Goud op de 100 meter HTA: van zorginnovaties voor chronisch zieken

Rede, in verkorte vorm uitgesproken bij de aanvaarding van het ambt van buitengewoon hoogleraar Economische Evaluatie van Zorginnovaties voor Chronisch Zieken aan het instituut Beleid & Management Gezondheidszorg van de Faculteit der Geneeskunde en Gezondheidswetenschappen, Erasmus Universiteit Rotterdam, op vrijdag 22 juni 2012. ‘Goud op de 100 meter HTA (Health Technology Assessment) van zorginnovaties voor chronisch zieken’ gaat in op de mogelijkheden van HTA onderzoek om de doelmatigheid van concrete interventies op de speerpunten van het beleid ten aanzien van chronische ziekten te onderbouwen. Deze speerpunten zijn preventie, vroege opsporing en vroege behandeling, optimaliseren van medicatie gebruik, verschuiving van ziekenhuiszorg naar thuiszorg, eHealth / telemedicine en disease management. Maar zelfs met de uitkomsten van gedegen economisch evaluatieonderzoek in de hand, blijft het maken van keuzes in de zorg moeilijk. Op de 100 meter HTA blijk

Economic appraisal of asthma and COPD care: A literature review 1980–1991

Despite the considerable burden and costs of illness and despite the increasing need to set priorities on the basis of efficiency considerations, only 20 economic appraisals of asthma and COPD care have been published during the past 11 years. This paper provides a detailed summary of the cost-effectiveness ‘evidence’ given by these studies and a discussion of relevant methodological issues. The studies comparing programme costs of delivery methods for oxygen and for aerosol bronchodilator drugs, provide the most straightforward evidence in favour of the concentrator and the metered dose inhaler respectively. There also seems to be evidence in favour of hospital-based home care programmes as compared to community-based home care programs. Health education, especially directed at asthmatic children seems to reduce health care costs and improve attitude, compliance behaviour and self-management skills. Information on the cost-effectiveness of pharmacotherapy and diagnostic technologies, both important interventions in asthma and COPD, was found to be totally lacking

Mix and match. A simulation study on the impact of mixed-Treatment comparison methods on health-economic outcomes

Background Decision-Analytic cost-effectiveness (CE) models combine many parameters, often obtained after meta-Analysis. Aim We compared different methods of mixed-Treatment comparison (MTC) to combine transition and event probabilities derived from several trials, especially with respect to health-economic (HE) outcomes like (quality adjusted) life years and costs. Methods Trials were drawn from a simulated reference population, comparing two of four fictitious interventions. The goal was to estimate the CE between two of these. The amount of heterogeneity between trials was varied in scenarios. Parameter estimates were combined using direct comparison, MTC methods proposed by Song and Puhan, and Bayesian generalized linear fixed effects (GLMFE) and random effects models (GLMRE). Parameters were entered into a Markov model. Parameters and HE outcomes were compare

Cost-effectiveness analysis of face-to-face smoking cessation interventions by professionals

Objectives: To estimate the cost-effectiveness of five face-to-face smoking cessation interventions: 1) Telephone Counseling (TC), 2) Minimal counseling by a general practitioner (H-MIS), 3) Minimal counseling by a general practitioner combined with Nicotine Replacement Therapy (H-MIS+NRT), 4) Intensive Counseling combined with Nicotine Replacement Therapy (IC+NRT) and 5) Intensive Counseling combined with Bupropion (IC+Bupr), in terms of costs per quitter, costs per life-year gained and costs per quality-adjusted life-year (QALY) gained. Methods: Scenarios on increased implementation of smoking cessation interventions were compared to current practice. Base-case scenarios assumed that one of the five interventions was implemented for a period of either 1 year, 10 years or 75 years and reached 25% of the smokers. A computer simulation model, the RIVM Chronic Disease Model, was used to project future gains in life-years and Quality Adjusted Life Years (QALYs), and savings of health care costs from a decrease in the incidence of smoking-related diseases. Regardless of the duration for which the intervention was implemented, our time horizon was 75 years, i.e. costs and effects were studied over a period of 75 years. Intervention costs were computed based on bottom up estimates of resource use and costs per unit of resource use. Cost calculations of smoking cessation interventions were carried out from a health care perspective, i.e. total direct medical costs were calculated based on estimates of real resource use. Effectiveness in terms of cessation rates was obtained from Cochrane meta-analyses. For the base-case scenarios, future costs and effects were discounted at an annual percentage of 4%. Incremental cost-effectiveness ratios were calculated as: (additional intervention costs minus the savings from a reduced incidence of smoking related diseases) / (gain in health outcomes). A series of one-way sensitivity analyses were performed to assess the robustness of the cost-effectiveness ratios with regard to variations in cessation rates, intervention costs, discount rates, time horizon, and the percentage of smokers reached by the intervention. Results: Base-case estimates for costs per quitter ranged from Euro 443 for H-MIS to Euro 2800 for IC+NRT. Compared to current practice H-MIS is a dominant intervention regardless of the duration of implementation. This means that H-MIS not onl

