessment of the nursing workload. Survey among care givers in Germany]. / Versorgungssituation von Palliativpatienten und Einschätzung der Arbeitsbelastung der Pflegenden. Befragung unter ambulant tätigen Pflegenden in Deutschland.

INTRODUCTION: The aim of this study is to explore aspects of the health care situation of outpatient palliative patients in Germany as well as effort and workload of care from the viewpoint of involved care givers. Additionally the future development with regard to the cooperation with other health care providers is assessed. METHODS: A detailed questionnaire was developed and sent to 188 outpatient care givers, all members of the German Association for Palliative Medicine, in January 2009. All data was analyzed via SPSS version 16. RESULTS: 69 questionnaires were included into statistical analyses. Care givers estimate the effort of care of palliative patients as very high. 28 per cent of working time is spent on administration. Responders consider general and quantitative workload to be the highest. Most care givers assess the SAPV-directive of the Federal Joint Committee as well as the future health care situation of palliative patients and cooperation with other health care providers as good. DISCUSSION: Further studies should focus on the collection of longitudinal patient data for a more comprehensive insight.

Application study of the EQ-5D-5L in oncology: linking self-reported quality of life of patients with advanced or metastatic colorectal cancer to clinical data from a German tumor registry.

BACKGROUND: The EQ-5D-5L questionnaire is used in oncology to generate health-related quality of life (HRQoL) weights and corresponding health states. The purpose was to explore the relationship between demographic and clinical characteristics and HRQoL among advanced or metastatic colorectal cancer (CRC) patients by linking clinical data of a German CRC registry to self-reported HRQoL measures from the EQ-5D-5L. METHODS: The study sample included patients with advanced or metastatic CRC currently recruited in the German Tumor Registry Colorectal Cancer. The EQ-5D-5L was administered once to patients who were at the start or at later stages of palliative treatment. Data on comorbidities, disease-specific health states, symptoms, and treatment status were drawn from the registry. Multivariate regression analyses were performed to explore the impact of patient and disease characteristics on HRQoL. RESULTS: In total, n = 433 questionnaires were included in the data analysis. Mean age of patients was 66.3 years and 61.2% were male. The mean EQ-5D-5L utility score was 0.82 and the mean EQ-5D-5L VAS score was 62.05. The regression analyses revealed that none of the demographic characteristics and few of the clinical characteristics, such as fatigue and pain, had a significant impact on the HRQoL. CONCLUSIONS: The study demonstrated a reduced HRQoL of patients with advanced or metastatic CRC when compared to the general population. The symptoms fatigue and pain negatively affected the HRQoL, whereas other characteristics such as age, gender, and comorbidities did not have a significant impact on HRQoL.

Quality of life as an outcome in Alzheimer's disease and other dementias--obstacles and goals.

BACKGROUND: The number of individuals at risk for dementia will probably increase in ageing societies as will the array of preventive and therapeutic options, both however within limited economic resources. For economic and medical purposes valid instruments are required to assess disease processes and the efficacy of therapeutic interventions for different forms and stages of illness. In principal, the impact of illness and success of an intervention can be assessed with biomedical variables, e.g. severity of symptoms or frequency of complications of a disease. However, this does not allow clear judgement on clinical relevance or comparison across different diseases. DISCUSSION: Outcome model variables such as quality of life (QoL) or health care resource utilization require the patient to appraise their own well-being or third parties to set preferences. In Alzheimer's disease and other dementias the evaluation process performed by the patient is subject to the disease process itself because over progress of the disease neuroanatomical structures are affected that mediate evaluation processes. SUMMARY: Published research and methodological considerations thus lead to the conclusion that current QoL-instruments, which have been useful in other contexts, are ill-suited and insufficiently validated to play a major role in dementia research, decision making and resource allocation. New models integrating biomedical and outcome variables need to be developed in order to meet the upcoming medical and economic challenges.

Cross-sectional validity of the EQ-5D-Y as a generic health outcome instrument in children and adolescents with cystic fibrosis in Germany.

