In search for more confidence in health economic modelling : reducing uncertainty associated with modelling studies

Every government is eager to control the increase of expenses by the implementation of central cost containment policies particularly in relation to pharmaceuticals. For the most part. those measures have relied on budgeting or price controls. including negotiated prospective budgets for hospitals. centralized negotiated budgets for ambulatory physicians including drug prescriptions, and limitations on payments for particular medications. Because those traditional central cost containment measures were only partially successfuL due to lack of incentives. the health authorities in Europe started to establish incentives for efficient healthcare delivery. Both traditional and recent containment measures focus especially on the pharmaceutical drugs sector in many countries. as these constitute a health technology that is relatively easy to introduce and implement compared to other forms of care. Financing prescription medicines in ambulatory care has been a central responsibility based on the traditional clinical trial outcomes (efficacy/safety parameters) used for registration. Although there is large variety betvveen the various countries. there are three related trends: decentralization of the healthcare decision-making process. prescription restrictions, and extra data requirements. One can distinguish various extra data requirements which all relate to the use of the drug in real daily practice. while the traditional clinical trial outcomes are only derived from randomised clinical trials. At a central level the demand for cost-effectiveness and budgetary impact data is increasing. The requirement for health economic data resulted to formal reporting requirements in some countries already (e.g. Canada, Australia. The Netherlands. UK. Portugal and Finland). Although the most evident impact of health economic studies is expected to be on central reimbursement audiences. evidence for the use of health economic studies by other audiences is expected to increase (e.g. patients, hospitals. insurers. formulary committees). This background information is described in more detail in Chapter 2. which is the introduction to this thesis

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

Costs of managing adverse events in the treatment of first-line metastatic renal cell carcinoma: Bevacizumab in combination with interferon-α2a compared with sunitinib

Background: Bevacizumab plus interferon-α2a (IFN) prolongs progression-free survival to>10 months, which is comparable with sunitinib as first-line treatment of metastatic renal cell carcinoma (RCC). The two regimens have different tolerability profiles; therefore, costs for managing adverse events may be an important factor in selecting therapy.Methods: Costs of managing adverse events affecting patients with metastatic RCC eligible for treatment with bevacizumab plus IFN or sunitinib were evaluated using a linear decision analytical model. Management costs were calculated from the published incidence of adverse events and health-care costs for treating adverse events in the United Kingdom, Germany, France and Italy.Results: Adverse event management costs were higher for sunitinib than for bevacizumab plus IFN. The average cost per patient for the management of grade 3-4 adverse events was markedly lower with bevacizumab plus IFN compared with sunitinib in the United Kingdom (\[euro]1475 vs \[euro]804), Germany (\[euro]1785 vs \[euro]1367), France (\[euro]2590 vs \[euro]1618) and Italy (\[euro]891 vs \[euro]402). The main cost drivers were lymphopaenia, neutropaenia, thrombocytopaenia, leucopaenia and fatigue/asthaenia for sunitinib; and proteinuria, fatigue/asthaenia, bleeding, anaemia and gastrointestinal perforation for bevacizumab plus IFN.Conclusion: The costs of managing adverse events are lower for bevacizumab plus IFN than for sunitinib. The potential for cost savings should be considered when selecting treatments for RCC

Workshop Report: Concepts and methods in the economics of nutrition - Gateways to better economic evaluation of nutrition interventions

Improving health through better nutrition of the population may contribute to enhanced efficiency and sustainability of healthcare systems. A recent expert meeting investigated in detail a number of methodological aspects related to the discipline of nutrition economics. The role of nutrition in health maintenance and in the prevention of non-communicable diseases is now generally recognised. However, the main scope of those seeking to contain healthcare expenditures tends to focus on the management of existing chronic diseases. Identifying additional relevant dimensions to measure and the context of use will become increasingly important in selecting and developing outcome measurements for nutrition interventions. The translation of nutrition-related research data into public health guidance raises the challenging issue of carrying out more pragmatic trials in many areas where these would generate the most useful evidence for health policy decision-making. Nutrition exemplifies all the types of interventions and policy which need evaluating across the health field. There is a need to start actively engaging key stakeholders in order to collect data and to widen health technology assessment approaches for achieving a policy shift from evidence-based medicine to evidence-based decision-making in the field of nutrition