Cost-effectiveness of face-to-face smoking cessation interventions: A dynamic modeling study

Objectives: To estimate the cost-effectiveness of five face-to-face smoking cessation interventions (i.e., minimal counseling by a general practitioner (GP) with, or without nicotine replacement therapy (NRT), intensive counseling with NRT, or bupropion, and telephone counseling) in terms of costs per quitter, costs per life-year gained, and costs per quality-adjusted life-year (QALY) gained. Methods: Scenarios on increased implementation of smoking cessation interventions were compared with current practice in The Netherlands. One of the five interventions was implemented for a period of 1, 10, or 75 years reaching 25% of the smokers each year. A dynamic population model, the RIVM chronic disease model, was used to project future gains in life-years and QALYs, and savings of health-care costs from a decrease in the incidence of 11 smoking-related diseases over a time horizon of 75 years. This model allows the repetitive application of increased cessation rates to a population with a changing demographic and risk factor mix. Sensitivity analyses were performed for variations in costs, effects, time horizon, program size, and discount rates. Results: Compared with current practice, minimal GP counseling was a dominant intervention, generating both gains in life-years and QALYs and savings that were highe

Current and Future Incidence and Costs of Osteoporosis-Related Fractures in the Netherlands

This study aims to estimate the incidence and costs of osteoporosis-related fractures in The Netherlands in 2010 and project them to 2030. The incidence and costs of five different types of fractures (spine, hip, upper extremity, lower extremity, wrist/distal forearm, other) were derived from claims data of all Dutch healthcare insurers. Given that fracture-codes in claims data do not indicate whether fractures are related to osteoporosis, we used a large dataset with DXA measurements to attribute fractures to osteoporosis. Future projections used four scenarios: (1) demographic, (2) demographic ? annual trend in incidence rates, (3) demographic ? annual trend in incidence rates ? annual trend in costs, and (4) treatment. Of all registered fractures, 32 % was attributed to osteoporosis (36 % in women and 21 % in men). Over time (2010–2030) the increase in incidence of osteoporosis-related fractures was estimated to be 40 % (scenario 1); for the hip 60–79 % (scenario 1–2). In 2010, approximately €200 million was spent on treatment of osteoporosis-related fractures, most on fractures of the hip followed by wrist/distal forearm. In both men and women, the excess costs due to osteoporosis-related fractures were highest for hip fractures (€11,000–€13,000 per person), followed by spine fractures (€6000–€7000).The costs for osteoporosisrelated fractures were projected to increase with 50 % from 2010 to 2030 (scenario 1); for the hip 60–148 % (scenario 1–3). Pharmacotherapeutic prevention can lead to costsavings of €377 million in 2030 (scenario 1 and 4 combined). The projected increase in incidence and costs of osteoporosis-related fractures calls for a wider use of prevention and treatment

Short- and long-term efficacy of a community-based COPD management programme in less advanced COPD: a randomised controlled trial

BACKGROUND: The effectiveness of pulmonary rehabilitation in advanced COPD is well established, but few data are available in less advanced disease. METHODS: In a 2 year randomised controlled trial, 199 patients with an average moderate airflow obstruction but impaired exercise capacity (mean (SD) forced expiratory volume in 1 s (FEV(1)) 60 (16)%, peak work load (Wmax) <70%) were randomised to the INTERdisciplinary COMmunity-based COPD management programme (INTERCOM) or usual care. Intervention consisted of 4 months multidisciplinary rehabilitation followed by a 20-month maintenance phase. Outcomes (4, 12, 24 months): health-related quality of life (St George's Respiratory Questionnaire (SGRQ)), exacerbation frequency, MRC dyspnoea score, cycle endurance time (CET), 6-minute walking distance (6MWD), skeletal muscle strength and patients' and caregivers' perceived effectiveness. RESULTS: Between-group comparison after 4 months revealed the following mean (SE) significant differences in favour of INTERCOM: SGRQ total score 4.06 (1.39), p = 0.004; activity and impact subscores, p<0.01; MRC score 0.33 (0.13), p = 0.01; Wmax 6.0 (2.3) Watt, p = 0.02; CET 221 (104) s, p = 0.04; 6MWD 13 (6) m, p = 0.02; hand grip force 4.3 (1.5) lb, p<0.01; and fat-free mass index 0.34 (0.13) kg/m(2), p = 0.01. Between-group differences over 2 years were as follows: SGRQ 2.60 (1.3), p = 0.04; MRC score 0.21 (0.10), p = 0.048; CET 253 (104) s, p = 0.0156; 6MWD 18 (8) m, p = 0.0155. Exacerbation frequency was not different (RR 1.29 (95% CI 0.89 to 1.87)). Patients' and caregivers' perceived effectiveness significantly favoured the INTERCOM programme (p<0.01). CONCLUSIONS: This study shows that a mu

