Challenges of consolidating evidence collected during a pandemic and lessons for the future.

OBJECTIVE: To illustrate the challenges encountered when gathering rapidly synthesized evidence in response to the coronavirus disease 2019 (COVID-19) pandemic. METHODS: In this article, we describe the challenges encountered when we performed a systematic literature review (SLR) of randomized controlled trials (RCTs) on the efficacy and safety of treatments for severe COVID-19. The methods of the SLR are described in full, to show the context of our objectives. Then we use the results of the SLR to demonstrate the problems of producing synthesized evidence in this setting. RESULTS: Various challenges were identified during this SLR. These were primarily a result of heterogeneity in the study methodology of eligible studies. Definitions of the patient populations and outcome measurements were highly variable and the majority of studies demonstrated a high risk of bias, preventing quantitative synthesis of the collated evidence. CONCLUSION: Consolidating evidence from RCTs evaluating COVID-19 interventions was problematic. Guidance is needed for scenarios with high rapid output in primary research.

Clinical and economic outcomes of adding [18F]FES PET/CT in estrogen receptor status identification in metastatic and recurrent breast cancer in the US.

BACKGROUND AND OBJECTIVES: Correct identification of estrogen receptor (ER) status in breast cancer (BC) is crucial to optimize treatment; however, standard of care, involving biopsy and immunohistochemistry (IHC), and other diagnostic tools such as 2-deoxy-2-[18F]fluoro-D-glucose or 2-[18F]fluoro-2-deoxy-D-glucose ([18F]FDG), can yield inconclusive results. 16α-[18F]fluoro-17ß-fluoroestradiol ([18F]FES) can be a powerful tool, providing high diagnostic accuracy of ER-positive disease. The aim of this study was to estimate the budget impact and cost-effectiveness of adding [18F]FES PET/CT to biopsy/IHC in the determination of ER-positive status in metastatic (mBC) and recurrent breast cancer (rBC) in the United States (US). METHODS: An Excel-based decision tree, combined with a Markov model, was developed to estimate the economic consequences of adding [18F]FES PET/CT to biopsy/IHC for determining ER-positive status in mBC and rBC over 5 years. Scenario A, where the determination of ER-positive status is carried out solely through biopsy/IHC, was compared to scenario B, where [18F]FES PET/CT is used in addition to biopsy/IHC. RESULTS: The proportion of true positive and true negative test results increased by 0.2 to 8.0 percent points in scenario B compared to scenario A, while re-biopsies were reduced by 94% to 100%. Scenario B resulted in cost savings up to 142 million dollars. CONCLUSIONS: Adding [18F]FES PET/CT to biopsy/IHC may increase the diagnostic accuracy of the ER status, especially when a tumor sample cannot be obtained, or the risk of a biopsy-related complication is high. Therefore, adding [18F]FES PET/CT to biopsy/IHC would have a positive impact on US clinical and economic outcomes.

A comprehensive analysis of clinical, quality of life, and cost-effectiveness outcomes of key treatment options for benign prostatic hyperplasia.

Treatment options for men with moderate-to-severe lower urinary tract symptoms (LUTS) due to benign prostatic hyperplasia (BPH) have variable efficacy, safety, and retreatment profiles, contributing to variations in patient quality of life and healthcare costs. This study examined the long-term cost-effectiveness of generic combination therapy (CT), prostatic urethral lift (PUL), water vapor thermal therapy (WVTT), photoselective vaporization of the prostate (PVP), and transurethral resection of the prostate (TURP) for the treatment of BPH. A systematic literature review was performed to identify clinical trials of CT, PUL, WVTT, PVP, and TURP that reported change in International Prostate Symptom Score (IPSS) for men with BPH and a prostate volume ≤80 cm3. A random-effects network meta-analysis was used to account for the differences in patient baseline clinical characteristics between trials. An Excel-based Markov model was developed with a cohort of males with a mean age of 63 and an average IPSS of 22 to assess the cost-effectiveness of these treatment options at 1 and 5 years from a US Medicare perspective. Procedural and adverse event (AE)-related costs were based on 2021 Medicare reimbursement rates. Total Medicare costs at 5 years were highest for PUL ($9,580), followed by generic CT ($8,223), TURP ($6,328), PVP ($6,152), and WVTT ($2,655). The total cost of PUL was driven by procedural ($7,258) and retreatment ($1,168) costs. At 5 years, CT and PUL were associated with fewer quality-adjusted life years (QALYs) than WVTT, PVP, and TURP. Compared to WVTT, the incremental cost-effectiveness ratios (ICERs) for both TURP and PVP were above a willingness-to-pay threshold of $50,000/QALY (TURP: $64,409/QALY; PVP: $87,483/QALY). This study provides long-term cost-effectiveness evidence for several common treatment options for men with BPH. WVTT is an effective and economically viable treatment in resource-constrained environments.

