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ABSTRACT: In addition to medical treatment, deep brain stimulation has become an alternative therapeutic option in advanced Parkinson's disease. High initial costs of surgery have to be weighted against long-term gains in health-related quality of life. The objective of this study was to assess the cost-effectiveness of deep brain stimulation compared with long-term medical treatment. We performed a cost-utility analysis using a lifetime Markov model for Parkinson's disease. Health utilities were evaluated using the EQ-5D generic health status measure. Data on effectiveness and adverse events were obtained from clinical studies, published reports, or meta-analyses. Costs were assessed from the German health care provider perspective. Both were discounted at 3% per year. Key assumptions affecting costs and health status were investigated using one-way and two-way sensitivity analyses. The lifetime incremental cost-utility ratio for deep brain stimulation was €6700 per quality-adjusted life year (QALY) and €9800 and €2500 per United Parkinson's Disease Rating Scale part II (motor experiences of daily living) and part III (motor examination) score point gained, respectively. Deep brain stimulation costs were mainly driven by the cost of surgery and of battery exchange. Health status was improved and motor complications were reduced by DBS. Sensitivity analysis revealed that battery life time was the most influential parameter, with the incremental cost-utility ratio ranging from €20,000 per QALY to deep brain stimulation dominating medical treatment. Deep brain stimulation can be considered cost-effective, offering a value-for-money profile comparable to other well accepted health care technologies. Our data support adopting and reimbursing deep brain stimulation within the German health care system. © 2013 Movement Disorder Society.
Movement Disorders 04/2013; · 4.51 Impact Factor
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Judith Dams,
Jens Klotsche, Bernhard Bornschein,
Jens P Reese,
Monika Balzer-Geldsetzer,
Yaroslav Winter,
Anette Schrag,
Andrew Siderowf,
Wolfgang H Oertel,
Günther Deuschl,
Uwe Siebert,
Richard Dodel
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ABSTRACT: BACKGROUND: Clinical studies employ the Unified Parkinson's Disease Rating Scale (UPDRS) to measure the severity of Parkinson's disease. Evaluations often fail to consider the health-related quality of life (HrQoL) or apply disease-specific instruments. Health-economic studies normally use estimates of utilities to calculate quality-adjusted life years. We aimed to develop an estimation algorithm for EuroQol- 5 dimensions (EQ-5D)-based utilities from clinical (UPDRS) or disease-specific HrQoL data in the absence of original utilities estimates. METHODS: Linear and fractional polynomial regression analyses were performed with data from a study of Parkinson's disease patients (n=138) to predict the EQ-5D index values from UPDRS and Parkinson's disease questionnaire eight dimensions (PDQ-8) data. German and European weights were used to calculate the EQ-5D index. The models were compared by R2, the root mean square error (RMS), the Bayesian information criterion, and Pregibon's link test. Three independent data sets validated the models. RESULTS: The regression analyses resulted in a single best prediction model (R2: 0.713 and 0.684, RMS: 0.139 and 13.78 for indices with German and European weights, respectively) consisting of UPDRS subscores II, III, IVa-c as predictors. When the PDQ-8 items were utilised as independent variables, the model resulted in an R2 of 0.60 and 0.67. The independent data confirmed the prediction models. CONCLUSION: The best results were obtained from a model consisting of UPDRS subscores II/III/IV a-c. Although a good model fit was observed, primary EQ-5D data are always preferable. Further validation of the prediction algorithm within large, independent studies is necessary prior to its generalised use.