Economic evaluation of tramadol/paracetamol combination tablets for osteoarthritis pain in the Netherlands

Objective: To compare the costs of treating osteoarthritis (OA) pain using combination tramadol/paracetamol tablets, NSAIDs alone, NSAIDs plus proton pump inhibitors (PPIs), or NSAIDs plus histamine H2-receptor antagonists (H2RAs) from the perspective of the Dutch healthcare system. Design and methods: A decision-analytical model was constructed to model the cost outcomes of the four treatment strategies over 6 months. A cost-minimisation approach was used, which considered data related to resource utilisation, medication costs and costs for the treatment of adverse events. Data, derived mainly from the clinical literature, were supplemented by inputs from a Delphi panel as well as official price and tariff lists. The base-case analysis considered direct medical costs, including those for treating all adverse events with tramadol/paracetamol and gastrointestinal (GI) adverse events with NSAIDs. Separate scenario analyses explored costs of NSAID-based regimens: (i) according to 21 levels of risk for GI adverse events, and (ii) when renal events attributable to NSAIDs were considered. Robustness of the model was tested using univariate sensitivity analysis. Results: In the base-case analysis, costs for 6 months' treatment of OA pain using tramadol/paracetamol were €244.45, compared with €317.32 for NSAIDs + PPIs, €200.67 for NSAIDs + H 2RAs, and €125.86 for NSAIDs alone. This provided a cost saving of €72.87 per patient over 6 months for tramadol/paracetamol compared with NSAIDs + PPIs. Tramadol/paracetamol became cost saving compared with NSAIDs alone and NSAIDs + H2RAs for GI risk levels >13 and >10, respectively. When renal adverse events of NSAIDs were considered, tramadol/paracetamol was cost saving compared with all NSAID-based regimens (saving €228.40 vs NSAIDs, €418.42 vs NSAIDs + PPIs, and €302.69 vs NSAIDs + H2RAs [year of costing 2005]). Sensitivity analysis confirmed the model was robust to wide-ranging changes in the value of input parameters. Conclusion: Tramadol/paracetamol is cost saving compared with NSAIDs + PPIs for the treatment of OA pain over a period of 6 months regardless of the risk of GI or renal complications. Tramadol/paracetamol is also cost saving compared with treatment with NSAIDs alone and NSAIDs + H2RAs for patients at medium and high risk of GI adverse events and in all cases if considering renal adverse events. Despite not being quantified in monetary terms, the lower incidence of adverse events with tramadol/paracetamol is a clinical benefit

Chronic kidney disease Markov model comparing paricalcitol to calcitriol for secondary hyperparathyroidism: A US perspective

Objective: The objective of this study was to determine the cost effectiveness of paricalcitol versus calcitriol for the treatment of secondary hyperparathyroidism in patients with chronic kidney disease in the United States setting. Methods: A Markov process model was developed employing data sources from the published literature, paricalcitol clinical trials and observational studies, official US price/tariff lists and national population statistics. The comparator was calcitriol, a non-selective vitamin D receptor activator (VDRA) medication. The primary perspective of the study was that of the third-party payer in the US. The efficacy outcomes (reduction in secondary hyperparathyroidism (SHPT), reduction in proteinuria, complications and mortality) were extrapolated to: number of life-years gained (LYG) and number of quality-adjusted life-years (QALYs). Clinical and economic outcomes were discounted at 3.5%. Results: The reference case analysis was a 10-year time horizon based on a comparison of paricalcitol with calcitriol, which is started in chronic kidney disease (CKD) stage 3 and continued in CKD stage 4 and CKD stage 5. The use of paricalcitol leads to a cost saving of US$1941. The inclusion of indirect costs leads to a cost saving of US$2528. The use of paricalcitol leads to an increase in life-years gained (0.47 years) and a gain in QALYs (0.43). The use of paricalcitol results in a dominant outcome from the perspective of the third-party payer, as well as from the societal perspective. One-way sensitivity analyses and probabilistic sensitivity analyses confirmed the robustness of the model. Conclusion: This model showed that the favorable clinical benefit of paricalcitol results in positive short and long-term health economic benefits. This study suggests that the use of paricalcitol in patients with early chronic kidney disease may be cost-effective from the third-party payer perspective in the US versus calcitriol. Additional comparative studies are necessary to validate these results