Cost-Effectiveness and Budget Impact of Emerging Minimally Invasive Surgical Treatments for Benign Prostatic Hyperplasia.

Background: Benign prostatic hyperplasia (BPH) is one of the most prevalent and costly chronic conditions among middle-aged and elderly men. Prostatic urethral lift (PUL) and convective water vapor thermal therapy (WVTT) are emerging minimally invasive surgical treatments as an alternative to traditional treatment options for men with moderate-to-severe BPH. This study evaluated the cost-effectiveness and budget impact of PUL and WVTT for men with BPH using long-term clinical outcomes. Methods: The cost-effectiveness and budget impact models were developed from a US Medicare perspective over a 4-year time horizon. The models were populated with males with a mean age of 63 and an average International Prostate Symptom Score (IPSS) of 22. Clinical inputs were extracted from the LIFT and Rezum II randomized controlled trials at 4 years. Utility values were assigned using IPSS and BPH severity levels. Procedural, adverse event, retreatment, follow-up, and medication costs were based on 2019 Medicare payment rates and Medicare Part D drug spending. One-way and probabilistic sensitivity analyses (PSAs) were performed. Results: At 4 years, PUL was associated with greater retreatment rates (24.6% vs 10.9%), lower quality-adjusted life-years (QALYs) (3.490 vs 3.548) and higher total costs (US$7393 vs US$2233) compared with WVTT, making WVTT the more effective and less costly treatment strategy. The 70% total cost difference of PUL and WVTT was predominantly driven by higher PUL procedural (US$5617 vs US$1689) and retreatment (US$976 vs US$257) costs. The PSA demonstrated that relative to PUL, WVTT yielded higher QALYs and lower costs 99% and 100% of the time, respectively. Conclusions: Compared to PUL, WVTT was a cost-effective and cost-saving treatment of moderate-to-severe BPH. These findings provide evidence for clinicians, payers, and health policy makers to help further define the role of minimally invasive surgical treatments for BPH.

Budget impact of niraparib as maintenance treatment in recurrent ovarian cancer following platinum-based chemotherapy.

Aim: To estimate financial implications of adopting niraparib as maintenance treatment in recurrent ovarian cancer. Materials & methods: A model was developed to estimate the budget impact of treating patients with niraparib compared with alternative maintenance treatment options (olaparib, rucaparib, bevacizumab or 'watch and wait') over 3 years. Results: For a hypothetical plan with 1 million lives representative of US/Medicare-only populations, projected cost savings with niraparib were US$78,721/$293,723, $276,671/$1,009,729 and $353,585/$1,289,712 at years 1, 2 and 3, respectively. Sensitivity analyses showed prices of niraparib, rucaparib and olaparib to have the most significant impact on the budget. Conclusion: Factoring in all treatment-related costs, the use of niraparib could result in significant cost savings compared with other maintenance treatment options.

Economic assessment of traditional surgical intervention versus use of a new innovative radiofrequency based surgical system in device replacements.