Health and Quality of Life Outcomes 03/2013; 11(1):35. · 2.11 Impact Factor
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ABSTRACT: The objective of this review was to assess models of cost effectiveness for Parkinson's disease (PD) published after July 2002 and to derive recommendations for future modelling. A systematic literature search was performed in the databases PubMed, Current Contents, EMBASE, EconLit, the Cochrane Database of Systematic Reviews, and DARE (Database of Abstracts of Reviews of Effectiveness), NHS EED (Economic Evaluation Database) and HTA (Health Technology Assessment) of the UK NHS Centre for Review and Dissemination (July 2002 to March 2010). Only fully published studies using decision trees, Markov models, individual simulation models or sets of mathematical equations were included. Most of the 11 studies identified used Markov models (n = 9) and two employed were based on decision trees. Based on the Hoehn & Yahr (HY) scale, authors evaluated the cost effectiveness of drug treatments (n = 6), surgical approaches such as deep brain stimulation (n = 1) or striatal cell grafting (n = 1), and diagnostic procedures such as single photon emission computed tomography (SPECT) testing (n = 3) over a time horizon of 1 year to lifetime. Costs were adapted to address a societal and/or healthcare provider/third-party payer perspective. All but one of the interventions investigated were considered cost effective or cost saving. Cost-effectiveness modelling in PD between 2003 and 2010 showed only minor improvement when compared with our earlier review of models published from 1998 up to 2003. Cost-effectiveness modelling recommendations were complied with to only a limited extent, leaving room for quality improvement. More advanced modelling approaches may, so far, be under-represented, but may be used in the future, driven by the research question. Adverse events of treatment, co-morbidities or disease complications are not yet sufficiently included in the models to adequately represent clinical reality.
PharmacoEconomics 12/2011; 29(12):1025-49. · 2.66 Impact Factor
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ABSTRACT: The Fractional Flow Reserve Versus Angiography for Multivessel Evaluation (FAME) study demonstrated significantly improved health outcomes at 1 year in patients randomized to multivessel percutaneous coronary intervention guided by fractional flow reserve (FFR) compared with percutaneous coronary intervention guided by angiography alone. The economic impact of routine measurement of FFR in this setting is not known.
In this study, 1005 patients were randomly assigned to FFR-guided or angiography-guided percutaneous coronary intervention and followed up for 1 year. A prospective cost-utility analysis comparing costs and quality-adjusted life-years was performed with a time horizon of 1 year. Quality-adjusted life-years were calculated with the use of utilities determined by the EuroQuol 5 dimension health survey with US weights. Direct medical costs included those of the index procedure and hospitalization and costs for major adverse cardiac events during follow-up. Confidence intervals for both quality-adjusted life-years and costs were estimated by the bootstrap percentile method. Major adverse cardiac events at 1 year occurred in 13.2% of those in the FFR-guided arm and 18.3% of those in the angiography-guided arm (P=0.02). Quality-adjusted life-years were slightly greater in the FFR-guided arm (0.853 versus 0.838; P=0.2). Mean overall costs at 1 year were significantly less in the FFR-guided arm ($14 315 versus $16 700; P<0.001). Bootstrap simulation indicated that the FFR-guided strategy was cost-saving in 90.74% and cost-effective at a threshold of US $50 000 per quality-adjusted life-years in 99.96%. Sensitivity analyses demonstrated robust results.
Economic evaluation of the FAME study reveals that FFR-guided percutaneous coronary intervention in patients with multivessel coronary disease is one of those rare situations in which a new technology not only improves outcomes but also saves resources. Clinical Trial Registration- URL: http://ClinicalTrials.gov. Unique identifier: NCT00267774.
Circulation 12/2010; 122(24):2545-50. · 14.74 Impact Factor
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Nico H J Pijls,
William F Fearon,
Pim A L Tonino,
Uwe Siebert,
Fumiaki Ikeno, Bernhard Bornschein,
Marcel van't Veer,
Volker Klauss,
Ganesh Manoharan,
Thomas Engstrøm,
Keith G Oldroyd,
Peter N Ver Lee,
Philip A MacCarthy,
Bernard De Bruyne
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ABSTRACT: The purpose of this study was to investigate the 2-year outcome of percutaneous coronary intervention (PCI) guided by fractional flow reserve (FFR) in patients with multivessel coronary artery disease (CAD).
In patients with multivessel CAD undergoing PCI, coronary angiography is the standard method for guiding stent placement. The FAME (Fractional Flow Reserve Versus Angiography for Multivessel Evaluation) study showed that routine FFR in addition to angiography improves outcomes of PCI at 1 year. It is unknown if these favorable results are maintained at 2 years of follow-up.
At 20 U.S. and European medical centers, 1,005 patients with multivessel CAD were randomly assigned to PCI with drug-eluting stents guided by angiography alone or guided by FFR measurements. Before randomization, lesions requiring PCI were identified based on their angiographic appearance. Patients randomized to angiography-guided PCI underwent stenting of all indicated lesions, whereas those randomized to FFR-guided PCI underwent stenting of indicated lesions only if the FFR was <or=0.80.
