Health Technology Assessment of Medical Interventions in the Prevention and Treatment of Disease: Directions of Further Research and Policy Implications

Health technology assessment (HTA) originated from the spread of costly medical equipment and growing concerns over the ability and willingness of taxpayers and health insurers to pay for them. The main aim of HTA is to provide a range of stakeholders, typically those involved in funding, planning, purchasing, and investing in healthcare, with accessible, useable, and evidence-based information that will guide decisions about technology and the efficient allocation of resources. The present thesis addresses some of the methodological challenges of the health technology assessment and evaluates the impact of economic evaluations in healthcare decision-making process. Systematic reviews have a central role in evidence based-medicine. The quantitative systematic review, also known as meta-analysis provides a logical structure for quantifying the existing evidence. Meta-analysis offers the opportunity to critically evaluate and statistically combine results of comparable studies or trials. The aim is to get a consistent estimation of the global effect of a procedure on a specified outcome by increasing the number of observations and statistical power. This thesis presents the results of a systematic literature review with meta-analysis on the effectiveness of lifestyle intervention in the prevention and treatment of obesity. The systematic review provides new information on the effectiveness of lifestyle interventions by assessing the mid- to long-term effects on weight and cardiovascular risk profile in overweight and obese people. Patient reported outcomes provide the patient perspective on the effectiveness of treatment. This thesis presents the data collected from a study that establishes the validity of the electronic versions of three quality of life measures in comparison to the existing paper versions. For health technology manufacturers it is important to provide evidence of the validity of the quality of life instruments in the electronic format as compared to paper version. Mathematical modeling is used widely in economic evaluations of medical interventions. Health economics models represent an important analytic framework to generate estimates of cost-effectiveness. We addressed the modeling challenges in two decision analytic models developed to inform decision-making process. One model presents the development and results of a cost-effectiveness model of zoledronic acid versus risedronate in Paget’s disease of bone. The second model presents the development and results of an economic analysis that evaluates the lifetime effects of three-year lifestyle intervention in the prevention and treatment of obesity. The model estimates the cost-effectiveness of lifestyle intervention versus standard treatment in overweight and obese people in Switzerland. The importance of health economics research utilization in policy-making and of understanding the mechanisms involved is increasingly recognized. The existence of relevant research, though necessary, is not sufficient. Evidence-based policy is difficult to achieve and it is widely agreed that health policies do not reflect research evidence to the extent that in theory they could. In this thesis we assessed the use of research evidence relating to economic analyses in healthcare decision-making. We conducted a literature review to summarize and synthesize published literature on self-reported attitudes of healthcare decision-makers towards economic evaluations of medical technologies. The aims of this literature review was to determine the extent to which economic evaluations are used in health policy decision-making, and to consider factors associated with the utilization of such research findings. Examination of the policy-making process confirms it to be complex, with man! y genuine obstacles to evidence-based policy-making at the same time as there are factors that could increase research utilization

Validation of Electronic Data Capture of the Irritable Bowel Syndrome—Quality of Life Measure, the Work Productivity and Activity Impairment Questionnaire for Irritable Bowel Syndrome and the EuroQol

ABSTRACTObjectivesTo assess the comparability, reliability, and subject acceptability of electronic data capture (EDC) versions of Irritable Bowel Syndrome—Quality of Life (IBS-QOL), EuroQoL (EQ-5D) and Work Productivity and Activity Impairment (WPAI:IBS) instruments.MethodsComparability of EDC and paper questionnaires was evaluated in 72 subjects with IBS who completed a baseline EDC or paper questionnaire, a crossover questionnaire 24 hours later, and a retest of the crossover version at 1 week. The EDC version was presented on a hand-held device. Comparability was assessed using paired t-test statistics, intraclass correlation coefficients (ICC) and tests for internal consistency (Cronbach's alpha).ResultsNo significant differences were found between scores obtained by paper questionnaire and EDC at the baseline and crossover assessments. ICCs between baseline and crossover assessments ranged from 0.83 to 0.96 for the IBS-QOL scores, 0.82 to 0.96 for the WPAI:IBS scores, and 0.77 to 0.82 for the EQ-5D. Internal consistency was comparable for the two data collection methods for the IBS-QOL overall score (0.96) and subscales and the EQ-5D Index (0.70 vs. 0.74). Retest statistics (ICC) were generally comparable between the EDC and paper versions for all scores. Ease of use was comparable for the two modes of administration, but more patients preferred EDC (47.2%) than the paper questionnaire (23.6%).ConclusionsEDC versions of the IBS-QOL, EQ-5D, and WPAI:IBS are comparable to paper questionnaires in internal consistency and test–retest reliability, and have greater patient acceptability

