Improving economic evaluations in stroke: A report from the ESO Health Economics Working Group.

INTRODUCTION: Approaches to economic evaluations of stroke therapies are varied and inconsistently described. An objective of the European Stroke Organisation (ESO) Health Economics Working Group is to standardise and improve the economic evaluations of interventions for stroke. METHODS: The ESO Health Economics Working Group and additional experts were contacted to develop a protocol and a guidance document for data collection for economic evaluations of stroke therapies. A modified Delphi approach, including a survey and consensus processes, was used to agree on content. We also asked the participants about resources that could be shared to improve economic evaluations of interventions for stroke. RESULTS: Of 28 experts invited, 16 (57%) completed the initial survey, with representation from universities, government, and industry. More than half of the survey respondents endorsed 13 specific items to include in a standard resource use questionnaire. Preferred functional/quality of life outcome measures to use for economic evaluations were the modified Rankin Scale (14 respondents, 88%) and the EQ-5D instrument (11 respondents, 69%). Of the 12 respondents who had access to data used in economic evaluations, 10 (83%) indicated a willingness to share data. A protocol template and a guidance document for data collection were developed and are presented in this article. CONCLUSION: The protocol template and guidance document for data collection will support a more standardised and transparent approach for economic evaluations of stroke care.

An Epidemiological Model for First Stroke in Saudi Arabia.

BACKGROUND: The Kingdom of Saudi Arabia has a young but ageing population, leading to concerns for planning for future growth in the number of strokes to provide necessary care. An understanding of the expected evolution of stroke incidence is therefore necessary to plan infrastructure changes. Our aim was to predict the number of first strokes occurring in Saudi Arabia over a 10-year period. METHODS: An epidemiological model was developed, using local mortality and population data to model changes in the population. Gender- and age-specific stroke rates were then applied to the population projections to estimate the number of first strokes occurring over a 10-year period. Stroke incidence data from a range of sources were applied to obtain a plausible range for the change in expected number of first strokes. RESULTS: The model predicted population growth of 12.8% over the 10-year period. Depending on the stroke incidence data applied, the number of first strokes occurring during this time was predicted to increase within the range 57%-67%. CONCLUSIONS: A growing and ageing population is expected to lead to a substantial increase in the number of first strokes occurring in Saudi Arabia in the coming decade. Our results suggest that stroke care services will need to be expanded to continue to ensure high quality care, and that strategies for stroke prevention will play an important role in reducing the overall burden. This type of analysis can be applied to other countries' stroke policy planning.

Workforce requirements for comprehensive ischaemic stroke care in a developing country: the case of Saudi Arabia.

BACKGROUND: Ischaemic stroke care requires a co-ordinated multi-disciplinary approach to optimise patient outcomes. Current care provision in Saudi Arabia is below international recommendations, and with increasing patient numbers, variable access to new therapies, and sub-optimal co-ordination of staff, the Kingdom's Ministry of Health has prioritised strategies to develop stroke care. Our objective was to use local epidemiological data to predict stroke incidence and to combine this with international staffing recommendations to estimate future staff requirements and their costs over a 10-year period. METHODS: We researched existing stroke services and staff availability within Saudi Arabia to establish current provision, undertook epidemiological modelling to predict stroke incidence, and used international staffing recommendations for acute and rehabilitation services to develop a care pathway to provide state-of-the-art stroke services. This information was used to determine the additional staff requirements, and their costs, across the Kingdom. RESULTS: Our research concluded that current staff numbers and services are inadequate to cope with the projected increase in the number of stroke cases. In order to provide acute and rehabilitation services which use the latest technologies, re-organisation of existing staff and services would be required, together with significant investment in new staff across several disciplines. An estimated additional 43.8 full-time equivalent stroke neurologists would be required, plus 53.5 full-time equivalent interventional neuroradiologists in addition to expansion of occupational therapy and psychology services. The total cost of additional staff over 10 years was estimated to be 862 390 778 Saudi Riyals ($229 970 874). CONCLUSIONS: Providing high-quality care for ischaemic stroke patients would involve significant investment in new staff in Saudi Arabia. Further research is required on the applicability of international staffing ratios to countries where there is a significant workforce gap. Nevertheless, this analysis provides a framework to inform stroke care planning and can be adapted to other regions or countries.