The number of indicated lesions was 2.7+/-0.9 in the angiography-guided group and 2.8+/-1.0 in the FFR-guided group (p=0.34). The number of stents used was 2.7+/-1.2 and 1.9+/-1.3, respectively (p<0.001). The 2-year rates of mortality or myocardial infarction were 12.9% in the angiography-guided group and 8.4% in the FFR-guided group (p=0.02). Rates of PCI or coronary artery bypass surgery were 12.7% and 10.6%, respectively (p=0.30). Combined rates of death, nonfatal myocardial infarction, and revascularization were 22.4% and 17.9%, respectively (p=0.08). For lesions deferred on the basis of FFR>0.80, the rate of myocardial infarction was 0.2% and the rate of revascularization was 3.2 % after 2 years.
Routine measurement of FFR in patients with multivessel CAD undergoing PCI with drug-eluting stents significantly reduces mortality and myocardial infarction at 2 years when compared with standard angiography-guided PCI. (Fractional Flow Reserve Versus Angiography for Multivessel Evaluation [FAME]; NCT00267774).
Journal of the American College of Cardiology 07/2010; 56(3):177-84. · 14.16 Impact Factor
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ABSTRACT: Coronary artery disease (CAD) is one of the leading causes of premature death in Germany. Percutaneous coronary interventions (PCI) are frequently performed in patients with angiographically intermediate stenoses. However, the necessity of PCI has not been proven for all patients. Pressure-based fractional flow reserve (FFR) is an invasive test that can be used to assess the functional significance of intermediate coronary stenoses in order to guide decisions on PCI.
This health technology assessment (HTA) aims to evaluate (1) the diagnostic accuracy, (2) the risk-benefit trade-off and (3) the long-term cost-effectiveness of FFR measurement to guide the decision on PCI in patients with stable angina pectoris and intermediate coronary stenoses.
We performed a literature search in medical and HTA databases. We used the DIMDI instruments (DIMDI = Deutsches Institut für Medizinische Dokumentation und Information/German Institute for Medical Information and Documentation) to assess study quality and to extract and summarize the information in evidence tables. We performed a meta-analysis to calculate the pooled overall estimate for sensitivity and specificity of FFR with 95% confidence intervals (95% CI). Individual studies' case numbers were used as weights. The influence of single studies and important covariates on the results was tested in sensitivity analyses. We developed the German Coronary Artery Disease Outcome Model (German CADOM), a decision-analytic Markov model, to estimate the long-term effectiveness and cost-effectiveness of FFR measurement in the context of the German healthcare system.
Our literature search identified twelve studies relevant to this HTA-report including ten diagnostic accuracy studies of FFR measurement, one randomized clinical trial (RCT) investigating the clinical benefits of this technique as well as one economic evaluation. Pooled estimates for sensitivity and specificity were 81.7% (95% CI: 77.0-85.7%) and 78.7% (95% CI: 74.3-82.7%). Sensitivity analyses indicated robust results. The RCT investigating the efficacy of an FFR-based treatment strategy provided evidence of the advantages of this strategy for patients with respect to freedom from angina and major adverse cardiac events. The published cost-effectiveness study demonstrates that the FFR-based strategy is cost-saving in the US context. Based on our own decision analysis for the German context, the FFR-based strategy improves (quality-adjusted) life-expectancy when compared to universal PCI and is cost-effective in the German healthcare context. This HTA is limited by the use of poor gold standards in several of the included diagnostic studies as well as the ongoing advance of technology and treatment options in interventional cardiology. Results of the decision analysis are limited by the necessary underlying assumptions and the uncertainty regarding long-term mortality reduction associated with PCI. Further research should focus on the acquisition of long-term data for disease progression in patients with and without functional coronary stenoses as well as the benefits and risks of PCI.
Based on actual evidence and our decision analysis, the use of FFR measurement to guide the decision on PCI should lead to better short- and long-term clinical outcomes in patients with stable angina and single-vessel disease without documented myocardial ischemia and it should provide a cost-effective use of resources in the German healthcare system. FFR measurement should be introduced in routine clinical practice. However, appropriate reimbursement strategies are necessary to avoid wrong incentives.
