Challenges in modelling the cost effectiveness of various interventions for cardiovascular disease

Objectives: Decision analytic modelling is essential in performing cost-effectiveness analyses (CEAs) of interventions in cardiovascular disease (CVD). However, modelling inherently poses challenges that need to be dealt with since models always represent a simplification of reality. The aim of this study was to identify and explore the challenges in modelling CVD interventions. Methods: A document analysis was performed of 40 model-based CEAs of CVD interventions published in high-impact journals. We analysed the systematically selected papers to identify challenges per type of intervention (test, non-drug, drug, disease management programme, and public health intervention), and a questionnaire was sent to the corresponding authors to obtain a more thorough overview. Ideas for possible solutions for the challenges were based on the papers, responses, modelling guidelines, and other sources. Results: The systematic literature search identified 1,720 potentially relevant articles. Forty authors were identified after screening the most recent 294 papers. Besides the challenge of lack of data, the challenges encountered in the review suggest that it was difficult to obtain a sufficiently valid and accurate cost-effectiveness estimate, mainly due to lack of data or extrapolating from intermediate outcomes. Despite the low response rate of the questionnaire, it confirmed our results. Conclusions: This combination of a review and a survey showed examples of CVD modelling challenges found in studies published in high-impact journals. Modelling guidelines do not provide sufficient guidance in resolving all challenges. Some of the reported challenges are specific to the type of intervention and disease, while some are independent of intervention and disease

Adjuvant Trastuzumab Therapy for Early HER2-Positive Breast Cancer in Iran: A Cost-Effectiveness and Scenario Analysis for an Optimal Treatment Strategy

Introduction: Clinical guidelines have recommended a 1-year trastuzumab regimen as standard care for early human epidermal growth factor receptor 2 (HER2)-positive breast cancer; however, th

De meerwaarde van HKZ-certificering voor GGZ-instellingen

Het certificeren van instellingen is een instrument geworden om de kwaliteit van zorg van instellingen te verbeteren. Certificeren behelst het beoordelen van een organisatie op voorgeschreven normen door een onafhankelijke derde en het afgeven van een certificaat wanneer aan de normen wordt voldaan. Instellingen worden in toenemende mate verplicht certificaten te halen. Maar heeft certificeren wel meerwaarde? Het onderzoek waarover wordt gerapporteerd stelt deze vraag centraal. En wel: Wat is de meerwaarde van HKZ-certificering op de geleverde kwaliteit van zorg in GGZ-instellingen? Gekozen is voor HKZ als certificeringinstrument en de geestelijke gezondheidszorg als sector. Deelvragen van het onderzoek zijn: 1. Welke waarde ontlenen zorgprofessionals (hulpverleners), kwaliteitsfunctionarissen, managers en medewerkers van stafafdelingen aan HKZ-certificering? 2. Presteren GGZ-instellingen met (zicht op) een HKZ-certificaat beter op relevante indicatoren dan GGZ-instellingen die niet (of nog niet) HKZ-gecertificeerd zijn? 3. Vanwege welke redenen leidt HKZ-certificering tot betere of slechtere prestaties

Using meta-regression analyses in addition to conventional systematic review methods to examine the variation in cost-effectiveness results

Background: Systematic reviews of cost-effectiveness analyses summarize results and describe study characteristics. Variability in the study results is often explained qualitatively or based on sensitivity analyses of individual studies. However, variability due to input parameters and study characteristics (e.g., funding or study quality) is often not statistically explained. As a case study, a systematic review on the cost-effectiveness of drug-eluting stents (DES) versus bare-metal stents (BMS) using meta-regression analyses is performed to explore the usefulness of such methods compared with conventional review methods. Methods: We attempted to identify and review all modelling studies published until January 2012 that compared costs and consequences of DES versus BMS. We extracted general study information (e.g., funding), modelling methods, values of input parameters, and quality of the model using the Philips et al. checklist. Associations between study characteristics and the incremental costs and effectiveness of individual analyses were explored using regression analyses corrected for study ID. Results: Sixteen eligible studies were identified, with a combined total of 508 analyses. The overall quality of the models was moderate (59 % ± 15 %). This study showed associations (e.g., type of lesion) that were expected (based on individual studies), however the meta-regression analyses revealed also unpredicted associations: e.g., model quality was negatively associated with repeat revascularizations avoided. Conclusions: Meta-regressions can be of added value, identifying significant associations that could not be identified using conventional review methods or by sensitivity analyses of individual studies. Furthermore, this study underlines the need to examine input parameters and perform a quality check of studies when interpreting the results