How to Address Uncertainty in Health Economic Discrete-Event Simulation Models

__Background__. Evaluation of personalized treatment options requires health economic models that include multiple patient characteristics. Patient-level discrete-event simulation (DES) models are deemed appropriate because of their ability to simulate a variety of characteristics and treatment pathways. However, DES models are scarce in the literature, and details about their methods are often missing. __Methods__. We describe 4 challenges associated with modeling heterogeneity and structural, stochastic, and parameter uncertainty that can be encountered during the development of DES models. We explain why these are important and how to correctly implement them. To illustrate the impact of the modeling choices discussed, we use (results of) a model for chronic obstructive pulmonary disease (COPD) as a case study. __Results__. The results from the case study showed that, under a correct implementation of the uncertainty in the model, a hypothetical intervention can be deemed as cost-effective. The consequences of incorrect modeling uncertainty included an increase in the incremental cost-effectiveness ratio ranging from 50% to almost a factor of 14, an extended life expectancy of approximately 1.4 years, and an enormously increased uncertainty around the model outcomes. Thus, modeling uncertainty incorrectly can have substantial implications for decision making. __Conclusions__. This article provides guidance on the implementation of uncertainty in DES models and improves the transparency of reporting uncertainty methods. The COPD case study illustrates the issues described in the article and helps understanding them better. The model R code shows how the uncertainty was implemented. For readers not familiar with R, the model’s pseudo-code can be used to understand how the model works. By doing this, we can help other developers, who are likely to face similar challenges to those described here

Does the 2013 GOLD classification improve the ability to predict lung function decline, exacerbations and mortality: a post-hoc analysis of the 4-year UPLIFT trial

BACKGROUND: The 2013 GOLD classification system for COPD distinguishes four stages: A (low symptoms, low exacerbation risk), B (high symptoms, low risk), C (low symptoms, high risk) and D (high symptoms, high risk). Assessment of risk is based on exacerbation history and airflow obstruction, whatever results in a higher risk grouping. The previous system was solely based on airflow obstruction. Earlier studies compared the predictive performance of new and old classification systems with regards to mortality and exacerbations. The objective of this study was to compare the ability of both classifications to predict the number of future (total and severe) exacerbations and mortality in a different patient population, and to add an outcome measure to the comparison: lung function decline.METHODS: Patient-level data from the UPLIFT trial were used to analyze 4-year survival in a Weibull model, with GOLD stages at baseline as covariates. A generalized linear model was used to compare the numbers of exacerbations (total and severe) per stage. Analyses were repeated with stages C and D divided into substages depending on lung function and exacerbation history. Lung function decline was analysed in a repeated measures model.RESULTS: Mortality increased from A to D, but there was no difference between B and C. For the previous GOLD stages 2-4, survival curves were clearly separated. Yearly exacerbation rates were: 0.53, 0.72 and 0.80 for stages 2-4; and 0.35, 0.45, 0.58 and 0.74 for A-D. Annual rates of lung function decline were: 47, 38 and 26 ml for stages 2-4 and 44, 48, 38 and 39 for stages A-D. With regards to model fit, the new system performed worse at predicting mortality and lung function decline, and better at predicting exacerbations. Distinguishing between the sub-stages of high-risk led to substantial improvements.CONCLUSIONS: The new classification system is a modest step towards a phenotype approach. It is probably an improvement for the prediction of exacerbations, but a deterioration for predicting mortality and lung function decline.TRIAL REGISTRATION: ClinicalTrials.gov NCT00144339 (September 2, 2005)