INTRODUCTION: Intra-operative complications like mechanical damages to the leads, infections and hematomas during generator replacements of implantable pacemakers and defibrillators contribute to additional costs for hospitals. The aim of this study was to evaluate operation room use, costs and budget impact of generator replacements using either a traditional surgical intervention (TSI) with scissors, scalpel and electrocautery vs. a new radiofrequency energy based surgical system, called PEAK PlasmaBladeTM (PPB). MATERIALS AND METHODS: We conducted a retrospective analysis of a population including 508 patients with TSI and 254 patients with PPB who underwent generator replacement at the Kepler University Hospital in Linz or the St. Josef Hospital in Braunau, Austria. The economic analysis included costs of resources used for intra-operative complications (lead damages) and of procedure time for TSI vs. PPB. RESULTS: Proportion of males, mean age and type of generator replaced were similar between the two groups. Lead damages occurred significantly more frequent with TSI than with PPB (5.3% and 0.4%; p< 0.001) and the procedure time was significantly longer with TSI than with PPB (47.9±24.9 and 34.1±18.1 minutes; p<0.001). Shorter procedure time and a lower rate of lead damages with PPB resulted in per patient cost savings of €81. Based on estimated 2,700 patients annually undergoing generator replacement in Austria, the use of PPB may translate into cost savings of €219,600 and 621 saved operating facility hours. CONCLUSION: PPB has the potential to minimize the risk of lead damage with more efficient utilization of the operating room. Along with cost savings and improved quality of care, hospitals may use the saved operating room hours to increase the number of daily surgeries.

Description of a New Predictive Modeling Approach That Correlates the Risk and Associated Cost of Well-Defined Diabetes-Related Complications With Changes in Glycated Hemoglobin (HbA1c).

The modeling approach described here is designed to support the development of spreadsheet-based simple predictive models. It is based on 3 pillars: association of the complications with HbA1c changes, incidence of the complications, and average cost per event of the complication. For each pillar, the goal of the analysis was (1) to find results for a large diversity of populations with a focus on countries/regions, diabetes type, age, diabetes duration, baseline HbA1c value, and gender; (2) to assess the range of incidences and associations previously reported. Unlike simple predictive models, which mostly are based on only 1 source of information for each of the pillars, we conducted a comprehensive, systematic literature review. Each source found was thoroughly reviewed and only sources meeting quality expectations were considered. The approach allows avoidance of unintended use of extreme data. The user can utilize (1) one of the found sources, (2) the found range as validation for the found figures, or (3) the average of all found publications for an expedited estimate. The modeling approach is intended for use in average insulin-treated diabetes populations in which the baseline HbA1c values are within an average range (6.5% to 11.5%); it is not intended for use in individuals or unique diabetes populations (eg, gestational diabetes). Because the modeling approach only considers diabetes-related complications that are positively associated with HbA1c decreases, the costs of negatively associated complications (eg, severe hypoglycemic events) must be calculated separately.

An electrical plasma surgery tool for device replacement--retrospective evaluation of complications and economic evaluation of costs and resource use.

BACKGROUND: Device replacements bear many potential risks for patients. Electrocautery should be used cautiously because of heating of the cautery tip which may lead to insulation melting. The PEAK PlasmaBlade™ (Medtronic Inc., Minneapolis, MN, USA) uses a novel technology to cut tissue. The objective of this study was to evaluate if this instrument is less destructive and can decrease complications and shorten procedure times. METHODS AND RESULTS: Two groups were compared: in group 1, surgery was done with scissors and conventional electrocautery, whereas the PEAK PlasmaBlade™ was used in group 2. Procedure time and complication rates were retrospectively investigated. Group 1 comprised 509 patients, while group 2 consisted of 102 patients. Procedure time in group 2 was significantly shorter with 28.4 ± 8.9 minutes than in group 1 with 47.5 ± 24.5 minutes (P < 0.001). The hospital stay was also reduced (2.1 ± 2.2 days versus 3.1 ± 2.4 days, P < 0.001). One major complication occurred in only 2.4% (two patients) of group 2 and in 6.9% (35 patients) of group 1. There were no damaged leads in group 2 compared to 5.7% in group 1 (P = 0.008). Cost analyses showed that the use of The PEAK PlasmaBlade™ instead of conventional electrocautery resulted in cost savings of €120/patient. CONCLUSION: Device replacement is associated with a notable complication risk. Our investigation showed that a new device for generator replacement resulted in significantly reduced procedure time and length of hospital stay while completely avoiding lead damage, which translated into considerable cost savings in a real world setting.