A national economic and clinical model for ischemic stroke care development in Saudi Arabia: A call for change.

BACKGROUND: Stroke is a significant burden in Saudi Arabia and the Saudi Ministry of Health's stroke committee has identified an urgent need to improve care. AIM: The purpose of this study was to undertake a health-economic analysis to quantify the impact of developing stroke care in the country. METHODS: An economic model was developed to assess the costs and clinical outcomes associated with an ischemic stroke care development program compared with current stroke care. Based on Saudi epidemiological data, cohorts of ischemic stroke patients enter the model each year for the first 10 years based on increasing incidence. Four treatment options were modeled including reperfusion and non-reperfusion treatments. The development scenario estimates the impact of gradually increasing uptake of more effective treatments over 10 years. Changes in the stroke care organization are considered along with resources required to increase capacity, allowing more patients to be admitted to stroke hospitals and access effective treatments. RESULTS: The stroke care development program is associated with an increase in functionally independent patients and a decrease in disabling strokes compared with current stroke care. Additionally, the development program is associated with estimated cost savings of $602 million over 15 years ($255 million direct costs, $348 million indirect costs). CONCLUSIONS: The model predicts that the stroke care development program is associated with improved patient outcomes and lower overall costs compared with the current stroke care program.

Understanding the relationship between costs and the modified Rankin Scale: A systematic review, multidisciplinary consensus and recommendations for future studies.

BACKGROUND AND PURPOSE: Cost-of-illness studies often describe a single aggregate cost of a disease state. This approach is less helpful for a condition with a spectrum of outcomes like stroke. The modified Rankin Scale is the most commonly used outcome measure for stroke. We sought to describe the existing evidence on the costs of stroke according to individual modified Rankin Scale categories. This may be useful in future cost effectiveness modelling studies of interventions where cost data have not been collected, but disability outcome is known. METHODS: Systematic review of the published literature, searching electronic databases between 2004 and 2015 using validated search filters. Results were screened to identify studies presenting costs by individual modified Rankin Scale categories. RESULTS: Of 17,782 unique identified articles, 13 matched all inclusion criteria. In only four of these studies were costs reported by modified Rankin Scale categories. Most studies included direct medical costs only. Societal costs were assessed in two studies. Overall, studies had a high methodological and reporting quality. The heterogeneity in costing methods used in the identified studies prevented meaningful comparison of the reported cost data. Despite this limitation, the costs consistently increased with greater severity (increasing modified Rankin Scale score). CONCLUSIONS: Few cost studies of stroke include information based on stroke recovery measured by individual modified Rankin Scale categories and the existing data are limited. To reliably capture this information, future studies are needed that preferably apply standardised costing methods to promote greater potential for use in cost-effectiveness analyses whereby direct collection of patient-level resource use has not been possible.

Early dialogue with health technology assessment bodies: a European perspective.

INTRODUCTION: Evidence requirements may differ across HTA bodies, and so pharmaceutical companies must plan to synergize their evidence generation strategy, across global regulatory and HTA bodies. Until recently, companies had no official platform to discuss the clinical development of a drug with HTA bodies; however, this is changing. OBJECTIVES: To achieve broad usage in the EU, products must achieve both regulatory and reimbursement approval, the latter of which is based on HTA appraisal in many markets. The objective of this study is to present and evaluate the different options available for early HTA consultation (during drug development/Phase III) in the major European markets from the industry perspective. METHODS: An exploratory (nonsystematic) literature review was performed to identify the European markets offering early HTA consultations, and each process was analyzed using a set of predefined metrics that are relevant to industry (the ability to consult with the regulatory body in parallel, consultation fees, length of consultation meeting, language of consultation meeting, maximum number of pharmaceutical company employees attending, procedural timelines, nature of data for which consultative advice can be sought, the output of the process, and the ability to involve external experts). RESULTS: Four different types of early HTA consultation processes were identified across the major European HTA markets. The nature of these processes varied in terms of the types and number of questions that can be addressed, the length of the meeting, the reporting output, and the ability to involve external experts. CONCLUSIONS: The availability of various options for early HTA consultation may help to avoid a mismatch between the evidence generated by means of a product's clinical development program, and the evidence expected by HTA bodies and payers, which can facilitate the pricing and reimbursement process upon a product's market authorization.