GMS health technology assessment. 01/2008; 4:Doc07.
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ABSTRACT: Few prospective data on the clinical progression of Parkinson's disease (PD) in patient groups outside treatment trials in selected patients are available, and controversy exists on the rate of clinical disease progression with advancing disease. In this study, we investigated the rate of clinical progression of PD in a clinic-based sample of 145 patients over 1 year and in a community-based sample of 124 patients over 4 years. Depending on the sample and clinical scale used, mean deterioration of motor and disability scores ranged from 2.4 to 7.4% of the maximum possible score per year, and standard deviations indicated that there was considerable variability of progression rates between individuals. The progression of motor scores decreased with follow-up over 4 years and significantly decreased in more advanced disease stages. Deterioration of disability scores did not differ between disease stages; this may reflect the increasing rate of disease complications, which contribute to increasing disability in addition to motor impairment alone, in more advanced disease. Thus, motor fluctuations, hallucinations, depression, memory problems, and bladder symptoms were all reported more often at follow-up in the community-based sample (all P < 0.01), and dyskinesias, motor fluctuations, falls, and hallucinations were more common and cognitive and depression scores worse in higher disease stages in the clinic-based sample (all P < 0.001). We conclude that progression of motor scores in PD decreases with advancing disease in PD. However, disability continues to deteriorate with advancing disease and with the development of disease complications that are likely to be related to additional extrastriatal pathology.
Movement Disorders 05/2007; 22(7):938-45. · 4.51 Impact Factor
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ABSTRACT: To provide an overview on the prevalence and incidence of Parkinson's disease (PD) in selected European countries.
PD is a common disease of unknown etiology. Accurate information on the epidemiology of PD is critical to inform health policy. An aging population will lead to more patients with PD; thus, the high financial burden PD places on society will increase.
A systematic literature search was performed to identify studies on the prevalence and incidence of PD in the following European countries: Austria, the Czech Republic, France, Germany, Italy, The Netherlands, Portugal, Spain, Sweden and United Kingdom. Only published studies were included. Abstracts, reviews, meta-analyses and letters to the editor were excluded. There were no language restrictions. Data were extracted using a standardized assessment form, and evidence tables were used to systematically report and compare the data.
Of 39 identified studies, most (87%) reported estimates of PD prevalence rates, while only a few (13%) reported estimates of PD annual incidence rates. Crude prevalence rate estimates ranged from 65.6 per 100,000 to 12,500 per 100,000 and annual incidence estimates ranged from 5 per 100,000 to 346 per 100,000. No publications could be identified for Austria or the Czech Republic.
The observed variations in prevalence and incidence rates may result from environmental or genetic factors, but might also be a consequence of differences in methodologies for case ascertainment, diagnostic criteria, or age distributions of the study populations. The comparability of existing studies is limited.
European Neuropsychopharmacology 09/2005; 15(4):473-90. · 4.05 Impact Factor
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Annika E Spottke,
Martin Reuter,
Olaf Machat, Bernhard Bornschein,
Sonja von Campenhausen,
Karin Berger,
Rudolf Koehne-Volland,
Jürgen Rieke,
Alexander Simonow,
Dirk Brandstaedter,
Uwe Siebert,
Wolfgang H Oertel,
Gudrun Ulm,
Richard Dodel
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ABSTRACT: To prospectively evaluate the health economic burden of patients with Parkinson's disease (PD) in Germany over a 6-month observation period and to identify the predictors of these costs.
Direct and indirect costs were evaluated in 145 patients with PD (mean age 67.3 +/- 9.6 years). PD patients were recruited from an outpatient department for movement disorders, a specialised PD clinic, two office-based neurologists and general practitioners, all located in Germany, and were enrolled between January and June 2000. Relevant economic data were documented in a patient diary over the 6-month period. Clinical evaluations (Unified Parkinson's Disease Rating Scale [UPDRS]) were performed at baseline and at 3 and 6 months. Costs were derived from various German medical economic resources. Costs were calculated from the perspective of healthcare and transfer payment providers and the individual patient. Indirect costs for lost productivity were also calculated. Costs are presented as means +/- standard deviation (SD). Multivariate regression analyses were performed to identify independent cost predictors. Costs are in year 2000-02 values.
