Budget impact analysis-principles of good practice: report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force.

BACKGROUND: Budget impact analyses (BIAs) are an essential part of a comprehensive economic assessment of a health care intervention and are increasingly required by reimbursement authorities as part of a listing or reimbursement submission. OBJECTIVES: The objective of this report was to present updated guidance on methods for those undertaking such analyses or for those reviewing the results of such analyses. This update was needed, in part, because of developments in BIA methods as well as a growing interest, particularly in emerging markets, in matters related to affordability and population health impacts of health care interventions. METHODS: The Task Force was approved by the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council and appointed by its Board of Directors. Members were experienced developers or users of BIAs; worked in academia and industry and as advisors to governments; and came from several countries in North America and South America, Oceania, Asia, and Europe. The Task Force solicited comments on the drafts from a core group of external reviewers and, more broadly, from the membership of the International Society for Pharmacoeconomics and Outcomes Research. RESULTS: The Task Force recommends that the design of a BIA for a new health care intervention should take into account relevant features of the health care system, possible access restrictions, the anticipated uptake of the new intervention, and the use and effects of the current and new interventions. The key elements of a BIA include estimating the size of the eligible population, the current mix of treatments and the expected mix after the introduction of the new intervention, the cost of the treatment mixes, and any changes expected in condition-related costs. Where possible, the BIA calculations should be performed by using a simple cost calculator approach because of its ease of use for budget holders. In instances, however, in which the changes in eligible population size, disease severity mix, or treatment patterns cannot be credibly captured by using the cost calculator approach, a cohort or patient-level condition-specific model may be used to estimate the budget impact of the new intervention, accounting appropriately for those entering and leaving the eligible population over time. In either case, the BIA should use data that reflect values specific to a particular decision maker's population. Sensitivity analysis should be of alternative scenarios chosen from the perspective of the decision maker. The validation of the model should include at least face validity with decision makers and verification of the calculations. Data sources for the BIA should include published clinical trial estimates and comparator studies for the efficacy and safety of the current and new interventions as well as the decision maker's own population for the other parameter estimates, where possible. Other data sources include the use of published data, well-recognized local or national statistical information, and, in special circumstances, expert opinion. Reporting of the BIA should provide detailed information about the input parameter values and calculations at a level of detail that would allow another modeler to replicate the analysis. The outcomes of the BIA should be presented in the format of interest to health care decision makers. In a computer program, options should be provided for different categories of costs to be included or excluded from the analysis. CONCLUSIONS: We recommend a framework for the BIA, provide guidance on the acquisition and use of data, and offer a common reporting format that will promote standardization and transparency. Adherence to these good research practice principles would not necessarily supersede jurisdiction-specific BIA guidelines but may support and enhance local recommendations or serve as a starting point for payers wishing to promulgate methodology guidelines.

The use of cost per life year gained as a measurement of cost-effectiveness in Spain: a systematic review of recent publications.

OBJECTIVES: The objective of this study was to evaluate the methodological characteristics of cost-effectiveness evaluations carried out in Spain, since 1990, which include LYG as an outcome to measure the incremental cost-effectiveness ratio. METHODS: A systematic review of published studies was conducted describing their characteristics and methodological quality. We analyse the cost per LYG results in relation with a commonly accepted Spanish cost-effectiveness threshold and the possible relation with the cost per quality adjusted life year (QALY) gained when they both were calculated for the same economic evaluation. RESULTS: A total of 62 economic evaluations fulfilled the selection criteria, 24 of them including the cost per QALY gained result as well. The methodological quality of the studies was good (55%) or very good (26%). A total of 124 cost per LYG results were obtained with a mean ratio of 49,529 and a median of 11,490 (standard deviation of 183,080). Since 2003, a commonly accepted Spanish threshold has been referenced by 66% of studies. A significant correlation was found between the cost per LYG and cost per QALY gained results (0.89 Spearman-Rho, 0.91 Pearson). CONCLUSIONS: There is an increasing interest for economic health care evaluations in Spain, and the quality of the studies is also improving. Although a commonly accepted threshold exists, further information is needed for decision-making as well as to identify the relationship between the costs per LYG and per QALY gained.

[Innovative patient access schemes for the adoption of new technology: risk-sharing agreements]. / Esquemas innovadores de mejora del acceso al mercado de nuevas tecnologías: los acuerdos de riesgo compartido.