BACKGROUND: Quality of life is recognized as an important additional outcome measure in clinical trials and health economic evaluations. The EQ-5D is an important generic health outcome instrument often used for economic evaluations as a complement with disease-specific outcome measures. In this study quality of life data was assessed using the EQ-5D-Y (new EQ-5D version for children and adolescents) and the Cystic Fibrosis Questionnaire (CFQ). The objective of the study is to evaluate the cross-sectional validity of the EQ-5D-Y as a generic health outcome instrument in children and adolescents with cystic fibrosis in Germany. METHODS: In 2006 a multi-centre study was conducted in four cystic fibrosis centres in Germany. Quality of life data from 96 patients between eight and seventeen years was collected using the EQ-5D-Y as a generic outcome instrument and the Cystic Fibrosis Questionnaire (CFQ) as a disease-specific instrument. Results of both instruments were compared by statistical analyses using Spearman's rank correlations. RESULTS: 44.6% of the patients stated that they had no problems in any of the EQ-5D-Y dimensions. Several low to high correlations between separate dimensions and the visual analogue scale of the EQ-5D-Y and the different scales of the CFQ for children, their parents and adolescents can be presented in this paper. Looking at the five EQ-5D-Y dimensions the highest correlation (rS = 0.625, p = 0.01) was found between the dimension 'mobility' and the CFQ scale 'physical functioning' in adolescent patients. The overall highest correlation was found between the 'subjective health perception' and the visual analogue scale (rS = 0.744, p = 0.01) in adolescent patients older than 13 years. CONCLUSION: The EQ-5D-Y can be considered a cross-sectional valid generic health outcome instrument which reflects differences in health according to the progression of the life-long chronic disease cystic fibrosis.

[Comparative cost analysis of outpatient and inpatient rehabilitation for cardiac diseases]. / Kostenvergleichsanalyse der ambulanten und stationären kardiologischen Rehabilitation.

PURPOSE: : To examine costs of inpatient and outpatient rehabilitation for cardiac disorders from the perspective of a major statutory health insurance fund in Germany. METHODS: : A nationwide database from a major health insurance fund in Germany was used to evaluate all rehabilitation cases in 2005. In addition to all direct cost domains of the rehabilitation itself, costs incurred in the preceding and the following year for hospital treatment, drugs and physical therapy were analyzed. A cost-cost analysis in different institutional settings was chosen for the cost comparison of in- and outpatient rehabilitation. To minimize the influence of possible confounders, a statistical control system was implemented. RESULTS: : Mean costs were 2,006 Euros for inpatient rehabilitation and 1,502 Euros for outpatient rehabilitation. No systematic differences were identified between in- and outpatient rehabilitation in costs for drugs and physical therapy in the year preceding and the year directly following the rehabilitation. The costs for hospital treatment in the year before rehabilitation were higher in inpatient rehabilitation, whereas in the year after rehabilitation no differences between in- and outpatient rehabilitation were seen. CONCLUSION: : Assuming comparable medical outcome, outpatient rehabilitation seems to pose a superior alternative compared to inpatient rehabilitation from an economic perspective. Hence, from the perspective of the statutory health insurance, fostering a higher market share of outpatient rehabilitation may add to a better allocation of overall health-care resources.

[Incremental cost-effectiveness of dipyridamole + acetylsalicylic acid in secondary prevention of ischemic noncardioembolic stroke]. / Inkrementelle Kosteneffektivität von Dipyridamol + Acetylsalicylsäure in der Sekundärprävention bei ischämischem nichtkardioembolischem Schlaganfall.

BACKGROUND AND PURPOSE: The aim of secondary prevention in stroke is to avoid restrokes. The current standard treatment in Germany is a lifelong therapy with low-dose acetylsalicylic acid (ASA). As the incidence of restrokes remains relatively high from a health-care payer's perspective, the question arises, whether the combination of dipyridamole + acetylsalicylic acid (Dip + ASA) is cost-effective in comparison with a therapy based on ASA only. METHODS: A decision-analytic cross-sectional epidemiologic steady-state model of the German population compares the effects of two strategies of secondary prevention with Dip + ASA (12 months vs. open end) and with ASA monotherapy. RESULTS: The model predicts the following estimates: the annual incidence of initial ischemic strokes in Germany is estimated at 130,000 plus an extra 34,000 restrokes (base year 2005). Additionally, there are 580,000 people that experienced a stroke > 12 months earlier, of whom 135,000 had a restroke. Every year, nearly 89,000 Germans die of the consequences of an ischemic stroke. If Dip + ASA would have been the standard therapy in secondary prevention of ischemic stroke, an additional 7,500 persons could have been saved in 2005. Statutory health insurance would have to spend 33,000 Euro for every additional life year gained with Dip + ASA as secondary prevention strategy. If secondary prevention with Dip + ASA would be limited to the first 12 months after an initial stroke, which is the time of the highest risk for a restroke, the incremental cost-effectiveness ratio is about 7,000 Euro per life year gained. The results proved to be robust in sensitivity analyses. CONCLUSION: Secondary prevention with Dip + ASA is cost-effective in comparison to treatment with ASA in monotherapy, because its incremental cost-effectiveness ratio is within common ranges of social willingness to pay. From the standpoint of the patient as well as the health-care payer, focusing on the first 12 months after the initial incident for intensified preventive drug treatment with Dip + ASA should be valuable from a medical as well as a health-economic perspective.

Methods for determining cost-benefit ratios for pharmaceuticals in Germany.