We estimated average per patient direct, indirect and total costs for the 6-month observation period. The costs from the perspective of statutory health insurance (Gesetzliche Krankenkversicherung [GKV]) consisted of direct medical costs 1370 euro +/- 3240 euro, including rehabilitation (420 euro +/- 1630 euro), hospitalisation (710 euro +/-2520 euro), outpatient treatment (40 euro +/- 30 euro), ancillary treatment (190 euro +/- 280 euro) and ambulatory diagnostic procedures (10 euro +/-30 euro). In addition, parkinsonian drug costs were 1520 euro +/-euro1250. Non-medical direct costs calculated from the GKV perspective were estimated to be euro480 +/-euro1710, which included transportation (10 euro+/- 20 euro), special equipment (420 euro +/- 1640 euro), social/home-help services (10 euro +/-110 euro) and sickness benefit (40 euro +/- 540 euro). The total medical (including drug costs) and non-medical direct costs for the GKV were 3380 euro +/- 4230 euro. Univariate predictors for GKV direct costs included occurrence of motor complications and falls, disease severity, nightmares and dementia. However, multivariate analyses only suggested disease severity and health-related quality of life as significant predictors. For nursing insurance, payments of 1330 euro +/- 2890 euro were calculated. For retirement insurance, payments were 650 euro +/- 1510 euro and there were patient (or caregiver) costs of 1490 euro +/- 2730 euro. Total indirect costs amounted to 3180 euro +/-6480 euro.
According to our study, PD puts a high financial burden on society and underscores the need for further economic and medical research to optimise treatment for PD.
PharmacoEconomics 02/2005; 23(8):817-36. · 2.66 Impact Factor
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Annika E. Spottke,
Martin Reuter,
Olaf Machat, Bernhard Bornschein,
Sonja von Campenhausen,
Karin Berger,
Rudolf Koehne-Volland,
Jurgen Rieke,
Alexander Simonow,
Dirk Brandstaedter,
Uwe Siebert,
Wolfgang H. Oertel,
Gudrun Ulm,
Richard Dodel
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ABSTRACT: Objective: To prospectively evaluate the health economic burden of patients with Parkinson's disease (PD) in Germany over a 6-month observation period and to identify the predictors of these costs. Study design and methods: Direct and indirect costs were evaluated in 145 patients with PD (mean age 67.3 +- 9.6 years). PD patients were recruited from an outpatient department for movement disorders, a specialised PD clinic, two office-based neurologists and general practitioners, all located in Germany, and were enrolled between January and June 2000. Relevant economic data were documented in a patient diary over the 6-month period. Clinical evaluations (Unified Parkinson's Disease Rating Scale [UPDRS]) were performed at baseline and at 3 and 6 months. Costs were derived from various German medical economic resources. Costs were calculated from the perspective of healthcare and transfer payment providers and the individual patient. Indirect costs for lost productivity were also calculated. Costs are presented as means +- standard deviation (SD). Multivariate regression analyses were performed to identify independent cost predictors. Costs are in year 2000-02 values. Results: We estimated average per patient direct, indirect and total costs for the 6-month observation period. The costs from the perspective of statutory health insurance (Gesetzliche Krankenkversicherung [GKV]) consisted of direct medical costs _1370 +- _3240, including rehabilitation (_420 +- _1630), hospitalisation (_710 +- _2520), outpatient treatment (_40 +- _30), ancillary treatment (_190 +- _280) and ambulatory diagnostic procedures (_10 +- _30). In addition, parkinsonian drug costs were _1520 +- _1250. Non-medical direct costs calculated from the GKV perspective were estimated to be _480 +- _1710, which included transportation (_10 +- _20), special equipment (_420 +- _1640), social/home-help services (_10 +- _110) and sickness benefit (_40 +- _540). The total medical (including drug costs) and non-medical direct costs for the GKV were _3380 +- _4230. Univariate predictors for GKV direct costs included occurrence of motor complications and falls, disease severity, nightmares and dementia. However, multivariate analyses only suggested disease severity and health-related quality of life as significant predictors. For nursing insurance, payments of _1330 +- _2890 were calculated. For retirement insurance, payments were _650 +- _1510 and there were patient (or caregiver) costs of _1490 +- _2730. Total indirect costs amounted to _3180 +- _6480. Conclusion: According to our study, PD puts a high financial burden on society and underscores the need for further economic and medical research to optimise treatment for PD.