The incorporation of new treatments, procedures and technologies into the services' portfolio of healthcare providers should aim to improve three areas equally: patient access to innovative solutions, the sustainability of the health system and compensation for innovation. However, traditional schemes based on fixed prices that fail to consider the product's appropriate use or its results in terms of effectiveness may lead to inefficient decision-making processes. Recently, risk-sharing agreements have appeared as new access schemes based on results that aim to reduce the uncertainty of the distinct health care players involved in reaching an agreement on new health technology financing and conditions of use. Key elements in the debate on these instruments are the huge variety of instruments available (especially those based on results), the implications for different players involved in their design and supervision, and their possible implementation in Spain. Our main conclusion is that risk-sharing agreements should be used in highly limited cases when standard conditions of access cannot be applied due to uncertainty about long-term effectiveness. These measures are aimed not only at regulating price but also at acting on the appropriate use of new technology. However, because international experience is limited, drawing a solid conclusion on the final results of the application of risk-sharing agreements would be premature.

[Cost-benefit analysis of concomitant atrial fibrillation management in Spain]. / Análisis de coste-utilidad del manejo de la fibrilación auricular concomitante en España.

OBJECTIVES: Atrial fibrillation (AF) is the most common arrhythmia in clinical practice; this disorder is a risk factor for stroke and is associated with substantial morbidity and mortality. Our objective was to develop a cost-utility analysis of the different treatment alternatives in patients aged 40 years old or more with concomitant AF with valve disease in Spain, from the National Health System perspective. METHODS: An economic evaluation through a Markov model with four health states (sinus rhythm, AF, dependent stroke, death) was developed to simulate the evolution of a cohort of 1,000 patients receiving each treatment alternative in addition to mitral valve surgery (drug therapy, surgical ablation and catheter ablation). The time horizon was 5 years, with a cycle length of 3 months. Data on costs and effects were obtained from the published literature and expert opinion and were discounted at 3.5%. A sensitivity analysis was developed to determine the robustness of the results. RESULTS: The quality-adjusted life years (QALY) gained were 3.29, 3.89, and 3.83, respectively, for the alternatives of no ablation, surgical ablation and catheter ablation. The costs per patient were 5,770euro, 10,034euro and 11,289euro, respectively. The surgical ablation cost/QALY rate compared with no ablation was 7,145euro. Surgical ablation was dominant versus catheter ablation. The probabilistic sensitivity analysis showed that the results were robust. CONCLUSIONS: Surgical ablation is a cost-effective treatment option in patients with concomitant AF, with a cost-effectiveness ratio under the efficiency threshold commonly accepted in Spain.

[Discrete-event simulation models in the economic evaluation of health technologies and health products]. / Los modelos de simulación de eventos discretos en la evaluación económica de tecnologías y productos sanitarios.

The use of mathematical models to assess therapeutic alternatives is increasing in the economic evaluation of health technologies and services and these models are becoming an increasingly important aid to decision making in health care. Until now, 2 types of model have been used, depending to some extent on the disease to be studied: decision trees have been used for acute diseases and Markov models in chronic or recurrent diseases. However, both models present major limitations when addressing complex processes or diseases. Consequently, interest in, and the use of, discrete-event simulation is growing. The present article aims to describe the main characteristics of discrete-event simulation, the state of the art in this field, and the advantages of these models with respect to other kinds of models in health economics, especially in the evaluation of health technologies and product assessment.

[Cost-utility analysis of insulin pumps compared to multiple daily doses of insulin in patients with type 1 diabetes mellitus in Spain]. / Análisis coste-utilidad de las bombas de insulina frente a múltiples dosis diarias en pacientes con diabetes mellitus tipo 1 en españa.

BACKGROUND: The use of continuous subcutaneous insulin infusion (CSII) for treating Type I diabetes mellitus (DM1) has been related to better metabolic control compared it to daily multiple insulin injections (DMI) and thus to a lowering of the related costs. However, this therapy is now being used to a lesser extent due, at least partially, to the higher initial cost of purchase. This study is aimed at estimating the clinical and economic consequences of using CSII as compared to DMI by means of a cost-utility analysis. METHODS: A mathematical simulation model was adapted using nationwide clinical and economic data to simulate the long-term clinical and economic consequences for a DM1 patient. The time horizon was the patient's lifetime, including only direct healthcare costs and updating both costs and benefits at an annual 3% rate. RESULTS: In the basecase, the patients treated using CSII gained 0.890 years (p < 0.05) and 0.852 QALYs (p < 0.05). CSII treatment gives rise to an incremental average cost of 25,523 Euro (p < 0.05) per patient treated, which gave us an incremental cost- utility ratio of 29,947 Euro-QALY [CI 95% (29,519; 30,375)]. CONCLUSIONS: The improvement in the glucose control among those patients treated using CSII was related to an overall lower cost in the handling of DM1 patients, which was found to have a favourable cost-utility ratio in comparison to conventional MDI treatment.