The aim of this methodological paper is to summarize evidence on how to implement cost-benefit assessment according to the new German legislative framework (Competition Enhancement Act). Given the complexity of existing health policy frameworks within industrialised countries in adapting health economics in their respective regulatory scheme, no clear international scientific consensus on which health economic methods should be chosen for assessment can be determined. Nevertheless, a broad consensus on the internal properties of methods itself can be found. Based on these common international standards in methodology, this work provides a minimum catalogue of methods and criteria that meet legal and local German requirements with regard to specific factors of its health care system. Aside from categorising clearly defined standards (e.g., study forms, cost and benefit categories) the suggested catalogue specifies some intensively debated areas in Germany (e.g., the QALY, modelling, the perspective used in the assessment). After the proposition of certain methods the paper leads to a first recommendation of a detailed assessment-process itself specific for the German way in implementing cost-benefit ratios within regulatory decision making in Germany.

Patient preferences in allergy immunotherapy (AIT) in Germany - a discrete-choice-experiment.

BACKGROUND: Allergic Rhinitis (AR) is a common disorder in Europe with Allergic Asthma (AA) as a frequent comorbidity. Allergy immunotherapy (AIT) is the only causal therapy of AR and AA, and can be administered as subcutaneous injections at the physician or as sublingual drops or tablets at home. The usual treatment duration is 3 years. OBJECTIVE: This study aimed to elicit patient preferences to identify the AIT administration mode preferred by patients. METHODS: A discrete-choice-experiment (DCE) was developed to determine how people weight different treatment options using a paper-based questionnaire from June to September 2014, including 16 study centres. Main inclusion criteria: >18 years, grass, birch and/or house dust mite AR with moderate to severe symptoms, AIT-naïve and AIT-indicated. DCE-attributes were: Administration form, number and duration of physician visits, frequency of life-threatening anaphylactic shocks, local side-effects and co-payments. RESULTS: Two-hundred thirty-nine subjects participated, resulting in analysable 1842 choices. All attributes were significant predictors for the treatment-choice. Ranked by importance, the following first three attributes are most preferred by patients: 1(st) Number and duration of physician visits: Fewer visits with shorter duration preferred (0.658*) 2(nd) Frequency of life-threatening anaphylactic shocks: Lower risk of shocks preferred (0.285*) 3(rd) Local side-effects: Preference for rash/swelling on upper arm over itching/swelling under the tongue (0.210*) (*coefficient-size represents relative importance of the attributes) CONCLUSION: The most important attribute is the number and duration of visits to a physician. A lower risk of life-threatening anaphylactic shocks was ranked as the second whereas co-payments and administration form play a limited role.

[Costs of ambulatory care for RA patients in Germany]. / Arztkontaktpreise bei der ambulanten Versorgung der chronischen Polyarthritis in Deutschland.

BACKGROUND: In 2000, the Regional Cooperative Center for Rheumatic Conditions in Hannover, Germany, initiated a project to introduce and evaluate a case-based clinical quality management system to enhance the quality of care for patients with rheumatoid arthritis. Building on that, it was possible to combine all resource uses, as paid by the sickness funds, including those in ambulatory care. METHODS: The purpose of this study was to reach average "per-visit prices" for single physician visits, not only for rheumatologists, but also for other specialists as well as generalists. Those visits all had to be connected to the rheumatic condition of the patients. All ambulatory care visits for 1 year (2001) were evaluated. RESULTS: Due to the high number of physician visits of the 338 patients with clinically assured rheumatoid arthritis included in the study (rheumatologist visits 4,488, generalist visits 3,901), statistically significant subgroups could be built. The average costs per visit associated with rheumatoid arthritis are: rheumatologist euro 22.71; generalist euro 8.02; as well as other specialties ranging from euro 5.81 to euro 19.48. The low price for generalists care is due to the selection of the cohort, as all patients are under constant specialist care. The prices were further broken down to certain subgroups of care. CONCLUSION: The prices are calculated under the premises that no budgetary constraints apply to ambulatory care. This is not the reality in Germany. Hence, looking at the average frequency of rheumatologist visits in this cohort (13.2/year), the conclusion has to be that not all rheumatologic ambulatory care is being covered by the reimbursement system within the Statutory Health Insurance in Germany.

Diffusion of innovations: treatment of Alzheimer's disease in Germany.