The effect of nurse-led diabetes self-management education on glycosylated hemoglobin and cardiovascular risk factors: a meta-analysis.

PURPOSE: The purpose of this meta-analysis was to determine the effect of nurse-led diabetes self-management education (DSME) on blood glucose control and cardiovascular risk factors. METHODS: The electronic databases PubMed and ISIS Knowledge were searched for relevant randomized controlled studies published between 1999 and 2009. Effect size was calculated for change in A1C, blood pressure, and lipid levels using both fixed- and random-effects models. Subgroup analyses were performed on patient age, gender, diabetes type, baseline A1C, length of follow-up, and study setting. RESULTS: A total of 34 randomized controlled trials with a combined cohort size of 5993 patients was identified. Mean patient age was 52.8 years, 47% were male, and mean A1C at baseline was 8.5%. Mean change in A1C was a reduction by -0.70% for nurse-led DSME versus -0.21% with usual care (UC). This corresponded to an effect size of 0.506, using a random-effects model for nurse-led DSME versus UC. Effect size was significantly associated with patient age older than 65 years and with duration of follow-up. Nurse-led DSME was also associated with improvements in cardiovascular risk factors, particularly among male patients, among those with good glycemic control, and in studies conducted in the United States. CONCLUSIONS: Nurse-led DSME is associated with improved glycemic control, demonstrating that programs are most effective among seniors and with follow-up periods of 1 to 6 months. Future programs tailored to the needs of patients younger than 65 years may improve the impact of DSME on blood glucose.

Glucose information for tight glycemic control: different methods with different challenges.

Rigorous glucose control is essential for prevention of diabetes-related complications in diabetes patients. Even without diabetes, tight glucose control is beneficial in hospitalized, critically ill patients. Actually, three different glucose measurement methods are used: (1) hand held devices, (2) blood-gas analyzers, and (3) laboratory analyzers in core laboratories. Each method is subject to specific challenges and limitations that can affect the overall system performance. In this article, we aim to demonstrate that even glucose measurement results from core laboratories (professional laboratory systems) do not necessarily reflect the absolute "true" glucose level of a patient.

Attitudes towards insulin pump therapy among adolescents and young people.

PURPOSE: This study investigated reasons for the rejection and discontinuation of insulin pump therapy and explored general attitudes towards this mode of therapy among young patients with type 1 diabetes. METHODS: A questionnaire was developed using a focus group of young people with diabetes. It was then used to survey a random sample of adolescents and young people identified by physicians specializing in diabetes care and participating in a voluntary quality improvement initiative in Germany. The physicians were also surveyed. RESULTS: Eighty-eight patients participated in the survey (22 had never used the pump, 20 had formerly used the pump, and 46 were using the pump at that time, with an average age of between 20 and 22 years, depending on the group). Those who had never used the pump had had diabetes for a significantly shorter length of time and had undergone their first diabetes education more recently. Current pump users were significantly younger at the time of the first diabetes education. There were no significant differences between patients concerning where they obtained their information about the condition and treatment options. Although clinical factors were named, social and psychological factors were prominent as reasons both in reluctance to try the pump therapy and in discontinuing therapy. Technical problems as a disadvantage of the pump (aside from the catheter) were less likely to be named. Responses among physicians confirmed discipline and compliance were essential prerequisites for this therapy and supported findings that patients discontinuing pump therapy at their own request tend to do so for nonclinical reasons. CONCLUSIONS: Although the technical reliability of the insulin pump was generally accepted by all patients regardless of current treatment, clinical disadvantages relating to the use of the pump but more commonly social/psychological factors were named, which resulted in patients being reluctant to try this therapy or discontinuing use of it.

Impact of self-measurement of blood glucose on complications of type 2 diabetes: economic analysis from a Czech perspective.