A cost-effectiveness analysis of subcutaneous interferon beta-1a 44mcg 3-times a week vs no treatment for patients with clinically isolated syndrome in Sweden.

OBJECTIVE: To assess the cost-effectiveness of subcutaneous interferon (sc IFN) beta-1a 44 mcg 3-times weekly (tiw) vs no treatment at reducing the risk of conversion to multiple sclerosis (MS) in patients with clinically isolated syndrome (CIS) in Sweden. METHODS: A Markov model was constructed to simulate the clinical course of patients with CIS treated with sc IFN beta-1a 44 mcg tiw or no treatment over a 40-year time horizon. Costs were estimated from a societal perspective in 2012 Swedish kronor (SEK). Treatment efficacy data were derived from the REFLEX trial; resource use and quality-of-life (QoL) data were obtained from the literature. Costs and outcomes were discounted at 3%. Sensitivity analyses explored whether results were robust to changes in input values and use of Poser criteria. RESULTS: Using McDonald criteria sc IFN beta-1a was cost-saving and more effective (i.e., dominant) vs no treatment. Gains in progression free life years (PFLYs) and quality-adjusted life-years (QALYs) were 1.63 and 0.53, respectively. Projected cost savings were 270,263 SEK. For Poser criteria cost savings of 823,459 SEK were estimated, with PFLY and QALY gains of 4.12 and 1.38, respectively. Subcutaneous IFN beta-1a remained dominant from a payer perspective. Results were insensitive to key input variation. Probabilistic sensitivity analysis estimated a 99.9% likelihood of cost-effectiveness at a willingness-to-pay threshold of 500,000 SEK/QALY. CONCLUSION: Subcutaneous IFN beta-1a is a cost-effective option for the treatment of patients at high risk of MS conversion. It is associated with lower costs, greater QALY gains, and more time free of MS. LIMITATIONS: The risk of conversion from CIS to MS was extrapolated from 2-year trial data. Treatment benefit was assumed to persist over the model duration, although long-term data to support this are unavailable. Cost and QoL data from MS patients were assumed applicable to CIS patients.

An economic analysis of using rhBMP-2 for lumbar fusion in Germany, France and UK from a societal perspective.

Recombinant human Bone Morphogenetic Protein-2 (rhBMP-2) can replace autogenous bone grafting in single-level lumbar interbody fusion. Its use is associated with a higher initial price for the intervention; 2,970 euros in Germany, 2,950 euros in France and 2,266 euros (1,790 pounds sterling) in UK. The aim of this study was to calculate the financial impact of rhBMP-2 treatment in Germany, UK and France from a societal perspective with a two-year time horizon. Based on clinical findings of a previously published study with a pooled data analysis, a health economic model was developed to estimate potential cost savings derived from reduced surgery time and secondary treatment costs, and faster return-to-work time associated with rhBMP-2 use compared with autogenous bone grafting. Country-specific costs are reported in 2008 Euros. From a societal perspective, overall savings from the use of rhBMP-2 in ALIF surgery compared with autograft are 8,483 euros, 9,191 euros and 8,783 euros per case for Germany, France and UK, respectively. In all the three countries savings offset the upfront price for rhBMP-2. The savings are mainly achieved by reduced productivity loss due to faster return-to-work time for patients treated with rhBMP-2. Use of rhBMP-2 in anterior lumbar fusion is a net cost-saving treatment from a societal perspective for Germany, France and UK. Improved clinical outcome for the patient combined with better health-economic outcome for the society support rhBMP-2 as a valuable alternative compared with autograft.