Systematic barriers seem to slow down the market penetration of innovative acethylcholinesterase (AChE) inhibitors in Alzheimer's disease. The goal of our study was to examine the diffusion of AChE inhibitors into the German market in more detail. On the basis of using the ongoing surveillance panel of the Institute of Medical Statistic (IMS) Health, the prescription patterns of 100 physicians (72 general practitioners, 28 neurologists) were examined. In addition, structured telephone interviews with the same 100 physicians were conducted. The interview included the assessment of a hypothetical treatment situation (i.e. physicians were asked what they would prescribe if a close relative of theirs had Alzheimer's disease) as well as qualitative items examining the physicians' attitudes towards AChE inhibitors and the perceived impact on drug budgets. As a major result, the analysis revealed that neurologists prescribed AChE inhibitors to 44.6% of their patients, while general practitioners only treated 9.0% of their patients with AChE inhibitors. The analysis of the qualitative items revealed positive attitudes regarding the safety and efficacy of AChE inhibitors, but negative attitudes regarding the budgetary limitations to prescribing these drugs. A correlation of r=0.21 (P<0.05) was found between the perceived impact on drug budgets and the adoption of AChE inhibitors and a correlation of r=0.32 (P<0.002) was seen between the physician's specialty and the adoption of AChE inhibitors. These data show that, while the AChE inhibitor adoption process has passed the early stages, various barriers slow down the final stages of AChE inhibitor adoption. The drug budget in particular seems to inhibit the adoption of the innovation by the majority of general practitioners. This leads to a more short-term cost control strategy instead of long-term disease management and cost saving approaches.

Comparison of estimated medical costs among patients who are defined as having rheumatoid arthritis using three different standards.

Accurate estimation of medical care costs raises a host of challenging issues. We examined whether pure administrative claims data without clinical validation of diagnosis allow reasonable estimation of disease-related costs in rheumatoid arthritis (RA). Three patient groups were examined: patients with clinically confirmed RA (group A, n=338), patients with likely RA (administrative claims data reported the diagnosis of RA and patients were treated with disease modifying antirheumatic drugs, DMARDs; group B, n=303), and patients with possible RA (same as group B but patients had no DMARD treatment; group C, n=685). The payer's perspective was taken for this analysis. Only direct costs were included in the analysis. Cost data and data for several covariates were obtained from a major German statutory health insurance plan, the AOK Niedersachsen. A patient-per-patient microcosting approach was performed. A repeated measures, fixed effects model was applied to examine differences between the three study groups. Mean+/-SEM annual RA-related direct costs were euro 2,017+/-183 per patient-year in group A, euro 1,763+/-192 in group B, and euro 805+/-58 in group C. Outpatient (inpatient) costs were euro 1,636 (328) in group A, euro 1344 (340) in group B, and euro 546 (136) in group C. DMARD costs were by far the single most important cost driver in groups A and B. The difference in total RA-related direct cost between groups A and B was not significant whereas the differences between groups A and C (group B and C respectively), were significant. Pure administrative claims data allowed for an accurate estimate of disease-related costs in RA patients that received DMARD therapy. However, the high number of patients for whom administrative claims data listed the diagnosis RA, but no DMARD treatment was given poses a challenge for further research.

Modeling rheumatoid arthritis using different techniques - a review of model construction and results.

BACKGROUND: Rheumatoid arthritis (RA) is a chronic, inflammatory disease with severe effects on the functional ability of patients. Due to the prevalence of 0.5 to 1.0 percent in western countries, new treatment options are a major concern for decision makers with regard to their budget impact. In this context, cost-effectiveness analyses are a helpful tool to evaluate new treatment options for reimbursement schemes. OBJECTIVES: To analyze and compare decision analytic modeling techniques and to explore their use in RA with regard to their advantages and shortcomings. METHODS: A systematic literature review was conducted in PubMED and 58 studies reporting health economics decision models were analyzed with regard to the modeling technique used. RESULTS: From the 58 reviewed publications, we found 13 reporting decision tree-analysis, 25 (cohort) Markov models, 13 publications on individual sampling methods (ISM) and seven discrete event simulations (DES). Thereby 26 studies were identified as presenting independently developed models and 32 models as adoptions. The modeling techniques used were found to differ in their complexity and in the number of treatment options compared. Methodological features are presented in the article and a comprehensive overview of the cost-effectiveness estimates is given in Additional files 1 and 2. DISCUSSION: When compared to the other modeling techniques, ISM and DES have advantages in the coverage of patient heterogeneity and, additionally, DES is capable to model more complex treatment sequences and competing risks in RA-patients. Nevertheless, the availability of sufficient data is necessary to avoid assumptions in ISM and DES exercises, thereby enabling biased results. Due to the different settings, time frames and interventions in the reviewed publications, no direct comparison of modeling techniques was applicable. The results from other indications suggest that incremental cost-effective ratios (ICERs) do not differ significantly between Markov and DES models, but DES is able to report more outcome parameters. CONCLUSIONS: Given a sufficient data supply, DES is the modeling technique of choice when modeling cost-effectiveness in RA. Otherwise transparency on the data inputs is crucial for valid results and to inform decision makers about possible biases. With regard to ICERs, Markov models might provide similar estimates as more advanced modeling techniques.