OBJECTIVE: A growing body of evidence indicates that self-measurement of blood glucose (SMBG) also has beneficial effects in people with type 2 diabetes, irrespective of the type of therapy. The objective of this analysis was to determine the economic impact of SMBG by comparing the cost share of self-monitoring and the direct costs of diabetes-related complications in users and non-users. RESEARCH DESIGN AND METHODS: A matched-pair analysis based on the cohorts of a large retrospective study of patients with type 2 diabetes (ROSSO) was conducted. The average annual direct costs of diabetes monitoring, treatment-related services, complications and follow-up costs of the disease for SMBG users versus non-users were calculated from the perspective of the Czech statutory health insurance system. Univariate sensitivity analysis was performed to determine the main cost drivers. Limitations of this study are: (1) differences in medical facilities/practice between Germany and the Czech Republic, (2) causal relationship between SMBG and health outcomes is missing, (3) ROSSO underestimated the number of test strips used, (4) Czech cost data are scarce. RESULTS: In patients treated with oral antidiabetic drugs (OAD) only, total annual costs in Czech koruna (CZK) were CZK 16 476 for SMBG users and CZK 19 440 for non-users. In patients treated with OAD + insulin, total annual costs were CZK 32 590 and CZK 48 600, respectively. The main cost drivers were stroke and myocardial infarction in patients treated with OAD only, and stroke, dialysis and myocardial infarction in patients treated with OAD + insulin. CONCLUSION: Cost analysis indicated that SMBG provides a rapid return on initial investment. By increasing the number of patients using SMBG, the statutory health insurance system in the Czech Republic may save several million CZK annually.

Cost impact of self-measurement of blood glucose on complications of type 2 diabetes: the Spanish perspective.

BACKGROUND: Despite the increasing prevalence of type 2 diabetes, its financial burden on the Spanish healthcare system remains unclear. This study was conducted to determine the cost share of self-measurement of blood glucose (SMBG) by comparing the direct costs of reduced complications of diabetes in SMBG users versus nonusers in the Spanish statutory health insurance system. METHODS: Matched-pair analysis was done of the average annual total direct cost of diabetes monitoring, treatment-related services, complications, and follow-up in the RetrOlective Study Self-Monitoring of Blood Glucose and Outcome in Patients with Type 2 Diabetes (ROSSO) study cohort, updated to 2008 from the year of occurrence or diagnosis of diabetes. RESULTS: In patients treated with oral antidiabetes drugs (OADs) only, total annual costs were euro1,934 in SMBG users and euro1,982 in nonusers. In those treated with OADs plus insulin, total annual costs were euro3,451 and euro4,167, respectively. By increasing the number of patients using SMBG, the Spanish statutory health insurance system might save several million Euros annually. CONCLUSIONS: The analysis showed that the promotion of SMBG in patients with type 2 diabetes is associated with considerable cost savings for the Spanish healthcare system.

Prevention of diabetic ketoacidosis and self-monitoring of ketone bodies: an overview.

OBJECTIVE: Diabetic ketoacidosis (DKA) is associated with significant morbidity and mortality. Self-monitoring of ketone bodies by diabetes patients can be done using blood or urine. We compared the two self-monitoring methods and summarized recent developments in the epidemiology and management of DKA. METHODS: MEDLINE and EMBASE were searched for relevant publications addressing the epidemiology, management and prevention of DKA up to 2009. The current, relevant publications, along with the authors' clinical and professional experience, were used to synthesize this narrative review. FINDINGS: Despite considerable advances in diabetes therapy, key epidemiological figures related to DKA remained nearly unchanged during the last decades at a global level. Prevention of DKA - especially in sick day management - relies on intensive self-monitoring of blood glucose and subsequent, appropriate therapy adjustments. Self-monitoring of ketone bodies during hyperglycemia can provide important, complementary information on the metabolic state. Both methods for self-monitoring of ketone bodies at home are clinically reliable and there is no published evidence favoring one method with respect to DKA prevention. CONCLUSIONS: DKA is still a severe complication potentially arising during prolonged hyperglycemic episodes with possibly fatal consequences. Education of patients and their social environment to promote frequent testing - especially during sick days - and to lower their glucose levels, as well as to recognize the early symptoms of hyperglycemia and DKA is of paramount importance in preventing the development of severe DKA. Both methods for self-monitoring of ketone bodies are safe and clinically reliable.