PharmacoEconomics 01/2005; 23(8):817-836. · 2.66 Impact Factor
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ABSTRACT: As a diagnostic test for patients with suspected Parkinson's disease (PD), single photon emission computed tomography (SPECT) using [(123)I]FP-CIT tracer has better sensitivity but is more expensive than regular clinical examination (CE). Our objective was to evaluate the clinical and economic impacts of different diagnostic strategies involving [(123)I]FP-CIT SPECT. We developed a decision tree model to predict adequate treatment-month equivalents (ATME), costs, and incremental cost-effectiveness ratio (ICER) during a 12-month time horizon in patients with suspected PD referred to a specialized movement disorder outpatient clinic. In our cost- effectiveness analysis, we adopted the perspective of the German health care system and used data from a German prospective health care utilization study (n = 142) and published diagnostic studies. Compared strategies were CE only (EXAM+), SPECT only (SPECT+), SPECT following negative CE (SINGLE+), and SPECT following positive CE (DOUBLE+). Costs of SPECT amounted to euro;789 per investigation. Based on our model, expected costs (and ATME) were euro;946 (52.85 ATME) for EXAM+, euro;1352 (53.40 ATME) for DOUBLE+, euro;1731 (32.82 ATME) for SINGLE+, and euro;2003 (32.96 ATME) for SPECT+; performance of SPECT was induced in 0%, 54%, 56%, and 100% of the patients, respectively. DOUBLE+ was more effective and less expensive than SINGLE+ or SPECT+; thus these two do not offer reasonable choices. The ICER of DOUBLE+ compared to EXAM+ was euro;733 per ATME gained. In sensitivity analyses, the ICER of DOUBLE+ versus EXAM+ ranged from euro;63 to euro;2411 per ATME gained. Whether the diagnostic work-up of patients referred to a specialized movement disorder clinic with a high prevalence of PD should include [(123)I]FP-CIT SPECT depends on patient preferences and the decision maker's willingness to pay for adequate early treatment. SPECT should be used as a confirmatory test before treatment initiation and limited to patients with a positive test result in the clinical examination. These results should be adjusted to the specific setting and individual patient preferences.
Movement Disorders 11/2003; 18 Suppl 7:S52-62. · 4.51 Impact Factor
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ABSTRACT: To give an insight into the structural and methodological approaches used in published decision-analytic models evaluating interventions in Parkinson's disease (PD) and to derive recommendations for future comprehensive PD decision models.
A systematic literature review was performed to identify studies that evaluated PD interventions using mathematical decision models. Using a standardized assessment form, information on the study design, methodological framework, and data sources was extracted from each publication and systematically reported. Strengths and limitations were assessed.
We identified eight studies that used mathematical models to evaluate different pharmaceutical (n=7) and surgical (n=1) treatment options in PD. All models included economic evaluations. Modeling approaches comprised mathematical equations, decision trees, and Markov models with a time horizon ranging from 5 years to lifetime. All based progression on the evolution of clinical surrogate endpoints. Treatment effects were either modeled via reduction of symptomatic progression and/or initial symptomatic improvement or via reduction of adverse effect rates. No model is currently available that encompasses both the underlying biologic disease progression and the spectrum of all relevant complications and also links them to patient preferences and economic outcomes.
Models have been successfully applied to evaluate PD treatments. However, currently available models have substantial limitations. We recommend that a comprehensive, generic, and flexible decision model for PD that can be applied to different treatment strategies should consider a large spectrum of clinically relevant outcomes and complications of the disease during a sufficiently long time horizon, include PD-specific mortality, systematically evaluate uncertainty including heterogeneity effects, and should be validated by independent data or other models. Approaches to model treatment effects included reduction of symptomatic progression, initial symptomatic improvement, or reduction of adverse effects. We believe that structural bias could be avoided if underlying disease progression and treatment effects on symptoms are modeled separately.
Value in Health 7(5):610-26. · 2.19 Impact Factor