Uncertainty in decision-making: value of additional information in the cost-effectivenss of lifestyle intervention in overweight and obese people

AbstractObjectivesTo quantify the uncertainty in the cost-effectiveness of lifestyle intervention versus standard care in overweight and obese people in Switzerland and to determine if further research is necessary based on current information. There has been an increasing interest in using value of information analysis in medical decision-making to quantify the uncertainty in decision-making, and to identify the need for further research.MethodsValue of information analysis was applied on a probabilistic cost-effectiveness model to evaluate the uncertainty by calculating the patient expected value of perfect information (EVPI), population EVPI, and partial EVPI. The costs were expressed in Swiss Francs (CHF), price year 2006.ResultsOverall, the EVPI was higher in overweight than in obese people. The maximum population EVPI was CHF 6.8 million in overweight people and CHF 3.2 million in moderate obese people, representing the upper limit on costs associated with decision uncertainty. The partial EVPI estimated a higher uncertainty in the model parameters such as utilities, body mass index, cardiovascular risk factors, and systolic blood pressure in overweight and moderate obese subjects.ConclusionsThe EVPI analysis indicates that there is some uncertainty regarding the choice between lifestyle intervention and standard care. The parameter EVPI suggests that if further research is commissioned, this should focus on the effectiveness of lifestyle intervention on cardiovascular risk factors and utilities

Burden of illness of people with persistent symptoms of schizophrenia: A multinational cross-sectional study.

BACKGROUND Few studies have examined the impact of persistent symptoms of schizophrenia, especially with respect to patient-reported outcomes (PROs), carer burden and health economic impact. AIMS Analyse data relating to burden and severity of illness, functional impairment and quality of life for patients with persistent symptoms of schizophrenia. METHODS A cohort of stable outpatients with persistent symptoms of schizophrenia across seven countries were assessed in a multicentre, non-interventional, cross-sectional survey and retrospective medical record review using PRO questionnaires, clinical rating scales and carer questionnaires. RESULTS Overall, 1,421 patients and 687 carers were enrolled. Approximately two-thirds of patients had moderate/mild schizophrenia with more severe negative symptoms predominating. Patients showed impaired personal/social functioning and unsuitability for work correlated with various patient factors, most notably symptom-related assessments. Quality-of-life assessments showed 25% to ⩾30% of patients had problems with mobility, washing or dressing. Carer burden was also considerable, with carers having to devote an average of 20.5 hours per week and notable negative impact on quality-of-life measures. Healthcare resource utilisation for in-hospital, outpatient and other care provider visits was significant. CONCLUSION These results demonstrate the significant burden of schizophrenia for patients, carers and society and highlight the need for improved treatment approaches

Uncertainty in Decision-Making: Value of Additional Information in the Cost-Effectiveness of Lifestyle Intervention in Overweight and Obese People

ABSTRACT Objectives: To quantify the uncertainty in the costeffectiveness of lifestyle intervention versus standard care in overweight and obese people in Switzerland and to determine if further research is necessary based on current information. There has been an increasing interest in using value of information analysis in medical decision-making to quantify the uncertainty in decision-making, and to identify the need for further research. Methods: Value of information analysis was applied on a probabilistic cost-effectiveness model to evaluate the uncertainty by calculating the patient expected value of perfect information (EVPI), population EVPI, and partial EVPI. The costs were expressed in Swiss Francs (CHF), price year 2006. Results: Overall, the EVPI was higher in overweight than in obese people. The maximum population EVPI was CHF 6.8 million in overweight people and CHF 3.2 million in moderate obese people, representing the upper limit on costs associated with decision uncertainty. The partial EVPI estimated a higher uncertainty in the model parameters such as utilities, body mass index, cardiovascular risk factors, and systolic blood pressure in overweight and moderate obese subjects. Conclusions: The EVPI analysis indicates that there is some uncertainty regarding the choice between lifestyle intervention and standard care. The parameter EVPI suggests that if further research is commissioned, this should focus on the effectiveness of lifestyle intervention on cardiovascular risk factors and utilities