Cost impact of blood glucose self-monitoring on complications of type 2 diabetes: a Swiss perspective (ROSSO study No.11).

QUESTION UNDER STUDY: despite the increasing prevalence of type 2 diabetes, its financial burden on the Swiss healthcare system remains unclear. Our aim was to determine the cost of self-monitoring of blood glucose (SMBG) in reducing diabetic complications by comparing the direct costs to the Swiss statutory health insurance system of diabetic complications in SMBG users vs. nonusers. METHOD: matched pair analysis of the average annual total cost of diabetes monitoring, treatment-related services, complications and followup in the RetrOlective Study Self-Monitoring of Blood Glucose and Outcome in Patients with Type 2 Diabetes (ROSSO) study cohort, updated to 2005 from the year of occurrence or diagnosis of diabetes, applying an annual inflation rate of 5%. RESULTS: in those patients treated with oral antidiabetic drugs only, total annual costs were CHF 5,140 in SMBG users and CHF 5,654 in non - users. In those patients treated with oral antidiabetic drugs plus insulin, total annual costs were CHF 8,254 and CHF 11,776, respectively. SMBG accounted for 1.6% to 1.7% of total costs. CONCLUSION: cost analysis indicates that SMBG provides a rapid return on initial investment.

Cost-effectiveness analysis of a quality-controlled mammography screening program from the Swiss statutory health-care perspective: quantitative assessment of the most influential factors.

OBJECTIVES: Quality-controlled mammography screening programs (MSP) have led to a reduction in breast cancer mortality. The purpose of this economic analysis was to assess the cost-effectiveness of MSP compared with an established opportunistic screening strategy (OS) in Switzerland, to identify the major factors influencing the economic outcome. METHODS: Using cancer registries and clinical data, a Markov-based decision model was designed to compare MSP with OS in the Swiss female population, considering the main screening-specific performance parameters. RESULTS: The discounted incremental life expectancy amounted to 0.022 life-years gained in favor of MSP when screening started at age 40 years and decreased to 0.008 years at the age of 70 years (number needed to screen to avoid one death over 10 years ranged from 10,000 to 2439 women depending on the baseline age). The total discounted life-time cost for screening, treatment at the baseline age of 40 years amounted in MSP to $4366 (OS: $2802) and decreased with the baseline age of 70 years to $2412 (OS: $1446). The discounted incremental cost-effectiveness ratio comparing MSP versus OS ranged from $73,018 (age 40 years) to $118,193 (age 70 years) per life-year gained. Testing all model variables confirmed that both incidence and mortality of breast cancer play the most important role in the health economic outcome, whereas cost and performances (sensitivity, specificity) of screening had a minor impact on the efficiency. CONCLUSION: This analysis, performed under conservative assumptions, supports that MSP in Switzerland enables a relevant reduction of breast cancer mortality, at moderate additional cost, compared with OS.

Self-measurement of blood glucose in patients with type 2 diabetes: a health economic assessment.

BACKGROUND: The clinical role and the potential benefit of self-measurement of blood glucose (SMBG) for patients with type 2 diabetes are still under discussion. Even less information is available on the cost-effectiveness of performing SMBG by this patient group. The goal of this study was to establish cost-effectiveness ratios of performing SMBG by patients afflicted by this disease. METHODS: We assessed the benefit and cost-effectiveness of SMBG in type 2 diabetes from a third-party payer perspective based on results of both a large epidemiologic cohort study reflecting the reality of care, and a Markov model calculation. RESULTS: Analysis of cohort study data revealed that total costs cumulated over the observation period of 8 years were lower in the SMBG group than in the non-SMBG group according to savings of euro 1'714 [oral antidiabetic drugs (OAD) only] and euro 13'815 (OAD + insulin) per patient. Several scenarios were considered in the model-based calculation. The cost-effectiveness ratio varied from euro 20'768/life year gained to domination of SMBG use compared to nonusers in OAD treated patients and from euro 59'057/life year gained to domination of SMBG use compared to nonusers in OAD + insulin treated patients. CONCLUSION: Results indicate that SMBG in type 2 diabetes offers an excellent opportunity to get a high investment-outcome ratio in the treatment of this pandemic disease.