Systematic review of available evidence on 11 high-priced inpatient orphan drugs

__Background__: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, costeffectiveness and budget impact for orphan drugs. __Methods__: A systematic review was performed in PubMed, Embase, NHS EED and HTA databases for 11 inpatient orphan drugs listed on the Dutch policy rule on orphan drugs. For included studies, we determined the type of study and various study characteristics. __Results__: A total of 338 studies met all inclusion criteria. Almost all studies (96%) focused on clinical effectiveness of the drug. Of these studies, most studies were case studies (41%) or observational studies (39%). However, for all orphan diseases at least one experimental or quasi-experimental study was found, and a randomized clinical trial was available for 60% of the orphan drugs. Eight studies described the cost-effectiveness of an orphan drug; an equal number described an orphan drug’s budget impact. __Conclusions__: Despite the often heard claim that RCTs are not feasible for orphan drugs, we found that an RCT was available in 60% of o

Economic evaluation in stratified medicine: Methodological issues and challenges

Background: Stratified Medicine (SM) is becoming a practical reality with the targeting of medicines by using a biomarker or genetic-based diagnostic to identify the eligible patient sub-population. Like any healthcare intervention, SM interventions have costs and consequences that must be considered by reimbursement authorities with limited resources. Methodological standards and guidelines exist for economic evaluations in clinical pharmacology and are an important component for health technology assessments (HTAs) in many countries. However, these guidelines have initially been developed for traditional pharmaceuticals and not for complex interventions with multiple components. This raises the issue as to whether these guidelines are adequate to SM interventions or whether new specific guidance and methodology is needed to avoid inconsistencies and contradictory findings when assessing economic value in SM. Objective: This article describes specific methodological challenges when conducting health economic (HE) evaluations for SM interventions and outlines potential modifications necessary to existing evaluation guidelines /principles that would promote consistent economic evaluations for SM. Results/Conclusions: Specific methodological aspects for SM comprise considerations on the choice of comparator, measuring effectiveness and outcomes, appropriate modeling structure and the scope of sensitivity analyses. Although current HE methodology can be applied for SM, greater complexity requires further methodology development and modifications in the guidelines

Productivity Loss Related to Neglected Tropical Diseases Eligible for Preventive Chemotherapy

Neglected Tropical Diseases (NTDs) not only have impact on health and life expectancy of mostly disadvantaged populations, but can also lead to economic consequences, including reduced ability to work. Investments in health improvement of the populations affected by NTDs would also help to increase economic growth of the affected regions, since healthier populations are more economically productive. We performed a systematic literature review to better understand how much NTDs affect people’s economic welfare. Here we present the results for the NTDs that are controlled with preventive chemotherapy (PCT): lymphatic filariasis, onchocerciasis, schistosomiasis, soil-transmitted helminths (ascariasis, trichuriasis, and hookworm infection) and trachoma. Our findings show that PCT NTDs clearly affect productivity, although the actual impact depends on the type and severity of the NTD as well as on the context where the disease occurs. Variation in estimated productivity loss is also caused by differences in research methods. Publications should provide enough information to enable readers to assess the quality and relevance of the study for their purposes

European survey on acute coronary syndrome diagnosis and revascularisation treatment

Rationale, Aims, and Objectives: While different imaging and treatment options are available in acute coronary syndrome (ACS) care, there is a lack of data regarding their use across Europe. We examined the diagnostic and treatment strategies in patients with known or suspected ACS as reported by physicians and identified variations in responses across European countries and geographical areas. Method: A web-based clinician survey focusing on ACS imaging and revascularization treatments was circulated through email distribution lists and websites of European professional societies in the field of cardiology. We collected information on respondents' clinical setting and specialty. Reported percentages of patients receiving imaging or treatment modalities and percentages of clinicians reporting to use modalities in a range of clinical scenarios were analyzed. Statistical comparisons were performed. Results: In total, 69 responses were received (Sweden [n = 20], United Kingdom [n = 16], Northern/Western Europe [n = 17], Southern Europe [n = 9], and Central Europe [n = 7]). Considerable variations between geographical areas were seen in terms of reported diagnostic modalities and treatment strategies. For example, when presented with the scenario of a theoretical 45-year-old smoking female with a suspected ACS, 56% of UK clinicians reported to use coronary computed tomography angiography, compared to only 10% of Swedish clinicians (P =.002). Large variations were observed regarding the reported use of fractional flow reserve by physicians for non-culprit lesions during invasive management of myocardial infarction patients (44% in Sweden, 31% in the United Kingdom, and 30% in Northern/Western Europe vs non-use in Central and Southern Europe). Conclusions: In this survey, respondents reported different diagnostic and treatment strategies in ACS care. These variations seem to have geographic components. Larger studies or real world data are needed to verify these observations and investigate their causes. More research is needed to compare the quality and efficiency of ACS care across countries and explore pathways for improvement