Using individualized predictive disease modeling to identify patients with the potential to benefit from a disease management program for diabetes mellitus.

Diabetes is an increasing health problem, but efforts to handle this pandemic by disease management programs (DMP) have shown conflicting results. Our hypothesis is that, in addition to a program's content and setting, the choice of the right patients is crucial to a program's efficacy and effectiveness. We used individualized predictive disease modeling (IPDM) on a cohort of 918 patients with type 2 diabetes to identify those patients with the greatest potential to benefit from inclusion in a DMP. A portion of the patients (4.7%) did not have even a theoretical potential for an increase in life expectancy and would therefore be unlikely to benefit from a DMP. Approximately 16.1% had an increase in life expectancy of less than half a year. Stratification of the entire cohort by surrogate parameters like preventable 10-year costs or gain in life expectancy was much more effective than stratification by classical clinical parameters such as high HbA1c level. Preventable costs increased up to 50.6% (or 1,010 per patient (1 = US dollars 1.28), p < 0.01) and life expectancy increased up to 54.8% (or 2.3 years, p < 0.01). IPDM is a valuable strategy to identify those patients with the greatest potential to avoid diabetes-related complications and thus can improve the overall effectiveness and efficacy of DMPs for diabetes mellitus.

Cost effectiveness of combination therapy with pioglitazone for type 2 diabetes mellitus from a german statutory healthcare perspective.

BACKGROUND: Pioglitazone has been approved in Europe for oral combination therapy for type 2 diabetes mellitus. Along with other agents of the thiazolidinedione class, it has a novel intracellular mechanism of action. Clinical trials with pioglitazone have confirmed a strong product profile in terms of control of blood glucose and lipids. However, the drug acquisition cost for pioglitazone is greater than standard medications for type 2 diabetes. Long-term data regarding the cost effectiveness of pioglitazone-based combination therapy are not available. OBJECTIVE: To evaluate, using a decision analysis model, the cost effectiveness of pioglitazone-based combination therapy compared with relevant alternative medications for the treatment of type 2 diabetes in Germany. METHODS: This study compared the clinical effects and costs of pioglitazone 30 mg added to metformin in patients who failed metformin monotherapy and pioglitazone added to a sulphonylurea in patients who failed sulphonylurea monotherapy, with the most relevant treatment alternatives. A published and validated Markov model was adapted to reflect the management of type 2 diabetes. This simulated the number of severe complications occurring and the mean life expectancy of a diabetic cohort, which was based on the overweight group of the UK Prospective Diabetes Study at year 6 of follow-up. Drug treatment costs, other costs for general management of type 2 diabetes and the costs of complications were combined to compute overall lifetime treatment costs from the perspective of the German statutory healthcare system in 2002. RESULTS: Combination therapy with pioglitazone/metformin was associated with a higher life expectancy (15.2 years) relative to sulphonylurea/metformin (14.9 years) or acarbose/metformin (14.7 years). Likewise, pioglitazone/sulphonylurea (15.5 years) was superior to metformin/sulphonylurea (14.9 years) and acarbose/sulphonylurea (14.8 years). Undiscounted incremental cost-effectiveness ratios in comparison to the next best strategy were euro20,002 per life-year gained (LYG) for pioglitazone/metformin versus sulphonylurea/metformin, and euro8707 per LYG for pioglitazone/sulphonylurea versus metformin/sulphonylurea. After discounting costs and life expectancy at 5% per year, the incremental cost-effectiveness ratio was euro47 636 per LYG for pioglitazone/metformin versus sulphonylurea/metformin, and euro19 745 per LYG for pioglitazone/sulphonylurea versus metformin/sulphonylurea. CONCLUSIONS: In this model, with its underlying assumptions and data, combination therapy with pioglitazone increased life expectancy in overweight type 2 diabetes patients at acceptable cost compared with other well established medications in Germany. These findings should be re-evaluated as soon as additional evidence becomes available from the currently ongoing long-term clinical and economic studies.